Q4 2019 Earnings Call
Unknown Executive: Good afternoon, and welcome to Editas Medicine's fourth quarter and full year 2019 conference call. All participants are now in listen-only mode.
Good afternoon, and welcome to Eddie Trust medicines fourth quarter, and full year 2019 conference call.
All participants are now in 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 Mullikin, Vice President of Finance and Investment Relations at Editas Medicine. Thank you, Operator.
There will be a question and answer session at the end of this call.
Be advised to this call is being recorded at the company's request.
I'd now like to turn the call over to Mark Mclaughlin, Vice President Finance and Investor Relations at a Chinese medicine.
Thank you operator, good afternoon, everyone and welcome to our fourth quarter and full year 2019 conference call.
Mark Mullikin: Good afternoon, everyone, and welcome to our fourth quarter and full year 2019 conference call. Shortly after the market closed, we issued a press release providing our financial results and corporate updates for the fourth quarter and full year 2019. A replay of today's call will be available on the investors and media section of our website approximately two hours after its completion.
Shortly after the market closed we issued a press release, providing our financial results in corporate update for the fourth quarter and full year 2019.
A replay of todays call will be available on the Investor and media section of our website approximately two hours after its completion.
Mark Mullikin: 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 annual report on Form 10-K, which is on file with the FCC. 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. Unless 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 Collins.
After our prepared remarks, well open the call for acuity.
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 up 1995.
Actual results may differ materially from those indicated by these forward looking statements as a result, a very it's important factors, including those discussed in the risk factor section of our most recent annual report on form 10-K, which is on file with the FCC.
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 Collins.
Cindy Collins: Thank you, Mark. Good afternoon, and thank you, everyone, for joining us for our corporate update call for the fourth quarter and full year 2019. In addition to Mark, I'm joined by several members of the Editas executive team, including Judith Abrams, our Chief Medical Officer; Charlie Albright, our Chief Scientific Officer; Michelle Robertson, our new Chief Financial Officer; and Tim Hunt, our Senior Vice President of Corporate Affairs. We are at an exciting point at Editas Medicine, developing differentiated, transformational medicines across a range of serious diseases and nearing the point of seeing them work in patients On today's call, we will review some of what we have achieved over the past year, then look ahead to 2020 and beyond. So let's start with some of our accomplishments over the past year.
Thank you Mark good afternoon, and thank you everyone for joining us for a corporate update call for the fourth quarter and full year 2019. In addition to Mark I'm joined by several members of the added Todd executive team, including Judith Abrams, Our Chief Medical Officer, Charlie Albright, our Chief Scientific Officer.
Hi, Michelle Robertson, our news Chief Financial Officer, and Tim Hot our senior Vice President of corporate Affairs.
We are at an exciting point at a child's medicine developing differentiated transformational medicines across a range of serious diseases and nearing the point of same them work in patients.
On today's call. We will review some of what we haven't changed over the past here, then look I had to 2020 and beyond.
So let's start with some of our accomplishments over the past year.
Cindy Collins: We initiated the first ever clinical trial of an in vivo CRISPR medicine in collaboration with Allergan of Edit 101 for patients with LCA-10. We've started IND-enabling studies for EDIT 301 as a potentially best-in-class medicine for the treatment of Sickle Cell Disease. We amended our collaboration with Celgene, now part of Bristol-Myers Squibb, to focus on developing engineered alpha-beta T-cell medicines for cancer. We are excited to continue our work with the leader in treating blood cancer. With the amendment, we received a $70 million cash payment and regained rights to develop engineered gamma T cell medicines in oncology. We also accelerated our efforts to develop engineered allogeneic NK cell medicines for cancer through newly formed research collaborations. We enabled our development of healthy donor NK cell medicines for solid tumors with cell expansion technology from Sandhill Therapeutics.
We initiated the first ever clinical trial of in Nvvault, Chris for Medicine in collaboration with Allergan and have added one elwen for patients with L.C. eight huh.
We started I N D, enabling studies for editorial one as they potentially best in class medicine for the treatment of sickle cell disease.
We amended our collaboration with Celgene now part of Bristol Myers Squibb to focus on developing engineered Alpha beta T cell medicines for cancer. We are excited to continue our work with the leader in treating blood cancers.
With the amendment, we received a 70 million dollar cash payment and regains right to develop engineered gamma T cell medicines in oncology.
We accelerated our efforts to develop engineered allogeneic NK cell medicines for cancer. Your newly formed research collaborations we enabled our development of healthy donor NK cell medicines for solid tumors with cell expansion technology from sandhill therapeutics and we are advancing ideas.
Cindy Collins: And we are advancing iPSC-derived NK cell medicine using technology from Blue Rock Therapeutics. And we added outstanding executive leadership with the appointment of Judith Abrams as our Chief Medical Officer, Michelle Robertson as our Chief Financial Officer, and Harry Gill as our Senior Vice President of Operations. I am pleased to have Judith and Michelle join me on the call today. These accomplishments give us momentum into the coming year.
You derived NK cell medicines using technology from Blue Rhino therapeutic.
And we added outstanding executive leadership with the appointment of Judith Abrams as our Chief Medical Officer, Michelle Robertson as our Chief Financial Officer, and Harry Gil as our senior Vice President of operations I am pleased to have Judith and Michelle joining me on the call today.
These accomplishments give us momentum into the coming here in 2020, we plan to announce dosing of patients in the first quarter and the beryllium phase one two trial of at at 101 in collaboration with our partner allergy on.
Cindy Collins: In 2020, we plan to announce dosing of patients in the first quarter of the Brilliance Phase 1-2 trial of EDIT101 in collaboration with our partner Allergan. Additionally, we plan to nominate a development candidate for Autosomal Dominant Retinitis Pigmentosa IV, or RPIV. File an IND for Edit 301 for the treatment of sickle cell disease. Initiate IMD-enabling studies for an allogeneic, healthy donor NK cell medicine candidate to treat solid tumors. And finally, present in vivo preclinical proof-of-concept data for an engineered, iPSC-derived NK cell medicine to treat solid tumors. Now, let me turn the call over to our Chief Medical Officer, Judith Abrams, to update you on our Phase 1-2 clinical trial of EDIT-101.
We plan to nominate a development candidate for autosomal dominant retinitis pigmentosa four or RP for.
Alan I, indeed for editorial one for the treatment of sickle cell disease.
Initiate I envy, enabling studies for an allogeneic healthy donor in Haynesville medicine candidate to treat salary solid tumors.
And finally present Nvvault preclinical proof of concept data for an engineered I.P.S.C. derived NK cell medicine to treat solid tumors.
Now, let me turn the call over to our Chief Medical Officer, Judith Abrams to update you on our phase one two clinical trial of at at 101.
Judith Abrams: Thanks, Cindy. It's my pleasure to join all of you on the call today. As Cindy mentioned, last year we opened a brilliant Phase I-II study for patients with LCA-10 for enrollment. Enrollment activity has accelerated in recent months, and we expect an announcement on the first patient dosing in the first quarter of this year. As a reminder, the first patient dose in the Billings clinical trial will mark a significant milestone toward delivering on the promised potential of CRISPR medicine to durably treat devastating diseases such as LCA-10. I'm also pleased to share that as our research pipeline continues to progress to the clinic, we are building on our senior leadership in our clinical organization. I'll now turn over the call to our Chief Scientific Officer, Charlie Albright, to discuss our broader pipeline.
Thanks, Andy.
My pleasure to do it all you want to call today.
Cindy mentioned.
Sure. We opened it really is great. Once you study for patients with LT 10 for enrollment.
Moment activity has accelerated in recent months and we expect to announce but until the first patient dosing in the first quarter of this year.
As a reminder, the first patient dose is going to come up with horrible more of a significant milestone towards delivering all the promise potential CRISPR medicine to durably tree devastating diseases, such as LTAC and.
I'm also pleased to share that was originally pipeline continues to progress to the clinic.
We are building or senior leadership in our clinical organization.
I'll now turn over the call to our Chief Scientific Officer, Charlie Albright to discuss our broader pipeline.
Charlie Albright: Thank you, Judith, and thank you all for joining us on the call. Following EDIT 101, our next in vivo ocular program is EDIT 102 for the treatment of Usher Syndrome 2A or USH 2A. Like LCA-10, S2A is an inherited retinal disease that affects photoreceptors and leads to blindness. At the product level, EDIT102 is nearly identical to EDIT101 in that EDIT102 uses the same AEV5 delivery vector, proprietary Staph aureus Cas9 enzyme, and photoreceptor-specific promoter as EDIT101. Preclinical studies support the advancement of EDIT-102 into IND-enabling studies since we have demonstrated editing levels, mRNA transcriptional levels, and phenotypic restoration that are consistent with therapeutic benefit. Based on these data, we delivered a data package for EDIT-102 to Allergan for potential licensing and development as part of the strategic alliance we formed in 2017.
I can do this and thank you all for joining us on the call.
Following I don't want to one or next in vivo akio programs at one or two for the treatment of most recent room to a rush that's true.
Like LCD turned out situation inherited retinal disease that affects photo receptors of leads to blindness.
That's a product level at a one or two is nearly identical to at a one on one in that at one or two uses the same D.V. five delivery vector proprietary staff worries casnine enzyme and photo <unk> receptor specific promoter as does I didn't want to one.
The clinical studies support the advancement of it at one or two into R&D, enabling studies since we have demonstrated editing levels.
Already transcriptional levels and phenotypic restoration never consistent with therapeutic benefit.
Some of these data we delivered a data package spread of one or two to our Dan for potential licensing and development as part of our strategic Alliance we formed in 2017.
Our learnings with that I want to one another one or two are being levers for other in vivo editing medicines, but your baby delivery of stuff, whereas casnine is used in our medicine aimed at RP for another inherited retinal disease, where we plan to declare development candidate or for R&D, enabling studies later this year.
Charlie Albright: Our learnings with EDIT101 and EDIT102 are being leveraged for other in vivo editing medicines. In particular, AAV delivery of Staph aureus Cas9 is used in our medicine aimed at RP4, another inherited retinal disease, where we plan to declare a development candidate for IND enabling studies later this year. Finally, we've expanded our in vivo research pipeline into neurologic diseases in collaboration with Ask Bio and hope to present initial data this year.
Although we've expanded our vivo research pipeline into neurologic diseases in collaboration with ask Bob Hope to present additional data this year.
Charlie Albright: Transitioning to our engineered cell medicines programs, we're developing EDIT-301 as a potential best-in-class medicine for sickle cell disease and beta thalassemia. Our program uses the Cas12 enzyme to edit the HBG1-2 promoter in the beta-globin locus to induce fetal hemoglobin in hematopoietic stem cells. CAST-12A is proprietary to Editas and previously known as CPF
For existing to our engineered cell medicines programs were developing editorial one is a potential best in class medicine for sickle cell disease beta thalassemia.
Our program uses the cast 12 enzyme to edit the H.P.G. one to promoter and the beta globin logos to these fetal hemoglobin in them out of politics stem cells.
12 day as proprietary to edit toss in previously known as CPF one.
Charlie Albright: For those unfamiliar with our program, our approach is differentiated from competitors who either edit the BC11A enhancer focus or use gene therapy. We shared our latest data at the American Society of Hematology conference in December. 301, Edits of the genomic region where human mutations are found to increase fetal hemoglobin. This genetic support is important as these data reduce the risk of human efficacy and safety. In contrast, the BC11A enhancer approach does not have human genetic validation. Credit 301.
Well those I'm familiar with our program or approaches differentiated from competitors, who either edit to be selevend enhancer, well because were you gene therapy.
We shared our latest David The American Society of Hematology Conference in December.
Three to one edit to the genomic region were human mutations are found to increase fetal hemoglobin.
This genetic support is important as these data reduced the risk with human efficacy and safety.
In contrast to be Selevend enhancer approach does not have human genetic validation.
Credit three or one preclinical data shows that HPG everything or the metal products themselves is durable produces high levels of fetal hemoglobin. It does not negatively impact blood cell lineage.
Plan for it I am for another 301, I'd filing for the treatment of patients in sickle cell disease by yearend.
Charlie Albright: Preclinical data shows that HPG editing in hematopoietic stem cells is durable, induces high levels of fetal hemoglobin, and does not negatively impact blood cell lean. Plan for an EDIT 301 IND filing for the treatment of patients with sickle cell disease by year-end. The other major focus for engineered cell medicines programs is to treat cancer. We're developing an engineered alpha-beta T-cell medicine for cancer in collaboration with Bristol Myers Squibb. The leader in treating blood cancer, where we believe alpha beta T cell medicine has the potential to be particularly effective. In addition, we are advancing our wholly owned programs by editing innate immune cells, including NK and gamma delta T cells, to treat solid tumors. We plan to begin IMD-enabling studies mid-year for an edited healthy donor NK cell medicine to treat solid tumors. We recently announced work with Hand Health Therapeutics to accelerate our healthy donor medicine. Sandhill brings established processes, manufacturing infrastructure, and proprietary expansion methods.
The other major focus for engineered cell medicines programs is treated as a treat cancer, we're developing an engineered alpha beta T cell medicines for cancer in collaboration with Bristol Myers Squibb.
The leader in treating blood cancers, where we believe alpha beta T cell medicines other potential to be particularly effective.
In addition, we are advancing our wholly owned programs by editing innate immune cells, including NK and gamma Delta T cell to treat solid tumors.
We plan to begin I'd, enabling studies mid year for edited healthy donor NK cell medicine to treat solid tumors.
We recently announced worked with sandhill therapeutics to accelerate are healthy donor medicines.
Sandhill brings established processes manufacturing infrastructure and proprietary expansion nothing.
In parallel to healthy donor program, we are advancing engineered I.P.S.C. derived NK cells or I NK cells as medicines for solid tumors in partnership with Blue Rock Therapeutics now part of bar, we've made great progress editing a differentiating IPO see self perform I NK cells.
Combining the technologies of IPO season, CRISPR gene editing brings together two platforms that can revolutionize engineered cell therapies.
Michelle Robertson: In parallel with the Healthy Donor Program, we are advancing engineered iPSC-derived NK cells, or INK cells, as medicines for solid tumors. In partnership with Blue Rock Therapeutics, now part of Bayer, we've made great progress editing and differentiating iPSC cells to form INK cells. Combining the technologies of IOTSCs and CRISPR gene editing brings together two platforms that can revolutionize engineered cells. We're excited about this potential and look forward to updating you on our progress in the near future. Now I'll turn the call over to our newly appointed Chief Financial Officer, Michelle Robertson.
We're excited about this potential and look forward to updating you on our progress in the near future.
Now I'll turn the call over to our newly appointed Chief Financial Officer Michel Robertson.
Thanks, Charlie.
I guess to join you all today introduce myself and presents a company latest financials it though.
I've been working in finance in the biotech industry for more than 25 years. Most recently as the CFO hooman pharmaceutical and prior to that and a number of leadership a little Genzyme Walter environment.
For me what differentiated has dropped from other companies infinite possibility develop medicine, we are working on how patient I'm hard pressed to think of another company. It is much potential that Uh huh developed truly transformative medicine for patients with the season of unmet need and this will be opportunity to make a difference in the future of the.
Michelle Robertson: I'm pleased to join you all today to introduce myself and present the company's latest financial results. I've been working in finance in the biotech industry for more than 25 years, most recently as the CFO of Omento Pharmaceuticals, and prior to that, in a number of leadership roles at Genzyme, StatsAltar, and Ironman. For me, what differentiated Editas from other companies was the infinite possibilities to develop medicines that we are working on to help patients. I'm hard-pressed to think of another company with as much potential as Editas to develop truly transformative medicines for patients with diseases of unmet need.
He was a big drawer.
Now turning to the number we've summarized our financial results for the fourth quarter and full year 2019 in the press release that we issued earlier today.
Our cash cash equivalent in marketable securities increased $88 million in 2000 $19 million to $457 million as of December 31st 2019, some $369 million as of December 31st 2018.
Our uses of cash <unk> hundred $24 million include cash operating expenses.
Let me $10 million and capital expenditures 6 million dollar.
Michelle Robertson: In this role, the opportunity to make a difference in the future of the company was a big draw. Now turning to the numbers, we have summarized our financial results for the fourth quarter and full year of 2019 in the press release that we issued earlier today. Our cash, cash equivalents, and marketable securities increased $88 million in 2019 to $457 million as of December 31, 2019, and $369 million as of December 31, 2018. Our uses of cash total $124 million and include cash operating expenses of $118 million and capital expenditures of $6 million. Over the course of the year, we grew the size of our organization by approximately 48%, increasing to 195 full-time employees from 132 at the end of 2018.
Over the course of a year, we grew the size of our organization by approximately 48% increasing 295 full time employee.
<unk> 32 at the end of 2018.
The growth in our spending in 2019 expansion maturation of the pipeline and advancement of our platform. We expect these to continues to be the primary driver spending world in 2020.
Our sources of cash in 2019 totaled $212 million consisted primarily of $160 million raised equity instruments $76 million milestone payments from our business development partner and $15 million proceeds from stock option exercises.
I was talking to the strong financial condition at least 24 month runway funds.
And with that I'll hand, it back.
Thank you Michelle we're confident in our strong leadership to guide the company as we become a clinical stage biotech and embark on our next phase of growth.
It's been a busy past few months for at a time and an exciting time as we look toward the coming here.
Michelle Robertson: The growth in our spending in 2019 was due to the expansion and maturation of the pipeline and advancement of our platform. We expect these to continue to be the primary driver of spending growth in 2020. Our sources of cash in 2019 totaled $212 million and consisted primarily of $116 million raised through equity, $76 million in milestone payments from our business development partners, and $15 million in proceeds from stock option exercises.
We have filled out our executive team and our confidence to strengthen leadership will support the long term growth of the organization our best in class programs, coupled with the unparalleled discovery research from our lab represent a pipeline of transformational medicines for diseases of unmet need we are eager to.
To see what 2020 M. beyond will hold as we look to deliver on the promise of CRISPR to transform patients' lives. We thank all of you for your interest and support with that we will open up the call for today operator.
Thank you, ladies and gentlemen to ask questions, we need to press star one order telephone to withdraw your question. Please press the pound key please stand by all the composite can when a roster.
Michelle Robertson: All staff are in a strong financial position.
Michelle Robertson: And with that, I will hand it back to you.
Cindy Collins: Thank you, Michelle. We are confident in our strong leadership to guide the company as we become a clinical stage biotech and embark on our next phase of growth. It has been a busy past few months for Editas and an exciting time as we look toward the coming year. We have filled out our executive team and are confident that its strength and leadership will support the long-term growth of the organization. Our best-in-class programs, coupled with the unparalleled discovery research from our labs, represent a pipeline of transformational medicines for diseases of unmet need. We are eager to see what 2020 and beyond will hold as we look to deliver on the promise of CRISPR to transform patients' lives. We thank all of you for your interest and support. With that, we will open up the call for Q&A. Operator?
And our first question comes from a line of Steve's seed House with Raymond James Your line is now.
Hi, good thank you.
My question is ash. So the data that was presented for editorial one it's a nice day to send you included sort of like the integrations on optimizing the protocol, whether it was the enzyme varian or the guide or the complication conditions and electroporation conditions as well. So there's basically there's a lot of moving pieces there and.
I wanted to understand if we set aside the factor you guys are targeting a different locus than some of the first movers in the gene editing approaches I'm curious if you could characterize to what extent you believe the other optimize conditions. So like the electroporation into R&D complex section.
Our novel or are an improvement over what's already been done or does the best in class pitch for editorial one just boil down basically to the different locus that you're targeting.
Operator: Thank you. And ladies and gentlemen, to ask a question, you will need to
This is Charlie the up the best in class comes from a couple of things. One we believe we have superior levels of fetal hemoglobin induction and secondly, we believe we don't carry or the potential bag as it comes with adding that the feel of an answer as you. Appreciate you can't knockout dceleven I because that causes dire consequences and we in our preclinical models.
Operator: To withdraw your question, please press the pound key. Please stand by while we compile the Q&A roster. And our first question comes from the line of Steve Seedhouse with Raymond James. Your line is now open.
Steven James Seedhouse: Hi, good afternoon. Thank you. My question is, Ash, so the data that was presented for EDIT301, it's a nice data set, and you included sort of like the iterations on optimizing the protocol, whether it was the enzyme variant or the guide or the complexation conditions and electroporation conditions as well. So basically there's a lot of moving pieces there, and I wanted to understand, if you set aside the fact that you guys are targeting a different locus than some of the first movers in the gene editing approaches, I'm curious if you could characterize to what extent you believe the other optimized conditions, so like the electroporation and the RNP complexation, are novel or are an improvement over what's already been done, or does the best-in-class Pitch for Edit 301 just boiled down basically to the different locus that you're targeting.
On.
Issues with even the these 11 I enhancer. So we've gone out after a site in front of hemoglobin locus. We know is genetically validated and and we scan the entire hemoglobin locus and we found size. So we've talked to get the most fetal hemoglobin induction then we look for the enzyme combinations would do that so as you appreciate there.
Both productive and nonproductive edits anytime you could we found a cutting at the site. We did with cast 12, a yield on a lot more productive at its than did cutting with casnine.
But to make that work as a product we needed to optimize cutting Cas twelvei and we did that as you mentioned with a variety of a methods. We we have oh enzyme of the various accounts 12 day that has increased activity. We have variations of the guy that increase the ability to do the editing and we've optimized electroporation can.
Additionally, in so all of those things went into building the building the product.
Okay. Thanks that helps hopes just to understand the sequence of events in the thought process quite a bit.
[music].
I don't want also ask on at a 301 if.
Charlie Albright: Steve, it's Charlie. The best in class comes from a couple of things. One, we believe we have superior levels of fetal hemoglobin induction. And secondly, we believe we don't carry any of the potential baggage that comes with adding the BC11A enhancer. But, as you know, you can't knock out BC11A because that causes dire consequences.
The studies that remain ongoing or the gating factors to an eye Andy.
Comprise any primate studies and.
Is it.
No. It is it worth testing the beta globin targeting approach in prime minutes or our rodent model sufficient to sort of she differences in biology between that versus the first generation approaches.
Charlie Albright: And we, in our preclinical models, have found issues with even the BC11A enhancer. So we've gone at it after a site in front of the hemoglobin locus. We know it's genetically validated. And we scanned the entire hemoglobin locus, and we found sites that we thought could get the most fetal hemoglobin induction.
We don't have any ongoing primate studies, so do the primate studies, you actually have to redesign the entire product.
Because it has to be able to cut the primates you know.
The it's not clear yet with the primate studies are going to be more predictive and the road and studies or not and so at this point, we feel like the thing to do as they get into the clinic as quickly as possible. It because it's really the clinical data that actually determines the course with all of these with all these.
Charlie Albright: And we looked for the enzyme combinations that would do that. So, as you can see, there are both productive and nonproductive edits any time you cut. And we found that cutting at the site we did with Cas12a yielded a lot more productive edits than did cutting with Cas12a. But to make that work as a product, we needed to optimize cutting with Cas12a, and we did that, as you mentioned, with a variety of methods. We have an enzyme, a variant of Cas12a that has increased activity. We have variations of the guide that increase the ability to do the editing, and we've optimized the electroporation conditions. And so all of those things went into building the product.
This class of medicines.
Okay. Thank you and just last quick one it just looks like operating expenses went up considerably in the quarter.
Just curious if you could shed some light on how that breaks out and what would carry forward into next year on a go forward basis.
Thanks for taking the questions.
I'm sure [laughter] freedoms advance will continue to invest especially as they go into the clinic and we expect that to continue in 21.
Thank you.
And our next question comes from a line of Amanda Murphy with BTI G. Your line is now open.
Hey, good afternoon, that's what a few more questions around the oncology business and.
Charlie Albright: Okay, thanks. That helps. [inaudible] I wanted to also ask on edit 301 if the studies that remain ongoing are the gating factors for an IND, comprise any primate studies, and if not, is it worth testing the beta-globin targeting approach in primates or are rodent models sufficient to sort of see differences in biology between that versus the first generation approaches?
Wanted to start out with the the I guess now BMS relationship you kind of specifically talked about allogeneic cells and I'm not sure. What you consider there in terms of how that's progressing but just curious overall you know.
There's obviously a lot of competition there you've been working on that you know for a number of years. They clearly re upped with you are not interested in the program.
Charlie Albright: We don't have any ongoing primate studies. So to do the primate studies, you actually have to redesign the entire product, because I have to be able to cut the primate genome. It's not clear yet whether the primate studies are going to be more predictive than the rodent studies are going to be. And so at this point, we feel like the thing to do is to get into the clinic as quickly as possible because it's going to be the clinical data that actually determines the course for all of these, with all these, this class of medicines.
So just wondering.
But you're well your thoughts there on on on either there's a ton of data coming this year, how that's progressing there for braskem and I want to shift after that on to all the.
Yes, there in house programs that your that you're working on but let's start with the alpha beta if we can't fairness.
Okay.
Charlie the yeah. We did we did re up with CMS, we they are the leaders and.
Michelle Robertson: Okay, thank you. And just last quick one, it just looks like operating expenses went up considerably in the quarter. I was just curious if you could shed some light on how that breaks out and what would carry forward into next year on a going forward basis. Thanks for taking the questions.
And so base medicines for hematologic indications that I think there was a good chance that their cdnineteena VCM I programs are going to be Indra industry, leading.
We can't sure the details of what's going on there we can say in in a more generic way that that.
Michelle Robertson: Sure, as the programs advance, we'll continue to invest, especially as we go into the clinic, and we expect that to continue in 2020.
I think that from a lay perspective, they're going to want to back up those programs and maintain their leadership and so we've disclosed a lot of data through the years about the types of that it's we've been able to make and T cells and.
I think it's safe to assume they're going to want to do some of those some of those same targets, but we're not at liberty to disclose what the detail products or.
Amanda Murphy: Thank you. And our next question comes from the line of Amanda Murphy with BTIG. Your line is now open.
Amanda Murphy: Good afternoon. I just had a few more questions about the oncology business. I wanted to start out with the, I guess, now BMS relationship. You kind of specifically talked about allogeneic cells, and I'm not sure what you can share there in terms of how that's progressing. There's obviously a lot of competition there, you've been working on that, you know, for a number of years, but they clearly re-upped their view and are interested in the program. So just wondering what your thoughts are on, on, on, you know, there's a ton of data coming this year about how this program might progress, and I want to shift after that on to all the in-house programs that you're working on, but let's start with alpha and beta, if we can.
Yeah fair enough.
But then I mean, then obviously you've been building out quite as.
Well you know sizeable portfolio when you when you think about what you've done with but NK cells in the sands I'll now with expansion and I am I understanding as that's kind of one is the key.
[noise] challenges and using NK cells as expands in technology and so you're talking about you know obviously working with Townhomes Iraq.
And now and then I think it's part of the BMS rise agreement you got back Cam adults itself.
So would love, it's kind of get a high level view of how you're thinking about.
Do you sort of a neat effector cells as backbone you talked about solid tumor as a target but.
Just looking at the back from me for me high level pipeline perspective, you know how do you see this evolving over time and and and and what else. Do you think is interesting I think T. Regs. You've also been discussed I, it's not a company et cetera.
Charlie Albright: Okay, I'm Anna Charlie the Yeah, we did. We did re up with PMS.
Charlie Albright: We they are the leaders in Cell-based medicines for hematologic indications, and I think there's a good chance that their CD19 and BCMI programs are going to be industry-leading. We can't share the details of what's going on there. We can say in a more generic way that, I think that from a lay perspective they're going to want to back up those programs and maintain their leadership and so we've disclosed a lot of data through the years about the types of edits we've been able to make and T-cells and and I think it's safe to assume they're going to want to use some of those some of those same targets but we're not at liberty to disclose what the detailed products are.
So does it gets a perspective there.
Yes, it will take a stab at that so we like the innate immune system and so we we feel like NK cells or the place to start and I'll come back to gamma Delta in a minute. So we know that NK cells are part of antibody directed salyer cytotoxicity or ADCC, which is part of the mechanism of your therapeutic antibodies things such as.
Her something an early talk some other among others.
And.
And we also know that NK cells have a very low propensity for graft versus host so when it comes to making truly allogeneic status and say they come already in a good place. We further knows that the path to making IPO see derived in case is is reasonably well worked out at this point. So all those things we've taken together.
Charlie Albright: Yeah, fair enough. And then, obviously, you've been building out quite a sizable portfolio when you think about what you've done with NK cells and the Sandhill now with expansion, and I, my understanding is that's kind of one of the key, so we'd love to kind of get a high-level view of how you're thinking about it.
And ER and our leading us to go after the major unmet need which is solid tumors.
We think that the on the and NK cells. They are exhausted is the T cells are exhausted in any of the solid tumor indications as well and there are things that we can do about that via gene editing.
And so among other things we want to do is is increased sensitivity I'll 15.
Charlie Albright: And we also know that NK cells have a very low propensity for graft versus host. So when it comes to making truly allogeneic medicines, they are already in a good place. And we further know that the path to making iPSC-derived NKs is reasonably well worked out at this point. So all those things we've taken together are leading us to go after the biggest part of that need, which is solid timber. We think that the NK cells are exhausted as are the T cells.
Targeting like the city 16 pathway better recover the tumor microenvironment and make them truly allogeneic. So all those things are possible gene editing. The number those you can do is limited with healthy donor and therein lies the the advantage of the IPO see platform not only do they easily.
Making the cells ultimately become a much better but to your ability to construct highly engineered medicines, which we think are gonna be needed to be successful in this space or enable so we are in the process of industrializing the platform, which was a combination of editing platform. We built over the last five years with an IPO CP.
Charlie Albright: http://www.thevenusproject.com And so among the things we want to do is increase sensitivity to IL-15, add targeting, make the CD16 pathway better, overcome the tumor microenvironment, and make them truly allogeneic. So all those things are possible with gene editing. But the number of those you can do is limited with Healthy Donor.
Platform that we've got a jump start with probably getting cell lines from Bluerock therapeutics.
Does that get you where you want to go yeah, I'd now like way too many questions in there, but I think also just talking about expansion that seems to be.
Charlie Albright: And therein lies the advantage of the IPSC platform. Not only does the ease of making the cells ultimately become much better, but your ability to construct highly engineered medicines, which we think are going to be needed to be successful in this space, is enabled. So we are in the process of industrializing the platform, which is a combination of the editing platform we've built over the last five years with an IPSC platform that we've got to jumpstart with by getting cell lines from Blue Rock Therapeutics.
It's something that is a challenge with using NK cells. So wanted to get a little more perspective, there and then and then also gamma Delta.
Yeah. So that's part of what we get for that it's a more of an issue for healthy donor than it is for IPO see derived cells, where you can just grow a whole lot. Yeah. So that's part of what sand Hill brings to the play for us So and that's kind of help enable our healthy donor program, which will begin to let us learn about what these different.
That's due in the context of solid tumor.
That's fair doesn't live game itself.
Yeah, Sorry go ahead I'm sorry.
But give him a delta is another part of the innate immune system. Obviously, it's less the biology of game adult themselves is less well worked out and NK cells are but the but they remain an interesting area and among the reasons are interesting is.
Charlie Albright: Yeah, I think I asked like way too many questions in there, but I also just...
Transcription Error: Transcribed by https://otter.ai
Charlie Albright: Yeah, so that's part of what we get for that. It's more of an issue for healthy donors than it is for iPSC-derived cells, where you can just grow a whole lot.
Closely.
Relationship between the number again with Delta cells, you see in your solid tumor and the control that tumor or among the best things that are correlated in the control of solid tumor growth. So it's a it's an interesting area. We're glad to have the ability to now work in that area and you'll hear more about that in the in the coming months.
Charlie Albright: And so that's part of what Sandhill brings to the plate for us. So, and that's.
Charlie Albright: And that's going to help enable our Healthy Donor Program, which will be again to let us learn about what these different edits do in the context of solving problems.
Okay fair to say youre using different approaches to really tackle.
Charlie Albright: And then Gamma Tau. Yeah, sorry. Go ahead.
Salads humor.
Using edited engineered.
Charlie Albright: Gamma Delta is another part of the innate immune system; obviously, it's less, the biology of Gamma Delta cells is less well worked out than NK cells are, but they remain an interesting area, and among the reasons they're interesting is because the relationship between the number of Gamma Delta cells you see in your solid tumor and the control of that tumor is among the best things that are correlated with the control of solid tumor growth. So it's an interesting area; we're glad to have the ability to now work in that area, and you'll hear more about that in the coming months.
In fact yourself so that it doesn't have anything about it as as a strategic yeah in Korea, Yeah, Yeah, and we're trying to bring them I would further say, we're trying to take them. The natural properties of NK cells, and and then make them every store them and make them better and so NK cells as I told to participate and antibody directed sellers.
Why the talks with theory VCC. They also recognized cells are lacking M. A C.
And so therefore, thereby don't express T cell antigens and so one of the major resistance mechanisms to PD. One inhibitors is the law so T cell antigen expression.
Amanda Murphy: It's fair to say you're using different approaches to really tackle Solid Tumor Using Edited Engineered,...
If you had a therapeutic that could specifically target those cells you'd have a nice add on to PD one.
Charlie Albright: Yeah.
Charlie Albright: Yeah, and we're trying to, and I would further say we're trying to take the natural properties of NK cells and make them, and restore them, and make them better. And so, NK cells, as I told you, participate in antibody-directed cellular cytotoxicity or ADCC. They also recognize cells that are lacking MHC. Hence, if you had a therapeutic that could specifically target those cells, you'd have a nice add-on to PD-1.
Yep, Okay. Thank you so much.
Thank you.
And our next question comes from the line of Matthew Harrison with Morgan Stanley. Your line is now.
Hi, guys. This is Tom is slated for your from Matthews team I've a question about and it went out to what does your partner Allegan need to decide on next steps that they don't opt in what would be your plans to pursue a trial alone. Thank you.
Amanda Murphy: Okay, thank you so much.
Sure.
Gregory Allen Harrison: And our next question comes from the line of Matthew Harrison with Morgan Stanley. Your line is now open. Hi guys, this is Thomas Lavery from Matthew's team. I have a question about Edit 102. When does your partner, Allegan, need to decide on next steps? And if they don't opt in, what would your plans be to pursue a trial alone? Thank you.
So the way the the way the deal is structured is the.
We agree on the targets in the option criteria with our again before we started a program and then we would go away we developed a medicine that fulfills. The criteria then we deliver that option package essentially it is what would be rude known as a development candidates stage. So it's ready for I'd, enabling Tox study and then our again.
Charlie Albright: Sure. So the way the deal is structured is that we agree on the targets and the option criteria with Allergan before we start a program, and then we go away, we develop the medicine that fulfills the criteria, and then we deliver that option package, essentially at what would be known as a development candidate stage, so it's ready for an IND-enabling toxic study. And then Allergan has a period of time to evaluate that package and make a decision about whether they will opt-in or not opt-in. And then after that, we have a window to decide whether we co-opt-in with them to co-develop the product with them. We did that for LCA-10. And then, if they decide not to opt-in, the medicine comes back to us, and we have the right to develop it on our own.
And has a period of time to evaluate that package.
And make a decision about where they often are not often.
And then after that we'd have a window to decide whether we co often with them to co develop in the.
The product with them, we did that for LCD 10.
And then and if they decide not to opt in the no medicine comes back, though so we have the right to developing on or.
Thank you.
Thank you.
Our next question comes from amount of Gena Wang with Barclays. Your line is open.
[laughter].
Hi, Thanks for taking my question that this June four Gina. So my first question. So we'd have followed the LSTA 10 could you give us a first of all some guidance about when the clinical data will be released.
That's first and Dallas, So I can't give us the cotter if all the differential fiorina showed dose you wouldn't pay.
And also a while to conduct a biomarker she would be looking for data will help you to decide whether you're going to move.
Huidong Wang: Thank you. Our next question comes from the line of Jenna Wang with Barclays. Your line is now open.
With the higher dose I have a follow up after day. Thank you.
Okay. Thank you for the question so I'll start with it the first part of it necessarily to talk a little bit more about biomarkers and Dennis range, but the overall trial is going very well Oh, we are actively screening patients and.
Huidong Wang: Hi, thank you for taking my question. This is Jun for Gina.
Huidong Wang: So my first question is really about the LCA10. Could you give us, first of all, some guidance about when the clinical data will be released? That's first. And then also, can you give us a little bit of information about the range of your initial dose you would take? And also, what kind of biomarkers would you be looking for that will help you to decide whether you're going to move on to the higher dose? I will have a follow-up after this. Thank you.
And have not disclose when we will announce data at this point in time as I mentioned earlier, we expect to announce dosing of the first patient this quarter, but we have not made any commitment around when we might think about I'm sharing data tell anyone else.
Sure. So we pick those is based on our preclinical studies say the low dose with low me that at a high dose study or are they spend the dose to choose by spark and the let's turn to trials was found to be epic safe and efficacious.
Charlie Albright: Okay, thank you for the question. So I'll start with the first part of it and ask Charlie to talk a little bit more about biomarkers and dose range. But the overall trial is going very well. We are actively screening patients, and And have not disclosed when we will announce data at this point in time. As I mentioned earlier, we expect to announce the dosing of the first patient this quarter, but we have not made any commitments about when we might think about sharing data. Charlie, do you want to?
The and it's a relatively narrow dose range. The are the lowest dose has a realistic chance of showing efficacy based on the preclinical studies and you have to do that for a a gene therapy trial.
The we're looking at a range of clinical outcome some of them or clinical there they're visual acuity.
As well as electrophysiological structural markers, including a year or G.
Charlie Albright: Sure. And so we pick doses based on our preclinical studies. The low dose, we have a low, mid, and a high dose. They span the dose that's used by SPARC and the Lexterna trials. It was found to be extremely safe and efficacious.
And Oh Sweetie.
We'll take all that they live into consideration as well as the safety and as we decide how to advance the program.
Great just hold on bad the how long do general going away. After you don't feel Georgia, George will always Filipino patient before you think that Youre happy enough information to decide whether to move onto what they are higher dose. What's the time, we can time between the two doses.
Charlie Albright: It's a relatively narrow dose range; the lowest dose has a realistic chance of showing efficacy based on preclinical studies, and you have to do that for a gene therapy trial. We're looking at a range of clinical outcomes. Some of them are clinical, they're visual acuity, as well as electrophysiologic and structural markers, including ERG and OCT. And we'll take all that data into consideration as well as safety as we decide how to advance the program.
[laughter]. So we have a safety review at the conclusion of Oh cohort one.
And Oh that review period can be a as I said.
Huidong Wang: Great. Just to follow on that, how long are you generally going to wait after you dose your lowest dose in your patient before you think that you will have enough information to decide whether to move on to the higher dose? What's the waiting time between the two doses?
Program to be approximately six cents in duration.
Okay. So we'll have another crushing about the sequence. So disease, obviously, you have to you'll preclinical data flash and the only so you'd operate that about they advantages you have so just a four I'm you know what kind of give myself believe benchmark understanding what level off I did not.
Charlie Albright: So we will have a safety review at the conclusion of Cohort 1.
Charlie Albright: And that review period can be a program to be approximately six weeks in duration.
Huidong Wang: Okay. So I have another question about sickle cell disease. Obviously, you have your preclinical data from ASH, and you also elaborated about the advantages you have. So just for, you know, kind of giving myself a benchmark understanding, what level of editing efficacy do you think would make you confident your program would be better, or like, you know, based on class, as you suggested? What percentage of gene editing are you looking for to move forward?
If I could say you would think will be something I get you a continent June jewel profound would be better or like you know best in class as you suggested what about the person chip a gene editing you are looking for two more forward.
I think it's not so much the gene editing insist fetal hemoglobin and documents were obviously going to optimize the the percent editing based on the.
Charlie Albright: I think it's not so much gene editing as fetal hemoglobin induction. So we're obviously going to optimize the percent editing based on the technical features. But really, what we're looking for are levels of fetal hemoglobin, and then that would be Predictive of Clinical Outcomes.
On the technical features of but really what we're looking for our levels with fetal hemoglobin and then that would be.
Predictive clinical outcomes.
Thank you.
And our next question comes from the line of Joe tone with Cowen and company. Your line is no.
Hi, there and thanks for taking my questions on the that are one on one study.
Joe Tong: Thank you. And our next question comes from the line of Joe Tong with Cowen and Company. Your line is now open. Hi there, and thank you for taking my questions.
Can you just let US know if has the first patient surgery and scheduled and maybe what what needs to happen before that patient is dosed.
And then looking for it kind of towards the end of the year do you still expect that you will have dose.
Joe Tong: On the EDA-101 study, can you just let us know when the first patient's surgery has been scheduled, and maybe what needs to happen before that patient is dosed? And then looking forward, kind of towards the end of the year, do you still expect that you'll have dosed the first two cohorts of patients in that study?
The first two cohorts of patients in that study.
By year end.
Yeah. Thank you for the question. So as I mentioned the trial is going very well we are actively screening patients as you heard from US previously the screening and enrollment has been a little bit more complicated than other types of trials just getting patients.
Cindy Collins: Thank you for the question. So, as I mentioned, the trial is going very well. We are actively screening patients. As you heard from us previously, the screening and enrollment has been a little bit more complicated than other types of trials, just getting patients lined up with family members, caregivers, things like that. But we are in daily contact with our partner Allergan and working very collaboratively to
Wind up with family members caregivers things like that but we are in daily contact with our partner allergy and working very collaboratively to it to continue to screen. The patients I feel very good about where we are we had said earlier.
Or that we.
Cindy Collins: And so I feel very good about where we are. We had said earlier that we do hope to treat both the first two cohorts by year end, and we're still tracking toward that.
Do you Oh to treat both the first two cohorts by year end and we're still tracking supports that.
Okay, Great and then and then will more kind of just on on more broad strategy.
Joe Tong: Okay, great. And then one more kind of just on a more broad strategy. You know, obviously, you have a lot of partnered and collaborative programs. When you're looking at going into a new therapeutic area, I guess how important is it to have a partner that you can, you know, kind of lend their expertise to and combine your strengths versus deciding to do a program alone?
Obviously, you have a lot of partnered and collaborative programs when you're looking at going into a new therapeutic area. I guess, how important is it to have a partner that you can kind of lend on their expertise in combined or strength versus defined drill program. Although now.
Yeah, we've taken its obviously a each one in isolation and and so we had partners such as allergy and where are we you'll have the extreme where we are co developing and commercializing a potential therapies. The partnership now with BMS Farley Celgenes you know.
Cindy Collins: We've taken those, obviously, each one in isolation, and so we have partners such as Allergan where we, you know, have the extreme where we are co-developing and co-commercializing potential therapies. The partnership now with BMS, formerly Celgene Juno, was a little bit different in that it was a development collaboration, but our role was [inaudible] And to some degree, Ask Bio, those were really purposeful in terms of getting access to either technology or capabilities that we thought were important to facilitate getting the programs up and running and going much more quickly.
With a little bit different end that it was a developing collaboration but our role as.
Unique they are we weren't compelling product per se with some of them. Our recent deals that we've done sand Hill and Blue rock and to some degree asked bio those were really purposeful in terms of getting access to either technology or.
Capabilities that we've thought were important to facilitate.
Getting the programs up and running and going much more quickly.
Great. Thank you so much and graphic on the progress.
Joe Tong: Great, thank you so much, and congrats again on the progress.
Joe Tong: Thank you.
Thank you.
Unknown Attendee: Thank you. And our next question comes from the line of Zsuzsanna Lipschitz with Chardon. Your line is now open.
Thank you and our next question comes from a line of journalism Lipschitz with Chardan. Your line is an open.
Unknown Attendee: Good afternoon, guys, and thanks for taking my questions. So to follow up on some of the prior questions on Edit 101, I think that in the third quarter results, it was mentioned that a patient had been identified. So can you speak to, essentially, some of the factors around the timing of dosing, and is that same patient the one that's still expected to be dosed?
Hi, Good afternoon, guys and thanks for taking my questions. So here's how often.
This is higher.
And it went on one so I think I first quarter results. It was mentioned dedication had been identified.
[laughter]. Unlike some other factors around the timing of dosing is that <unk> is that same patients. The one that's still expected to be dose.
And then I have a color.
Cindy Collins: and Then I have a couple of more.
So I I'm not sure I recall, specifically the the statement and about first patients being identified a per se, but we are as I said actively screening patients we have identified a patients.
Cindy Collins: So, I'm not sure I recall specifically the statement about first patients being identified per se, but we are, as I said, actively screening patients. We have identified patients for the first cohort and are working even, you know, towards the other cohorts. So, the best I can say at this point is that we do expect to announce dosing of the first patients this quarter.
For the first cohort and are working even you know towards via the other cohorts I'm. So that's I can say at this point is that we do expect to announce a dose thing I first patient this quarter.
Great.
Unknown Attendee: And then Actually, to follow up on some of the questions regarding the oncology program, can you elaborate a little bit more on the Sandhill collaboration and what drew you to work with that company? And then I have a follow-up question on that.
And then.
And then actually it's a follow up on some of my questions regarding young allergy programs can you elaborate a little bit more on the Santos collaboration and wait for you to.
With that company that I've a follow up on.
Sure.
Charlie Albright: Sure. Van Hill has developed a technology they call Binate, which allows the efficient expansion of NK cells, which was, as referred to earlier, a significant issue in the field. So that's the primary driver of that. It's a group that has experience in developing cell-based medicines as part of earlier companies, and we felt like they could be a good partner in the NK field because of the technology they developed there.
Said hills developed a technology, they called <unk>, which allows ER.
Fishing the expansion of NK cells, which is it was as referred to earlier significant issuing that feels so that that's the primary driver of that it's a group that has experienced in developing cell based medicines as part of a as part of earlier companies and we felt like they could be a good partner in the.
Okay.
Field or because of the technology they developed there.
Charlie Albright: And is that tech also applicable to the Gamma Delta cells, or is that primarily for the NK program?
Yeah, Hi Tech also for a couple to the gamut delta cells or is that.
Thank you.
Are there are elements of that may be applicable to gamma delta, but the primary uses a short run is for NK cells.
Charlie Albright: There are elements that may be applicable to Gamma Delta, but the primary use in the short run is for NK cells.
Charlie Albright: And I think at some point, you previously had a collaboration with GAMI to sell, I think, NK technology. So, to your mind, is that something that's still active, or is this Sandhill collaboration supersedes that, or do both approaches come into play for a donor drive program?
And then I think at some point you previously had a collaboration with Damnedest now I think around and key technologies. So you have minus is that something that's still active or is this the santo collaboration supersede that or do you put their purchase come into play for us owner I program.
Yeah, those were independent a collaboration than we had we had an M.T.J. with can either so.
Charlie Albright: Yeah, those were independent collaborations, and we had an MTA with Camille Dessau.
Charlie Albright: Got it. Got it. I think that's it for me. Thanks.
Got it right.
That's it for me thanks.
Sylvan Twerken: Thank you. And our next question comes from the line of Sylvan Twerken with Oppenheimer. Your line is now open.
Thank you.
Our next question comes from a line of so Vince working.
With Oppenheimer. Your line is an open.
Sylvan Twerken: Well, congratulations on the quarter, and thank you for taking my question. My first question is again about the LCA-10 data when we eventually get it, probably towards the end of the year.
Thank God congrats on the quarter and thank you for taking my question. My first question is if I get about Delphine, a 10 data when we eventually got.
Maybe towards the end of a year, how comparable or what can we compare across to the phase one two clinical trial for.
Sylvan Twerken: How comparable, or what can we compare across the phase one, two clinical trials by ProQR? Just in terms of are they similar patients, is it not? What's comparable, what's not?
Procure.
Just to jump or is it similar patients is that not so what's comparable what's not.
There was some color there.
Charlie Albright: ..
Sylvan Twerken: To comment on data, I do anticipate that there's the potential to have some data to share by the end of the year. That's been our previous guidance. I'll ask Charlie to comment on the ProQR portion of the question.
[laughter] ER to comment on data, Okay, Yeah, I do anticipate but there's the potential to have yeah. Some data to share by the end of the year. That's that's than our previous guidance I'll ask Charlie to comment on a pro QR a portion of the question.
Charlie Albright: The patients are relatively comparable. That's the short answer to your question. There are not huge differences. I wouldn't know if there are detailed differences, but they're in the same ballpark.
Oh, it's just the patients are relatively comparable that's the short answer your question, there's not huge differences.
I wouldn't know if they're detail differences, but the them the same ballpark.
Sylvan Twerken: Great, thanks. And maybe one question for Michelle. Thank you for taking on the role of CFO. Could you maybe help us understand how you view the different programs in eye, oncology, and CNS now and Sickle Cell in terms of maybe the dimension of risk versus payoff versus how you would allocate assets to them?
Great. Thanks, and maybe one more question for Michelle.
Taking the on the roll up this year could you maybe help us understand how how you view the different programs and I I'll call. It.
Yes now.
And sickle cell in it jumps off maybe on the dimensional risk versus pay off versus how you would allocate assets towards them.
[noise] I'm sure.
Michelle Robertson: I'm sure. I'm still getting up to speed on the portfolio, but I think that we're comfortable with the two pillars that we've talked about publicly at J.P. Morgan and about the programs and the relationships that we have with our partners. As we think about financing and supporting the portfolio, I believe that prioritization is going to be sort of key for the next 12 to 24 months because we have such a good pipeline and that we're going to have to, I think, focus on the hybrid.
Hi, I'm still AG getting and the does not on the portfolio, but I think that we're comfortable with.
The two pillars that we've talked about publicly at JP Morgan.
And if the programs and not the relationships that we have with our partners I think that what we'll continue to do is assessed the portfolio as a whole Ah. So that we can prioritize programs are in our investment and Ah in map out you know our war.
Our timeline you know any events.
And you know as we think about financing and supporting portfolio I believe that prioritization, it's gonna be so the key.
For the next 12 to 24 months I'm because the we have such a a good pipeline and that we're gonna have to I think focus in I'm not at the high value programs are working closely with our partners.
Sylvan Twerken: Great. Thank you so much. And maybe one last question here on this AskBio CNS partnership: What are the kind of indications that you're going after in the long term?
Great. Thank you for much of it maybe one last question.
Ask <unk> CMS partnership.
What are kind of be.
Indications that are you going after a in the long term.
Sylvan Twerken: We have not yet disclosed those, but the indication that we're pursuing with them.
And we have not yet disclosed that.
The indication that were pursuing with them.
Sylvan Twerken: Okay, great. Thanks for taking my questions.
Okay, great. Thanks, Thanks for taking my question.
Sylvan Twerken: Thank you.
Thank you.
Yanan Zhu: Thank you. And as a reminder, to ask a question, you need to press star one on your telephone. And our next question comes from the line of Yanan Zhu with Wells Fargo Securities. Your line is now open.
Okay.
Thank you and as a reminder to ask a question you need to press star one on your telephone.
And our next question comes from the line of NN issue with Wells Fargo Securities. Your line is open.
Yanan Zhu: Hi, thanks for taking the question. So, the first question is on EDIT 301.
Hi, Hi, thanks for taking the questions.
So first question is on added real one would you share or at some point share a preclinical data comparing a youre chemical olden promoter targeted approach versus the PC all 11, a enhancer approach and Oh, So just hypothetically speaking it.
Charlie Albright: Would you at some point share preclinical data comparing your gamma-globin promoter targeted approach versus the BCL11A enhancer approach? And also, just hypothetically speaking, is there anything to be gained if the two targeting approaches are combined? Or do you think the gamma-globin promoter approach achieves maximum HPF induction already? Thanks.
Sure anything could be gain the if the two targeting approaches a combined.
Or do you think the common closed then promoter approach achieve maximum H.P.F. induction already.
Charlie Albright: Sure, the answer to your first question is yes, at some point, we'll disclose the comparative data, but I'm not sure when that'll be. Combining them is an interesting question. I think there are some technical..., challenges of doing that, I guess. That wouldn't be our first. [inaudible]
Sure the answer to your first question is yes at some point, we'll disclose or the comparative data, but I'm not sure one that'll when that will be.
[laughter] combining them is an interesting question the I think theres some technical.
Challenges to doing that I guess.
The wouldn't be or furstenberg. This I guess you'd have to edit you'd have to do both editing events at the same time that'd be there'll be some technical challenges that come with it.
Yanan Zhu: Right, got it. The purpose of that question is mainly to see whether the true approach has, there are
Right.
Got it.
Just a yeah, that's because the [laughter]. The purpose of that question is mainly to see whether the to approach has.
There are <unk> or <unk>.
Transcription Error: The University of Georgia College of Agricultural and Environmental Sciences, 2015
Uh Huh H.P.H.B.S. induction that could be a you know much actually sit by independent three different mechanisms or perhaps the are you know working well in the same that pathway and you know one is better than the other but yeah I agree that [laughter] if not every.
Yanan Zhu: HBF inductions that could be achieved by independently different mechanisms, or perhaps they are working in the same pathway, and one is better than the other. But yeah, I agree that it's not a real proposal, but rather to understand the pathways. But maybe a quick question on the NK-Cell program. In terms of the kind of edits you're doing, I think you mentioned you're editing to increase the potency of NK-Cell killing. Would any edits to increase persistence also be something that you're considering? Or do you see this mainly as a multiple dosing approach, and therefore, persistence doesn't really come into the equation? Thank you.
Ill Ah proposal, but rather to understand the pathways, but maybe a.
Quick question on the NK cell program in terms of or the kind of thing kind of added Youre doing a I think you mentioned that you're adding to increase potency of NK cell, killing I would.
I just any I just to increase persistence also something that you're considering or do you see this mainly as a multiple dosing or approach and are therefore puts this kind of doesn't.
Really come into the equation. Thank you.
Charlie Albright: We will make edits to increase persistence, and multiple dosing still remains on the table as well.
So we will make yet as to increase persistency was even and multiple dosing still remains on the table as well.
Yanan Zhu: I see. Thank you very much.
I see thank you very much.
Cindy Collins: Thank you. And this concludes today's question and answer session. I would now like to turn the call back to CEO Cindy Collins for closing remarks.
Thank you.
And this concludes today's question and answer session I would now like to turn the call back to C O Cindy Collins for closing remarks.
Cindy Collins: Great, so with that, we thank you for participating in today's call and for your support as we work to bring transformative new medicines to patients. Have a great evening.
Great. So with that we thank you for participating in today's call and for your support as we work to bring transformative new medicines to patients have a great evening.
Ladies and gentlemen. This concludes today's conference call. Thank you for participating you may now disconnect [noise].
Operator: Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.
Transcription Error: The Bulletproof Executive, 2013
[noise].