Mark Mullikin - IR Katrine Bosley - CEO Gerry Cox - CMO Charlie Albright - CSO Andrew Hack - CFO Vic Myer - CTO.

Cory Kasimov - JPMorgan Matthew Harrison - Morgan Stanley Peter Lawson - SunTrust Gena Wang - Barclays Gbola Amusa - Chardan Capital Markets.

Good afternoon and welcome to Editas Medicine’s Second Quarter 2018 Conference Call. At this time, all participants are in a 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 Editas’s request.

I would now like to turn the call over to Mark Mullikin, Senior Director of Finance and Investor Relations at Editas Medicine..

Thank you, operator, Good afternoon everyone and welcome to our second quarter 2018 conference call. Shortly after the market closed we issued a press release providing our financial results and corporate updates for the second quarter.

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 Chief Executive Officer, Katrine Bosley..

Thanks, Mark. Good afternoon, everybody, and thank you for joining us for our second quarter conference call.

I am joined today by several members of our executive team, including Charlie Albright, our Chief Scientific Officer; Gerry Cox, our Chief Medical Officer; Andrew Hack, our Chief Financial Officer; Vic Myer, our Chief Technology Officer; and Tim Hunt, our Senior Vice President of Corporate Affairs.

We are at a particularly exciting time for our company and for the field of medicine broadly. Our medicine is strong as we advance the first in vivo CRISPR medicine into the clinic and progress our broader pipeline of experimental ocular and engineered cell medicines.

I'd like to start by discussing our medicines program to treat Leber Congenital Amaurosis type 10 with our experimental medicine EDIT-101. First, let's talk about our progress towards this important clinical trial.

As we've discussed before, prior to dosing the first patients in the United States, our clinical trial protocol will need to be registered with the National Institute of Health or NIH and we expect this process will involve the review by NIH's Recombinant DNA Advisory Committee which is also known as the RDAC.

Registration of human gene transfer protocols with the NIH is required in addition to the customary IND process with the FDA. Our plan has always been to conduct these filings roughly in parallel with each other and we continue to take that approach.

In July, we filed the requisite data package with the NIH in order to be eligible for the quarterly RDAC meeting which is scheduled for late September. As many of you know, the NIH recently revised their guidelines to corporate recommendations of the Institute of Medicine regarding their review of human gene transfer protocol.

This has resulted in fewer RDAC meetings and a higher bar essentially for RDAC review. We will learn later this month if the RDAC will conduct a public review of EDIT-101 at that September meeting and we will let you know in advance if they do. We then expect to file our IND with the FDA in October.

Although this timing is slightly later than our original plan, given the timing of the RDAC we decided that this is the best approach to having a successful IND submission and to getting our study up and running efficiently after IND allowing. And Gerry will share more details on the program with you shortly.

We are very pleased to announce today that Allergan has exercised its option to develop and commercialize EDIT-101 globally as part of the strategic alliance we formed early last year. Concurrently we have exercised our option to co-develop and share profits and losses in the United States.

Allergan has a long and deep history as an ophthalmology innovator for over 50 years. It is very close to this program since we established the alliance and their development, manufacturing, and commercial capabilities meaningfully enhance the EDIT-101 program and maximize the opportunity to bring the transformative medicine to patients.

Andrew will share more details on this development later in the call. And with that, I would like to turn the call over to our Chief Medical Officer, Gerry Cox to provide further detail on the RDAC as well as our LCA10 and natural history study..

Thanks, Katrine. First, to give a bit more color on the RDAC. This is a federal advisory committee that advises the NIH Director on clinical trial protocol that involves human gene transfer and may benefit from discussions. EDIT-101 falls under the purview of the NIH because it is recombinant DNA molecule delivered by a viral vector.

In the case of EDIT-101, the recombinant DNA encoding the gene so the Staphylococcus aureus Cas9 protein and the true guide-RNA is delivered in an AAV vector. The RDAC has substantial experience of gene editing having reviewed several product candidates that are currently in the clinic.

The RDAC is convened at the discretion of the NIH when requested by the Institutional Review Board or Institutional Biosafety Committee and the investigational product meet certain novelty criteria. Our first anticipated clinical trial site is Massachusetts design here has made such as request.

Of course as Katrine noted in her comments, not long ago NIH revised their guidelines for the review of human gene transfer protocol and it is possible that we will not be asked to present to the RDAC. We have a deep regard for the NIH and their oversight processes and will respect whichever approach they deem most appropriate.

If a RDAC review is scheduled we expect that this meeting will be open to the public and available via live webcast. In addition of the RDAC we're advancing our natural history study for LCA10. As a reminder, this study aims to prospectively evaluate patients to inform trial design and facilitate enrollment for interventional trial.

However, its completion does not get into first in human study and we plan to continue to evaluate patients in the natural history study as we ramp up our Phase 1/2 interventional trial. We now have five sites enrolling patients in the natural history study, including four in the U.S. and one in Germany.

We expect to add two more sites in Europe by the end of the year and plan to share initial data from the study in the first half of next year. Now to discuss our pipeline progress I'll turn the call over to our Chief Scientific Officer, Charlie Albright..

Thanks Gerry. As we approach the IND filing for EDIT-101, let me review some of the most recent data that supports a strong submission. At the American Society of Gene & Cell Therapy, or ASGCT Meeting we presented tolerability and immunogenicity data for EDIT-101 for a pilot study in nonhuman primates.

In that study we found that EDIT-101 was well tolerated over the duration of the study based on a panel or clinical test. Furthermore, either preexisting nor induced immunity to either AAV or Staphylococcus aureus Cas9 impacted productive editing in these nonhuman primates.

To date our comprehensive set of pharmacology, specificity, tolerability and immunogenicity data gives us substantial confidence in EDIT-101 as an experimental medicine for LCA10. Beyond EDIT-101 we are advancing our next inherited retinal disease program for Usher Syndrome type 2A patients.

We presented in vitro data at the ASGCT Meeting with our collaborators in Massachusetts Eye and Ear. These data showed that deletion of exon 13 in the human USH2A gene successfully rescue cilia formation in cultured cochlear cells from USH2A knockout mice which normally lacks such cilia.

Since both USH2A and LCA10 affect cell receptors, the USH2A product will benefit from our EDIT-101 experience. We expect the USH2A experiental medicine will use the same AV stereo type promoters for Cas9 and gRNA expression route of delivery, pharmacology models and toxicology programs that we developed for EDIT-101.

The USH2A program is currently in lead optimization. Switching gears to our other major therapeutic focus on engineered cell medicines, in the second quarter we revised our relationship with Celgene to develop engineered T-cell medicines for cancer.

This revision was done to more fully enable the lead program and the collaboration with targets human papilloma virus associated solid tumors. The collaboration is making steady progress and we look forward to sharing more over the balance of the year.

Additionally, we continue to drive forward our program to develop a superior medicine for sickle cell disease and beta thalassemia, to durably induce higher levels of fetal hemoglobin expression our novel strategy targets the native beta-globin locus, in contrast, others that are targeting the erythroid enhancer in the BCL11A gene.

We presented in vitro data at ASGCT on the discovery of these novel hemoglobin inducing sites we plan to present in vivo data later this year demonstrating that targeting these sites can robustly repopulate the blood system. This data will include a comparison to results from editing the BCL11A erythroid enhancer.

Based on this data we are working toward a medicine that will be safer and more effective than other generic medicines currently under development for hemoglobin opportunites. In summary, we are very pleased with the progress in our pipeline over the second quarter and we look forward to a busy second half of the year.

With that, I'll turn the call to our Chief Financial Officer, Andrew Hack to discuss progress in building the business and to review our financial results..

Thanks Charlie. It's my pleasure to update you on how we're building the business and to summarize our financial results that we reported today. As Katrine mentioned earlier, we're excited to announce today that Allergan has exercised its option to development EDIT-101 for LCA10.

Concurrently we exercised our option to a 50-50 financial and operational partnership for EDIT-101 in the United States. We ratified that Allergan has decided to join us in developing EDIT-101 in the U.S. and that they have taken responsibility for the development of EDIT-101 outside the U.S. as well as for its commercialization globally.

As a result of this decision we have received a $15 million option exercise fee from Allergan which will be recorded in the third quarter. We're eligible to receive another $25 million from Allergan upon acceptance of the IND application for EDIT-101.

Going forward Editas and Allergan will share all profits and losses from the development and commercialization of EDIT-101 in the U.S. In addition, Editas will receive high single-digit royalties as well as regulatory and commercial milestones for activities outside the U.S.

I'll now turn to our second quarter 2018 financials which are summarized in the press release we issued roughly an hour ago. We remain very well capitalized with $344 million of cash, cash equivalents and marketable securities as of June 30, 2018, a decrease of $15 million from March 31, 2018.

As I mentioned above, this quarter and cash balance does not include the $15 million we received recently from Allergan. Net cash used in operations in the second quarter were $17 million, which includes $10 million received from Celgene related to the recent expansion of our agreement with them.

Capital expenditures in the quarter totaled approximately $1 million. Moving to the income statement, we recognized $7.4 million of revenue consisting of $3.9 million related to our license agreement with Beam Therapeutics and $3.5 million related to our collaboration with Celgene.

Research and development expenses of $32.7 million included $14.7 million in success and sublicense payments and $6.9 million in process and platform development expenses.

Key non-cash items recorded in our income statement include $7 million of stock-based compensation, $12.5 million of success-based payments for sponsored research that were settled in equity and as well as $0.8 million of depreciation.

We remain confident in the fundamentals of our pipeline and platform and given current trends we believe that our cash position will provide us with at least 24 months of funding to advance our business through a series of important value inflection points. Now, let me turn the call back to Katrine for closing comments..

Thanks Andrew. Before we close it, I'd like to step back and summarize our progress in executing against both our short-term objectives and our long-term EM22 goals. First, we are on the verge of the IND filing for EDIT-101 which is poised to be the first in vivo CRISPR medicine to enter human clinical trial.

Second, with advanced additional experimental ocular and engineered cell medicines for USH2A ocular HSV Sickle Cell Disease and HPV associated solid tumors. Third, we've added important guide RNA engineering and manufacturing capabilities through the acquisition of assets from I2 Pharmaceuticals.

Fourth, we continue to be active under business development plans with the expansion of our Celgene collaboration and our decision to opting to co-develop EDIT-101 with Allergan.

Fifth, and finally, we're building our standing organization adding industry leaders to our Board of Directors and successfully scaling our organization to over 130 employees today. I'd like to thank you for your interest and support and with that let's open it up to Q&A.

Operator?.

[Operator Instructions] Our first question comes from Cory Kasimov with JPMorgan. Your line is now open..

Hey, good afternoon guys, thanks for taking my questions. I've got two of them for you.

I guess, first one is just bigger picture, I'm curious to get your thoughts on the recent safety controversies I guess in the CRISPR field and whether these publications have had any impact on the progress you're making towards the clinic or just your general approach with your technology? And then I have one followup after that..

Sure, thanks for that question. Certainly, something that's garnered a lot of attention, I'd actually like to ask Vic Myer, our Chief Technology Officer to speak to this because as I think you and others who follow us closely know we've spent a lot of time thinking about and working on specificities since the very founding of the company.

Vic?.

Yes, thanks. Cory, we have been thinking about this talk all along and we don’t think the recent publications from the Bradley Lab are specifically problematic in our work to make CRISPR based medicines.

Presumably if the Bradley paper you are talking about has gotten a lot of press, in this paper the investigators were looking at actively dividing cells and they saw a low level of unexpectedly large deletions, insertions and versions from the cut site [ph].

The reason these observations are considered unexpected is the vast majority of the field looks at a very narrow window directly around the cut site and so when you look more broadly they saw more events. These events are likely the result of a natural site or repair process called resection.

This is part of the ophthalmology directed repair pathway and it's generally on an actively dividing cells and I think that's ultimately an important point.

So maybe to method on to what we are doing here at Editas, the first is this research finding likely doesn’t impact LCA10 because photoreceptors are certainly differentiated, they are not dividing this pathway is not active in these cells.

Second piece is we've always embraced strategies using multiple [indiscernible] methods to look not only at the small insertions and deletions around the cut site but also much more broadly at larger inversions, deletions and translocation.

So we've been ahead of this type of analysis for quite some time and we've shared a lot of that data externally.

And finally for all of our programs we've developed a deep preclinical safety package looking now we have the molecular outcomes of the genome, but we also look for any phenotypical alterations in the tissue or cell type of interest and these are of course clinical experiments to couple with any molecular analysis to reduce the risk for experimental novel medicine..

Okay, that's very helpful. And then my second question is I want to go back and ask about the logistics of a potential RDAC meeting.

I guess I'm wondering is there any – is there a reason or any precedent for why there wouldn't be a RDAC meeting in this case given the novelty of the program, and that your first center has requested one, and is this potential RDAC meeting and the subsequent follow up to that the only gating factor at this stage to your IND or is there other work you're still wrapping up? Thanks..

One thing that Gerry mentioned in his comments is that the RDAC sort of changed how it worked about a year and a half ago and so they haven’t had as many meetings and that is why the precedent is perhaps a bit harder to call in this win.

We're certainly as Gerry mentioned comfortable if they do or don’t choose to review this publicly and we'll let folks know and if there is interest in that. And if they do review publicly, that will be a public event and we should now that in the next week or so if that's going to occur or not. So stay tuned, we're staying tuned as well.

Basically in parallel we're continuing to finalize everything towards the IND so it is not strictly gating, there's obviously some resilience across the work in these two streams of work.

So we are finishing reports, finishing documentation, et cetera, all of the nuts and bolts which they are finalizing a full IND is that it's actively underway here..

Okay, good luck with it and thank you too for taking the questions..

Thank you..

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

Sorry, I was on mute, thanks for taking the question. I think two from me as well.

So the first one if I could just followup on Cory's question, regarding the RDAC, I mean given the lack of precedence in how it's changed do you have a view on the meaningfulness of whether or not they choose to publicly review EDIT-101 as part of a RDAC meeting? And then I have a followup then..

Yes, I think meaningfulness is probably a number of ways to interpret that, but I think at the end of the day we will have to refer back to – we submitted a data package to them, that's part of this process. So the Massachusetts Eye and Ear committees have looked at our data, they've requested – they sent a request to the RDAC to review it.

We then submitted a data package to the RDAC. So they're receiving quite a bit of information about this program. It's not just – it's a fairly substantial amount of data.

And so if they choose not to review it, then I think it's really just I'm feeling like between Institutional Review Board, the IBC and the FDA, all the appropriate questions will be covered and if they don’t think there's a need for an additional layer to this, they may choose to go that way or they may say, hey this is the first in vivo CRISPR gene editing medicine and a public review is a good idea.

Otherwise their judgment – remember they've been around for 40-odd-years. They've seen a lot of evolution in technology over the decade and I think are really consciously thinking about how they fit into the evolutionary field of medicine..

Okay, perfect that's helpful, thank you.

And then I guess the second question is just around to the extent that you've engaged in any pre-IND meeting with the FDA, I wonder if you could just comment broadly about whether or not you think there are broad agency concerns with CRISPR medicines or that most of the agency concerns around IND filings are product specific? Thanks..

You know the FDA engages in public commentary themselves at various meetings that we've been at and Editas has been at and I think that they've reviewed a number of Gene editing INDs to date and have allowed a number of Gene editing INDs to date.

Certainly what we hear from them is consistent with what they say publicly and we haven't been surprised by anything we've heard from them. I think we're comfortable that our data packages coming together..

Thank you. And our next question comes from Peter Lawson with SunTrust. Your line is now open..

Hey Katrine, just on the modest layer on the IND filings, has that delay mostly caused potential for the RDAC Committee or were there any internal products?.

Yes, really just looking at how all these different trends stick together and with the potential for RDAC meeting that also then give us the opportunity if there is feedback from that’s incorporated.

Also as you can imagine while Allergan is very close to this the whole time and it’s been looking at every piece of data that emerges with them opting in and of course we’re operationally bring all these pieces together. So really it's just a bit of a logistical call more than anything.

We’re certainly going to continued to work to make sure that we're - as we work towards treating the first patient in a position to do that as rapidly as possible after IND allowance..

Okay, thank you.

And then just on the Celgene Juno expansion of the collaboration when do you think we'll see the first color [ph] around that program?.

So, as Charlie mentioned in his comments, we expect later this year to be sharing more data from that program, so look for that later this year..

All right, okay thanks so much. Thanks for taking the questions..

Thank you. And our next question comes from Gena Wang with Barclays. Your line is now open..

Thank you for taking my questions.

I also have two questions and the first one for everyone, so asking a little bit about IND filing and the RDAC, my understanding of the RDAC and IND filing will be two independent process, just wondering the timing, you said in October slightly after a RDAC meeting just wondering if you can share with us what you would expect to learn from the RDAC meeting to help you prepare for the IND meeting?.

Yes, so you're right, they are independent processes and as we said we always expected to run them roughly in parallel with one another as. You look at the scheduled meetings for the RDAC the late September date is the one that kind of made sense for us to file in advance and that’s why we filed our RDAC package in July.

That was the deadline to be eligible for that September meeting. And if you think about, what the RDAC reviewed they do focus they look at the whole data package but they have a particular focus on things like informed consent and bio safety and things like that.

And so, I think as we look at it, it just seemed prudent to avail ourselves of any insights that may come from that given just the way the dates fall with respect to one another is pragmatic as much as anything..

Okay, so related question is just there are recently a few IND hold clinical hold, just wondering what that - anything you wanted to prepare extra data sets or anything just to be extra careful with the IND preparation to avoid any potential delay on the IND filing?.

I’ll ask Charlie, our CSO to speak to that..

We have been preparing a thorough package and obviously cannot comment on other people's clinical holds that we don't know anything about but we feel confident the data set were assembling based on what we have been planning to do for many months and based on our interactions with regulatory, that's what we're acting upon..

Okay, one last question as for beta thalassemia and Sickle Cell Program, I know there are also a few other companies approach, not exactly the same approach with Gene editing technology, just wondering if you can highlight your differentiation or differentiate approach versus others?.

Sure, there are several approaches being pursued for beta hemoglobinopathies and the gene editing approach is that our competitors are approaching are by targeting the erythroid enhancer that lies upstream the BCL11A gene and they're targeting that enhancer because a knock out of the BCL11A gene is not tolerated.

We've decided to focus instead on the promoter region and the general genomic region of the beta globin locus and that's partly because that's where the human genetics points us, but that's also partly based on a comprehensive screen of sites within that broad locus that will allow us to upregulate fetal hemoglobin more directly.

And we've started pursuing some of that data. We discussed our Lente viral screen at the ASGCT meeting and we'll discuss more of the data including the in vivo data at the upcoming ASH meeting in December..

Okay, great thank you..

Thank you. [Operator Instructions] Our next question comes from Gbola Amusa with Chardan Capital Markets. Your line is now open..

Hi, it’s Gbola Amusa at Chardan. Thanks for taking my call. Just a couple of questions on the update on Usher Syndrome, that was super helpful so far.

Do you have estimates on what the therapeutic thresholds for editing there might be for example mutation be similar to those LCA10? And then I had a ASH, do you have any specifics on what you might present at ASGCT in October?.

Charlie will capture those..

So let me start with the USH2A, so we do expect to target the same level of editing because our rationale for setting therapeutic targets for LCA10 were based on studies in humans that were generalized [indiscernible].

So by the same logic we expect that improving 10% photoreceptors will have a dramatic effect on the ability of these patients' vision and so that will be the target for inherited retinal disease programs.

And of course that's one of the advantages to running multiple programs in related areas so that we can take learnings from one area and apply them to others and I highlight some of those similarities in my talk. At ASGCT we will be talking as well about the LCA10 program..

Right, thank you..

Thank you. And our next question comes from Joseph [indiscernible] with Cowen and company. Your line is now open..

Hi there and thank you for taking my questions.

The first one is on the Allergan opt-in, was there something that Allergan saw in either the preclinical data packet or the nonhuman primates or as you went through the submission of the NIH materials of the IND that sort of triggered more interest or is it simply a timeline sort of decision? And then second, I know we're going to get the natural history data in the first half of 2019, but are you anticipating going into a subset of patients in the Phase 1 study and can the decisions from the RDAC meeting sort of guide the initial clinical patient experience?.

So I’ll ask Andrew to speak to how the opt-in worked in the Allergan relationship..

Sure. Yes, hey thanks for the question. So the timing of the Allergan opt in is driven by various events in the program. We haven't disclosed fully for this relationship, what the opt-in triggers are but it's not purely at their discretion any point in time to opt-in.

They do have substantial access to the information in the program on an ongoing basis and so they were absolutely well aware of everything that's going on with respect to the program when they made that opt-in and I think they made as a result of their informed decision..

And I'll ask Gerry to speak to your natural history study question?.

Sure, thanks Katrine.

We are learning things from the natural history study that are really helping us and formed the design of the Phase 1/2 study and part of that includes feasibility of [indiscernible] what ages patients may be able to perform certain assessments and all those will factor into that the design of the Phase 1/2 as well thinking a bit about the risks, benefits in adults and children and then how we enroll those patients..

Great, thank you..

Thank you. This concludes today's Q&A session. I would now like to turn the call back over to Katrine Bosley for closing remarks..

Great, thank you. And so with that I'd like to thank all of you for participating in today’s call and for your support as we work to bring the transformative new medicines to patients. So, thanks again and have a great evening. Bye..

Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program. You may all disconnect. Everyone have a great day..