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Edited Transcript of EDIT earnings conference call or presentation 12-Nov-19 1:00pm GMT

Q3 2019 Editas Medicine Inc Earnings Call

Cambridge Dec 2, 2019 (Thomson StreetEvents) -- Edited Transcript of Editas Medicine Inc earnings conference call or presentation Tuesday, November 12, 2019 at 1:00:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Charles Albright

Editas Medicine, Inc. - Executive VP & Chief Scientific Officer

* Cynthia L. Collins

Editas Medicine, Inc. - CEO, President & Director

* Eric Ek

Editas Medicine, Inc. - Interim CFO, Principal Accounting Officer & Principal Financial Officer

* Judith R. Abrams

Editas Medicine, Inc. - Chief Medical Officer

* Mark J. Mullikin

Editas Medicine, Inc. - Senior Director of Finance & IR

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Conference Call Participants

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* Amanda Louise Murphy

BTIG, LLC, Research Division - MD & Senior Biotechnology Equity Analyst

* Anvita Gupta

Guggenheim Securities, LLC, Research Division - Associate

* Geulah Livshits

Chardan Capital Markets, LLC, Research Division - Senior Research Analyst

* Joseph John-Charles Thome

Cowen and Company, LLC, Research Division - VP of Healthcare

* Konstantinos Biliouris

Morgan Stanley, Research Division - Research Associate

* Silvan Can Tuerkcan

Oppenheimer & Co. Inc., Research Division - Associate

* Timur Ivannikov

Raymond James & Associates, Inc., Research Division - Senior Research Associate

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Presentation

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Operator [1]

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Good morning and welcome to Editas Medicine's Third Quarter 2019 Conference Call.

(Operator Instructions) 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 Investor Relations at Editas Medicine.

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Mark J. Mullikin, Editas Medicine, Inc. - Senior Director of Finance & IR [2]

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Thank you, operator. Good morning, everyone, and welcome to our third quarter 2019 conference call. Earlier this morning, we issued 2 press releases that will be discussed on this call. The first announces an amendment to our long-standing collaboration with Celgene. The second press release provides our financial results and corporate updates for the third quarter of 2019.

A replay of today's call will be available on the Investors and Media section of our website approximately 2 hours after its completion. After our prepared remarks, we will open the call for Q&A.

As a reminder, various remarks that we make during this call about the company's future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the Risk Factors section of our most recent quarterly report on Form 10-Q, which is on file with the 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 date. 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.

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Cynthia L. Collins, Editas Medicine, Inc. - CEO, President & Director [3]

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Thank you, Mark. Good morning, and thank you, everyone, for joining us for our third quarter 2019 corporate update. In addition to Mark, I'm joined by several members of the Editas executive team, including Charlie Albright, our Chief Scientific Officer; Judith Abrams, our new Chief Medical Officer; and Eric Ek, our Interim Chief Financial Officer.

Editas has had a productive quarter, and we are excited to share some of the highlights with you today.

First and foremost, I am pleased to welcome Dr. Judith Abrams as our new Chief Medical Officer. Judith has tremendous experience and an outstanding reputation. She is a valued addition to our executive team and her arrival is ideal as we prepare to dose the first patient in the Brilliance Phase I/II clinical trial. Second, we announced this morning an amendment to our long-standing collaboration with Celgene to develop engineered T cell medicines for cancer. As you may recall, our original collaboration spanned the entire field of engineered T cells, the amended collaboration will focus on alpha-beta T cells, freeing up Editas to develop its own medicines using non alpha-beta T cells.

As a result of this amendment, we are entitled to receive a payment of $70 million, with the potential for future milestones and royalties on alpha-beta T cell medicines developed by Celgene using our technology.

Third, we are making solid progress with EDIT-301, our preclinical candidate to treat sickle cell disease and beta thalassemia. We look forward to sharing in vivo data at the upcoming ASH Meeting, demonstrating why we believe it has the potential to be a best-in-class medicine.

Fourth, we recently formed a new collaboration with AskBio, a leader in AAV gene therapy to develop in vivo CRISPR medicines to treat neurological diseases. This marks our second therapeutic focus for our in vivo CRISPR medicine portfolio in an area with high unmet need.

Finally, our ophthalmology portfolio is advancing with the first patient dosing of EDIT-101 for LCA10, anticipated by early 2020. With that, I'd like to turn the call over to Judith Abrams, our new Chief Medical Officer, to introduce herself.

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Judith R. Abrams, Editas Medicine, Inc. - Chief Medical Officer [4]

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Thanks, Cindy, for your warm introduction, and thank you all for being with us on the call today. Over the course of my career, spanning more than 25 years, I've had the opportunity to lead the development of medicines through all stages of clinical development across a number of therapeutic areas, including immunology and neurology. I'm thrilled to join Editas Medicine during this pivotal time and look forward to advancing EDIT-101 as well as additional experimental medicines in our pipeline through clinical development, including EDIT-301 for the treatment of sickle cell disease and future engineered cell medicines for the treatment of cancer.

Now let me turn the call over to our Chief Scientific Officer, Charlie Albright, to update you on our pipeline.

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [5]

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Thank you, Judith. As Cindy mentioned, we have amended our collaboration with Celgene to focus on developing autologous and allogeneic, engineered alpha-beta T cell medicines to treat cancer and autoimmune diseases. We've been working with our partners at Juno Therapeutics and now Celgene, since 2015, to develop engineered T cell medicines to treat cancer. As the original research term was set to expire in May of next year, we've extended and focused our collaboration to enable the advancement of medicines into the clinic. As a result of the amendment, we have regained rights to develop non alpha-beta T cells in all disease areas.

These rights were previously exclusive to Celgene for oncology. For example, we now have the right to pursue gamma delta T cells for oncology.

Gamma-delta T cells are part of the innate immune system, as are NK cells, where we have a significant [effort] using both donor-derived and iPSC-derived NK cells.

We believe that cells from the innate immune system, both complement alpha-beta T cells and increase the potential to treat solid tumors, an area of significant unmet need.

Moving on to our other area of focus in engineered cell medicines, we are developing EDIT-301 as a best-in-class medicine to treat sickle cell disease in beta thalassemia. EDIT-301 uses an engineered Cpf1 enzyme to edit hematopoietic stem cells at the beta-globin locus, we are confident in this program as other editing approaches target the BCL11A erythroid enhancer, which we believe may impact overall survival of the erythroid lineage.

At last year's American Society of Hematology Meeting, our work showing this effect was recognized as the best of ASH award. We will present additional data from our program at the upcoming ASH Meeting, describing our product configuration, in vivo efficacy data and off-target analysis. The totality of the data captures why we are so encouraged by this program and we think clinicians and patients will be as excited as well.

Switching gears to our in vivo CRISPR medicines' pipeline. In partnership with Allergan, we are developing a portfolio of gene-edited ophthalmology treatments for inherited retinal diseases. Our lead program EDIT-101 as a treatment for patients suffering from LCA10 and the Brilliance Phase I/II interventional study is underway to evaluate its safety, tolerability and efficacy.

The first potential patients that we anticipate participating in the trial has been successfully screened and confirmation of a surgery date by early 2020 is pending.

With that being said, drug product has been manufactured and is available for dosing. Multiple sites are recruiting, and we are actively engaged with clinicians to identify, screen and schedule patients for the initial cohort. Our ophthalmology portfolio also includes an experimental medicine -- treatment for Usher Syndrome Type 2A. To restore fully functional usherin protein and correct the disease, we knock out exon 13, which contains the most common disease-causing mutation. At the European Society of Gene and Cell Therapy meeting, we demonstrated a therapeutically relevant editing of up to 60% in human retinal explants. This data gives us confidence in our ability to treat the disease and supports further preclinical development of our lead candidate. The program draws heavily on the work we've done to research and develop EDIT-101, utilizing the same AAV vector, promoter and Cas9 enzyme, leveraging many of the same functional assays and models and following on the regulatory path paved by EDIT-101.

On ophthalmology, we have recently formed a collaboration with AskBio to develop in vivo CRISPR medicines to treat neurologic diseases. AskBio brings significant expertise in AAV development and manufacturing. With AskBio, we target cells in the peripheral and central nervous system, a well-known challenge for drug development. The combination of AskBio's expertise and knowledge in vector engineering and manufacturing, coupled with Editas' gene editing expertise could address a host of neurologic diseases with high unmet need. For example, we are excited about the potential for gene editing to treat debilitating pain in Huntington's disease.

We look forward to providing additional updates on our programs on the next -- in the near future as Editas works to translate gene editing into transformative treatments for patients with serious diseases.

With that, I'd like to turn the call over to Eric, who will provide a financial update.

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Eric Ek, Editas Medicine, Inc. - Interim CFO, Principal Accounting Officer & Principal Financial Officer [6]

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Thanks, Charlie. I'm pleased to update all of you on our business and present the latest financial results. These numbers are summarized in the press release that we issued an hour ago, and full details will also be available in our Form 10-Q. Our cash, cash equivalents and marketable securities, increased by $15 million in the third quarter to $333 million as of September 30, 2019, from $318 million as of June 30, in 2019.

Our uses of cash totaled $29 million and include cash operating expenses of $27 million and capital expenditures of $2 million. Our sources of cash totaled $44 million and consisted of $41 million related to the at-the-market offerings, $2 million of stock option exercises and $1 million of interest income.

We believe our cash, cash equivalents and marketable securities of $333 million as of September 30, 2019, provides at least 24 months of capital to fund our business.

And with that, I will hand it back to Cindy.

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Cynthia L. Collins, Editas Medicine, Inc. - CEO, President & Director [7]

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Thanks, Eric. It has been a busy past few months for Editas as we work to deliver the promise of CRISPR into treatments for patients with serious diseases. It is truly an exciting time for us with the work we have been doing to advance our programs and explore new possibilities for genome editing.

Editas' programs have the potential to revolutionize the treatment of genetic blindness, cancer, sickle cell disease and neurological conditions. But our work is only getting started. We are

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with what the future holds with upcoming data readouts, scientific achievements and program advancement.

Thank you all for your interest and support during this journey we have been making alongside researchers, clinicians, investors and, most importantly, patients.

With that, we are happy to take your questions. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Steve Seedhouse with Raymond James.

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Timur Ivannikov, Raymond James & Associates, Inc., Research Division - Senior Research Associate [2]

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This is Timur Ivannikov on for Steve Seedhouse. And we have a couple of questions for EDIT-101 based on a recent competitive readout. So they had a couple of super responders in LCA10. And then -- they had the worst vision at baseline in the low dose cohort. And it turns out the best responder took the longest time to lose his vision and the company didn't specifically say what the genetic or epigenetic differences were. And in addition, there was one patient whose contralateral eye improvement of 0.2 logMAR was relatively significant. So given the potential for super responders and potentially high placebo response, we were wondering how you address those issues in your study?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [3]

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You're right -- this is Charlie. Those are things we will be monitoring, and they're not obvious explanations for some of the phenomena they've been seeing to-date.

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Timur Ivannikov, Raymond James & Associates, Inc., Research Division - Senior Research Associate [4]

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Okay. And I guess, do you also -- in terms of a safety follow up, do you have an expectation for how many patients will develop cataract in your study, just to see what the baseline rate would be?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [5]

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We don't have any expectations it'll be any different than the other therapies in this space. The other AAV-based therapies. So it's something we'll clearly be monitoring.

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Timur Ivannikov, Raymond James & Associates, Inc., Research Division - Senior Research Associate [6]

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Okay. And then just a quick housekeeping question on your updated Celgene partnership. So it looks like you're getting additional $70 million. Is that mostly just for the expanded time line? not clear Is it -- how much after 2020? It is because it seems like you're also getting back some of your rights. So if you could explain a little bit better.

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [7]

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The $70 million is an upfront payment in recognition of the work we've done to date and the contributions going forward. And yes, you're right, we are getting some rights back.

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Operator [8]

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Our next question comes from Matthew Harrison with Morgan Stanley.

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Konstantinos Biliouris, Morgan Stanley, Research Division - Research Associate [9]

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This is Kostas on for Matthew Harrison. My first question is, can you explain a little bit what caused the timeline for the first dosing of EDIT-101 to be pushed back into 2020 from 2019?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [10]

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Sure. This is a -- as you can appreciate, this is a rare inherited retinal disease. And as with all rare diseases, accruing patients is challenging, particularly challenging since the entry criteria for this first patient is restrictive because that's the right thing to do. It has to be an adult with light perception only. And it's further complicated by challenges with the holidays. The patients typically need to bring a caregiver, need to arrive before the surgery and they can't fly for 10 days after the surgeries. The combination of those things has just made it complicated as we approach the holiday season.

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Konstantinos Biliouris, Morgan Stanley, Research Division - Research Associate [11]

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And how is the screening progressing for other patients beyond the first patient?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [12]

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We're progressing well. We've -- in addition to the patients we've identified in the natural history study, we've opened 2 sites, 2 more sites are pending, and we continue to screen for patients outside the natural history study. And so we have, as part of our screening, identified other patients, which are potential patients in the subsequent cohorts.

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Konstantinos Biliouris, Morgan Stanley, Research Division - Research Associate [13]

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Okay. And one more question, please. Can you talk about the reason for Cpf1 in your hemoglobinopathies program?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [14]

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Sure. And so our general strategy is to use the best enzyme for the application. And in this -- the hemoglobin program is a fascinating one because you're knocking out a transcription factor binding site. And as we'll show at the ASH meeting, not all edits of the transcription factor site are actually productive edits, where they knock out the -- they knockout the binding of the transcription factors. So it turns out in this case that when we edit with Cpf1, we get a much higher fraction of productive edits in the long-term hematopoietic stem cells. And so what we've done there is really to create a relatively unique product where we maximize the ability to induce fetal hemoglobin, which is, of course, the objective of this program. So it's really been an interesting journey there, and we look forward to the upcoming ASH Meeting.

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Konstantinos Biliouris, Morgan Stanley, Research Division - Research Associate [15]

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Okay. And one last question, a follow-up on this. Given that we expect data from another CRISPR medicine in hemoglobinopathies again before year-end. Can you talk a little bit about how this data could inform your strategy in this area of diseases?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [16]

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Well, we carefully monitor the competitors in this space as we do in every program we have, we do have reason to believe that we have developed a best-in-class medicine, and we clearly monitor that with time, and it will be interesting to see the upcoming data, particularly in light of the abstracts we've seen to date.

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Operator [17]

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Our next question comes from Whitney Ijem with Guggenheim Securities.

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Anvita Gupta, Guggenheim Securities, LLC, Research Division - Associate [18]

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This is a Anvita Gupta on for Whitney this morning. Could you please give us more color on the vectors that you may gain access to under your AskBio collaboration and the initial indications that you maybe [countering] in this CNS space? And then what attracted you to AskBio in particular versus other next-gen vectors in the space? And then I have an additional follow-up.

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [19]

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Sure. We're very impressed with AskBio. And not only are they a leader in AAV and have a track record. The people involved in AskBio have a significant track record of putting medicines into the clinic that are efficacious. They've also developed a nice infrastructure. They have an excellent manufacturing facility. They've acquired some significant businesses that go with their core business and complement the -- the core expertise they've developed in AAV manufacturing. So it's a very impressive group, and we're super excited to be working with them on a neurologic disease. Our initial efforts will be pointed to severe pain. And so we don't want to say a tremendous about it now, but it's an area of high unmet need where the targets are genetically validated, and we think we can do something very unique.

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Anvita Gupta, Guggenheim Securities, LLC, Research Division - Associate [20]

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Okay. And then in your beta thalassemia and sickle cell program. What is the HbF that target that you're aiming for? And is it any different between the 2 diseases?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [21]

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We believe we need to get more than 30% fetal hemoglobin [deducts in --] increases, and it needs to be significantly pancellular, obviously. And so we'll show data at ASH that we've exceeded those targets. And we believe we have a very interesting experimental medicine.

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Operator [22]

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Our next question comes from Gena Wang with Barclays.

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Unidentified Analyst, [23]

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This is David on for Gena.

My first question is regarding LCA10 data read-through. How do you think that the LCA data read-through to the Usher Syndrome in terms of initial dosing?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [24]

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We -- for each of these, we will -- we are doing the pharmacology modeling to correlate the editing with the dose. We haven't shown that data. But we do anticipate that dose responses between medicines will be quite similar. And we've seen some of that preclinically. We'll show more of that next year. So as with all molecular medicines, the pharmacodynamic, pharmacokinetic relationship is typically reasonably well maintained across therapies in different spaces. We anticipate the same thing here.

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Unidentified Analyst, [25]

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Got it. It's very helpful. Second question is regarding the preclinical data for beta thalassemia and sickle cell disease. Your preclinical data at ASH, how does that compare to the BCL11A approach? And what do we expect in terms of timing to Phase I?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [26]

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Taking them in reverse order, we haven't disclosed the timing to Phase I, what we said, is that we're in a position to begin IND-enabling studies and are advancing it as rapidly as possible. And we, of course, stand by that. The data compares favorably, I think you'll be able to judge that when you see the poster as far as fetal hemoglobin is.

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Unidentified Analyst, [27]

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Okay, very helpful. And then last question on the recent prime editing news that came out. What do you think is the impact to CRISPR?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [28]

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We monitor the competition across the space, both the direct gene editing approaches and the modified gene editing approaches. The prime has many similarities to the base editing has many of the same -- we anticipate many of the same issues, quite frankly.

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Operator [29]

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Our next question comes from Amanda Murphy with BTIG.

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Amanda Louise Murphy, BTIG, LLC, Research Division - MD & Senior Biotechnology Equity Analyst [30]

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Just a few questions, I guess, on the Celgene change or (inaudible) and then also just kind of what you've been up to on that front.

I know you've been presenting for a while now at various conferences, preclinical data on Cpf1 and T cells. So just curious, kind of, if you could update us on what, I guess, a, what happens to the work that's been done previously with Cpf1 and T cell that you've presented? And then I'm assuming that also includes engineered TCRs in terms of taking that sort of back, if you will. So curious if you can just update us on both of those programs, where you're at with those and when you think you might have an update on timing and indications, and things like that.

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [31]

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Yes, I wish I could be more clear about the timing in the details of the Celgene programs. Unfortunately, that's not possible. Suffice it to say that the work that we have shown will carry forward into the new collaboration. And we have shown a lot of data on multiplexing, targeted integration, knock out of the what one would say, are the typical genes in this space. All of that work gets rolled into the new collaboration with Celgene. As you can imagine, as a leader in, we believe, the leader in T-cell medicines for oncology, including arguably the best CD19 and the best BCMA CAR-T programs. They have a strong interest in maintaining their leadership position and gene editing is certainly an important part of what are likely to be the next-generation of T cell medicines for oncology. So we are excited to continue to work with them in that space, and we'll be able to use the knowledge we've gained to date to do that. And unfortunately, we just can't be transparent about the nature of the products or the timing of the products.

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Amanda Louise Murphy, BTIG, LLC, Research Division - MD & Senior Biotechnology Equity Analyst [32]

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Fair enough. And then just on the NK program? I mean, I'm sorry if I missed this. Have you kind of talked about there, what your timing is? I know you had sort of an NK cell program that was sort of running individually and then you obviously have the iPSC collaboration. And I think you've talked about iPSC-derived NK cell as well. So just looking for an update there, if you can provide one?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [33]

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We've provided a general update without the specifics that you probably really want. The -- suffice to say that the science is going really well, and you'll hear a lot more about that next year as we go into the oncology and the stem cell meeting. We are able to with our collaborator BlueRock, identify well-defined and characterized iPSC lines. We've been able to edit those iPSC lines. We've been able to differentiate them into NK cells that have potent killing activity in vitro. We're starting to combine all those things now into what we believe will be interesting experimental medicines. And accumulate the data as rapidly we can to advance those into the clinic. We have a similar effort in the healthy donor-derived NK cells that we think complement the iPSC-derived NK cells. So we do anticipate providing significant update next year that illustrates that progress in a scientific venue.

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Amanda Louise Murphy, BTIG, LLC, Research Division - MD & Senior Biotechnology Equity Analyst [34]

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Got it. Again, if I can sneak in one more about EDIT-101. So it just reminds us again from a protocol perspective, what's required for, sort of, a second patient dosing. Is that going to be a little more [indiscernible] just a reminder on the timing there?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [35]

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Yes. So the entry criteria for both patient 1 and patient 2 are quite similar, light perception only adults. There's an interval between the dosing of those two patients, which I believe is 6 weeks. And so we are obviously trying to accrue both the first and the second patient, because that's what's required in the first cohort.

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Operator [36]

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Our next question comes from Joe Thome with Cowen & Company.

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Joseph John-Charles Thome, Cowen and Company, LLC, Research Division - VP of Healthcare [37]

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The first one on the 101 program. In terms of patient screening, I know you mentioned that, obviously, the first cohort we want patients with only light perception and kind of advanced disease. Have you screened patients that maybe just didn't have advanced disease enough that would be applicable for a later cohort? Or kind of how is patient interest in the program [sitting?]

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [38]

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Yes. Yes. The simple answer is yes. We have screened patients that do not fit the criteria for cohort 1, that could fit the criteria for cohort 2 and 3. And so obviously, we're interested in them. The interest of patients and the physicians remains high. And we're optimistic that we're going to start to get the study dosing and further enrolled.

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Joseph John-Charles Thome, Cowen and Company, LLC, Research Division - VP of Healthcare [39]

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And then one more on that program, I know it's looking ahead, but in terms of data disclosure? I know you're [following] for a year, but there is the potential for some interim, how do you anticipate releasing data? Would you release data from the first cohort? Or would you wait for your -- until you had a few cohorts of patients dosed for a meaningful amount of time.

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [40]

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Right. It depends on the data, quite frankly. And so I remind you that we are partnering with Allergan in this study, and they're going to have an important say in the release of data. We have said we're not going to release data on a patient-by-patient basis because we don't believe that data will be representative and as illustrated by one of the earlier questions, there are some super responders here and you could be significantly misled by releasing patient-by-patient data. And so I think it will very much depend on what the data is and our discussions with our partners, Allergan.

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Joseph John-Charles Thome, Cowen and Company, LLC, Research Division - VP of Healthcare [41]

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Great. And then just one last kind of housekeeping question on the Celgene deal. The $70 million milestone, do you expect to receive that in Q4? And maybe how are you going to account for that? And in terms of when Celgene can opt-in on some of these programs? Is there a specific time after proof-of-concept or before pivotal? Or how are they thinking about that?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [42]

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So yes, we are going to accrue in the fourth quarter. The deal is structured so they opt in -- they can opt-in at any point in time.

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Operator [43]

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(Operator Instructions) Our next question comes from Geulah Livshits with Chardan.

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Geulah Livshits, Chardan Capital Markets, LLC, Research Division - Senior Research Analyst [44]

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Congrats on the progress.

So a quick one on LCA10 and then 1 on the oncology. So with respect to the LCA10 program just to make sure I understand. Was that first patient that was identified rolled in from the natural history study?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [45]

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Yes.

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Geulah Livshits, Chardan Capital Markets, LLC, Research Division - Senior Research Analyst [46]

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Great. And then with respect to the Celgene collaboration in terms of overall oncology strategy. Kind of a broader question. So you have a number of tools at your disposal now. And I'm just wondering how you're thinking about the decision process towards deploying a particular [T] or NK or gamma delta T cell therapy, I should say, towards a given target or towards solid versus liquid tumors and internal versus Celgene collaboration. Yes, if there's any color that you provide on that?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [47]

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Yes. So the collaboration with Celgene is very much about working with somebody who's a leader in alpha-beta T cells. And as you know, they're a leader in alpha-beta T cells directed at hematologic indications. CD19 and BCMA are at the top of their portfolio, as we believe, and I said they arguably have the best medicines in both of those spaces. And I can -- I think that maintaining that leadership will be at the top of their list of things to do. We are going to focus most of our effort on solid tumors, an area of significant unmet need, where engineered cell medicines have been relatively unsuccessful to date, but we believe we can get there and that's going to need significant genetic changes and editing to do that. And the iPSC platform really provides that ability to make the genetic changes that are going to be required. For instance, you need targeting, you're going to need increased persistence, evasion of the immune system, evasion of the tumor microenvironment and probably ability to stimulate the endogenous immune system. And there's no way you're going to get there with any cell type from -- derived from healthy donors. And so we're building up a platform, we believe, is unique in this space and affords the ability to make complex products, which will get us into a space that has a significant unmet need.

Right now, our efforts internally are focused primarily on NK cells. They are one of the easier cell types to make from the iPSC platform. And there's emerging clinical data that they are indeed efficacious in patients, albeit in hematologic indications to date. So we are very excited about that. We've made a lot of progress in that area, and we look forward to disclosing all that at the meetings next year.

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Geulah Livshits, Chardan Capital Markets, LLC, Research Division - Senior Research Analyst [48]

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And so -- this is kind of theoretical, but then with Celgene's potential opt-in rights include something like an iPSC-derived alpha beta T cell-based on the structure of the agreement?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [49]

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It could. That's not the initial focus of the agreement.

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Geulah Livshits, Chardan Capital Markets, LLC, Research Division - Senior Research Analyst [50]

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Great. And then just one last one on the AskBio collaboration. So you mentioned what drew you to AskBio and so just out of curiosity, would under the collaboration, do you have access to the Synpromics informatics platform?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [51]

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We do. We have access to whatever is under the AskBio umbrella. And that's a great example of the way they're expanding their technology footprint to really become, what we believe, is one of the leaders in the AAV field.

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Operator [52]

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Our next question comes from Silvan Tuerkcan with Oppenheimer.

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Silvan Can Tuerkcan, Oppenheimer & Co. Inc., Research Division - Associate [53]

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Well, congrats on the quarter. First off, I wanted to ask you about Matthew Porteus' approach, which is academic. That was presented at ESGCT in sickle cell disease. He will be starting up a Phase II trial next year. Is there anything -- could you maybe highlight the differences between your approach and his. And is there potential read-across from his trial that could apply to EDIT-101?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [54]

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Sure. His approach, as I understand it, is to actually target correction of the sickle cell mutation, and that has some -- obviously, has some theoretical appeal because you're going right after the -- right at the mutation. As you can appreciate, though, not all of the -- not all of the genetic changes at the sickle cell allele are productive, i.e., not all of them lead to correction of the mutation and the problematic aspect of that strategy is when you don't correct, you actually make an indel, which is highly likely to be nonfunctional so those alleles and potentially, those cells will now not contribute hemoglobin to their patient. And so if you don't get enough correction, you actually have the potential to make beta-thalassemia patients -- take sickle cell patients and make them beta-thalassemia patients, which we have found to be a significant risk.

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Silvan Can Tuerkcan, Oppenheimer & Co. Inc., Research Division - Associate [55]

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Great. And could you please remind us of what's left to do to file an IND for Usher and what could be a potential timeline?

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Charles Albright, Editas Medicine, Inc. - Executive VP & Chief Scientific Officer [56]

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We haven't disclosed the time line. What we have said is that at the end of the year, we anticipate having a molecule that will be ready for an IND-enabling study. And so you'll recall that the Usher program is part of our collaboration with Allergan, and it's an option agreement. So we provide packages at the end of the discovery phase to Allergan, and then they have the right to opt in. So we anticipate delivering that package to them at the end of the year. We remain on track for that. And at that point, they'll have a decision to make. And once they've made their decision, we will have -- potentially have a decision to make as well.

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Operator [57]

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And at this time, I'm showing no further questions. I'd like to turn the call back over to Mark Mulligan for any closing remarks.

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Cynthia L. Collins, Editas Medicine, Inc. - CEO, President & Director [58]

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Great. So with that, we thank all of you for participating in today's call and for your support as we work to bring transformative new medicines to patients. Have a great day.

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Operator [59]

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Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.