Q2 2020 Editas Medicine Inc Earnings Call

August 6, 2020

[music].

Good afternoon, and welcome to edit cosmetics in second quarter 2020 conference call. All participants are now in listen only mode.

Operator: Good afternoon, and welcome to Editas Medicine's second quarter 2020 conference call. All participants are now in a listen-only mode.

Operator: There will be a question and answer session at the end of this call. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Mark Mulliken, Vice President of Finance and Investor Relations at Editas Medicine. Thank you, operator. Good afternoon, everyone, and welcome to our second quarter 2020 conference call. Earlier this afternoon, we issued two press releases.

There will be a question and answer session at the end of this call.

Please be advised that this call is being recorded at the company's request.

Now I'd like to turn the call over to Mark Mclaughlin, Vice President of Finance and Investor Relations at Editas Medicine.

Thank you operator, good afternoon, everyone and welcome to our second quarter 2020 conference call.

Earlier. This afternoon, we issued two press releases.

Mark Mulliken: The first press release announces the termination of our agreement with Allergan for the development of ocular medicines, returning full control of these programs to Editas Medicine. The second press release provides our financial results and corporate updates for the second quarter of 2020. A replay of today's call will be available on the investor section of our website approximately two hours after its completion.

The first press release announcing the termination of our agreement with Allergan for the development of ocular medicine.

Returning full control of these programs to Editas medicine.

The second press release provides our financial results and corporate updates for the second quarter of 2020.

A replay of todays call will be available on the Investor section of our web site approximately two hours after its completion.

After our prepared remarks, we will open the call for Q any.

As a reminder, various remarks that we make during this call about the company's future expectations plans and prospects constitute forward looking statement.

For purposes of the Safe Harbor provisions under the private Securities Litigation Reform Act of 1995.

Actual results may differ materially from those indicated by these forward looking statements as result of various important factors.

Including those discussed in the risk factor section of our most recent quarterly report on form 10-Q, which is on file with the FCC.

Mark Mulliken: In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. Except as required by law, we specifically disclaim any obligation to update or revise any forward-looking statements, even if our views change. Now, I will turn the call over to our Chief Executive Officer, Cindy Cohn. Thank you, Mark. Good afternoon, and thank you everyone for joining us for our corporate update call for the second quarter of 2020. In addition to Mark, I am joined by Charlie Albright, our Chief Scientific Officer, and Michelle Robertson, our Chief Financial Officer. The second quarter of 2020 has been an important period for the advancement of our pioneering gene editing programs, the continuation of building out the development organization and CMC, and strengthening the balance sheet.

In addition, any forward looking statements represent our views only as of today.

And should not be relied upon as representing our views as of any subsequent date.

Except as required by law, we specifically disclaim any obligation to update or revise any forward looking statement, even if our views changed.

Now I will turn the call over to our Chief Executive Officer, Cindy Collins.

Thank you Mark good afternoon, and thank you everyone for joining us for our corporate update call for the second quarter of 2020.

In addition to Mark I am joined by Charlie Albright, Our Chief Scientific Officer, and Michelle Robertson, our Chief Financial Officer.

The second quarter of 2020 has been an important period for the advancement of our pioneering gene editing programs continuation of building out the development organization and CMC and strengthening the balance sheet.

Mark Mulliken: All of this leaves us well positioned to continue to revolutionize treatments for genetic blindness, cancer, sickle cell disease, and neurological conditions. Our momentum is even more significant given the considerable challenges we and the broader scientific community are still facing with the continuing COVID-19 pandemic. I am proud of our team, whose dedication and implementation of a robust business continuity plan have enabled us to remain on track with the guidance we shared last quarter.

All of this leaves us well position to continue to revolutionize treatments for genetic blindness.

Cancer sickle cell disease.

Neurological condition.

Our momentum is even more significant given the considerable challenges, we and the broader scientific community are still facing what the continuing cobot 19 pandemic.

I am proud of our team, whose dedication and implementation of a robust business continuity plan has enabled us to remain on track with the guidance, we shared last quarter.

I would now like to review some of our key recent accomplishments.

As Mark mentioned, we are thrilled to regain full control of our ocular medicine, including at at 101, the first Nvvault Christopher medicine to be administered to patients.

Our 2017 agreement with allergies and has been terminated and we have entered into a new agreement with Abbvie that return development and commercialization rights for ocular medicine to out as Hal.

Cindy Cohn: Over the coming months, we will transition activities previously carried out by Allergan, including regulatory and clinical activities, as well as manufacturing activities for EDIT101 and EDIT102 to Editas. Additionally, we will purchase inventory for EDIT 101 and EDIT 102 programs and add staff in clinical development, research, manufacturing, and quality. For our lead program, EDIT 101 for LCA-10, we will resume dosing in the Phase 1-2 Brilliance Clinical Trial now that sites have been cleared to dose patients after the COVID-19 related pause.

Over the coming months, we will transition activities previously carried out by al or Gan, including regulatory and clinical activities as well as manufacturing activities for at at 101 and at one or two two at a time.

Additionally, we will purchase inventory for at at 101, and at one or two programs and add staff in clinical development research manufacturing and quality.

For our lead program at at 101 for LCH, then we will resume dosing in the phase one two beryllium clearly clinical trial now that sites have been clear to dose patients. After the cobot 19 related pause.

We remain on track to complete the adult low dose and dose at least one patient in the adult mid dose cohort by the end of this year.

I'm pleased to report that we are continuing to progress our differentiated engineered cell medicines for cancer and Hemoglobinopathies.

Cindy Cohn: We remain on track to complete the adult low-dose and dose at least one patient in the adult mid-dose cohort by the end of this year. I am pleased to report that we are continuing to advance our differentiated engineered cell medicines for cancer and hemoglobinopathies. We remain on track to file our IND by the end of the year for Edit 301, our cell medicine for sickle cell disease. We have identified the lead investigator, as well as our CRO, and are beginning to identify clinical sites. We continue to advance IND-enabling studies for our lead oncology candidate, Edit 201, an allogeneic healthy donor in K-cell medicine for the treatment of solid tumors.

We remain on track for filing our I NZ by the end of the year for edit Threeo won our cell medicine for sickle cell disease.

We have identified the lead investigator as well as our COO and are beginning to identify clinical site.

We continue to advance I envy, enabling studies for our lead oncology candidate edit to a one and allogeneic healthy donor NK cell medicine for the treatment of solid tumors.

Additionally, we continue to advance our IPO see derived NK programs for solid tumors as well.

We believe our proprietary Caf 12, a engineering for both our healthy down or an IPO see derived NK cell medicine will propel at a top to generate transformative medicines for solid tumors and aereo with oppressing unmet need for innovative treatment solution.

As we advance our pipeline towards the clinic, we continued to build our manufacturing capabilities, both internally and externally.

To support this robust pipeline in the years ahead, we recently announced two important agreement.

Cindy Cohn: Additionally, we continue to advance our iPSC-derived NK programs for solid tumors as well. We believe our proprietary Cas12a engineering for both our healthy donor and iPSC-derived NK cell medicines will propel Editas to generate transformative cell medicines for solid tumors, an area with a pressing unmet need for innovative treatment solutions. As we advance our pipeline towards the clinic, we continue to build our manufacturing capabilities both internally and externally. To support this robust pipeline in the years ahead, we recently announced two important agreements. We signed a multi-year lease agreement for a clean room space with Azure to perform preclinical and early phase clinical manufacturing activities for our engineered cell medicines. Editas employees will perform all manufacturing and analytical work.

We signed a multi year lease agreement for a clean room space with as Youre to perform preclinical and early phase clinical manufacturing activities for our engineered cell medicines.

Editas employees perform all manufacturing and analytical work.

Securing dedicated GMP manufacturing base to support the development of our cell medicines, including editorial one and add it to a one and our I NK program provides us with needed flexibility and control as we advance these programs into the clinic.

We also entered into a strategic partnership with Catalent, who will provide critical manufacturing infrastructure and support from their facilities in Baltimore, Houston and Philadelphia.

Catalents integrated support will include supplying raw material viral vectors and engineered cell medicine production as well as storage and distribution of finished product preclinical trials.

Lastly, we continued to build out our manufacturing facility in Boulder to manufacture guide are in a.

Cindy Cohn: Securing dedicated GMP manufacturing space to support the development of our cell medicines, including EDIT-301 and EDIT-201, and our INK program, provides us with needed flexibility and control as we advance these programs into the clinic. We have also entered into a strategic partnership with Catalan, who will provide critical manufacturing infrastructure and support from their facilities in Baltimore, Houston, and Philadelphia. Catalan's integrated support will include supplying raw materials, viral vectors, and engineered cell medicine production, as well as storage and distribution of finished products for clinical trials. Lastly, we continue to build out our manufacturing facility in Boulder to manufacture guide RNA. On the organizational front, we hired 18 employees over the past quarter, primarily in development and CMC, to continue to advance our programs toward the clinic. At the executive level, we hired a chief business officer, Gad Berdugo, who has more than 25 years of experience in biotech, and we continue our search for a chief medical officer. We could not accomplish any of this without the support of our investors. A recent offering of common stock resulted in gross proceeds of approximately $216 million.

On the organizational front, we onboarded 18 employees over the past quarter, primarily in development and CMC to continue to advance our programs toward the clinic.

At the executive level, we hired a chief business Officer, Gad, Berdugo, who has more than 25 years of experience in biotech and we continue our search for our Chief Medical Officer.

We could not accomplish any of the without the support of our investors. Our recent offering of common stock resulted in gross proceeds of approximately $216 million.

These funds significantly strengthen our balance sheet, enabling the continued development of our best in class Nvvault and engineered cell medicine pipeline.

While demonstrating the continued confidence among the investment community and our strategic approach and execution. We thank you.

With that overview I would now like to turn the call over to our Chief Scientific Officer, Charlie Albright to discuss additional pipeline update.

Thanks, Andy and thanks, everyone for joining the call today.

I'll start with or in vivo, ending medicines, which constitute the first pillar of our therapeutic strategy.

Earlier this year, we're proud to report dosing of the first patient with an EBIT in vivo CRISPR medicine with dosing of at one or one of the brilliance clinical trial.

On our last call we share that the first six weeks of safety data from the first patient dose supported dosing for the second patient.

We're pleased to report today that the first patient remained stable following their three month follow up evaluation.

We look forward to dosing more patients presenting additional clinical data out at Warner one in the near future.

Following added 101 or next ocular per room is at one or two for us or syndrome tied to way.

With the termination of our agreement with our again, we will transition manufacturing processes materials from our June two editas should dances program into I'd and enabling studies.

Cindy Cohn: These funds significantly strengthen our balance sheet, enabling the continued development of our best-in-class, in vivo, and engineered cell medicine pipeline, while demonstrating the continued confidence among the investment community in our strategic approach and execution. We thank you. With that overview, I would now like to turn the call over to our Chief Scientific Officer, Charlie Albright, to discuss additional pipeline updates. Thanks, Cindy, and thanks, everyone, for joining the call today. I'll start with our in vivo editing medicines, which constitute the first pillar of our therapeutic strategy. Earlier this year, we were proud to report dosing of the first patient with an in vivo CRISPR medicine, EDIT101, in the BRILLIANT clinical trial. On our last call, we shared that the first six weeks of safety data from the first patient dose supported dosing for the second patient. We are pleased to report today that the first patient remained stable following their three-month follow-up evaluation.

We are expanding our in vivo pipeline indications beyond our dealer using our knowledge from akio programs as previously disclosed we're collaborating with asphalt.

In neurology program.

Switching to engineered cell medicines, the other strategic pillar of our therapeutic strategy.

We are encouraged with our progress with better three of one or potentially best in class medicine for Hemoglobinopathies.

Three or one remains on track for 90 falling for sickle cell disease. This year.

As a reminder, editorial one increases fetal hemoglobin by leveraging our proprietary cast twelvei to edit the beta globin locus of this site or naturally occurring human mutations increase fetal hemoglobin and suppress sickle cell disease symptoms.

The human genetics support for our approach.

Decreases potential safety risks importantly, this human genetics support is not exist for the DC eliminate enhancer site.

Further our approach induces more fetal hemoglobin in preclinical studies and editing at the DCIO M&A enhancer region.

We believe this increase fetal hemoglobin will translate into improved efficacy in the clinic.

We presented preclinical proof of concept and oral presentation at the hub in this presentation. We showed we could officially added hematopoietic stem cells from sickle cell disease patients and that these edited cells had improved properties indicative of reduced circling propensity.

Further we showed cells from healthy donor derived amount affording stem cells were maintained in vivo and elevated and pans failure fetal hemoglobin expression following editorial one treatment.

These data add to our package supporting the best in class potential of editorial one for these three homes. We are eager to test at a 301 in the clinic and remain on track to file 90 for sickle cell disease. This year.

The preparation for this phase one two trial, we've identified the lead principal investigator engaged with zero begun site selection and are planning an investigator meeting in the fourth quarter further our recent agreement with as Youre provide some necessary infrastructure to manufacture the clinical trial material.

As we've shared edits Ross is growing its investment in oncology.

Cell based medicines have shown transformational activity in liquid tumors, the major unmet need though exists in solid tumors, where we aim to develop differentiated off the shelf medicines.

Proprietary editing capabilities position us uniquely to deliver on our vision for oncology.

Charlie Albright: filing for sickle cell disease this year. As a reminder, EDIT 301 increases fetal hemoglobin by leveraging our proprietary Cas12a to edit the beta-globin locus at a site where naturally occurring human mutations increase fetal hemoglobin and suppress sickle cell disease symptoms. The human genetic support for our approach decreases potential safety risks. However, importantly, this human genetic support does not exist for the BC11A enhancer site. Further, our approach induces more fetal hemoglobin in preclinical studies and editing at the BC11A enhancer region. We believe this increased fetal hemoglobin will translate into improved efficacy in the clinic. We presented preclinical proof of concept in an oral presentation at EHOP. In this presentation, we showed we could efficiently edit hematopoietic stem cells from sickle cell disease patients and that these edited cells had improved properties indicative of reduced sickling propensity.

We've made substantial progress with our lead candidate added to a one the healthy donor derived allogeneic NK cell medicine for the treatment of solid tumors. We continue R&D, enabling studies as part of these efforts our collaboration with sand Hills accelerated the acquisition and expansion of healthy donor NK cells.

And 10% to contend to present updated preclinical data from this program and the scientific Congress later this year.

We also continue to advance our engineered IPO see derived NK cell medicine program were similarly, presenting in vitro David CCT.

These data showed that our CRISPR Cas 12, a editing platform enhanced IPO see derived NK cell tumor, killing properties and support the transformative potential these cells and some office shelf medicine.

We believe these treatments can be an important new treatment option that explores the intrinsic properties are NK cells, while avoiding the side effects of T cell therapies, such as graft versus host disease and cytokine release syndrome.

Our lead experimental medicine will contain multiple genetic changes and will provide further updates on this program later in the year.

Now I'll turn the call over to our Chief Financial Officer Michel Robertson.

Thank you Charlie and good afternoon, everyone.

Edits costs remain concerned financial position as we advance our programs forward, our cash cash equivalents and marketable securities as of June Thirtyth, turning 20 or $598.7 million compared to 415 known as of March 30, Onest from 28.

Charlie Albright: Further, we showed cells from healthy donor-derived hematopoietic stem cells were maintained in vivo and that elevated and pan-cellular fetal hemoglobin expression following EDIT301 treatment. These data add to our package supporting the best-in-class potential of ETA-301. For these reasons, we are eager to test ETA-301 in the clinic and remain on track to file an IND for sickle cell disease this year. In preparation for this Phase 1-2 trial, we've identified the lead principal investigator, engaged the CRO, begun site selection, and are planning an investigator meeting in the fourth quarter. Further, our recent agreement with Azure provides the necessary infrastructure to manufacture the clinical trial material. As we have shared, Editros is growing its investment in oncology. Cell-based medicines have shown transformational activity in liquid tumors.

The increase largely due to the $203.7 million net proceeds raised from the company's recent equity offering.

The proceeds we raise from our recent equity offering have strengthened our balance sheet and we expect our current cash balance will fund our operating plan into 2023.

We are well positioned to continue execution across our clinical and preclinical pipeline funding both our ongoing brilliance trial and also enabling the advancement of additional in vivo and ex vivo candidates into the clinic.

The use of proceeds includes further build out of the development organization enhancement of our fancy fancy analytical capability and the advancement of all of our programs.

Now I'd like to review our income statement for the second quarter turning from.

Revenue was 10.7 million compared to 2.3 million for the same period last year.

Revenue in the current quarter includes $7.6 million in cash revenues received in connection with and license agreement.

Total operating expenses were 42.1 million net of noncash stock compensation expense of $5.4 million compared to 38 million for the same period last year.

Research and development expenses were 28 million compared to 23.6 million for the same period last year.

Charlie Albright: A major unmet need, though, exists in solid tumors, where we aim to develop differentiated, off-the-shelf medicines. Our proprietary editing capabilities position us uniquely to deliver on our vision for oncology. We've made substantial progress with our lead candidate, Edit 201, a healthy donor-derived allogeneic NK cell medicine for the treatment of solid tumors. We continue IND-enabled studies. As part of these efforts, our collaboration with Sandhill has accelerated the acquisition and expansion of healthy donor NK cells. We intend to present updated preclinical data from this program at a scientific congress later this year. We also continue to advance our engineered IPSC-derived NK cell medicine program, recently presenting in vitro data at ASGCT. These data showed that our CRISPR-Cas12a editing platform enhanced iPSC-derived NK cell tumor killing properties and support the transformative potential of these cells as an off-the-shelf medicine. We believe these treatments could be an important new treatment option that exploits the intrinsic properties of NK cells while avoiding the side effects of T cell therapies such as graft versus host disease and cytokine release syndrome.

The increase in our R&D expenses was primarily attributable to the expansion of our development and innovation.

Total expenses related to R&D, enabling studies for editorial and program and nonrecurring fees related to licensing.

General and administrative expenses were $14.1 million compared to 14.4 million for the same period last year.

The decrease our DNA expenses was attributable primarily to lower professional services and patent related fees.

I reiterate for our strong balance sheet will provide support and flexibility for our pipeline progress into 2023 as well as our commitment to fiscal discipline with allocations of our capital.

With that I'll now turn the call back over to tender.

Thank you Michelle.

Im incredibly proud of the dedication from the leadership employees and trusted partners that at a cost who have enabled tremendous clinical scientific and corporate progress. Despite the challenges that remain from covered 19.

We're incredibly excited to advance the two pillars of additive.

To provide differentiated Christopher medicines that have the potential to revolutionize the treatment of genetic blindness cancer sickle cell disease and neurological diseases.

As the first and only company to treat a patient with an Nvvault Christopher gene editing medicine, we look forward to further strengthening our position as the leader in this space with additional programs advancing to the clinic and our expansion to broader indications beyond okcular.

We're also excited with our progress and engineered Paul Medicine, where our differentiated cast 12, a added AG has generated nvvault proof of concept preclinical data in our Hemoglobinopathies and oncology programs. Our recent manufacturing agreement enable the continuation.

Charlie Albright: Our lead experimental medicine will contain multiple genetic changes and will provide further updates on this program later in the year. Now, I'll turn the call over to our Chief Financial Officer, Michelle Robertson. Thank you, Charlie, and good afternoon, everyone. Editas remains in a strong financial position as we advance our programs forward. Our cash, cash equivalents, and marketable securities as of June 30, 2020 were $598.7 million, compared to $415 million as of March 31, 2020.

Of our rapid progress across these programs.

We will continue our efforts to expand the reach of gene editing across our platform and look forward to significant organizational velocity in partnership with our employees partners shareholder positions and of course patients.

We thank all of you for your continued interest and support.

With that we will open up the call for Q on a operator.

Thank you as a reminder to ask a question you will need to press Star then one on you touched on telephone to withdraw your question from the Q. Please press the pound key.

Michelle Robertson: The increase was largely due to the $203.7 million in net proceeds raised from the company's recent equity offering. The proceeds we raised from our recent equity offering have strengthened our balance sheet, and we expect our current cash balance will fund our operating plan into 2023. We are well positioned to continue execution across our clinical and preclinical pipeline, funding both our ongoing brilliance trial and also enabling the advancement of additional in vivo and ex vivo candidates into the clinic. The use of proceeds will include further build-out of the development organization, enhancement of our CMC and analytical capabilities, and the advancement of all of our programs. Now I'd like to review our income statement for the second quarter of 2020. Revenue was $10.7 million compared to $2.3 million for the same period last year. Revenue in the current quarter includes $7.6 million in cash revenues received in connection with an out-license agreement. Total operating expenses were $42.1 million, net of non-cash stock compensation expense of $5.4 million, compared to $38 million for the same period last year.

Please stand by what we compile the queuing up hey roster.

Our first question comes the Gena Wang with Barclays. Your line is now open.

Hello, Thanks for taking our question this is Peter or Gina.

I guess.

I had a question or two on edit Rio one.

My first one is.

Thank you showed potentially best in class.

Hps levels in preclinical studies, maybe you're at about 50%.

Yes levels versus maybe 30 to 40 by Chris for for example.

My question is I guess.

To what extent theme, though about having those additional beyond so called 30% threshold, which translates to additional clinical benefit.

And I have one more question after that.

Sure Thanks as Charlie so.

The clinical data with fetal hemoglobin induction across retrospectively shows that more fetal hemoglobin is better.

And I think you'll see is the trials progress that.

While some of the markers are our normalized or close to normal from the 30% hemoglobin inductions, you'll see that others are not in so markers of red cell on Wallace's for instance are unlikely to be normalize, although we haven't seen all that data.

And those.

The normalization of those markers of their increased elevation will have a will have an effect on those symptoms and the progression of the disease in sickle cell disease.

Got it. Thank you might want one one more question is also on editorial one.

As is there any.

The risk of being cutting out the H. GB I think it's to be too.

Is that an issue at all given that I guess cleavage side with the identical between let's say respect in front of age to do you want to issue too.

Michelle Robertson: Research and Development expenses were $28 million compared to $23.6 million for the same period last year. The increase in our R&D expenses was primarily attributable to the expansion of our development organization, external expenses related to IND-enabling studies for our EDIT 301 program, and non-recurring fees related to licensing. General and administrative expenses were $14.1 million compared to $14.4 million for the same period last year.

You're talking about making the deletion because of the.

Right the duplications HPG, one or two yet but the question yes, yes, that's what we know that's a really where again right now so we're not we're not number one we're not concerned in general and secondly, it's very rare event.

Okay and that very back at no safety issue.

No. It won't be initiative that will just 90 will be missing one of the two HPG wheels.

Okay, great. Thank you very much.

Thank you. Our next question comes from Matthew Harrison with Morgan Stanley. Your line is now open.

Hi, This is Mac score on from Matthew Harrison. Thank you for taking our questions could you provide any additional color regarding the royalty range and milestones associated with the new Abbvie deal and how should we think about gating factors or initial clinical timelines for the us to eight program. Thank you very much.

Michelle Robertson: The decrease in our G&A expenses was attributed primarily to lower professional services and patent-related fees. I reiterate that our strong balance sheet will provide support and flexibility for our pipeline progress into 2023, as well as our commitment to fiscal discipline with allocations of our capital. With that, I'll now turn the call back over to Cindy. Thank you, Michelle.

So I'll take the first yes, yes, the first part of that question. So.

We jointly agreed with Abbvie that we would not disclose the financial terms.

This quarter and however, the payment were obligated to pay in Q3.

He is not material to our cash runway, which will take us into 2023.

And I'll, let Charlie pick the second part of that I'll I'll answer the timeline question. So we will.

Hello.

Cindy Cohn: I am incredibly proud of the dedication of the leadership, employees, and trusted partners at Editas, who have enabled tremendous clinical, scientific, and corporate progress despite the challenges that remain from COVID-19. We are incredibly excited to advance the two pillars of Editas to provide differentiated, crisper medicines that have the potential to revolutionize the treatment of genetic blindness, cancer, sickle cell disease, and neurological diseases. As the first and only company to treat a patient with an in vivo CRISPR gene editing medicine, we look forward to further strengthening our position as the leader in the space with additional programs advancing to the clinic and our expansion to broader indications beyond the ocular. We are also excited with our progress in engineered cell medicines, where our differentiated Cas12a editing has generated in vivo proof-of-concept preclinical data in our Our recent manufacturing agreements enable the continuation of our rapid progress across these programs.

He would transition services associated with the transfer of the assets back.

Two additives and we are really thrilled to be able to regain.

Control of the LCH 10 timeline.

I'm not going to be impacted at all and we will be transferring as we indicated in his prepared remarks.

The manufacturing activities as well, so we're not going though.

Speak to timelines on today's call because we need to assimilate Ali transition activities that need to occur from a regulatory clinical and manufacturing perspective.

Great. Thank you.

Thank you and our next question comes from film the dealt with Cowen and company. Your line is now open.

Good afternoon, and thanks for taking my questions. First question is a follow up on the day.

Turning of the deal with Allergan can you talk a little bit about what gave rise to the decision to get the price back were presented to us.

Opportunistic and seeking those rights back or to the priorities and heavy algorithm change after the merger such that they were particularly interested in the program any longer.

So I certainly don't want to speak on behalf of Abbvie or speculate what the rationale was but we were certainly thrilled.

That they.

You know identified and believed that we were the best Tom for these assets and for the portfolio given that the technology has had originated with them at a time.

Okay Fair enough and then second questions on through one what remains to be completed before the R&D and or any of those activities at risk because of Cobi.

All the activities to get to the IDR. The are the typical activities, which we're at the at the tail end of all of them right now so were.

Operator: We will continue our efforts to expand the reach of gene editing across our platform and look forward to significant organizational velocity in partnership with our employees, partners, shareholders, physicians, and, of course, patients. We thank all of you for your continued interest and support. With that, we will open up the call for Q&A. Operator.

We remain on track to file the IB by the end of the year Thats required to really incredible effort from the team this year.

Great. Thanks for taking my questions.

Thank you. Our next question comes as Steve Steve House with Raymond James Your line is now open.

Great. Thanks for the question Charlie mentioned the first patient.

Densely remained stable following a three month follow up I just wanted to clarify.

Stable in that context is with respect to the safety overall health or are you referring to vision there.

Operator: Thank you. As a reminder, to ask a question, you will need to press star, then one on your touchtone telephone. To withdraw your question from the queue, please press the pound key.

The visions also stable in that and that patients and so we secretly away from next update from that in the dosing in the second patient.

Okay, so stable as and not increasing or decreasing to be clear.

Operator: Please stand by while we compile the Q&A roster. Our first question comes from Gina Wang with Barclays. Your line is now open. Hello, thanks for taking our questions. This is Peter on behalf of Gina Wang.

Right, Okay, and then on the.

New deal with Abbvie.

Could you just clarify also if that does that include known other editas programs beyond ophthalmology or is this just.

Restricted to the previously contemplated programs.

Peter (for Gina Wang): I guess I had a question or two on EDIT 301. The first one is, I think you showed potentially best-in-class HDF levels in preclinical studies. Maybe you had about 50% HDF levels versus maybe 30 to 40 by CRISPR, for example.

As of the programs that were part of the 2017 allergy and agreement. So they are LCH pan us to ebay.

An RFP for and.

Fourth fourth program to be identified.

Okay. Thanks, and last question I was hoping you could just comment on.

Base editing as a technology if other thats certainly interest in pursuing base everything to supplement your.

Already broad gene editing platform.

A couple from assigned standpoint, and leaves the business comments or somebody else. The we looked at base only early on its an interesting technology is its own listing platform.

Charlie Albright: And my question is, I guess... To what extent do you know that having those additional beyond the so-called 30% thresholds would translate to additional clinical benefits? And I have one more question after that. Sure, thanks. This is Charlie.

We feel we can do we need to do is the.

The combination of Casnine, an important recast twelvei.

And there's still a lot to be a lot to be understood about base everything is a technology.

Happy with where we are.

Hi, Thanks for the questions.

Charlie Albright: So, the clinical data with fetal hemoglobin induction retrospectively shows that more fetal hemoglobin is better. And I think you'll see as the trials progress that while some of the markers are normalized or close to normal from the 30% hemoglobin induction, you'll see that others are not. And so, markers of red cell hemolysis, for instance, are unlikely to be normalized, although we haven't seen all that data. And the normalization of those markers or their increased elevation will have an effect on those symptoms and the progression of the disease and sickle cell disease.

Thank you. Our next question comes from Yanzhou with Wells Fargo Securities. Your line is now open.

Hi, Thanks for taking the questions.

The first a question regarding the regaining of Rice just wanted to confirm has abbvie seen any clinical data generated from the first patients for patients.

Yes, as you might recall the LCH 10 program was the co development co commercialization program. So both at the top end Allergan had an active seat at the table as it related to the clinical development and the clinical trial.

Got it and could you give I do more color on the timing of data I think that's already mentioned near future for the added one on one program.

Charlie Albright: Thank you. And my one more question is also on 8301. Does there any risk of being cut out of the HGV, I think it's HGV-2? Is that an issue at all, given that, I guess, the cleavage type would be identical between the cleavage type in front of HGV-1 and HGV-2?

So if it's not.

It's difficult to be very specific on the timing could you share some of your decision criteria on when to report such as patient number or follow up time. Thanks.

So I'll start.

The.

Hope is that we will have data to share later this year as you know we've treated the first patient we are in the process of continuing to map and screen patients or dosing the second patient and we think will.

Charlie Albright: You're talking about making the deletion because of the duplication of the HPG-102, is that the question? Yes. Yeah, we know that's a really rare event.

Successfully dosed, the second patient and cohort one as well as the first patients in cohort two this year and the.

Charlie Albright: So we're not, we're not number one; we're not concerned in general. And secondly, it's a very rare event. Okay, and that rare event is no safety issue. No, it won't be an issue. It'll just mean we'll be missing one of the two HPG alleles. Okay, great. Thank you very much. Thank you. Our next question comes from Matthew Harrison with Morgan Stanley. Your line is now open. Hi, this is Max Skor on behalf of Matthew Harrison.

Clinical.

Trial sites are are open now and we have clearance through abbvie to.

Look forward and dosing the next patients so it will largely depend on what.

What the data that looks like the robustness on it and we'll have to take that decision nail on.

Patient by patient and collectively.

What we see.

From the from the study I don't know Charlie if you want to add any other color.

Gregory Allen Harrison: Thank you for taking our questions. Can you provide any additional color regarding the royalty range and milestones associated with the new AbbVie deal? And how should we think about dating factors or initial clinical timelines for the USH 2.8 program? Thank you very much. So I'll take the first part of that question. We jointly agreed with AbbVie that we would not disclose the financial terms of this quarter. And, however, the payment we're obligated to pay in Q3 is not material to our cash runway, which will still take us into 2023. And I'll let Charlie take the second part of that.

I think that's absolutely right. We're we're eager to eager to talk about the data will really sit in that timeframe that makes it makes sense from a.

From both an investor standpoint amounts and being accurate in a representation of what's happening.

Got it and.

On the added tool and a healthy donor NK program could you share some high level color on what's your.

First iteration of the product looks like.

Okay, how many added.

And.

I think you pre previously mentioned is not occurring pays rather it works with the.

Antibody therapeutics, such as I'd heard to anti PDGF are.

Any.

Cindy Cohn: I'll answer the timeline question. So we will have a few transition services associated with the transfer of the assets back to Editas, and we are really thrilled to be able to regain control of these. The LCA-10 timeline is not going to be impacted at all.

Further characterization of.

I didn't make will be very helpful. And also in with regard to date preclinical data later this year would you be able to be a little more specific on the timing.

Preclinical data thanks.

Sure, we do look forward to providing a.

Oh full preclinical package for added to a one later this year. So we're going to hold off on the on that because we are we hope to get on into assigned to the meeting.

Charlie Albright: And we will be transferring, as we indicated in the prepared remarks, some of the manufacturing activities as well. So we're not going to speak to timelines on today's call because we need to assimilate all the transition activities that need to occur from a regulatory, clinical, and manufacturing perspective. Great, thank you. Thank you, and our next question comes from Phil Nadeau with Cowan & Company. Your line is now open.

We're we're reluctant to two of the to tell you about which made it because it all depends on the abstracts and getting submitted accepted and when we're going to talk or a poster et cetera. So.

We wouldn't want to buyers.

Buys any of those things, but we really do want to talk about us and one way or the other we're going to talk about it this year hopefully they'll have a robust scientific meeting.

Philip M. Nadeau: Good afternoon, thanks for taking my questions. The first question is a follow-up on the ending of the deal with Allegan. Can you talk a little bit about what gave rise to the decision to get the rights back?

Great then last question.

Regarding the IPO see program could you speak too.

The method of.

The IP I see platform.

Cindy Cohn: Was Editas opportunistic in seeking those rights back, or did the priorities at Allegan change after the merger such that they weren't particularly interested in the program anymore? So I certainly don't want to speak on behalf of AbbVie or speculate what the rationale was, but we were certainly thrilled that they identified and believed that we were the best home for these assets and for this portfolio, given that the technologies had originated within Editas. Okay, fair enough.

Well.

Obviously its licensed from Bluerock, how is that platform.

Differentiated from other Mike.

Therapeutics method of.

The writing IPO Csos and also in terms of IP intellectual property position.

What's your thought there is there any overlap thanks.

Sure what was the first it's important to realize that what we got from Blue rock were GMP qualified IPO seeds lives and Thats really important because the.

Charlie Albright: And then the second question is on 301. What remains to be completed before the IND? And are any of those activities at risk because of COVID?

Accessing GNP qualified lines is not a is not a simple thing to do so that.

But alliance with Blue rock in that specific way allowed us to accelerate the program significantly. So we're in the middle of doing is merging a world class editing platform and creating a world class III PFC platform.

Charlie Albright: All the activities to get to the IND are the typical activities, and we're at the tail end of all of them right now. So we remain on track to file the IND by the end of the year, and that's required a really incredible effort from the team this year. Thank you. Our next question comes from Steve Seedhouse with Raymond James. Your line is now open.

We've talked quite a bit about our foundational IP position and.

Both cast nine as well as cast 12 day right now we're using cash twelvei for our cell based medicines and it's a very good enzyme and and that puts us in a great intellectual property position, we're developing our own methods, where we need to two.

Steven James Seedhouse: Great, thanks for the question. Charlie, you mentioned the first patient in the LCA-10 study remained stable following a three-month follow-up. I just wanted to clarify. Unstable in that context is with respect to safety and overall health, or are you referring to vision there? The vision is also stable in that patient, and so we eagerly await the next update on that and the dosing of the second patient. Okay, so stable as in not increasing or decreasing, to be clear.

To avoid the intellectual properties and others have a flow.

Thanks, Charlie I, sorry, I asked if I meant the IP position for Blue rocks, I PFC technology product platform, whether that how that whether that have any overlap with the other I can't see platform.

I wouldn't want to.

Probably not the right person to comment on election property for Blue rock. The I'll, just say that we feel comfortable with that position we're in.

Got it thank you.

Yes.

Operator are there any other question.

There is my apologies Ed question from Joon Lee with through Securities. Your line is now open.

Charlie Albright: Right, right. Okay. And then on the new deal with AbbVie, could you just clarify if that does that include now other editas programs beyond ophthalmology, or is this just restricted to the previously contemplated program? These are the programs that were part of the 2017 Allergan Agreement. So they are LCA-10, USH-2A, and RP4.

Hi, guys. Thanks for taking my question is and congrats on the progress.

Just wondering what started the the ocular program. Shortly you mentioned that division is stable.

It's stable and safety is a good thing, but as I recall procures program. The antisense program. They were able to see a visual improvement by month, two if I recall correctly.

You know how long would you need we do you think.

Cindy Cohn: 4th program to be identified. Okay, thanks. And last question.

And efficacy and have a follow up.

Yes, we've talked before about seeing efficacy in a small number of months I mean, I think the important thing to recognize it does this patient is like perception only and it's a low dose. So this is the this is going to be the most difficult place for us to see efficacy in the study.

Charlie Albright: I was hoping you could just comment on base editing as a technology and if Editas has any interest in pursuing base editing to supplement your already broad gene editing platform. I'll comment from a science standpoint and leave the business comments to somebody else. We looked at base editing early on. It's an interesting technology. It has its own distinct platform.

So we're we're eagerly awaiting a full somewhat to the data center.

Okay and regarding your AK steel strategy, you have an outflow and an induced encased programs.

Charlie Albright: We feel we can do what we need to do with the combination of Cas9 and importantly Cas12a, and there's still a lot to be understood about base editing as a technology. We're happy with where we are. Thank you. Our next question comes from Yanan Zhu with Wells Fargo Securities. Your line is now open.

Hi, how are you planning to.

Sort of divvy up indication or because they look very.

Yes.

Hey, already synergistic or are they more.

Ballistic.

We're going to make we're making as in other areas, we're making differentiated medicine. So we don't expect.

Yanan Zhu: Hi, thanks for taking the questions. The first question regarding the regaining of rights, just wanted to confirm: has AbbVie seen any clinical data generated from the first patients? Yes, as you might recall, the LCA 10 program was a co-development and co-commercialization program. Both Editas and Allergan had an active seat at the table as it related to clinical development and clinical trials. Got it. And could you give a little more color on the timing of the data?

Two products to cannibalize each other in fact, we'd expect to learn.

Things for me editorial one are healthy donor program to test some biological hypotheses that we will be relevant for.

Subsequent NJ products with a very healthy donor IPO Cedar Raj.

Okay.

So what would you be developing these allo and induced NK cells for the same diseases are different diseases.

We haven't disclosed indications yet that will come in due course again, there's we have sensitivity around those from a competitive perspective force.

And then.

Just.

Do you I mean, do you still benefit or get IP from.

Cindy Cohn: I think Charlie mentioned the near future for the Edit 101 program. So if it's not, if it's difficult to be very specific on the timing, could you share some of your decision criteria on when to report, such as patient number or follow-up? Okay.

They are starting to see co founders.

That all of your company.

And if so how do those discoveries it was academic labs.

Get decided whether it goes to you guys or some other company.

Cindy Cohn: The hope is that we will have data to share later this year. As you know, we've treated the first patient. We are in the process of continuing to map and screen patients for dosing the second patient, and we think we'll successfully dose the second patient in Cohort 1 as well as the first patient in Cohort 2 this year. I don't know, Charlie, if you want to add any other color.

Yes, we were were obviously in contact with the entire world to the extent we cannot founders are among the.

Among the folks we we talk too so.

I'll just leave it at about the we're constantly on the look out for technologies that would make sense to improve our platform.

Great. Thank you.

Thank you and our next question comes from Jay Olson with Oppenheimer. Your line is now open.

Charlie Albright: I think that's absolutely right. We're eager to eager to talk about the data, and we'll release it in a time frame that makes sense from a, from both an investor standpoint and as accurate a representation of what's happening. Got it. And on the Edit 201 Healthy Donor NK program, did you share some high-level color on what your first iteration of the product looks like, such as How Many Edits?

Oh, hi, thanks for taking the question.

Just a follow up on that Abbvie deal I was curious now that you regain full rights to that.

Ocular portfolio are there any.

Susan's that you would make differently or or things that you would change.

With regards to either the clinical development of one on one or the strategic direction that programs moving in now that you have the freedom to make those decisions independently.

We don't have any intention to change that strategic direction are the clinical development strategy.

Charlie Albright: And as you mentioned previously, it's not a CAR-NK. Rather, it works with antibody therapeutics, such as anti-HER2, and anti-EGFR. So any further characterization? [inaudible] Sure. We do look forward to providing a full preclinical package for EDIT 201 later this year, and so we're going to hold off on that because we hope to get that into a scientific meeting. We're reluctant to tell you about which meeting because it all depends on the abstracts and getting submitted, and accepted, and whether we get a talk or a poster, etc., so we wouldn't want to bias any of those things, but we So one way or the other, we're going to talk about it this year, hopefully at a robust scientific meeting. Great, then last question, regarding the IPSC program. Could you speak to the method of the IPSC platform? Obviously, it's licensed from Blue Rock.

I would say that.

I think we'll be able to make decision. So you know more quickly because we don't have to make joint decision and be able to advance the program more rapidly.

Okay, great. Thank you for that and then just on three year, one as you progress towards an eye. Andy are there any initial thoughts you can share with regards to the clinical trial design in terms of patient numbers or segmentation and patient population.

About age or Gino types.

Okay.

Yes, we would really caught want to comment on that at this point clearly I'm, giving you, our India and getting into patients as a top priority for us we don't really want to comment on the details in the past.

This quarter.

Okay understood. Thanks for taking the questions.

Thank you and I'm showing no further questions in the queue at this time I'd like to turn the call back to Cindy Collins for any closing remarks.

Great. So with that we thank you all are participating on today's call and for your support as we work to bring transformative new medicines to patients.

Take care NBC.

Ladies and gentlemen, thank you for your participation on today's conference. This does conclude your program and you may now disconnect.

Charlie Albright: How would that platform... Faith Therapeutics' method of deriving, And also, in terms of IP, intellectual property position, what's your thought there? Sure. Well, first, it's important to realize that what we got from Blue Rock were GMP qualified IPS seed lines. And that's really important because accessing GMP qualified lines is not a simple thing to do.

[music].

Charlie Albright: So that alliance with Blue Rock in that specific way allowed us to accelerate the program significantly. So what we're in the middle of doing is merging a world class editing platform and creating a world class IPSC platform. We've talked quite a bit about our foundational IP position in both Cas9 as well as Cas12a. Right now, we're using Cas12a for our cell-based medicines, and it's a very good enzyme, and it puts us in a great intellectual property position. We're developing our own methods where we need to avoid the intellectual property that others have filed. Thanks, Charlie.

Oh.

[music].

Charlie Albright: Sorry, I meant the IP position for Blue Rocks, platform, whether that, how the, you know, whether that Um, I'm probably not the right person to comment on intellectual property for Blue Rocks. I'll just say that we feel comfortable with the position we're in.

Operator: Thank you. Operator, are there any other questions? There is, my apologies.

Joon So Lee: A question from Joon Lee with Truist Securities. Your line is now open. Hi guys, thanks for taking my questions and congrats on the progress. Just following up on the ocular program, Charlie; you mentioned that the vision is stable. It's stable, and safety is a good thing. But you know, as I recall, the Pro QRS program, the antisense program, they were able to see a visual improvement by month three, if I recall correctly. You know, how long would you need to wait? Do you think?

Charlie Albright: Ian Eskesi and Kevin Fowler. Yeah, we've talked before about seeing efficacy in a small number of months. I mean, I think the important thing to recognize is this patient has light perception only, and it's a low dose. So this is going to be the most difficult place for us to see efficacy in this study. So we're eagerly awaiting a fulsome look at the data set there. And regarding your NK-Cell strategy, you have an Allow and an Induced NK programs. You know, how are you planning to do this? http://www.youtube.com. Are they synergistic, or are they more kind of elusive?

Charlie Albright: We're going to make, we're making, as in other areas, differentiated medicines, so we don't expect the two products to cannibalize each other. In fact, we expect to learn things from the Edit 201 or Healthy Donor Program to test some biological hypotheses that will be relevant for subsequent NK products, whether they're healthy donors or IPSC derived. OK.

[music].

Charlie Albright: So will you be developing these aloe and NK cells for the same diseases or different diseases? We haven't disclosed the indications yet. That'll come in due course. Again, we have sensitivity around this from a competitive perspective. And then, you know, just

Charlie Albright: Do you still benefit or get IP from the scientific co-founders of your company? And if so, how do those discoveries at those academic labs get decided, whether it goes to you guys or some other company? Yeah, we're obviously in contact with the entire world to the extent we can.

Charlie Albright: Our founders are among the folks we talk to. And so I just leave it at that, that we're constantly on the lookout for technologies that would make sense to improve our platform. Great. Thank you. Thank you, and our next question comes from Jay Olson with Oppenheimer. Your line is now open.

Jay Olson: Oh, hi, thanks for taking the question. Just to follow up on the AbbVie deal, I was curious, now that you regain full rights to that Ocular portfolio. Are there any decisions that you would make differently or things that you would change with regard to either the clinical development of 101 or the strategic direction that the program's moving in now that you have the freedom to make those decisions independently? We don't have any intention to change the strategic direction or the clinical development strategy.

Cindy Cohn: I would say that I think we'll be able to make decisions more quickly because we don't have to make joint decisions and be able to advance the programs more rapidly. Okay, great. Thank you for that. And then, just on 301, as you progress towards an IND, are there any initial thoughts you can share with regard to the clinical trial design in terms of patient numbers or segmentation of patient populations by age or genotype? Yeah, we wouldn't really want to comment on that at this point.

Charlie Albright: Clearly, getting the idea and getting the patients is, I think, a top priority for us, but we don't really want to comment on the details of that past, past that at this point. Okay, understood. Thanks for taking the question. Thank you, and I am showing no further questions in the queue at this time. I'd like to turn the call back to Cindy Collins for any closing remarks.

Cindy Collins: Great. So with that, we thank you all for participating in today's call and for your support as we work to bring transformative new medicines to patients. Take care and be safe. Ladies and gentlemen, thank you for your participation on today's conference. This does conclude your program and you may now disconnect. ??? ??? © BF-WATCH TV 2021, Thank you for watching! ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? ?? https://www.youtube.com.uk [inaudible] , , , , , , , , , , , , , , , , , Good afternoon, and welcome to Editas Medicine's second quarter 2020 conference call. All participants are now in a listen-only mode.

Mark Mulliken: There will be a question and answer session at the end of this call. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Mark Mulliken, Vice President of Finance and Investor Relations at Editas Medicine. Thank you, operator. Good afternoon, everyone, and welcome to our second quarter 2020 conference call. Earlier this afternoon, we issued two press releases.

Mark Mulliken: The first press release announces the termination of our agreement with Allergan for the development of ocular medicines, returning full control of these programs to Editas Medicine. The second press release provides our financial results and corporate updates for the second quarter of 2020. A replay of today's call will be available on the Investors section of our website approximately two hours after its completion.

Mark Mulliken: After our prepared remarks, we will open the call for Q&A. As a reminder, various remarks that we make during this call about the company's future expectations, plans, and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the risk factors section of our most recent quarterly report on Form 10-Q, which is on file with the FBC. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. Except as required by law, we specifically disclaim any obligation to update or revise any forward-looking statements, even if our views change.

[music].

Mark Mulliken: Now, I will turn the call over to our Chief Executive Officer, Cindy Cohn. Thank you, Mark. Good afternoon, and thank you everyone for joining us for our corporate update call for the second quarter of 2020. In addition to Mark, I am joined by Charlie Albright, our Chief Scientific Officer, and Michelle Robertson, our Chief Financial Officer. The second quarter of 2020 has been an important period for the advancement of our pioneering gene editing programs, the continuation of building out the development organization and CMC, and strengthening the balance sheet. All of this leaves us well positioned to continue to revolutionize treatments for genetic blindness, cancer, sickle cell disease, and neurological conditions. Our momentum is even more significant given the considerable challenges we and the broader scientific community are still facing with the continuing COVID-19 pandemic. I am proud of our team, whose dedication and implementation of a robust business continuity plan have enabled us to remain on track with the guidance we shared last quarter.

Cindy Cohn: I would now like to review some of our key recent accomplishments. As Mark mentioned, we are thrilled to regain full control of our ocular medicine, including EDIT101, the first in vivo CRISPR medicine to be administered to patients. Our 2017 agreement with Allergan has been terminated, and we have entered into a new agreement with AbbVie that returns development and commercialization rights for ocular medicine to Editas. Over the coming months, we will transition activities previously carried out by Allergan, including regulatory and clinical activities, as well as manufacturing activities for EDIT101 and EDIT102 to Editas. Additionally, we will purchase inventory for EDIT 101 and EDIT 102 programs and add staff in clinical development, research, manufacturing, and quality. For our lead program, EDIT 101 for LCA10, we will resume dosing in the Phase 1-2 Brilliance Clinical Trial now that sites have been cleared to dose patients after the COVID-19-related pause. We remain on track to complete the adult low-dose and dose at least one patient in the adult mid-dose cohort by the end of this year.

[music].

Cindy Cohn: I am pleased to report that we are continuing to progress our differentiated engineered cell medicines for cancer and hemoglobinopathies. We remain on track to file our IND by the end of the year for Edit 301, our cell medicine for sickle cell disease. We have identified the lead investigator, as well as our CRO, and are beginning to identify clinical sites. We continue to advance IND-enabling studies for our lead oncology candidate, Edit 201, an allogeneic healthy donor in K-cell medicine for the treatment of solid tumors.

Cindy Cohn: Additionally, we continue to advance our iPSC-derived NK programs for solid tumors as well. We believe our proprietary Cas12a engineering for both our healthy donor and iPSC-derived NK cell medicines will propel Editas to generate transformative cell medicines for solid tumors, an area with a pressing unmet need for innovative treatment solutions. As we advance our pipeline towards the clinic, we continue to build our manufacturing capabilities both internally and externally. To support this robust pipeline in the years ahead, we recently announced two important agreements. We signed a multi-year lease agreement for a clean room space with Azure to perform preclinical and early phase clinical manufacturing activities for our engineered cell medicine. Editas employees will perform all manufacturing and analytical work.

Good afternoon, and welcome to edit Medicine second quarter 2020 conference call.

All because they are now in the listen only mode.

I will be a question answer session at the end up this call.

Please be advised that this call is being recorded at the company's request.

No I could turn the call over to Mark Robertson, Vice President Finance and Investor Relations.

Medicine.

Thank you operator, good afternoon, everyone and welcome to our second quarter 2020 conference call.

Earlier. This afternoon, we issued two press releases.

The first press releases.

Combination of our agreement.

For the development occupying medicine.

Returning full control of these programs to edit house not.

The second press release provides our financial results and corporate updates for the second quarter of 2020.

A replay of todays call will be available on the Investor section of our website approximately two hours after its completion.

After our prepared remarks, well open the call for key money.

As a reminder, various remarks that we make during the call about the company's future expectations plans and prospects constitute forward looking statement.

Purposes, the Safe Harbor prevention under the private security.

Litigation Reform Act of 1995.

Actual results may differ materially used for most indicated by these forward looking statements as a result at various important factor.

Including those discussed in the risk factor section of our most recent quarterly report on form 10-Q, which is on file with the FCC.

In addition, any forward looking statements represent our views only as of today.

It should not be relied upon as representing our views as a base subsequent date.

Except as required by law, we specifically disclaim any obligation to update or revise any forward looking statement, even if argues change.

No I will turn the call over to our Chief Executive Officer, So any color.

Thank you Mark good afternoon, and thank you everyone for joining us for corporate update call for the second quarter of 2020.

In addition to Mark I'm joined by Charlie Albright, Our Chief Scientific Officer, and Michelle Roberts, our Chief Financial Officer.

The second quarter of 2020 has been an important period for the advancement of our pioneering gene editing programs continuation of building out the development organization L.C.M.C. and strengthening the balance sheet.

All of this leaves us well position to continue to revolutionize treatments for genetic blindness.

Canister sickle cell disease and neurological conditions.

Charlie Albright: And thanks, everyone, for joining the call today. I'll start with our in vivo editing medicines, which constitute the first pillar of our therapeutic strategy. Earlier this year, we were proud to report dosing of the first patient with an in vivo CRISPR medicine, dosing of EDIT101 in the BRILLIANT clinical trial. On our last call, we shared that the first six weeks of safety data from the first patient dose supported dosing for the second patient. We are pleased to report today that the first patient remained stable following their three-month follow-up evaluation.

Our momentum is even more significant given the considerable challenges, we and the broader scientific community are still facing what the continuing cobot night change hands on that.

I am proud of Archie him with his dedication and implementation of a robot business continuity plan has enabled us to remain on track with the guidance, we shared last quarter.

I would now like to review some of our key recent accomplishments.

As Mark mentioned, we are thrilled to regain full control of our ocular medicine, including at at one at one the first Nvvault CRISPR medicine to be administered to patients.

Our 2017 agreement with allergies, and how it's been terminated and we have entered into a new agree not with Abbvie that returns development and commercialization rights.

Charlie Albright: We look forward to dosing more patients and presenting additional clinical data on EDIT101 in the near future. Following EDIT 101, our next ocular program is EDIT 102 for Usher syndrome type 2a. With the termination of our agreement with Allergan, we will transition manufacturing processes and materials from Allergan to Editas to advance this program into IND-enabled studies. Additionally, we are expanding our InViva pipeline to indications beyond ocular using our knowledge from our ocular program. As previously disclosed, we're collaborating with Aspire on a neurology program. Switching to Engineered Cell Medicine is the other strategic pillar of our therapeutic strategy. We are encouraged with our progress with 03-M1, for Potentially Best-in-Class Medicine for Hemoglobin Opposites. EDIT 301 remains on track for an ID filing for sickle cell disease this year. As a reminder, EDIT 301 increases fetal hemoglobin by leveraging our proprietary Cas12a to edit the beta-globin locus at a site where naturally occurring human mutations increase fetal hemoglobin and suppress sickle cell disease symptoms. The human genetic support for our approach decreases potential safety risk. Importantly, this human genetic support does not exist for the BC11A enhancer site.

Ocular medicine to out its off.

Over the coming months, we will transition activities previously carried out by allergy and including regulatory and clinical activities as well as manufacturing activities for at at 101, and I don't want out to two out of Todd.

Additionally, we will purchase inventory for at at 101, and at one or two program and add staff in clinical development research manufacturing and quality.

For our lead program at at 101 for LCH had we will resume dosing in the phase one two beryllium <unk> clinical trial now that sites have been cleared to dose patients. After the cobot 19 related pause.

We remain on track to complete the adult low dose and dose at least one patient and the adult mid dose cohort by the end of this year.

I am pleased to report that we are continuing to progress our differentiated engineered cell medicines for cancer and Hemoglobinopathies.

We remain on track for filing our I NZ by the ended the year for editorial won our cell medicines for sickle cell disease.

We have identified the lead investigator as well as our see arrow and are beginning to identify clinical site.

We continue to advance I N D, enabling studies for our lead oncology candidate at it to a one and allogeneic healthy donor NK cell medicine for the treatment of solid tumors.

Additionally, we continue to advance our IPO see derived NK programs for solid tumors as well.

Charlie Albright: Further, our approach induces more fetal hemoglobin in preclinical studies than editing at the BC11A enhancer region. We believe this increased fetal hemoglobin will translate into improved efficacy in the. We presented preclinical proof of concept in an oral presentation at EHOP. In this presentation, we showed we could efficiently edit hematopoietic stem cells from sickle cell disease patients and that these edited cells had improved properties indicative of reduced sickling propensity. Furthermore, we showed cells from healthy donor-derived hematopoietic stem cells were maintained in vivo at an elevated and pan-cellular fetal hemoglobin expression following EDIT301 treatment.

We believe our proprietary Cafs 12, a engineering for both our healthy donor and IP FC derived NK cell medicine will propel how to talk to generate transformative medicines for solid tumors and area with a pressing unmet need for innovative treatment solutions.

As we advance our pipeline towards the clinic, we continue to build our manufacturing capabilities, both internally and externally.

To support this robust pipeline in the years ahead, we recently announced two important agreement.

We signed a multiyear lease agreement for our clean room space with as Youre to perform preclinical and early phase clinical manufacturing activities for our engineered cell medicines.

Charlie Albright: These data add to our package supporting the best-in-class potential of ETA-301. For these reasons, we are eager to test ETA-301 in the clinic and remain on track to file an IND for sickle cell disease this year. In preparation for this Phase 1-2 trial, we've identified the lead principal investigator, engaged the CRO, begun site selection, and are planning an investigator meeting in the fourth quarter. Further, our recent agreement with Azure provides the necessary infrastructure to manufacture the clinical trial material. As we have shared, Editros is growing its investment in oncology. Cell-based medicines have shown transformational activity in liquid tumors.

The top employees perform all manufacturing and analytical work.

We also entered into a strategic partnership with Catalent, who will provide critical manufacturing infrastructure and support from their facilities in Baltimore, Houston and Philadelphia.

Catalent integrated support will include supplying raw material viral vector and engineered cell medicine production as well as storage and distribution of finished product for clinical trials.

Lastly, we continued to build out our manufacturing facility in Boulder to manufacture guide our anyway.

On the organizational fraud, we onboarded 18 employees over the past quarter, primarily and development and the M.C. to continue to advance our program toward the clinic.

Charlie Albright: A major unmet need, though, exists in solid tumors, where we aim to develop differentiated, off-the-shelf medicine. Our proprietary editing capabilities position us uniquely to deliver on our vision for oncology. We've made substantial progress with our lead candidate, Edit 201, a healthy donor-derived allogeneic NK cell medicine for the treatment of solid tumors. We continue IND-enabled studies. As part of these efforts, our collaboration with Sandhill has accelerated the acquisition and expansion of healthy donor NK cells. We intend to present updated preclinical data from this program at a scientific congress later this year. We also continue to advance our engineered IPSC-derived NK cell medicine program, recently presenting in vitro data at ASGCT. These data show that our CRISPR-Cas12a editing platform enhances iPSC-derived NK cell tumor-killing properties and support the transformative potential of these cells as an off-the-shelf medicine. We believe these treatments could be an important new treatment option that exploits the intrinsic properties of NK cells while avoiding the side effects of T cell therapies such as graft versus host disease and cytokine release syndrome.

At the executive level, we hired a chief business Officer, Gad, Berdugo, who has more than 25 years of experience in biotech and we continue our search for a chief Medical officer.

We could not accomplish any of the without the support of our investors are resistant offering of common stock resulted in gross proceeds of approximately $216 million.

It's fun significantly strengthen our balance sheet, enabling the continued development of our best in class Nvvault and engineered cell medicine pipeline.

While demonstrating the continued confidence among the investment community and our strategic approach on execution. We thank you.

With that overview I would now like to turn the call over to our Chief Scientific Officer, Charlie Albright to discuss additional pipeline update.

Thanks, Andy and thanks, everyone for joining the call today.

I'll start with or in vivo editing medicines, which constitute the first pillar of our therapeutic strategy.

Earlier this year, we're proud to report dosing of the first patient with an eye in vivo CRISPR medicine with dosing of I didn't want to one of the brilliance clinical trial.

On our last call we share that the first six weeks of safety data from the first patient dose supported dosing for the second patient.

We're pleased to report today that the first patient remained stable following their three month.

A follow up evaluation.

Look forward to dosing more patients presenting additional clinical data out at Warner one in the near future.

Well I did want to one or next October program is that at one or two for us for syndrome type two way.

With the termination of our agreement with our again, we will transition manufacturing processes materials from Allergan, two editas sure advances program into R&D and enabling studies.

We are expanding our in vivo pipeline indications beyond art you are using our knowledge from our ocular programs as previously disclosed we're collaborating with asphalt.

Charlie Albright: Our lead experimental medicine will contain multiple genetic changes and will provide further updates on this program later in the year. Now, we'll turn the call over to our Chief Financial Officer, Michelle Robertson. Thank you, Charlie, and good afternoon, everyone. Editas remains in a strong financial position as we advance our programs forward. Our cash, cash equivalents, and marketable securities as of June 30, 2020, were $598.7 million compared to $415 million as of March 31, 2020.

And in Neurology program.

Switching to engineered cell medicines, the other strategic pillar of our therapeutic strategy.

We are encouraged with our progress would that are three of one or potentially best in class medicine for Hemoglobinopathies.

Three a one remains on track for 90 falling for sickle cell disease. This year.

As a reminder, editorial one increases fetal hemoglobin by leveraging our proprietary cast twelvei to edit the beta globin locus of the site or naturally occurring human mutations increase fetal hemoglobin and suppress sickle cell disease, sometimes human genetics support for our approach.

Decreases potential safety risks.

Certainly this human genetics support does not exist for the B C 11, a enhancer site.

Further our approach induces more fetal hemoglobin, a preclinical studies on editing it'll be selevend anti cancer research.

Michelle Robertson: The increase was largely due to the $203.7 million in net proceeds raised from the company's recent equity offering. The proceeds we raised from our regional equity offering have strengthened our balance sheet, and we expect our current cash balance will fund our operating plan into 2023. We are well positioned to continue execution across our clinical and preclinical pipeline, funding both our ongoing brilliance trial and also enabling the advancement of additional in vivo and ex vivo candidates into the clinic. The use of proceeds will include further build-out of the development organization, enhancement of our CMC and analytical capabilities, and the advancement of all of our programs. Now I'd like to review our income statement for the second quarter of 2020. Revenue is $10.7 million compared to $2.3 million for the same period last year. Revenue in the current quarter includes $7.6 million in cash revenues received in connection with an adult license agreement. Total operating expenses were $42.1 million, net of non-cash stock compensation expense of $5.4 million, compared to $38 million for the same period last year.

We believe this increase fetal hemoglobin will translate into improved efficacy in the clinic.

Were presented preclinical proof of concept at an oral presentation that huh.

This presentation, we show we could officially added hematopoietic stem cells from sickle cell disease patients that these edited cells had improved properties indicative of reduced circling propensity.

Further we showed cells from healthy donor derived amount affording stem cells were maintained in vivo and elevated and pans failure fetal hemoglobin expression following editorial one treatment.

These data add to our package supporting the best in class potential of editorial one for these three homes. We are eager to test set a three on one of the clinic and remain on track to file 90 for sickle cell disease. This year.

The preparation for this phase one two trial, we've identified the lead principal investigator engage the CRM BPO Big on site selection and are planning an investigator meeting in the fourth quarter further our recent agreement with as Youre provides a necessary infrastructure to manufacture the clinical trial material.

As we've shared edits Ross is growing its investment in oncology.

Cell based medicines have shown transformational activity and liquid tumors, a major unmet need though exists in solid tumors, where we aim to develop differentiated off the shelf medicines.

Our proprietary editing capabilities position us uniquely to deliver on our vision for oncology.

We've made substantial progress with our lead candidate added to a one.

Healthy donor derived Allergan age NK cell medicine for the treatment of solid tumors. We continue R&D, enabling studies as part of these efforts our collaboration with sand Hills accelerated the acquisition and expansion of healthy done rig Jay cells.

Michelle Robertson: Research and development expenses were $28 million compared to $23.6 million for the same period last year. The increase in our R&D expenses was primarily attributable to the expansion of our development organization, external expenses related to IND-enabling studies for our Editorial One program, and non-recurring fees related to licensing. General Administrative Expenses were $14.1 million compared to $14.4 million for the same period last year.

I mean temper that took intensive present updated preclinical data from this program and the scientific Congress later this year.

We also continue to advance our engineered IPO see derived NK cell medicine program recently presenting in vitro data at CCT.

These data showed that our CRISPR Cas twelvei editing platform enhanced I see derived NK cell tumor, killing properties and support the transformative potential. These cells is off the shelf medicine.

Our lead experimental medicine will contain multiple genetic changes and will provide further updates on this program later in the year.

Now I'll turn the call over to our Chief Financial Officer Michel Robertson.

Thank you Charlie and good afternoon, everyone.

Other costs remained strong financial position as we advance our programs forward, our cash cash equivalents and marketable securities as of June Thirtyth, turning 20 or $598.7 million compared to 415 Moreau as of March 30, Onest total total.

The increase largely due to the $203.7 million macrophage rates from the company's recent equity offering.

Cindy Collins: I am incredibly proud of the dedication from the leadership, employees, and trusted partners at Editas who have enabled tremendous clinical, scientific, and corporate progress despite the challenges that remain from COVID-19. We are incredibly excited to advance the two pillars of Editas to provide differentiated CRISPR medicines that have the potential to revolutionize the treatment of genetic blindness, cancer, sickle cell disease, and neurological diseases. As the first and only company to treat a patient with an in vivo CRISPR gene editing medicine, we look forward to further strengthening our position as the leader in the space with additional programs advancing to the clinic and our expansion to broader indications beyond the ocular. We are also excited with our progress in engineered cell medicine, where our differentiated Cas12a editing has generated in vivo proof-of-concept preclinical data in our hemoglobinopathies and oncology programs.

The proceeds we raise my Regal equity offering have strengthened our balance sheet and we expect our current cash balance will fund our operating plan into 2023.

The use of proceeds include further build out the development organization.

Yes, one of our fancy CMC and analytical capability and the advancement of all of our program.

Now I'd like to review our income statement for the second quarter 20 phone.

Revenue was 10.7 million compared to 2.3 million for the same period last year.

Revenue in the current quarter includes $7.6 million on cash revenues received in connection with an out license agreement.

Total operating expenses were 42.1 million net of noncash stock compensation expense of 5.4 million dollar compared to 38 million for the same carrier left here.

Research and development expenses were 28 million compared to 23.6 million for the same period last year.

Increase in our R&D expenses was primarily attributable to the expansion of our development and innovation.

Total expenses related to R&D, enabling studies for editorial one program and nonrecurring fees related to like okay.

Cindy Collins: Our recent manufacturing agreements enable the continuation of our rapid progress across these programs. We will continue our efforts to expand the reach of gene editing across our platform and look forward to significant organizational velocity in partnership with our employees, partners, shareholders, physicians, and, of course, patients. We thank all of you for your continued interest and support. With that, we will open up the call to Q&A. Operator.

General and administrative expenses were $14.1 million compared to 14.4 million for the same period last year.

The decrease our DNA expenses was attributable primarily to lower professional purposes and patent related fees.

I reiterate our strong balance sheet will provide support and flexibility for our pipeline progress into 2023 as well as our commitment to fiscal discipline with allocations of our capital.

With that I'll now turn the call back over to said.

Thank you Michelle.

Operator: Thank you. As a reminder, to ask a question, you will need to press star, then one on your touchtone telephone. To withdraw your question from the queue, please press the pound key. Our first question comes from Gina Wang with Barclays. Your line is now open. Hello. Thanks for taking our questions. This is Peter from GNAT.

We are incredibly excited to advance the two pillars of additive.

To provide differentiated Chris for medicine.

I have the potential to revolutionize the treatment of genetic blindness cancer sickle cell disease and neurological diseases.

As the first and only company to treat a patient with an nvvault Chris for gene editing medicine, we look forward to further strengthening our position as the leader in this space with additional programs advancing to the clinic and our expansion to broader indications beyond okcular.

Peter (for Gina Wang): I guess I had a question or two on EDIT 301. The first one is, I think you showed potentially best-in-class HPF levels in preclinical studies. Maybe you had about 50% HPF levels versus maybe 30 to 40 by CRISPR, for example.

We're also excited with our progress and engineered Paul Medicine, where our differentiated caps 12, a added dang has generated nvvault proof of concept preclinical data in our keven called in the opportunities and oncology programs. Our recent manufacturing agreement enable the continuation.

Some of our rapid progress across these programs.

We thank all of you for your continued interest and support.

With that we will open up the call for Q1 day operator.

Thank you as a reminder to ask a question you will need to press Star then one on you touched on telephone to withdraw your question from the Q. Please press the pound key.

Charlie Albright: So the clinical data with fetal hemoglobin induction retrospectively shows that more fetal hemoglobin is better. And I think you'll see as the trials progress that while some of the markers are normalized or close to normal from the 30% hemoglobin induction, you'll see that others are not. And so markers of red cell hemolysis, for instance, are unlikely to be normalized, although we haven't seen all that data. And the normalization of those markers or their increased elevation will have an effect on those symptoms and the progression of the disease and sickle cell disease.

Please stand by what we compile the Q and out hey roster.

Our first question comes to Gena Wang with Barclays. Your line is now open.

Hello, Thanks for taking our question Peter or Gina.

I guess.

I had a question or two on edit Rio one.

My first one is.

Thank you showed potentially best in class.

Hps levels in preclinical studies, maybe you had about.

Okay.

HTS levels versus maybe 30 to 40 by Chris for for example, and my question is I guess.

To what extent, so you know about having those additional beyond so call it 30% threshold, which translates to additional clinical benefit.

Charlie Albright: Thank you. And my one more question is also on 8301. Does there any risk of cutting out the HGV, I think it's HGV-2? Is that an issue at all, given that, I guess, the cleavage type would be identical between the cleavage type in front of the H2B1 and H2B2?

And I have one more question after that.

Sure Thanks as Charlie so.

The clinical data with fetal hemoglobin induction across retrospectively shows that more feet on globant is better.

And I think you'll see is the trials progress that.

Well some of the markers are our normalized or close to normal from the 30% hemoglobin inductions, you'll see that others are not so markers of red cell. Wallace's for instance are unlikely to be normalize all that we haven't seen all that data.

Charlie Albright: You're talking about making the deletion because of the duplication of the HPG-102, is that the question? Yeah, yeah. Yeah, we know that's a really rare occurrence.

And those.

Normalization of those markers of their increased elevation, we'll have we'll have an effect on the symptoms and the progression of the disease and sickle cell disease.

Got it. Thank you might want one one more question is outs on editorial one.

Hi, this is there any.

The risk of being.

Putting out the H. JB I think its tribute to.

Is that an issue at all given that I guess cleavage side with the identical between let's say you're stuck in front of HIV, one HIV assays.

You're talking about making the deletion because of the.

Right the duplications HPG, one or two yes. Good question, yes, yes, that's what we know that's really where again right now so we're not we're not number one we're not concerned in general and secondly, it's very rare event.

Gregory Allen Harrison: Thank you for taking our questions. Can you provide any additional color regarding the royalty range and milestones associated with the new AbbVie deal? And how should we think about gating factors or initial clinical timelines for the USH2A program? Thank you very much. So I'll take the first part of that question. We jointly agreed with AbbVie that we would not disclose the financial terms this quarter.

Okay and that Larry that no safety issue.

No it won't be initiative, it'll just nine will be missing one of the two HPG illegals.

Okay, great. Thank you very much.

Thank you. Our next question comes from Matthew Harrison with Morgan Stanley. Your line is now open.

Hi, This is Mac score on from Matthew Harrison. Thank you for taking your questions could you provide any additional color regarding the royalty range and milestones associated with the new Abbvie deal and how should we think about gating factors or initial clinical timelines for the us to eight program. Thank you very much.

So I'll first yes, yes, the first part of that question. So.

Cindy Cohn: And, however, the payment we're obligated to pay in Q3 is not material to our cash runway, which will still take us into 2023. And I'll let Charlie take the second part of that. I'll answer the timeline question. So we will have a few transition services associated with the transfer of the assets back to Editas. And we are really thrilled to be able to regain control of these. The LCA-10 timeline is not going to be impacted at all.

We jointly agreed with Abbvie that we would not disclose the financial terms.

This quarter.

And however, the payment were obligated to pay in Q3.

It's not material to our our cash runway, which will take us into 2023.

And I'll, let Charlie pick the second part of that Oh I'll answer the timeline question. So we will.

Hello.

He would transition services associated with the on transfer of the assets back.

Two out of top than we are really thrilled to be able to regain.

Control of the LCH 10 timeline is not going to be impacted at all and we will be transferring as we indicated in the prepared remarks.

The manufacturing activities as well, so we're not going though.

Speak to timelines on today's call because we need to assimilate all the transition activities that need to occur from a regulatory clinical and manufacturing perspective.

Great. Thank you.

Thank you and our next question comes from fell in the dealt with Cowen and company. Your line is now open.

Good afternoon, and thanks for taking my questions. First question is a follow up on the day.

Think of the deal with elegant can you talk a little bit about what gave rise to the decision to get the price back were presented to us.

Cindy Cohn: Was Editas opportunistic in seeking those rights back, or did the priorities at Allegan change after the merger such that they weren't particularly interested in the program anymore? So I certainly don't want to speak on behalf of AbbVie or speculate what the rationale was, but we were certainly thrilled that they, you know, identified and believed that we were the best home for these assets and for this portfolio, given that the technologies had originated within Editas. Okay, fair enough. And then the second question is on 301. What remains to be completed before the IND? And are any of those activities at risk because of COVID?

Opportunistic and seeking those rights back or to the priorities.

We're going to change after the merger such that they were particularly interested in the program any longer.

So I certainly don't want to speak on behalf of Abbvie or speculate what the rationale was that we were certainly thrilled.

That they.

You know identified in believed that we were the best talent for these assets and for the portfolio given that the technology has had originated within at a time.

Okay Fair enough and then second questions on through one what remains to be completed before the R&D and Ernie those activities at risk because of coking.

All the activities to get to the R&D are the are the typical activities, which we're at the at the tail end of all of them right now so were.

We remain on track to file the I'd by the end of the year and Thats required to really incredible effort from the team this year.

Great. Thanks for taking my questions.

Thank you. Our next question comes as Steve Steve House with Raymond James Your line is now open.

Steven James Seedhouse: Great, thanks for the question. Charlie, you mentioned the first patient in the LCA-10 study remained stable following a three-month follow-up. I just wanted to clarify. (Inaudible) The vision is also stable in that patient, and so we eagerly await the next update on that and the dosing of the second patient. Okay, so stable as in not increasing or decreasing, to be clear.

Great. Thanks for the question Charlie mentioned, the first patient and they also Tinsley remained stable following a three month follow up I just wanted to clarify.

Stable in that context is with respect to the safety overall health or are you referring to vision.

The visions also stable in the patient and so we secretly await the next update from that and the dosing in the second patient.

Okay, so stable as and not increasing or decreasing to be clear.

Right, Okay, and then on the.

New deal with Abbvie.

Could you just clarify also if that does that include no other editas programs beyond ophthalmology or is this just.

Restricted to the previously contemplated programs.

The programs that were part of the 2017 allergy and agreement. So they are LCH Pan us to AG.

Cindy Cohn: 4th program to be identified. Okay, thanks. And last question.

An RFP for and.

As for Fourq program to be identified.

Okay. Thanks, and last question I was hoping you could just comment on.

Charlie Albright: I was hoping you could just comment on base editing as a technology and if Editas has any interest in pursuing base editing to supplement your already broad gene editing platform. I'll comment from a science standpoint and leave the business comments to somebody else. We looked at face editing early on. It's an interesting technology, but it has its own distinct platform.

Editing as a technology.

So the interest in pursuing base editing to supplement your.

Already broad gene editing platform.

Thanks.

Got it from a sign of standpoint to leave the business comments or somebody else. The we looked at base owning early on its an interesting technologies its own listing platform.

Charlie Albright: We feel we can do what we need to do with the combination of Cas9 and, importantly, Cas12a. And there's still a lot to be understood about face editing as a technology. But we're happy with where we are.

We feel we can do we need to do is the.

With the combination of Casnine, an important recap twelvei.

And there's still a lot to be a lot to the understood about chase everything is a technology.

Charlie Albright: Alright, thanks for the question. Thank you. Our next question comes from Yanan Zhu with Wells Fargo Securities. Your line is now open.

Happy with where we are.

Hi, Thanks for the questions.

Thank you. Our next question comes when you add on Zoo with Wells Fargo Securities. Your line is now open.

Yanan Zhu: Hi, thanks for taking the questions. The first question regarding the regaining of rights, just wanted to confirm: has AbbVie seen any clinical data generated from the first patients? Yes, as you might recall, the LCA-10 program was a co-development and co-commercialization program. Both Editas and Allergan had an active seat at the table as it related to clinical development and clinical trials. Got it. And could you give a little more color on the timing of the data?

Hi, Thanks for taking the questions.

The first question regarding the regaining of Rice just wanted to confirm has abbvie seen any clinical data generated from the first patients first patient.

Yes, as you might recall the LCH 10 program was the co development co commercialization program. So.

Both at the top end Allergan had an active seat at the table has it related to.

The clinical development and the clinical trial.

Got it and could you give more color on the timing of data I think our tardy mentioned that near future for the added one program.

Cindy Cohn: I think Charlie mentioned that in the near future for the Edit 101 program. So if it's not, if it's difficult to be very specific on the timing, could you share some of your decision criteria on when to report, such as patient number or follow-up? So I'll start. The hope is that we will have data to share later this year. As you know, we've treated the first patient. We are in the process of continuing to map and screen patients for dosing the second patient. And we think we'll successfully dose the second patient in Cohort 1 as well as the first patient in Cohort 2 this year. I don't know, Charlie, if you want to add any other colors.

So if it's not.

It's difficult to be very specific the timing could you share. Some of your decision criteria I went to report such as patient number or follow up time. Thanks.

So I'll start.

The.

Hope is that we will have data to share later this year as.

As you know we've treated the first patient we are in the process of continuing to map and screening patients for dosing. The second patient and we think will successfully dosed the second patient and cohort one as well as the first patient in cohort two this year and.

The.

Clinical.

Charles slides are our open now and we have clearing through abbvie to.

Cindy Cohn: I think that's absolutely right. We're eager to eager to talk about the data, and we'll release it in a time frame that makes sense from both an investor standpoint and as accurate a representation of what's happening. Got it. End of, on the Edit 201 Healthy Donor NK Program. Did you share some high-level color on what your first iteration of the product looks like, such as How Many Edits?

Look forward and dosing the next patients so.

It will largely depend on what what the data that looks like the robustness of it and we'll have to take that decision. So on a.

Patient by patient and collectively.

What we see.

From the from the study I don't know Charlie if you want to add any other color.

No I think that's absolutely right. We're we're eager to eager to talk about the data in will really sit in that timeframe that makes it makes sense from a.

On both an investor standpoint.

Charlie Albright: And I think you previously mentioned it's not a CAR-NK. Rather, it works with antibody therapeutics, such as anti-HER2, anti-EGFR. So any further characterization. Edited by A.J.

And then being accurate in a representation of what's happening.

Got it and have.

On the added tool and a healthy donor NK program could you share some high level color on what Youre.

First iteration of the product look like such as how many added.

And.

I think you pre you previously mentioned is not a car NK.

Rather it works with.

Charlie Albright: Bork, The edit will be very helpful. And also with regard to data, preclinical data later this year, would you be able to be a little more specific on the timing? Sure. We do look forward to providing a full preclinical package for EDIT 201 later this year, and so we're going to hold off on that because we hope to get that into a scientific meeting. We're reluctant to tell you about which meeting because it all depends on the abstracts and getting them submitted, and accepted, and whether we get a talk or a poster, et cetera.

Antibody therapeutics, such as I'd heard to anti PDGF are so any further characterization of the added will be very helpful. And also in with regard to date preclinical data later this year.

Would you be able to be a little more specific on the timing.

For that preclinical data thanks.

Sure, we do look forward to providing a.

Full preclinical package for added to a one later this year. So we're going to hold off on the on that because we are we hope to get that into assigned to the meeting.

We're we're reluctant to.

So tells you about which made it because it all depends on the abstracts and getting submitted accepted and whether we're going to talk or a poster et cetera. So.

Charlie Albright: So we wouldn't want to bias any of those things, but we really do want to talk about them. So one way or the other, we're going to talk about it this year, hopefully at a robust scientific meeting. Great, then last question, regarding the IPSC program. Did you speak to the method of the IPSC platform? Obviously, it's licensed from Blue Rock.

We wouldn't want to buyers.

Hi, guys any of those things, but we really do want to talk about us one way or the other we're going to talk about it this year hopefully though.

Robust scientific meeting.

Great then last question.

Regarding the IPO see program.

You speak too.

The method.

The IPO see platform.

Well.

Obviously, it's a licensed from Bluerock how is that platform.

Charlie Albright: How would that platform... Faith Therapeutics' method of deriving, And also, in terms of IP, intellectual property position, what's your thought there? Sure. Well, first, it's important to realize that what we got from Blue Rock were GMP qualified IPS feed lines. And that's really important because accessing GMP qualified lines is not a simple thing to do.

Differentiated from other unlike.

Therapeutics method of.

Deriving IPO Etsy sellers and also in terms of IP intellectual property position.

What's your thoughts there is there any overlap thanks.

Sure first it's important to realize what we got from Blue rock were GMP qualified IPO seeds lines.

And thats really important because see.

Accessing GMP qualified lines is not a is not a simple thing to do so that.

Charlie Albright: So that alliance with BlueRock in that specific way allowed us to accelerate the program significantly. So what we're in the middle of doing is merging a world-class editing platform and creating a world-class IPSC platform. We've talked quite a bit about our foundational IP position in both Cas9 as well as Cas12a. Right now, we're using Cas12a for our cell-based medicines, and it's a very good enzyme, and it puts us in a great intellectual property position.

That.

Alliance with Blue rock in that specific way allowed us to accelerate the program significantly. So we're in the middle of doing is merging a world class editing platform and creating a world class III PFC platform.

We've talked quite a bit about our foundational IP position and.

Both cast nine as well as cast 12 day right now we're using cast Twelvei for our cell based medicines and it's a very good enzyme and and that puts us on a great intellectual property position, we're developing our own methods, where we need to too.

Charlie Albright: We're developing our own methods where we need to avoid the intellectual property that others have filed. Thanks, Charlie. Sorry, I meant the IP position for Blue Rocks, platform, whether that, how the, you know, whether that's Um, I wouldn't want to, I'm probably not the right person to comment on intellectual property for Blue Rocks. I'll just say that we feel comfortable with the position we're in. Got it. Thanks. Operator, are there any other questions? There is, my apologies.

Boy, the intellectual property that others have.

[music].

Thanks, Charlie I sorry.

I meant the IP position for Blue rocks, IP FC technology product platform, whether that how that whether that.

Have any overlap.

With the other IP FC platform.

So I wouldn't want to.

Probably not the right person to comment on the watch for property for Blackrock.

I'll, just say, we feel comfortable with that position we're in.

Got it thank you.

Yes.

Operator are there any other question.

There is my apologies question from Joon Lee with true Securities. Your line is now open.

Joon So Lee: A question from Joon Lee with Truist Securities. Your line is now open. Hi, guys. Thanks for taking my questions and congrats on the progress. Just following up on the ocular program, Charlie. You mentioned that the vision is stable. It's stable, and safety is a good thing.

Hi, guys. Thanks for taking my questions and congrats on the progress.

Just following up on the the ocular program. Shortly you mentioned that division is stable.

On a stable and safety is a good thing, but as I recall procures program. The antisense program they were able to see.

Charlie Albright: But, you know, as I recall, ProQR's program, the antisense program, they were able to see a visual improvement by month three, if I recall correctly. You know, how long would you need to wait, do you think? and SKC and have a follow-up. Yeah, we've talked before about seeing efficacy in a small number of months. I mean, I think the important thing to recognize is that this patient has light perception only, and it's a low dose.

Visual improvement by month lead if I recall correctly.

You know how long would you need to wait do you think.

The an efficacy.

And have a follow up.

Yeah, we we've talked before about seeing efficacy in a small number of months I mean, I think the important thing to recognize it does this patient as life perception only and into low dose. So this is the this is going to be the most difficult place for us to see efficacy in this study.

Charlie Albright: So this is the most difficult place for us to see efficacy in this study. So we're eagerly awaiting a fulsome look at the data set there. And regarding your NK-SELF strategy, you have an ALO and an induced NK program. You know, how are you planning to use them? Are they synergistic, or are they more kind of a list?

So we're we're eagerly awaiting a fulsome what are the data center.

Okay and regarding their AK steel strategy, you have an outflow and then induced encased programs.

Hi, how are you planning to.

You sort of divvy up indication or because they look very.

Charlie Albright: We're going to make, we're making, as in other areas, differentiated medicine, so we don't expect the two products to cannibalize each other. In fact, we expect to learn things from the Edit 201 or Healthy Donor Program to test some biological hypotheses that will be relevant for subsequent NK products, whether they're healthy donors or IPSC derived. OK. So will you be developing these aloe and NK cells for the same diseases or different diseases?

Yes.

Are these synergistic or are they more.

Cannibalistic.

We're going to make we're making as in other areas, we're making differentiated medicine. So we don't expect.

Two products to cannibalize each other in fact, we expect to learn.

Things from the editorial one are healthy donor program to test some biological hypotheses that will be relevant for.

Subsequent NJ products for the they're healthy donor IPO cederoth.

Okay.

So what would you be developing these allo and induced NK cells for the same diseases or different diseases.

Charlie Albright: We haven't disclosed the indications yet. That'll come in due course. Again, we have sensitivity around this from a competitive perspective. And then, you know, just, Do you still benefit or get IP from the scientific co-founders of your company? And if so, how do those discoveries at those academic labs get decided, whether they go to you guys or some other company?

We haven't disclosed indications yeah that'll come in due course again, there's we have sensitivity around this from a competitive perspective.

Sure.

And then.

Just.

Do you I mean, do you still benefit or get IP from.

The scientific Cofounders.

That up to a company.

And if so how do those discoveries it was academic labs.

Get decided whether it goes to you guys or some other company.

Yes, we were were obviously in contact with the entire world to the extent, we can our founders are among the.

Charlie Albright: Yeah, we're obviously in contact with the entire world to the extent we can. Our founders are among the folks we talk to. And so I just leave it at that. We're constantly on the lookout for technologies that would make sense to improve our platform. Great, thank you. Thank you, and our next question comes from Jay Olson with Oppenheimer. Your line is now open.

Among the folks we we talk too so.

I'll just leave it at that there were constantly on the look out for technologies that would make sense to improve our platform.

Great. Thank you.

Thank you and our next question comes from Jay Olson with Oppenheimer. Your line is now open.

Jay Olson: Oh, hi, thanks for taking the question. Just to follow up on the AbbVie deal. I was curious now that you regain full rights to that. Ocular portfolio. Are there any decisions that you would make differently or think that you would change with regard to either the clinical development of 101 or the strategic direction that the program's moving in now that you have the freedom to make those decisions independently? We don't have any intention to change the strategic direction or the clinical development strategy.

Hi, Thanks for taking the questions.

Just a follow up on that Abbvie deal I was curious now that you regain full rights to that.

Okcular portfolio are there any.

Susan's that you would make differently or or things that you would change.

In regards to either the clinical development of one on one or the strategic direction that programs moving in now that you have the freedom to make those decisions independently.

We don't have any intention to change the strategic direction are the clinical development strategy I would say that.

Cindy Cohn: I would say that I think we'll be able to make decisions more quickly because we don't have to make joint decisions and be able to advance the programs more rapidly. Okay, great. Thank you for that. And then, just on 301, as you progress towards an IND, are there any initial thoughts you can share with regard to the clinical trial design in terms of patient numbers or segmentation of patient populations by age or genotypes? Yeah, we wouldn't really want to comment on that at this point.

Thanks, well be able to make decision you know more quickly because we don't have to make joint decision and be able to advance the program with a more rapidly.

Okay, great. Thank you for that and then just on three year, one as you progress towards an eye. Indeed are there any initial thoughts you can share with regards to the clinical trial design in terms of patient numbers or segmentation and patient population.

Age or Gino types.

Yeah, we wouldn't.

I want to comment on that at this point clearly.

Charlie Albright: Clearly, getting the IND and getting any of the patients is, I think, a top priority for us, but we don't really want to comment on the details of that past that at this point. Okay, understood. Thanks for taking the question. Thank you, and I am showing no further questions in the queue at this time. I'd like to turn the call back to Cindy Collins for any closing remarks. Okay. So, with that, we thank you all for participating in today's call and for your support as we work to bring transformative new medicines to patients. Take care, and be safe. Ladies and gentlemen, thank you for your participation in today's conference. This does conclude your program, and you may now disconnect.

Given the R&D and getting into patients as a top priority for us where we don't really want to comment on the details in the.

But at this point.

Okay understood. Thanks for taking the questions.

Thank you and I'm showing no further questions in the queue at this time I'd like to turn the call back to Cindy Collins for any closing remarks.

Great. So with that we thank you all are participating on today's call and for your support as we work to bring transformative new medicines to patients take care and be safe.

Ladies and gentlemen, thank you for your participation on today's conference. This does conclude your program and you may now disconnect.

Q2 2020 Editas Medicine Inc Earnings Call

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