Q4 2020 Editas Medicine Inc Earnings Call
Ladies and gentlemen, good morning, and welcome to any type of medicine fourth quarter and for year 2020 Conference call All day.
Operator: Good morning, and welcome to Editas Medicine's fourth quarter and full year 2020 conference call. All participants are now in a listen-only mode.
Disciplined are now in a listen only mode. There will be a question and answer the session at the end of this call. Please be advised that this call is being recorded at the company's request.
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 Ron Mulvaver, Investor Relations, at Editas Medicine. Thank you, operator. Good morning, everyone.
I would like to turn the call all of which is on the Davis Investor Relations at Medicine.
Thank you operator, good morning, everyone and welcome to our fourth quarter and full year 2020 conference call earlier. This morning, we issued a press release, providing our financial results and corporate updates for the fourth quarter and full year.
Ron Mulvaver: And welcome to our fourth quarter and full year 2020 conference call. Earlier this morning, we issued a press release providing our financial results and corporate updates for the fourth quarter and full year 2020. A replay of today's call will be available on the Investors section of our website approximately two hours after its completion.
Our 2020, a replay of today's call will be available on the investors section of our website approximately two hours. After its completion after our prepared remarks, we will open the call for Q&A.
Ron Mulvaver: 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 the 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 SEC.
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 the purposes of the safe Harbor provision 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 SEC. 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 day.
Ron Mulvaver: 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, Jim Mullen.
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, Jim Mullen.
Jim Mullen: Thank you, Ron. Good morning, everyone.
Thank you Brian Good morning, everyone and thank you for joining us today.
Jim Mullen: And thank you for joining us today. With me this morning are several members of the Editas executive team, including Lisa Michaels, our new chief medical officer, and Michelle Robertson, our chief financial officer. I would like to start off by saying how thrilled I am to speak with everyone today on my first earnings call as Editas CEO. As many of you already know, I've been chairman of Editas Sports since 2008. Since assuming the role of CEO earlier this month, the most common question I've received, other than what this means for the company, is, why do this now?
With me this morning of several members of the head of tax executive team, including lease of Michaels, Our new Chief Medical Officer, and Michelle Robertson, our Chief Financial Officer.
Like to start off by saying, how thrilled I am the speak with every once a day the first earnings call.
The other tasks CEO.
As many of you already know I've been chairman of any kind of sports into 2018.
Since assuming the role of CEO earlier. This month. The most common question of angry received other than what this means for the company is why do this now.
Jim Mullen: The reason I was drawn to Editas originally was because of the remarkable gene editing technology and the promise of potentially curing diseases that were once thought to be uncurable. During the last three years, my enthusiasm for the company grew as quickly as the company itself. The chance to step in as CEO at this stage of the company is an incredible opportunity. When I started working at Biogen in 1989, the company was smaller than it is today.
The reason why withdrawing the edit tests of originally was because of the remarkable gene editing technology in the promise of potentially curing diseases. The rule once thought to be on the curable.
For the last three years my enthusiasm for the company grew as quickly as the company itself.
The chance of step in the CEO at this stage of the company is an incredible opportunity when I started working at Biogen of 1989, the company was smaller than the other taxes today.
Jim Mullen: The success and growth of Biogen are directly related to the people and components that were in place at each phase of that company. As Editas transitions from preclinical to clinical to development and commercialization, I look forward to strategically leading the company through each of these stages. The editing technology that is the foundation of Editas is fantastic. As this technology continues to advance, along with all the downstream technologies, we need to make sure the company is strategically positioned to maximize these capabilities and bring medicine to patients. Now I'd like to discuss 2020.
The success in growing for Biogen is directly related to the people in components that were in place for each phase of the company.
The other tests transitions from preclinical to clinical to the development and commercialization I look forward to strategically leading the company through each of these stages.
The editing technologies as the foundation of the other task is fantastic.
As this technology continues to advance along with all of the downstream technologies, we need to make sure. The company is strategically positioned to maximize these capabilities bring medicines to patients.
Now I'd like to discuss 2020, but before I do that I'd like to acknowledge all of the hard work that all of the associates did during 2020 are working.
Jim Mullen: But before I do that, I'd like to just acknowledge all of the hard work that all of the associates did during 2020, working through COVID, as everyone else in the world has had to do as well. We enter 2021 as the global leader in in vivo gene editing and are posed to bring our first ex vivo medicine to the, So let's begin with some of the accomplishments of Editas over the past. We made history one year ago by initiating and advancing the first ever clinical trial of an in-vivo gene-edited medicine with EDIT101 for LCA.
Working through Covid as every.
One else of the World has had to do as well.
We enter 2021 as the global leader in the in vivo gene editing and are posed to bring our first ex vivo medicine to the court.
So let's begin with some of the accomplishments of the other tests over the past year, we made history, one year ago by initiating and advancing the first ever clinical trial of an in vivo gene editing medicine with edit 101 for LCI channel.
Jim Mullen: We regained full operating control and rights of our ocular programs through a new agreement with AbbVie and transitioned the clinical contracts, IND for Edit 101, and manufacturing to Editas in the second half of the year. We presented preclinical data demonstrating the best-in-class potential of EDIT 301 for sickle cell disease, filed the IND, and received approval by the FDA to begin patient enrollment in the RUB We expanded our manufacturing capabilities through relationships with Azure and Catalan. And we extended our cash run rate at year-end with more than $500 million in cash and raised approximately $250 million in net proceeds earlier this year, enabling us to fund our operations well into 2023.
We regained full operating control and rights of our ocular programs through the new agree with the Abbvie and transition of the clinical contracts I N V for edit 101 and manufacturing to other tests in the second half of the year.
We presented preclinical data demonstrating the best in class the central of edit three of one for sickle cell disease filed the IMD and received approval by the FDA to begin patient enrollment in the Ruby clinical trial.
We expanded our manufacturing capabilities through relationships with Azure and catalyst and.
And we extended our cash run rate.
At year end with more than $500 million of cash and raised approximately $250 million of net proceeds earlier this year, enabling us to fund our operations well into 2023.
Jim Mullen: And lastly, we added outstanding executive leadership with the appointments of Lisa Michaels as Chief Medical Officer and Meeta Chatterjee to the Board of Directors. I'm pleased to have Lisa join me on the call. The momentum of these accomplishments puts Editas in a very good position to achieve a number of value-creating milestones in 2021. Our key goals for 2021 are, first, to continue dosing patients in a brilliance trial for EDIT101. I'm pleased to announce we've already dosed the first patient in Cohort 2.
And lastly, we added outstanding executive leadership for the appointments of lease of Michaels as Chief Medical Officer, and music chaired the G to the board of directors.
Pleased to have Lisa join me on the call today.
The momentum of these accomplishments puts out of tests in a very good position to achieve a number of value creating milestones in 2021 of our key goals for 2021 of our first to continue dosing patients from the brilliance trial for edit 101.
I am pleased to announce we've already dosed the first patient in cohort two we also plan to share initial clinical trial data from the phase one to bring its trial of edit 101 before the end of the year.
Jim Mullen: We also plan to share initial clinical trial data from the Phase 1-2 Brilliance trial of EDIT101 before the end of the year. We'll advance our ocular programs by declaring a development candidate for RP4, which is retinitis pigmentosa. Initiate dosing in the Phase 1-2 RUBY clinical trial of EDIT-301 for the treatment of sickle cell disease, and we expect to file an IND application for EDIT-301 for the treatment of beta thalassemia. We want to continue the development of our engineered IPSC-derived NK cell medicines for the treatment of solid tumor cancers and present new preclinical data at medical meetings. And finally, we will progress the Now, I will turn the call over to Liisa Michaels, our new Chief Medical Officer, to introduce herself, discuss EDIT 101 and EDIT 301 clinical trials, and review our broader pipeline. Liisa?
We will advance our ocular programs by declaring a development candidate for ERP for retinal pigment ptosis.
Initiate dosing in the phase one two Ruby clinical trial of the edit 301 for the truth of the sickle cell disease, and we expect to file an IND application for edit 301 for the treatment of beta thalassemia.
We wanted to continue the development of our engineered Ips derived NK cell medicines for the treatment of solid tumor cancers and present, new preclinical data at medical meetings later this year and for.
Finally, we will progress the company's collaboration with Bristol Myers Squibb to advanced Alpha Beta T cell medicines.
Now, let me turn the call over to lease the Michaels, our new Chief Medical officer to introduce herself discuss it at one of the one and edit 301 of the clinical trials and to review our broader pipeline Lisa.
Liisa Michaels: Thank you, Jim. Thank you both for your introduction, and thank you for being with all of you for being with us on the call today. I'm very pleased to have joined Editas and to have the opportunity to lead an extremely experienced and dedicated group of clinical trial professionals who are focused on bringing treatments to patients with debilitating diseases resulting from genetic causes. By training and in my early career, I was a pediatrician.
Thank you Jim. Thank you both for your introduction and thank you for being with all of you for being with US today on the call today.
Very pleased to have joined out of toss it to have the opportunity to lead an extremely experienced from dedicated group of clinical trial professionals.
Focused on bringing treatments to patients with debilitating diseases.
<unk> from genetic causes of training.
Training and in my early career I was the pediatric Hematologist oncologist.
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Liisa Michaels: My interest during my academic practice was in finding and creating treatment solutions for rare diseases across the spectrum of immunology, blood and bone marrow disorders, and malignancies. And as such, I have been involved with clinical research and drug development for more than 25 years in both industry and academia.
My interest during my academic practice was from finding and creating true solutions for rare diseases across the spectrum of immunology blood and bone marrow, the disorders and malignancies and as such I have been involved with clinical research and drug development for both the industry and academia for more than 25 years.
Liisa Michaels: I joined Editas from Bayer Pharmaceuticals, where, in my most recent position, I was the head of clinical development for the rare diseases cell and gene therapy therapy. I'm very excited about the transformative potential of Editas' Premier Gene Editing platform, and I'm excited to drive the development of these potentially curative treatments into the clinic, with the goal of transforming the future standard of care. I'll begin today's update with our in vivo gene edited medicine pipeline, which constitutes the first pillar of our therapeutic strategy.
I joined out of tasks from Bayer Pharmaceuticals, where in my most recent position I wish the.
Our head of clinical development of the rare disease of cell and gene therapy therapeutic area.
I'm very excited about the transformative potential of Ed cautious premier of gene editing for it.
The platform and I'm excited to drive development of these potentially curative treatments into the clinic onto approval with the goal of transforming the future standard of care.
I'll begin today's update with our in vivo gene edited medicine pipeline, which constitutes the first pillar of our therapeutic strategy.
Liisa Michaels: As Jim mentioned last year, the first patients ever to receive an in vivo gene editing product were dosed in the BRILLIANCE trial, a Phase 1-2 safety study of Edit 101 for the treatment of Leber's congenital amaurosis type 10, or LCA-10. The goal of the development program is to demonstrate a cure for this genetic retinal disease that results in blindness, usually in childhood. At the recent J.P. Morgan conference, we shared the very important findings that there were no dose-limiting adverse events in the first two patients that were treated, And this result has allowed us to escalate to the next plan, Joe's School. Additionally, the observed safety allowed us to modify the inclusion criteria to allow enrollment of Sentinel patients with visual acuity better than just light perception.
As Jim mentioned, the last year, the first patients ever to receive an in vivo gene editing product for just in the brilliance trial.
The phase one two safety study of edit 101 for the treatment of Leber congenital amaurosis type 10, or LCA 10.
The goal of the development program is to demonstrate the cure of the genetic disease that results from blindness, usually childhood at the.
Recent JP Morgan conference, we shared the very important findings the debris.
No dose limiting adverse events in the first two patients that were treated with.
Result has allowed us to escalate to the next planned dose cohort.
Additionally, the observed safety allowed us to modify the inclusion criteria to allow enrollment of Sentinel patients with visual acuity better than just light perception.
Liisa Michaels: Feedback from our investigators is that this change will facilitate enrollment and is expected to help the study regain momentum. As such, we're pleased to report that we have already dosed the first patient in the adult mid-dose cohort. We continue to follow all the treated patients for the primary endpoint of safety every three months for the first. We currently collect data to confirm the expected beneficial effects of the edits.
The feedback from our investigators that this change will facilitate enrollment and is expected to help the study we gained momentum as.
As such we are pleased to report that we have already dosed the first patient in the adult mid dose cohort.
We continue to follow all of the treated patients for the primary endpoint of safety every three months for the first year.
Currently collect data to confirm the expected beneficial.
For the beneficial effects of the edits.
Liisa Michaels: We expect to share clinical data from the BRILLIANCE trial by the end of this year. Now, following 101, our next in vivo ocular program is edit 102 for the treatment of Usher Syndrome 2A. ASH2A is a different inherited retinal disease that results in progressive loss of vision in childhood or later in life. It follows on her work on
We expect of share clinical data from the brilliance trial by the end of this year.
Now following 101, our next in vivo ocular program is edit one or two for the treatment of Usher syndrome <unk>.
I wish to weigh in sort of different inherited retinal disease that results in progressive loss of vision in childhood or later in life. This probe.
Program follows on her work on edit 101, instead of one or two use as much at the same editing machinery and delivery system of edit one of one.
Liisa Michaels: This EDIT102 uses much of the same editing machinery and delivery system as EDIT103. As we reported at the recent JPMorgan meeting, we have completed the transfer of manufacturing materials for EDIT-102 from AbbVie to our CDML, and we are progressing the program. Our third ocular program is aimed at another progressive cause of the, Autosomal Dominant Retinitis Pigmentosa Type 4, or RP4. RP4 is a more complex target as mutations are found throughout the gene.
As we reported also at the recent Jpmorgan meeting we have completed the transfer of manufacturing materials of edit one of 'em too from Abbvie to our CMO and we arent progressing the program forward.
Our third ocular program is aimed at another progressive cost of blindness.
All of the Soma dominant retinitis pigmentosa type for RP for RP.
RP for is a more complex target of mutations are found throughout the gene.
Liisa Michaels: Consequently, we are exploiting the strengths of our editing approach to replace the abnormal gene with a wall-type version. We've made excellent progress on this program and plan to identify a development candidate by year. Our programs targeting treatments for blindness aim to solve a significant need. Looking to the future, we plan to further expand our in vivo pipeline and aim to leverage the curative potential of genes to address other inherited causes or predispositions to blindness across all age groups.
Sequentially, we are exploiting the strength of our editing approach to replace the abnormal gene with the wild type version and.
And we've made excellent progress on this program and plan to identify a development candidate by year end.
Our programs targeting treatments for blindness aim to solve a significant need around the globe and looking to the future. We plan to further expand our in vivo pipeline and aimed at leverage the curative potential of gene editing to address other inherited the cautious our predispositions deadline is across all age groups.
Liisa Michaels: So now transitioning to our ex vivo gene edited medicine programs, which is our second area of focus. Our most advanced ex-vivo cell medicine program is Edith Rios. This is potentially a best-in-class, durable, autologous cell medicine for sickle cell disease and beta thalassemia. With the FDA approving the start of the Phase I-II Ruby study in sickle cell disease, we expect to enroll our first patient later this year. In addition, we will be moving EDIT 301 forward into beta thalassemia. The goal is to follow the IND by year end. Free clinical data that was presented at the recent American Society of
So now transitioning to our ex vivo gene edited medicine programs, which is our second area of focus.
Our most advanced ex vivo cell medicine program is edit 301.
Just the potentially best in class durable autologous cell medicine for sickle cell disease and beta thalassemia.
Does the FDA approving the start of the phase <unk> study in sickle cell disease, we expect to enroll our first patient later this year.
In addition, we will be moving edit 301 forward in to date of thalassemia goal to file the IND by year end.
Preclinical data that was presented at the recent American Society of Hematology.
Liisa Michaels: and the National Hematology Conference, or ASH, meeting in December showed that the editing of CD34 cells from healthy donors and from sickle cell patients using 3-O-1 was greater than 90% efficient and was specific, although all target effects were not detected.
Hematology conference for Ash meeting in December showed that the editing of CD 34 cells from healthy donors and from sickle cell patients using 301 was greater than 90% of efficient and with specific.
Off target effects were not detected.
Liisa Michaels: Predictive of the expected clinical benefit, the red cells derived from patients with sickle cell disease, edited with EDIT, showed effective switching of hemoglobin production in favor of. In the same presentation, Editas demonstrated the development of a successful, large-scale manufacturing system for Editas that will be used in the processing of patient cells as part of our clinical program. Ultimately, we believe that EDID-301 will provide a durable and differentiated treatment with the potential to transform the lives of patients with inguistical cell disease and transfusion-dependent beta thalassemia.
<unk> of the expected clinical benefits of red cells derived from patients with sickle cell disease and limited with edit 301 showed effect of switching of hemoglobin production in favor of fetal hemoglobin.
And then the same presentation at a cost of demonstrated the development of a successful large scale manufacturing system for edit 301 that will be used in the processing of patient sales as part of our clinical program.
Ultimately, we believe that edit 301 will provide the durable and differentiate of treatment with the potential to transform the lives of patients living with sickle cell disease and transfusion dependent beta thalassemia.
Liisa Michaels: Moving next to oncology, the other major focus of our gene-edited cell medicines program. Well, at the end of last year, we decided to discontinue our Healthy Donor Edited NK Cell Program, EDIT 201, in order to focus our resources on advancing iPSC-derived NK cell medicine. The learnings from non-clinical studies completed on the HDNK program are directly relevant to the development of iPSC-derived NK cells, which we believe have potentially superior benefits as a homogeneous, fully characterized, therapeutic that is off the shelf for the treatment of a variety of tumor types.
Moving next to oncology the other major focus of our gene edited cell medicine programs.
At the end of last year, we decided to discontinue our healthy donor NK cell program net of 201 in order to focus our resources on advancing Ips C derived NK cell medicines.
The learnings from non clinical studies completed on the HD NK program are directly relevant to the development of Ips derived NK cells, which we believe has potentially superior benefits as the homogeneous.
The characterized therapeutics, which is off the shelf for the treatment of a variety of tumor types.
Liisa Michaels: By targeting the use of INK cells, we hope to avoid the toxicities associated with other immunotherapies, such as graft-versus-host disease and cytokinesis. Editas also shared data at last December's ASH meeting showing that INK cells, which have the CISH and TGF-beta double knockout, were more effective in killing tumor cells than control INK cells in a model mimicking the in vivo tumor These data support the potential of our INK program and treatment for Solid.
By targeting use of NK cells, we hope to avoid the toxicities associated with other immunotherapies, such as graft versus host disease, the cytokine release syndrome.
<unk> also share data at last Decembers ash meeting showing that our NK cells, which have the fish and TGF beta double knockout for more effective in killing tumor cells and control I NK cells in our model.
The in vivo tumor microenvironment and these data support the potential of our I N K program as a treatment for solid tumors.
Liisa Michaels: Our INK program is complemented by our partnership with Bristol-Myers Squibb. This collaboration recently generated a milestone payment for Editas resulting from our Alpha Beta T cell medicines program, providing an important part of validation for our editing technology. Overall, the progress we've made across both in vivo and ex vivo gene editing has advanced our goals of developing differentiated, transformational medicines for people living with serious disease, and we look forward to building our momentum and providing updates on our progress.
Our other NK program is complemented by our partnership with Bristol Myers Squibb.
This collaboration recently generated of milestone payments for <unk>, resulting from our Alpha Beta T cell medicines program, providing an important part of validation for editing technology and expertise.
Overall, the progress we've made across both in vivo and ex vivo gene editing has advanced our goals of developing differentiated transformational medicines for people living with serious diseases, and we look forward to building our momentum in 2021, we will provide updates on our progress along the way.
Michelle Robertson: And now I'd like to turn the call over to our Chief Financial Officer, Michelle Robertson.
And now I would like to turn the call over to our Chief Financial Officer, Michelle Robertson.
Michelle Robertson: Thank you, Lisa. And good morning, everyone.
Thank you Lisa and good morning, everyone edits house remains in a strong financial position as we advance our portfolio of forward.
Michelle Robertson: Editas remains in a strong financial position as we advance our portfolio forward. As Jim mentioned, our recent financing resulted in net proceeds of approximately $250 million, substantially strengthening our balance. Combined with the capital raised from last year, we are well-positioned for continued execution, supporting the manufacturing and clinical objectives of the BRILLIANCE and RUBY clinical trials and also enabling the advancement of our pre-clinical in-devo and ex-devo candidates. Our cash equivalents and marketable securities as of December 31st were $512 million compared to $457 million as of December 31st, 2019.
As Jim mentioned, our recent financing resulted in net proceeds of approximately $250 million substantially strengthening our balance sheet.
Combined with the capital raise from last year, we are well positioned for continued execution supporting the manufacturing of clinical objectives of the brilliance and will be clinical trial.
And also enabling the advancement of our preclinical in vivo and ex vivo candidates.
Our cash cash equivalents in marketable securities as of December 31st for $512 million compared to $457 million as of December 31, 2019. This does not include the approximately $250 million in net proceeds raised earlier this year, which extends our cash runway well into 2023.
Michelle Robertson: This does not include the approximately $250 million in net proceeds raised earlier this year, which extends our cash runway well into 2023. Now turning to revenue and expenses, which we have also summarized in our financial results for the fourth quarter and full year 2020 in the press release that was issued earlier today. Revenue was $91 million compared to $21 million for the same period last year.
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Now turning to revenue and expenses, which we are also summarized in our financial results for the fourth quarter and full year 2020 in the press release that was issued earlier today.
Revenue was $91 million compared to 21 million for the same period last year. This increase was mostly attributed to previously deferred revenue of $57 million from the termination of our alliance with Allergan as well as revenue received from our other collaborations and out licensing agreements.
Michelle Robertson: This increase was mostly attributed to previously deferred revenue of $57 million from the termination of our alliance with Allergan, as well as revenue received from our other collaborations and out-licensing agreements. While our G&A expenses remained relatively flat in 2020 at $68 million compared to $65 million in 2019, R&D expenses for the full year 2020 were up $61 million to $158 million. This increase was primarily driven by our ramp-up in manufacturing and clinical-related costs for EDIT 101 and EDIT 301, as well as non-recurring charges related to collaborations and success payments to our licensors.
While our G&A expenses remained relatively flat in 2020 at $68 million compared to $65 million in 2019, R&D expenses for the full year 2020 were up $61 million to $158 million.
This increase was primarily driven by a ramp up in manufacturing and clinical related costs for edit 101, and edit 301 as well as non recurring charges related to collaborations of success payments to our license source.
These expenses combined with our expanding and maturing pipeline and advances in our platform were the primary drivers of growth in our spending in 2020, we.
We expect that these will continue to be the primary drivers of spending growth in 2021.
Over the course of the year, we grew the size of our organization by approximately 23% increasing to over 240 full time employees from 195 employees at the end of 2019.
Michelle Robertson: These expenses, combined with our expanding and maturing pipeline and advances in our platform, were the primary drivers of growth in our spending in 2020. We expect that these will continue to be the primary drivers of spending growth in 2021. Over the course of the year, we grew the size of our organization by approximately 23%, increasing to over 240 full-time employees from 195 employees at the end of 2019. At this time, we don't expect any material negative financial impact from COVID-19.
At this time, we don't expect any material negative financial impacts from COVID-19 in fact, as we sort of alluded to our brilliance and Ruby trial are both progressing and we're excited to be part of these potentially life changing solutions to patients.
And with that I'll hand, it back to Jim.
Thank you Michelle.
The company is incredibly proud of where the achieved over the last year. These accomplishments of solidified our position of the global leader in vivo gene editing and leaves us poised to expand our clinical pipeline.
Michelle Robertson: In fact, as Liisa alluded to, our Brilliance and Ruby trials are both progressing, and we are excited to be part of these potentially life-changing solutions for patients. And with that, I will hand it back to Jim. Thank you, Mary.
These accomplishments were achieved amidst the global pandemic is a testament to the hard work and unwavering commitment of our employees and partners.
Over the last three years and especially over the last three weeks I've learned a tremendous amount of about the diseases at the pass as trying to cure the.
Jim Mullen: Thank you, Michelle. The company is incredibly proud of what it has achieved over the last year. These accomplishments have solidified our position as a global leader in in-vivo gene editing and leave us poised to expand our clinical potential. That these accomplishments were achieved amidst a global pandemic is a testament to the hard work and unwavering commitment of our employees. Over the last three years, and especially over the last three weeks, I've learned a tremendous amount about the diseases Editas is trying to cure, the patients suffering from them, and the capabilities that this company has to potentially change these patients' lives.
The patients suffering from them and the capabilities of this company has to potentially change these patients' lives.
Both humbled and excited the served as head of task.
I'm the CEO.
Look forward to our progress from 2021 and the momentum it will generally towards the ultimate goal developing differentiated transforming of medicines across a range of serious diseases.
We thank all of you for your continued interest and support in.
With that we will open up the call for Q&A operator.
Thank you for a reminder, ladies and gentlemen to ask a question just press star one of your telephone to withdraw your question press the pound or hash scheme. Please standby, while we compile the Q&A roster.
And our first question comes from Gena Wang with Barclays.
Jim Mullen: I'm both humbled and excited to serve as Editas' permanent CEO. We look forward to our progress in 2021 and the momentum it will generate towards the ultimate goal of developing differentiated, transformative medicines across a range of serious, We thank all of you for your continued interest and support. And with that, we will open up the call for Q&A. Operator?
<unk> please.
Hi, this is sheldon.
The for Gena, Thanks for taking all of our questions I have two if.
If I may so first zone edit 101.
Could you remind us how much waiting period is required for the cohort two and beyond.
<unk>.
Interestingly, how many patients could we expect in the updates.
The.
Before year end.
And my second question is on them at the 301.
Operator: Thank you. And as a reminder, ladies and gentlemen, to ask a question, just press star one on your telephone. To withdraw your question, press the pound or hash key. Please stand by while we compile the Q&A roster. And our first question comes from Gina Wang with Barclays. Your question, please. Hi, this is Sheldon on behalf of Gina. Thanks for taking our question. I have two, if I may. So first, on Edit 101, could you remind us how much of a waiting period is required for the cohort to
So.
Given the recent.
Adverse events reported by Blue birds, how are you thinking about the conditioning regimen and the overall approach in sickle cell disease. Thanks.
Alright, I guess, that's me at least the Michaels and I will jump in for those two questions. So to the first one regarding <unk>.
You asked I think starting with what was the waiting period by the protocol. There is a four to six week waiting period that occurs after each first two patients are dosed in that protocol. So we're actually very happy to let everybody know that we were able to dose our first patient in the mid dose cohort of <unk>.
Back in January and so that patient will be coming back in for the first offer the one of the major safety follow up periods of time in order to determine if it's safe to move onto the second patient in the cohort.
Operator: and beyond.
Operator: How many patients could we expect in the updates before year-end? And my second question is, it is 301. Um, so, given the recent adverse events reported by Bluebirds, how are you thinking about the conditioning regimen and the overall approach to sickle cell disease? Thanks.
That patient then would be dosed sometime in the next several weeks if we have the for the approval or we see no safety concerns and then we have a second waiting period on that patient before we kind of over the next two so we're looking at for patients being dosed in the mid dose cohort based upon that timeline at the moment. We also have the follow up.
Liisa Michaels: All right, I guess that's me, that's Lisa Michaels, and I will jump in for those two questions. So, for the first one regarding LCA-10, you asked, I think, starting with what was the waiting period. By the protocol, there is a four to six week waiting period that occurs after each first two patients are dosed in that protocol. So we're actually very happy to let everybody know that we were able to dose our first patient in the mid-dose cohort back in January.
Thats being planned for the first two patients in cohort one.
Both of those patients are also scheduled for return visits in the next several weeks we continue to follow those patients on an every three month basis. After the initial safety follow up where were looking both for mid and long term safety as well as additional efficacy and so we're looking as we move forward with the second cohort of patients in combination with the first one to have a significant amount.
Liisa Michaels: And so that patient will be coming back in for one of the major safety follow-up periods of time in order to determine if it's safe to move on to the second patient in the cohort. [inaudible] So we're looking at four patients being dosed in the mid-dose cohort based upon that timeline. We also have follow-up visits planned for the first two patients in Cohort 1. Both of those patients are also scheduled for return visits in the next several weeks.
At robust enough data set regarding the safety and hopefully early signs of efficacy in combination we will be able to give some nice.
Some nice conclusion, so that's why we've pretty much targeted the second half of the year in order to share more data.
For the second question.
I think we're all watching bluebird very very carefully and very cautiously at the moment.
Liisa Michaels: We continue to follow those patients on an every-three-month basis after the initial safety follow-up, looking both for mid- and long-term safety as well as additional efficacy. And so we're looking, as we move forward with the second cohort of patients in combination with the first one, to have a significant amount of robust enough data regarding both safety and, hopefully, early signs of efficacy in combination, where we'll be able to give some nice conclusions. So that's why we've pretty much targeted the second half of the year in order to share more.
Field.
I feel I'm very sympathetic to my colleagues and friends, who is not only participated in the treatment of patients in the part of that study as well as also blue bird, which has spent substantial amount of data as well as the number of patients treated the suddenly have the setback several years into their program.
As much as we're watching them at the moment I think an important part to keep the clear here is that the bluebird as well as our program are not really interchangeable the actual even though its the same patient population similar procedures to treat the patients and also overlapping in very similar clinical endpoints the law.
Liisa Michaels: For the second question, I think we're all watching Bluebird very, very carefully and very cautiously at the moment. I feel very sympathetic to my colleagues and friends who've not only participated in the treatment of patients in part of that study, but also Bluebird, which has a substantial amount of data, as well as a number of patients treated, to suddenly have this setback several years into their program. As much as we're watching them at the moment, I think an important part to keep clear here is that the Blueberg, as well as our program, are not really interchangeable.
Tori endpoints as well as the approach to treating those patients are distinctly different approaches.
The Bluebird program is based on the rental virus injure.
Injection of of gene therapy, basically putting in a gamma globulin of the beta I'm, sorry of the normal functioning beta globin gene and so between mental virus and also being of gene therapy program. It actually is a very distinctly different approach than what we're doing with sterling.
Non viral delivery.
And also doing gene editing so it's not really clear at the moment of the risks that they have encountered will be the same as we move into the gene editing program, but we are watching very closely and very carefully because of any outcomes from this to have the potential to affect our approach moving forward.
Liisa Michaels: They actually, even though it's the same patient population, similar procedures to treat the patients and also overlapping and very similar clinical endpoints. However, the laboratory endpoints, as well as the approach to treating those patients, are distinctly different approaches. The Blue Bird program is based on lentivirus injection of gene therapy, basically putting in a gamma globulin gene, the beta gene, sorry, the normal functioning beta globin gene. And so between lentivirus and also being a gene therapy program, it actually is a very distinct and different approach than what we're doing with doing non-viral delivery and also doing gene editing.
Okay. Thank you also maybe just a quick clarification on the waiting for you already mentioned the floor for six weeks in the later emission of the second the waiting period. So the is the focus of the two waiting periods combined or each one.
So each each patient is treated sequentially. So we have to do one patient first wait for follow up second patient wait for follow up and then we have the ability to treat the subsequent patients.
Got it thank you so much.
Yeah.
Thank you. Our next question is from Corey pass them off with JP Morgan Your question. Please.
Hey, Good morning. This is Turner on for Cory. Thanks for taking my question. So just with respect to the Ruby trial, where are you with the improved potency assay that the FDA requested just prior to enrolling the efficacy portion and how do you see those timelines intersecting so that it won't cause any future delays.
Liisa Michaels: So it's not really clear at the moment that the risks that they have encountered will, but we are watching very closely and very carefully because any outcomes from this do have the potential to affect our approach moving forward.
So it's helpful for at least understands that the protocol.
Operator: Thank you. Also, maybe just a quick clarification on the waiting period. You mentioned that it would take four to six weeks, and then later you mentioned a second waiting period.
Basically is divided into a basically a rule and very similar to what I. Just described actually for LCA 10. The first couple of patients who were treated in that study or are part of the quote unquote safety cohort and that allows us to be able to provide long enough observation times between patients to ensure the what we're doing is.
Liisa Michaels: [inaudible] So, each patient is treated sequentially, so we have to do one patient first, wait for follow-up, the second patient, wait for follow-up, and then we have the ability to treat the next patient.
Operator: Got it. Thank you so much. Thank you. Our next question is from Corey Kazimoff with JPMorgan. Your question, please. Hey, good morning. This is Turner on for Corey.
The safe, but also efficacious. So that's really kind of the the period of time that the FDA has allowed us to just move forward exactly as planned and.
Operator: Thanks for taking my question. So just with respect to the Ruby trial, where are you with the improved potency assay that the FDA requested just prior to enrolling the efficacy portion? And how do you see those timelines intersecting so that it won't cause any future delays?
In the same timeframe that will continue to work through the potency assay in the meantime, we have a little bit more clarity from the agency regarding the type of questions that they actually had insisted we are planning to go back actually sometime mid this year with a clearly defined plan. So hopefully that the FDA will agree with our progress moving for it but it's not interfering with our timelines, it's something that can easily be done in <unk>.
Liisa Michaels: So it's helpful to at least understand that the protocol basically is divided into basically a rule and is very similar to what I just described for LCA-10. The first couple of patients who were treated in that study are part of the quote unquote safety cohort, and that allows us to be able to provide long enough observation times between patients to ensure that what we're doing is actually both safe and efficacious.
Parallel to the work that we're already starting.
Great. That's helpful. Thank you.
Yes.
Yes.
Thank you. Our next question is from Phil Nadeau with Cowen and company. Your question. Please.
Good morning, Thanks for taking my question.
First a question on edit 102 for two way can you talk a bit more about the program and what is necessary to get it into the clinic.
Liisa Michaels: So that's really kind of the period of time that the FDA has allowed us to just move forward exactly as planned, and in the same time frame that we'll continue to work through the potency assay. In the meantime, we have a little bit more clarity from the agency regarding the type of questions that they actually had, and so we are planning to go back actually sometime in the middle of this year with a clearly defined plan, so hopefully, the FDA will agree with our progress moving forward.
So anyway, we're basically moving forward with clinical candidate in that particular space.
And I think largely what we're really doing is just trying to get more of the non clinical confirmation of effect in various different models before we declare that clear candidate and we're hoping to have that progress done later this year.
Got it and then.
The second on 301.
Liisa Michaels: But it's not interfering with our timelines. It's something that can easily be done in parallel to the work that we're already starting.
In light of the part of question on Bluebird and maybe the concerns around the the.
For generally cost of the competition.
Operator: Great, that's helpful. Thank you. Thank you. Our next question is from Phil Nadeau with Cowan & Company. Your question, please. Good morning. Thanks for taking my question. First, a question on EDIT1024-2A. Can you talk a bit more about the program and what is necessary to get it into the clinic?
Can you talk about what will be the bar to moving that forward what type of proof of concept data do you need to see to advance into pivotal trials.
I'm a little confused or are we talking about let me see with length of viral of what we're seeing in our clinical trial.
So I think the concern is is also about the pre conditioning regimen. So there is concern about the pre conditioning regimen, plus it's a relatively credit field. So can you talk about the bar that you're.
Liisa Michaels: So anyway, we're basically moving forward with a clinical candidate in that particular space, and I think largely what we're really doing is just trying to get more non-clinical confirmation of effect in various different models before we declare that clear candidate. And we're hoping to have that progress made.
You're holding to three of one two to moving into a pivotal study and continue to investment I think.
Total lots of people during J P. Morgan that basically I see each one of these plants as the sequential improvement on the prior to it and we had talked even at that time, but that the theoretical concerns of safety and at the time. They were more theoretical concerns of safety related to using both the lentivirus and of gene therapy approach versus gene editing the <unk>.
Operator: got it, and then Second on 301, in light of the prior question on Bluebird and maybe the concerns around the approach generally, plus the competition, can you talk about what will be the bar to moving that forward? What type of proof of concept data?
Operator: Do you need to see to...
Operator: Do you need to see to advance that into a pivotal trial?
Sequence of the next level of proof here is really is gene editing going to be a sequential improvement over safety in that particular setting and also the particular targets that we're looking at such as are you, calling after bcl 11, a which I remind people meet piece.
Operator: I'm a little confused. Are we talking about what we've seen with the lentil virus or what?
Operator: Well, I...
Operator: So I think the concern...
Liisa Michaels: is also about the preconditioning regimen. So there's concern about the preconditioning regimen. Plus, it's a relatively crowded field. So can you talk about the bar that you're holding for 301 to move it into a pivotal study and continue to invest in the program?
B cell leukemia AML. It was defined as part of the known problem in the other clinical settings versus targeting of more physiologic type of points such as the gamma globulin, Lucas, which the index physiologic changes that take place for hemoglobin F. So I think one of the things that will differentiate with all of these over the long.
Liisa Michaels: I told a lot of people during J.P. Morgan that basically I see each one of these plans as a sequential improvement on the one prior to it, and we had talked even at that time about the theoretical concerns of safety, and at the time they were more theoretical concerns of safety related to using both the lentil virus and a gene therapy approach versus gene editing. In consequence, the next level of proof here is really, is gene editing going to be a sequential improvement over safety in that particular setting, and also the particular targets that we're looking at, such as, are you going after BCL11A, which I remind people means B-cell leukemia, you know, it was defined as part of a known problem in other clinical settings, versus targeting a more physiologic point such as the gamma globulin lucas, which mimics physiologic changes that take place for hemoglobin F. So I think one of the things that will differentiate with all of these, actually is going to be the potential related to safety.
Term actually is going to be the potential related to safety in the short term I think of lot of people are looking at the conditioning regimen, which has always been one of the biggest hurdles and even treating patients with.
What was considered the standard of care for cures of bone marrow transplant. Many patients did not go for it because of concerns related to the conditioning regimen and the advantage of the autologous programs of courses that you are able to use much less conditioning.
Obviously, the concerns that have come up recently with the length of Io program.
<unk> also a little bit to do with whether or not these are effects that are associated with alcohol alkylating agents such as peace Olson. So I think we're still kind of waiting and watching with all of that but I do think that as we move all of these programs for the main focus and goal is not only getting excellent clinical outcomes, but also being able to improve on safety long term.
Liisa Michaels: In the short term, I think a lot of people are looking at the conditioning regimen, which has always been one of the biggest hurdles in even treating patients with what was considered the standard of care for Bone Marrow Transplant. Many patients did not go for it because of the concerns related to it, and the advantage of the autologous programs, of course, is that you're able to use much less conditioning. Obviously, the concerns that have come up recently with the lentiviral program also have a little bit to do with whether or not these are effects that are associated with alkalinating agents such as busulfan.
Great. That's very helpful. Thank you.
Thank you. Our next question comes from Matthew Harrison with Morgan Stanley. Your question. Please.
Hello, everyone. This is cost of strong for my view of.
The quick question from Us on the NK cell program. When do you think it would be ready to file a night and day for this program and what steps out of needed to beat at the for the identified tangible.
Yeah.
I guess I'll jump in for the fact that we were making a huge amount of progress and we're actually from moving for two of <unk> filing for our HD NK program with the attitude of one program, but I think as we began to become much more familiar with the the process of doing the edits as well as the benefits of using Ips CS sales force.
Liisa Michaels: So I think we're still kind of waiting and watching with all of that, but I do think that as we move all these programs forward, the main focus and goal is not only getting excellent clinical outcomes but also being able to improve safety. Great, that's very helpful.
And Kate human derived NK cells, we began to be much more aware of the advantages of moving forward with an Ips C off the shelf type program.
Operator: Great, that's very helpful, thank you. Thank you. Our next question comes from Matthew Harrison with Morgan Stanley. Your question, please. Hello, everyone. This is Kostas.
So for right now what we're doing is that we're now taking a deep dive into our I N K program with those learnings and looking at what would be our best clinical candidates or the first approach to our proof of concept study. So I'm I think we're probably will provide a little bit more information on those specific targets before I'm able to give you the clears timeline for the <unk>.
Operator: I have a question for Matthew. A quick question from us on the NCASE sales program. When do you think you will be ready to file?
Operator: Thank you.
Liisa Michaels: I guess I'll jump in on the fact that we are making a huge amount of progress and we're actually moving forward with an IND filing for our HDNK program with the Edit 201 program.
<unk>.
Great. Thank you.
Thank you. Our next question is from jewelry with choice of Securities. Your question. Please.
Hello. This is an adult gorilla dominion for jewelry. Thank you for taking my questions regarding Elbit won the one Hollywood <unk> antibodies, one influence efficacy readout.
Liisa Michaels: But I think as we began to become much more familiar with the process of doing the edits, as well as the benefits of using iPSC cells versus human-derived NK cells, we became much more aware of the advantages of moving forward with an iPSC off-the-shelf program. So for right now, what we're doing is now taking that deep dive into our INK program with those learnings and looking at what would be our best clinical candidates or the first approach to a proof-of-concept study. So I think we'll probably need to provide a little bit more information on those specific targets before I'm able to give you a clear timeline.
How close do you think we can get from your normal vision.
Also according to our predictions per cent editing might be sufficient in the for the full gear, but.
But the more conservative estimate of what has gone negative.
Opex publications suggest 20% everything might be required to restore the normal vision what is your perspective here.
Okay. So I think there's two questions. There one is related to the 10% threshold for quality edits and the other one was what I'm sorry.
The other one is related to the <unk>, so basically how well the patients can see before the enrolling the trial and are treated because one of these debt youll see higher improvement in lower the CVI, but how will the influence the efficacy readout.
Operator: Great, thank you. Thank you. Our next question is from Joon Lee with Truist Securities. Your question, please. Hello, this is Miguel Coelho filling in for Joon Lee.
Operator: Thank you for taking our questions. Regarding Edit 101, how will the BCVA baseline influence the efficacy readout? And how close do you think we can get to near normal vision? Also, according to your predictions, 10% editing might be sufficient in the fovea, but a more conservative estimate based on adaptive optics publications suggests 20% editing might be required to restore near normal vision. What is your perspective on this? Thank you.
Okay. So so we're actually keeping a very open mind related to what would be defined as the primary efficacy readout for these patients.
E V E is only one of several different measurements that we're looking at we're also looking at whether or not we can get some improvement in terms of these patients look down of gun barrel. So we're also looking at any of potential improvements in visual fields as it can be measured with perimeter free and we're also looking at mobility and function very similar to the luxe turn of debt through the.
Liisa Michaels: OK, so I think there were two questions there. One was related to the 10% threshold for quality edits, and the other one was, sorry.
Use of amazed I think each one of those in themselves provide the potential real quality improvement for these patients vision.
Operator: The other one is related to the BCVI baseline, so basically how well the patients can see before they enroll in a trial and are treated, because one idea is that you'll see higher improvements in lower BCVI, but how will this influence the efficacy results?
And as of today as for collecting information on are patients I think we'll have a better chance to see where we're going to fall in terms of the true benefits of vision.
Liisa Michaels: Okay, so we're actually keeping a very open mind related to what would be defined as a primary efficacy readout for these patients. BCVA is only one of several different measurements that we're looking at.
The real benefits that the patients are having it may be across more than one measure. So another reason why I would like to be able to provide more of a hell of a robust efficacy readout on these patients as opposed to just focusing on just one end point to the second question related to the amount of editing.
Liisa Michaels: We're also looking at whether or not we can get some improvement in terms of these patients looking down a gun barrel. So we're also looking at any potential improvements in visual fields as they can be measured with perimetry. And we're also looking at mobility and function very similar to the luxernative through the use of a maze.
I think everybody is aware that the original modeling data was based upon how much of a vision had to be it could be lost and still maintain really good optical on the vision and those of the datas, where where if youre coming out with 10% versus 20%. What I can tell you. However is that 10% was only considered to be a threshold.
Liisa Michaels: I think each one of those in itself provides a potential real quality improvement for these patients' vision. And as of today, as we're collecting information on our patients, I think we'll have a better chance to see where we fall in terms of the true benefits of vision, which are the real benefits that the patients are getting. It may be across more than one measure. So another reason why I'd like to be able to provide more of a robust efficacy readout for these patients as opposed to just focusing on just one end point.
Response, and that is what we chose for the first two patients treated in the trial with our mid dose response, we're actually expecting editing frequencies grade in the 20%.
Excellent. Thank you so much.
Okay.
Thank you our net.
Next question is from Jay Olson with Oppenheimer. Your question. Please.
Liisa Michaels: To the second question related to the amount of editing, I think everybody is aware that the original modeling data was based upon how much vision could be lost and still maintain really good optical vision. And those are the data where you're coming out with 10% versus 20%. What I can tell you, however, is that 10% was only considered to be a threshold response, and that is what we chose for the first two patients treated in the trial. With our mid-dose response, we're actually expecting editing frequencies greater than 20%.
Oh, hi, Thank you for taking the questions.
Maybe on capital allocation.
Can you talk about your strategy for allocating resources across the ocular haemoglobinopathy and Io franchises and how you prioritize those and.
Then are there any gaps that you'd like to fill with inline seeing or acquisition and your technology or delivery platforms and then finally, maybe a question for Jim.
Congrats on all of the new leadership can you maybe talk about.
Liisa Michaels: Excellent. Thank you so much. Thank you.
Operator: Our next question is from Jay Olson with Oppenheimer. Your question, please. Oh, hi, thank you for taking the questions. Maybe on capital allocation, can you talk about your strategy for allocating resources across the Ocular Hemoglobinopathy and IO franchises and how you prioritize those? And then, are there any gaps that you'd like to fill with in-line fitting or acquisition in your technology or delivery platforms? And then, maybe, a question for Jim. Congratulations on all the new leadership. Can you maybe talk about any changes that you're making or planning to make in the direction of the company? Thank you.
Any changes that youre, making or planning to make in the direction of the company. Thank you.
Okay.
Hey, Thanks. This is Jim let me.
Troy.
Three of those and probably get a little bit of help from Michele on the first part so I think for.
One was the capital allocation.
As we go into 2021.
Against those three big platforms.
The real ex vivo in the Ips via the <unk>.
K platform.
It's roughly a third of third a third.
Not quite.
Actually that because we're also doing some investments in the platform technologies.
Which probably bears a little bit on your second question.
Which.
Yeah.
But let me finish the first question.
Jim Mullen: Okay, Jay, thanks. This is Jim.
Jim Mullen: Let me try all three of those and probably get a little bit of help from Michelle on the first part. So I think part one was capital allocation. As we go into 2021, it's Against those three big platforms, InVivo, ExVivo, and the IPSC, the IMK platform, you know, it's roughly a third, a third, a third. It's not quite, it's not exactly that, because we're also doing some investments in platform technologies, which probably bears a little bit on your second question, which and let me finish the first question. The way we think about that is, you know, just looking at the kind of progress we're making and how well each one of those platforms is unfolding.
The way, we think about that is.
Just looking at the kind of progress we're making.
Well the each one of those platforms.
Holding and so obviously as we recaptured the all.
All of the rights for ocular that's a nice space.
101, hopefully continues to move forward smoothly, you have actually quite of bit of synergy as you go into the future programs.
When you look at.
The HFC or the sickle cell.
Obviously, there is another opportunity that we're planning on there with beta thalassemia.
It'll be a different trial, but the same product.
And then the I N K is really a platform and the way I think about the platform.
As you know.
Starting from the PSC being rule of successfully added.
Jim Mullen: And so, you know, obviously, as we recaptured all the rights for Ocular, that's a nice space. If EDIT101, hopefully, continues to move forward smoothly, you actually have quite a bit of synergy as you go to the future programs. When you look at the HSC or the sickle cell, obviously, there's another opportunity that we're planning on there with beta thalassemia. It'll be a different trial, but the same product.
The.
Differentiate and expand the.
In the whatever cell type of we'd like in this particular case I okay.
That's a nice platform and we're focused on reducing that really the practice I think Lisa touched on.
Thinking through and how were addressing which edits we will start with there.
Sort of the debt.
Jim Mullen: And then INK is really a platform. And the way I think about that platform is, you know, starting from iPSCs, being able to successfully edit them, clone them, differentiate them, and expand them into whatever cell type we'd like. In this particular case, INK, that's a nice platform, and we're focused on reducing that really to practice. I think Lisa touched on, you know, thinking through and how we're addressing which edits we will start with there.
That's probably that's that is net settled yet, but it's the platform right. So once we start with a few edits we can continue on to add additional edits or change the edits.
To address different segments of the disease are different.
Tumor profiles, if you will.
Things that we want to fill the interim look I think we're always attentive to questions.
The advancement of the platform technology.
Trying to think about how we can improve the delivery aspects.
You saw beam two of the deal I think it was earlier this week sort of on delivery.
Jim Mullen: So I don't think that's, that's probably not, that's not settled yet, but it's a platform, right? So once we start with a few edits, we can continue on to add additional edits or change the edits to address different segments of the disease or different tumor profiles, if you will. Things that we, you know, want to fill in around. I think we're always attentive to questions about the advancement of platform technology, always trying to think about how we can improve the delivery aspects.
We probably are not going to chew up the lnp's right now because I think we've got plenty on our plate.
Interesting deal for them, but probably not one that we would of been excited about.
At this moment in time.
Certainly the manufacturing area is very complex for us with those three big platforms. We have a couple of partnerships there one with catalyst one with Azure, which is which is really.
Performing the activities of the manufacturing activities within their control spaces, and then of course, we're also making the guys in the rfps for ourselves in Boulder as well as doing in sourcing some of our outsourcing some of that.
Jim Mullen: You saw Beam do a deal, I think it was earlier this week, sort of on delivery. We probably are not going to chew off LNPs right now because I think we've got plenty on our plate. So it's an interesting deal for them, but probably not one that we would have been excited about at this moment in time.
So those are some of the specific as you think about these programs more broadly the I've been.
And around this business a long time.
Going to need more partnerships and whether those be manufacturing academic.
Jim Mullen: Certainly, the manufacturing area is very complex for us with those three big platforms. We have a couple of partnerships there, one with Catalan, one with Azure, which is really us performing the activities, the manufacturing activities within their controlled spaces. And then, of course, we're also making the guides and the RMPs for ourselves in Boulder, as well as outsourcing some of that. So, you know, those are some of the specifics.
The development partnerships for commercial partnerships.
We will for sure be.
Active in that space, it's really a question of the timing around the different programs.
And when is the best time to engage in those conversations.
For the near term to progress the.
The NK to progress the.
Sickle cell disease and for that matter for growth the ocular programs like we have access to the things we need in the near term.
Jim Mullen: As you think about these programs more broadly, I've been around this business a long time. You know, we're going to need more partnerships, and whether those be manufacturing, academic, development, or commercial partnerships, we will for sure be active in that space. It's really a question of timing for the different programs and when is the best time to engage in those conversations. For the near term, to progress INK, to progress sickle cell disease, and for that matter, to progress ocular programs, I think we have access to the things we need in the near term. It's sort of the medium and long term that I'm thinking about. In terms of direction for the company, You know, I probably just described how I'm thinking about that. It's early days.
It's sort of the medium and long term time, thank you Beth.
In terms of direction of the company.
Probably just describes how im thinking about that.
It's early days so.
I'm really trying to dig into each one of these platform areas, but broadly speaking I am thinking about these as three very broad platforms, which we need to.
If you will reduce the size of the products in practice.
And if we can do that successfully.
Not only do we have some initial very exciting product prospects.
But it really opens up the world to the other other applications for gene editing technology.
And.
For the sort of circle, all the way back business development of partnerships. It is clear to everybody needs them in this business.
You just have to be thoughtful about when you do them and who your partners are.
Even pfizer needed wanted to make the vaccine so.
Jim Mullen: So, you know, I'm really trying to dig into each one of these platform areas. But broadly speaking, I'm thinking about these as three very broad platforms, which we need to, you know, if you will, reduce the science to products and practice. And if we can do that successfully, then not only do we have some initial very exciting product prospects, but it really opens up the world to the other applications for genetic technology, and you know to sort of circle all the way back you know business development and partnerships it's clear everybody needs them in this business you just have to be thoughtful about when you do them and who your partners are you know even Pfizer needed one to make a vaccine so you know you just have to be very open to filling in those gaps and bringing in additional expertise and capabilities hope that answers the question.
You just have to be very open to filling in those gaps and bringing in additional expertise and capabilities I hope that answers the question.
Super helpful. Thanks for taking the question.
Thank you.
And our last question is from Steve The Hoop House with Raymond James Your question. Please.
Hi, This is Ryan Deschner on for Steve for your House.
In the LCA 10 could you talk a little bit more about the data review protocol and that study is specifically at what time points, you will be taking visual acuity where.
For the gun barrel assessment and other measurement.
And then also how do you see R&D spend increasing over 2021. Thank you.
Alright, so I'll I'll take the first one and then I'll hand off the second so for the first one actually the patients come in for routine visits every three months once we get past that first four to six week period of time will be primarily focusing on safety. The key question in that period of time of course is.
Operator: It's super helpful. Thanks for taking the question. Thank you. And our last question is from Steve Seedhouse with Raymond James. Your question, please. Hi, this is Ryan Deshner, author of Steve Seedhouse:
Some of the questions that were raised actually with the very first patients treated was on the potential to actually has the cash nine apparatus result in a very damaging or potentially not easily manage some inflammatory state.
Operator: In LCA-10, could you talk a little bit more about the data review protocol in that study specifically? At what time points will you be taking visual acuity or the gun barrel assessment and other measurements? And then also, how do you see R&D spend increasing over 2021? Thank you.
We have not seen that which for me is a huge positive finding and really has allowed us to be able to be a little bit more flexible on the type of patients that we were rolling of the study and as a consequence of what I'm just reminding folks is that the cost of that safety concerns. The first two patients were treated or treated at a low dose and they were also with patients who don't have the ability to act.
Liisa Michaels: All right, so I'll take the first one and then I'll hand off the second one. So for the first one, actually, the patients come in for routine visits every three months. Once we get past that first four to six week period of time where we're primarily focusing on safety, the key question in that period of time, of course, is Some of the questions that were raised with the very first patients treated were about the potential to actually have the Cas9 apparatus result in a very damaging or potentially not easily managed inflammatory state.
Discern.
They can only tell the difference between like dark and whether or not there's like flashing in front of their eyes. So the ability to be able to measure changes in those maybe very much physiologic measurements such as changes in pupil responses to light whether or not the can actually discern flashes of light in different parts of the eye, whether or not we can see anatomical changes but.
Liisa Michaels: We have not seen that, which for me is a huge and positive finding and really has allowed us to be a little bit more flexible in the type of patients that we see, parts of the eye, whether or not we can see anatomical changes. But we're also continuing to follow these patients go through routine walk through mazes, visualizing various different methods of seeing whether or not they concern shapes and sizes, as well as these physiologic measurements. So, all and, like I think I said, walking through the maze, I want to make sure I got it. So basically, every three months, consistently over time, we are continuing to measure. Does that answer your question?
We're also continuing to follow these patients go through routine of walked through mazes vishal.
Visual of various different message of the.
Seeing whether or not the concern shapes and sizes as well as also the sociological measurements. So all of it and like I think I sort of walking through the night. So what are the make sure I got everything so basically every three months consistently over time, we are continuing to measure these things moving forward does that answer the question.
Operator: Yes, thank you.
Yes. Thank you.
Operator: Okay, so I'll hand off the second one to you.
Okay I'll handle the second one.
Michelle Robertson: Yes, so I'll handle the second one, the R&D expense. So, you know, as you know, we put the rights back to the Ocular program. So we're fully responsible for those programs. So you'll see increased investment, obviously, in Ocular, as well as continued investment in the INK programs and sickle cell, particularly, as Jim mentioned, in manufacturing. So we'll continue to make significant investments in our manufacturing, both internally and externally, as well as continue to hire key positions in our clinical operations and regulatory department.
Yes, so I'll handle the second one of the R&D expense so.
As you know we got the rights back to the ocular programs that were fully responsible for those programs. The youll see increased investment obviously in ocular.
As well as continued investment in the.
NK program in sickle cell, particularly as Jim mentioned in manufacturing So we'll continue to make.
The significant investments in our manufacturing both internally and externally.
As well as continuing to.
The higher of key positions and our clinical operations and regulatory Department.
Michelle Robertson: Okay, thank you very much. Thank you. And, ladies and gentlemen, this concludes our Q&A session. I would like to turn the call back to James Newland for his final remarks.
Okay. Thank you very much.
Thank you and ladies and gentlemen, this concludes our Q&A session I would like to turn the call back to James Rowland for his final remarks.
James Newland: Thank you all for sitting through the call this morning. Appreciate all the questions. It's pretty clear what people are focused on, and we look forward to continuing the dialogue as the year goes on and talking about clinical data and progress on the other programs. So, appreciate it. Thank you very much. Everyone have a great weekend.
Thank you all for for sitting through the call. This morning appreciate all the questions pretty clear what people are focused on.
We look forward to continuing the dialogue as the year goes on.
Talking about clinical data and progress on the other programs. So I appreciate it. Thank you very much and everyone have a great weekend.
And ladies and gentlemen. This concludes today's presentation. Thank you once again for your participation you may now disconnect.
Operator: And ladies and gentlemen, this concludes today's presentation. Thank you once again for your participation. You may now disconnect. Thank you for watching!
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Okay.
Yes.
Net.
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Great.
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Operator: BF-WATCH TV 2021