Good afternoon, and welcome to Editas Medicine’s Second Quarter 2019 Conference Call. All participants are now in listen-only mode. 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 Investor Relations at Editas Medicine. Please go ahead..

Thank you, operator. Good afternoon, everyone, and welcome to our second quarter 2019 conference call. Shortly after the market closed, we issued a press release providing our financial results and corporate updates for the second quarter of 2019.

A replay of today’s call will be available on the Investors & Media section of our website approximately two 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 newly appointed Chief Executive Officer, Cindy Collins..

Thanks, Mark. Good afternoon, everyone, and thank you for joining us for our second quarter 2019 corporate update.

In addition to Mark, I’m joined today by several members of our executive team, including Charlie Albright, our Chief Scientific Officer; Vic Myer, our Chief Technology Officer; Eric Ek, our interim Chief Financial Officer, and Tim Hunt, our Senior Vice President of Corporate Affairs.

As we announced this afternoon, the board ran a comprehensive process over the past several months focused on identifying the right leader to take Editas into its next chapter, accelerate our achievement and focus on our EM22 goals.

The board's search was run by the nominating and governance committee with assistance from leading executive search firms.

Overall, the Board interviewed many candidates and was focused on executives with strong industry, scientific and development expertise, a proven ability to grow and scale companies and a passion for driving long-term success for Editas and patients.

As a veteran of the gene and cell medicine industry, I knew Editas well which is why I jumped at the chance to join the Editas Board last December. I believe that this company has an extraordinary opportunity to develop durable medicines that can dramatically transform patients lives in ways never imagined.

And as Interim CEO, my enthusiasm has grown as I have dug in and learned more about the people, programs and capabilities of the company. That is why I’m pleased and humbled to accept the Board's appointment as Permanent CEO.

We've had great momentum in 2019 and we are determined to build on that and establish Editas as the clear leader in the gene editing space. We have been driving programs, executing business development deals, hiring and building teams and strengthening our Inspiritas culture.

I am proud of what our team has accomplished and I am excited to update you on our latest progress. I will hit on a few of the highlights and then hand it over to Charlie and Eric to discuss in further detail.

First in partnership with Allergan, we have initiated patient screening in our Phase 1, 2 clinical trial with EDIT-101 for Leber Congenital Amaurosis 10 or LCA10 and we’re on track to dose patients in the second half as previously communicated.

This is a thrilling moment in the history of medicine as we are on the cusp of treating people for the first time ever with in vivo CRISPR medicine as well as first for Editas Medicine, second beyond EDIT-101 we’re making strong progress on our broader pipeline of in vivo CRISPR medicine starting with another ocular disease Usher Syndrome 2A.

This program is de-risk and accelerated by our work on EDIT-101 and we expect to be ready for IND enabling studies by the end of the year.

Third, we continue to advance our engineered cell medicines, our confidence in EDIT-301 as a potential best-in-class medicine for sickle cell disease and beta thalassemia continues to build and our team is making tangible headway on its development of engineered cell medicines for cancer including universal allogeneic cell medicines.

Finally, we’re investing in manufacturing infrastructure required to develop and commercialize CRISPR medicines including manufacturing capacity at our site in Boulder, Colorado. Now I will hand the call over to our Chief Scientific Officer, Charlie Albright to update you further on the pipeline..

Thanks Cindy. And thanks to everyone on the call for joining us today. I'll start with our first in vivo CRISPR -- I’ll start with our in vivo CRISPR medicine's beginning with our lead candidate EDIT-101 for LCA10.

As Cindy mentioned, we've initiated patient screening for our Phase 1, 2 interventional study called Brilliance in collaboration with Allergan. We remain on track to begin dosing in the second half of this year. Brilliance is an open label dose escalation study to evaluate the safety, tolerability and efficacy of EDIT-101 in approximately 18 patients.

Mass. Eye and Ear is currently recruiting and we plan to open three more sites in the weeks ahead. We also expect the first patients in the trial will come from our natural History study which enroll patients with very similar enrollment criteria to the Interventional study.

You will recall that accelerating the Interventional study was one of the objectives of the natural history study. For our second in vivo program, we are developing experimental medicine for patients with Usher syndrome 2A.

Like LCA10, USH2A is an inherited retinal disease that affects the photoreceptors, USH2A is caused by mutation in the gene encoding Usherin, a protein essential for photo transduction in the retina.

The most common mutation in the USH2A gene is a single nucleotide deletion in Exon 13 causing a frame shift that results in a truncated non-functional protein. Our therapeutic approach uses CRISPR gene editing to remove the Exon containing the mutation and thereby create a functional protein. Our collaborators from Mass.

Eye and Ear showed preclinical proof of concept for this approach at the recent American Society of Gene and Cell Therapy Conference and we are currently completing preclinical studies for our lead molecule. This medicine will share many features of EDIT-101 including the AV serotype, promoter and proprietary Staph aureus Cas9.

We anticipate having a molecule for USH2A patients that is ready for IND enabling studies by the end of the year. Finally we're working on additional programs and we'll speak further about these programs in the coming months. These programs will illustrate the power of our platform to rate in vivo editing medicines.

Transitioning to engineered cell medicines. The second pillar of our therapeutic strategy we’re developing EDIT-301 to be a best-in-class medicine for sickle cell disease and beta thalassemia. This program uses gene editing to induce fetal hemoglobin expression to treat hemoglobinapathy.

As a reminder, our therapy involves targeting the beta-globin locus and hematopoietic stem cells. This therapeutic approach is differentiated from other approaches which go after the BCL11A erythroid enhancer which only indirectly up regulates fetal hemoglobin. Our findings suggest adding the BCL11A enhancer may impact stem cell differentiation.

Ultimately the number and viability of edited red blood cells in vivo. We have found that adding the beta-globin locus on the other hand had no detrimental effect on erythroid output while inducing robust fetal hemoglobin.

IND enabling activities are underway, we plan to present additional data demonstrating the features and benefits of EDIT-301 in the Medical Conference later this year. Due to our confidence in this program, we are building manufacturing capacity at our site in Boulder, Colorado.

Particularly intend to use this facility as supply guide RNA and ribonucleic protein for preclinical and clinical studies with EDIT-301 as well as other engineered cell medicine oncology programs. We expect this site to be commissioned next year.

Beyond our work on Hemoglobinopathies, we are investing broadly to develop engineered cell medicines and oncology. We have a long standing partnership with Celgene to develop engineered T-cell medicines and in parallel we are advancing wholly owned efforts to develop engineered NK cells as potential therapies for cancer.

Last quarter, we announced a collaboration with BlueRock Therapeutics to develop cell medicines for oncology using induced pluripotent stem cells or iPSCs.

Engineered cell medicines from iPSCs may offer several potential advantages including the ability to make a highly engineered cell with total control of the genome and thereby create a truly off the shelf medicine. We believe these features will be essential to unlock the potential of cell medicines for solid tumors, an area of high unmet need.

Encouraged by the rapid progress we're making, we've made in just the past few months. Firstly the successfully generated iPSCs from fibroblasts and peripheral blood cells. Second we've achieved efficient editing of iPSCs and third we have differentiated iPSCs in the functional NK cells with potent killing activity.

This progress puts us on a good path to develop highly engineered off-the-shelf NK medicines that address the high unmet need for patients with solid tumors. I have more to say about these results at upcoming scientific meetings.

In conclusion, our team continues to make strong headway against both in vivo CRISPR medicines and engineered cell medicines. Now let me turn the call over to Eric to provide a business and financial update..

Thanks Charlie. I'm pleased to update all of you on our company's financial results. These numbers are summarized in the press release that we issued about an hour ago and full details will also be available in our Form 10-Q.

Our cash, cash equivalents and marketable securities decreased $24 million in the second quarter to approximately $318 million as of June 30, 2019 from approximately $342 million as of March 31, 2019. Our uses of cash totaled $32 million and include cash operating expenses of $31 million and capital expenditures of $1 million.

Key non-cash items recorded in our income statement include $6 million of stock-based compensation and $1 million of depreciation. Our sources of cash include $2 million of stock option exercises and $1 million of interest income.

We believe our cash, cash equivalents and marketable securities of $318 million as of June 30, 2019 provides at least 24 months of capital to fund our business. And with that, I will hand it back to Cindy..

Thank you, Eric. Before we close, I'd like to make a few comments on intellectual property and organizational development. In early June, the U.S.

Patent Office declared a second interference between certain patent applications submitted by the University of California Group and certain patents issued to the Broad Institute which are exclusively licensed to Editas Medicine.

We have the utmost confidence in our intellectual property and expect these issued patents to be confirmed and upheld in this proceeding. On the organizational side, executive searches remain active to bring on an excellent new CFO and Chief Medical Officer. We look forward to updating you at an appropriate time.

In closing, we are making history with the first ever treatment of patients with in vivo CRISPR medicine. Our pipeline is advancing to translate CRISPR gene editing into genomic medicines across a wide range of serious diseases from hemoglobinopathies to cancers to inherited blindness.

And we are further enhancing our end-to-end capability to bring medicines to patients by building manufacturing capacity to reliably supply CRISPR medicines. With that, we thank all of you for your interest and support and are happy to take your questions.

Operator?.

Thank you. [Operator Instructions] Our first question comes from the line of Gena Wang from Barclays. Gena your line is open. Our next question comes from the line of Matthew Harrison from Morgan Stanley..

Hello, this is Costus on for Matthew. I have two questions. The first one is how do you expect to report the data from the Brilliance study given that this is an open label study.

Will you announce data from few patients first or you will wait until the studies completed before you present data?.

Hi this is Charlie. So number one we're really excited to get the study going. As we’ve said this is the first in vivo CRISPR editing and how we report the data will very much depend on the circumstances and so we will be able to see the data in real time.

We will but we want to be careful about how the accuracy of our conclusions will make that ultimate call in collaboration with Allergan or T-collaborators in this area..

Okay. Thank you.

And one more can you detail what do you need to complete an IND filing for EDIT-301?.

It’s all the usual stuff and so we need to scale-up manufacturing with the IND-enabling Tox studies..

And do you know approximately how long will this take?.

No we have not disclosed timelines for EDIT-301 yet. Stay tuned you'll see more data on the program at an upcoming scientific meeting which I think you'll find quite interesting..

Okay, thank you..

Our next question comes from the line of Cory Kasimov from JPMorgan..

Hi this is Gavin on for Cory. I extend the congrats to Cynthia on the new appointment. And then we just had maybe a follow-up on the first question on EDIT-101. The disclosure strategy, do you guys see any potential headwinds from the pending deal with the partner and then just a follow-on for Usher syndrome.

What are the gating factors that remain for the IND-enabling studies? Thank you..

I didn't quite understand the first question about headwinds. I mean we have a partner and we will disclose in collaboration with them.

Are you talking about the AVI merger?.

Yes, correct.

It’s the disclosure on the Allergan side or?.

Right now Allergan is, Allergan is our partner right now and so that deal is obviously not closed. And until it does AVI is not a factor in any decision making. So it's business is usual very much on all of the ophthalmology programs that are partnered with Allergan and so we've got our head down and turn and deliver the portfolio.

Usher Syndrome 2A we aim to be ready for IND-enabling activities by the end of the year. So we have a variety of preclinical pharmacology studies going on now and completing some other studies that get us in a position to have found the package we need to proceed into the IND-enabling studies..

Great, thanks for the clarity..

Our next question comes from the line of Steve Seedhouse from Raymond James..

Yes, hi. This is Vanica on for Steve Seedhouse. And we would like to follow-up on patent interference question. So what is your estimate of the legal costs involved and also what is your view of the acquisitions being made by Berkeley regarding willfully making an accurate statements.

And finally what would be an ideal resolution if the other side wanted to come to the table and negotiate? Thank you..

So first of all, we're not going to speculate on what the overall cost of the litigation might be nor make any comment about acquisitions of the inventors. And your last question, I apologize was….

What would be an ideal resolution if the other side wanted to come to the table and negotiate, what would be an ideal situation?.

So I think an ideal situation for us would be that we continue to prevail with the intellectual property position that we have which we feel very confident around particularly as it relates to Cas9 and Cpf1..

Okay, thank you. And then we also have a follow-up on the Brilliance study. I think in the past you've talked about preliminary design. I don't know if that's still the case but I've seen two patients in the first cohort. I don't know if that's still the plan for you and they're going to be dose but low dose and then you will escalate to higher dose.

So the question is I mean is it still two patients or have you made any changes and also is the second patient is going to be younger than the first one, if you can disclose? Thank you..

Sure. And so obviously safety is of paramount concern, so two of their main objectives are safety and tolerability and the third objective is efficacy. So obviously the right thing to do is to put the risk benefit very much in our favor as we start the trial.

So we will start the trial in adults that have very poor vision and there will be two patients dose that at the low dose, a dose that we think can be will be pharmacologically active.

But on the low end of the range of pharmacologic activity and then based on the results from those two patients, we will escalate the dose and eventually we will escalate.

We will move into adults who have better vision and based on those results will eventually move into ideally children that have clear vision and then better vision because a priority would expect children with some vision to have the most benefit from this therapy.

But that's obviously not the right place to start this trial from a risk benefit standpoint..

Great, thank you very much..

Our next question comes from the line of Amanda Murphy from BTIG..

Thank you and congrats Cindy. I guess I have a question for you Cindy just since you've been there now for several months.

Just curious if you've now you have some contacts and thinking about your broader strategy in EM22 et cetera, any tweaks that you're thinking about making whether you have already or going forward and obviously the company is changing somewhat going into from a preclinical entity into clinical entity.

I mean there's some resource hiring things like that but just look curious now that you've had a year behind you or I guess almost a year, what is your perception?.

Thanks Amanda. It's been about six months but maybe it feels like a year. So we remain very committed to our current EM22 strategy and to our 2019 corporate goals and you’re correctly referring to the fact that the company is now moving into a clinical phase which is different for us.

We'll continue to hire the required resources that we need to support that growth as we scale, we talked a little bit about some of our efforts in manufacturing in our Boulder facility and we'll just continue along that trajectory for the near term..

Okay, got it. And then just one on the fully owned NK side of the world.

So I guess considering NK and iPSC drug, NK cells A is of solid tumor and it's maybe too early to ask you but just curious about your strategy there going forward with the view would you focus more on maybe starting out in some more well validated targets that ones that are a little more crowded competitively.

I know there's not that much in solid tumor really or would you kind of go, I was thinking high level, how you might approach from an indication perspective?.

Sure, this is Charlie, Amanda. So we obviously haven't disclosed the details of where we're going. I think you can draw some parallels from how we treated other disease areas and be assured will have a thoughtful approach to the indications, we pursue in that area to develop both differentiated and transformative medicines..

Okay. And I just have last one on the iPSC side, it seems like that iP landscape is almost as complicated as CRISPR quite but is there anything to factor in there as we think about your opportunities.

Do you feel like you have freedom to operate broadly there or is there anything from a restriction standpoint to think about?.

We feel very good about our position there and are very enthusiastic as hopefully gathered about advancing those medicines..

Okay, thanks very much..

Our next question comes from the line of Peter Lawson from SunTrust Robinson..

Hey Cynthia, I just want to offer my congratulations on the role. Maybe a question for Charlie, just how should we think about the timing for entering the clinic for beta thal and sickle cell produced beta first or sickle first.

And can we use I guess timing from peers as an example there?.

Yes, that’s good. Thanks Peter. Yes, I think that the precedence in this field are numerous and it's totally fair to use the existing trials as a guidepost. We haven't disclosed our strategy on sickle cell versus beta thal in U.S.

versus Europe either both key to strategic decisions as we advance what we feel is a really exciting medicine and look forward to really talking a lot more about it later this year..

And then just on LCA10 just considering the pace the initial patients appropriately the most difficult to treat.

Do you think you would have a therapeutic dose initially for those patients, so how long do you think we have to wait until we kind of see that?.

Well we believe that you'll see benefit in a small number of months. And we've gone through that scientific rationale for that and because it's based on the preclinical pharmacology and the doses are entirely based on the preclinical pharmacology as well.

So we did studies in mice and non-human primates which give a very similar relationship between dose and Cas9 levels and editing.

And so we feel as good as one can feel at this point about dose selection and that's been the basis for how we've chosen the starting dose because we do believe at that dose you have potential to have therapeutic benefit but obviously that's why we do the trials to understand what's going on there and we want to be conservative at the same time, we're trying to understand the efficacy to first understand safety and tolerability..

Great, okay. Thanks for taking the questions. Congratulations..

Our next question comes from the line of Kenneth Atkins from Cowen and Company..

Hi, thanks for taking my questions.

Can you comment on the pacing of patient dosing in EDIT-101 trial, how soon after the first patient dose can you dose the second patient and so on?.

Yes, this is Charlie again. So we haven't disclosed the details, it's a very typical design based on gene therapy. And so you're correct that there will be an interval between all the patients actually and that fits with the priorities of the study to ensure safety and tolerability before we expose additional patients to the drug..

Got it, okay. And then aside from safety and tolerability, what level of improvement on visual acuity and visual function navigation do you think would be meaningful for patients.

And would that differ depending on the patient's age or their disease severity?.

I think that -- I think that the SPA provides a reasonable precedent and what's considered clinically meaningful benefit in patients in general. And clearly a major objective of the trials to understand the benefit we can deliver with our medicine..

Got it, okay. That's helpful, thanks..

Our next question comes from the line of Whitney Ijem from Guggenheim Securities..

Hi this is Anita on for Whitney Ijem. Thanks for taking my question first. So as you look towards expanding the utility in vivo CRISPR technology can you talk about any other work ongoing or progress made on the delivery side. And also how do you think about targeting muscle and lung in DMD and CF programs? Thanks..

Thanks, this is Charlie. We are very excited to move into other tissues. And one of the big benefits we get out of the EDIT-101 is experience in delivering a complete package that was suitable to regulators in this space.

And so this does set us up to go into other tissues where AAV has been proven to be a good delivery vehicle and of course you realize we're the only company that actually has the ability to deliver as gene editing and a single AAV because of our access to the Staph aureus Cas9, so we do want to take advantage of that unique capability and move into other in vivo tissues.

We do think that the eye was a great place to start but we look forward to talking more about the other places we're going to go..

[Operator Instructions] Our next question comes from the line of Silvan Tuerkcan from Oppenheimer..

Thank you for taking my question and congratulations on the new role, Cynthia.

My question is could you -- I looked at the trial on clinical trials that got the Brilliance trial and so there's a middle -- low middle and high dose in adults and low dose and high dose in pediatrics, would that middle dose be the same as in adults for children?.

Yes. And it turns out that the eye is not all that different and so as you grow and so the same dose is totally appropriate..

Okay, great. And could you please remind us of the endpoints that you have in the natural history study for the LCA10.

And could that be kind of viewed as a control I mean these patients will get spontaneously better but to kind of quantify the clinical benefit at the end?.

Yes, so we’re looking at a variety of potential endpoints in the natural history study, all the usual suspects.

So that’s corrected visual acuity of right imaging endpoints which could provide substantial evidence that we actually had the functional benefit that we aim to achieve as well as a mobility maze which would be a more stringent test of functional ability of these patients.

So part of the whole point of that natural history study was to get familiarity with these endpoints and understand which would be most operationally and functionally relevant for this patient population. And we will be looking at several of those endpoints in the Interventional study..

And so my last question.

Could we get that data before you will disclose any efficacy data from the Brilliance trial or do you think that will be after?.

We've disclosed some of that data at this past year’s ARBO meeting. And so we disclosed baseline data in that that should be available on our website..

Great, thank you so much..

We have no further questions at this time. I will now turn the call over back to Ms. Cindy Collins..

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

Ladies and gentlemen, thank you for joining us. This concludes today’s conference call. You may now disconnect..