Gilmore O’Neill will become President and CEO of Editas Medicine effective June 1

Gilmore O’Neill, who will become President and CEO of Editas Medicine on June 1, joined current President and CEO Jim Mullen, who will become Executive Chairman, in an exclusive interview with GEN Edge. Read Part I here

In Part II of this interview, O’Neill and Mullen discuss Editas’ search for a new chief medical officer to succeed Lisa Michaels, MD, who was immediately terminated February 7 without explanation; which programs Editas will seek to partner; Editas’ response to an analyst who questioned how the company will assess the success of EDIT-101; and the company’s workforce expansion plans.

(This interview has been lightly edited for length and clarity.)

GEN Edge: One of the short-term priorities, which you’ve talked about publicly, is the need for a new chief medical officer. Where does that recruitment effort stand?

O’Neill: That process started before the announcement, but I am working with Jim on the recruiting of a chief medical officer. It’s a very important role. There are a couple of things I want to say. One, it’s a phenomenally exciting opportunity to use an incredibly exciting technology across a number of different therapeutic areas, with many more in the future.

I think that will be very attractive to a slew of candidates. Those right candidates are strong clinician scientists who are experienced in bringing medicines from the bench into the clinic and beyond. The nice thing is that, with my 20 years of experience in bringing new medicines across a number of different modalities, from small molecules through biologics and nucleotide-based medicines, as well as experience in gene therapy and rare diseases, that person can complement me and the executive team that I’ll be leading with all its skill set, to bringing these medicines forward.

I also have a network of contacts and colleagues that has lit up since [the announcement]. There’s a whole new network. We also have a broader pool of potential candidates when you have somebody like Gilmore in the CEO role. This is Gilmore’s search to run from here. I got it started, but he will finish it off.

I’m very confident that we’ll get a high-quality candidate that’s got the right profile. It’s something that this company has lacked in the past.

GEN Edge: Gilmore, the announcement mentioned your roles in developing Amondys 45®(casimersen) and Vyondys 53® (golodirsen) at Sarepta, as well as Spinraza® (nusinersen) and Tecfidera® (dimethyl fumarate) at Biogen. What similarities does Editas have with some of the other companies where you’ve been that you can draw upon?

O’Neill: I think that Editas has very strong science. It has a very good business development leader, and I think with the executive team and the various skill sets there, which I find very impressive, I think we’re very well poised to look at and evaluate, and frankly attract business development opportunities. Jim has also spoken about that, and what plays he can help enable in the executive chair room.

Mullen: When you’ve been through as many co-development programs and partnerships that Gilmore and I have done—some of them were the same ones, some of them were different kinds of deals. We’ve seen a lot of flavors of relationships, and there’s learning that comes from that. In the end, business development is all about amplifying our ability to move products forward more quickly, because we can access expertise, we can access people, we can access, capital in areas that we might not be able to advance something as quickly.

For example, we’ve put a priority on thinking about a partner for our iPSC-derived and edited NK (immune-oncology) program. It’s a big, competitive space. There’s one where we would like to have more expertise brought to the table. Similarly, if you look at our hemoglobinopathy [portfolio], the sickle cell disease and beta thalassemia programs.

We’re a company of 280 people or so right now. We’re thinking about how to do development—looking forward, commercializing a product outside of the US is really beyond what we should be thinking about. Finding a partner that can help us in that would be great. Having worked with big pharma over so many years, they want to see that some of the initial data, which will be coming shortly.

GEN Edge: You mentioned the desire to partner the NK cell programs. Will those programs be generating data this year?

Jim Mullen, Editas’ Chairman, President and CEO, who will become Executive Chairman on June 1

Mullen: Data this year would be actually our EDIT-101 in LCA, and we’ve guided to that. We’ll have 12-month data on the mid-dose, 12-cohort six-month data. And in the high dose, we’re extending that cohort and we’ve also initiated pediatrics—we’ll have some initial safety information from the pediatric cohort as well. We haven’t set the date exactly, but it’ll be the second half of 2022.

Similarly, the initial patients in the sickle cell trial, we’ll begin to see some data on that as the year goes on.

GEN Edge: You recently announced that Editas is the first company to test a CRISPR gene editing therapy, EDIT-101, in a pediatric patient?

Mullen: That’s a very exciting advancement for a couple of reasons, not least of which is, it’s a pediatric patient. We’re working in inherited diseases, and many of these have relatively early onset, and so one would hope that, if we can intervene earlier in life, we can have a greater impact on that life.

LCA is one of those where usually it’s the parents who observe within 9-12 months after birth that there’s an issue with the child’s eyesight. Then they start the journey to figure out what that is.

[We also have] a retinitis pigmentosa (RP) product moving in preclinical studies—that’s another one where a fairly large proportion of the patients have very early onset RP, so one would hope—and also for sickle cell disease—you can intervene earlier, while you can still have a dramatic impact on the overall life of that patient. So getting to that first pediatric patient is exciting. We’ll get learnings from that that we’ll be able to apply to these other programs as they come along.

GEN Edge: On EDIT-101, a competing clinical product, sepofarsen, showed some more disappointing results earlier this year, although one analyst (SVB Leerink’s Rick Bienkowski) suggested that restoring CEP290 expression may not be sufficient to achieve a disease-modifying effect in LCA. Do you agree with that assessment?

Mullen: No, There’s a few things, one can learn about clinical trial design that are useful, but CEP290 is pretty directly implicated in this disease and the correction of CEP290 can be done. We’ve seen it done, and it will restore some amount of function. Exactly how much function is a good question. But it’s pretty clear CEP290 is implicated in this particular disease pretty significantly.

GEN Edge: You mentioned before the sickle cell and beta-thalassemia program is EDIT-301, set to enter the clinic this year. We’ve seen several candidates in clinical trials for those indications. How does EDIT-301 stand out?

Mullen: We’ve chosen a slightly different way to intervene with this. We also use a different enzyme than Cas9. We use Cas12a, which is both highly targeted and highly efficient. More importantly, we chose a site that recapitulates a naturally occurring adult phenotype, which is an adult that has persistent fetal hemoglobin levels, because they’ve got a patient that effectively we’re just mimicking with—we’re intervening. I like to think that our approach has been validated by nature.

We’ll see. We’re in the clinic and we’ll start to collect the data. I think this is an area while it’s competitive, it’s really the long-term outcomes that people should be watching for. Short term, there’s a fairly high bar, but long term it’s about keeping these patients out of the hospital, and reductions of organ damage, stroke and early mortality—those things that will ultimately shape the most beneficial products.

GEN Edge: You mentioned Editas has a workforce of about 280 people now (up from the year-end number of 264 as reported in the Form 10-K annual report for 2021). The company said it expected its clinical and development organization to grow as the candidates advance through trials. How much will that workforce increase this year?

Mullen: It will follow success, so as we have positive data for the programs we have in the clinic today, there’s not a huge increase required in the workforce. It’s really more about programs coming behind in the clinic, so EDIT-103, EDIT-202, those programs that will require more support when they’re in the clinic and more support from the CMC [chemistry, manufacturing, and controls] side, and some from the research side.

GEN Edge: Any increase from now on the commercial side or will that have to wait further data?

Mullen: On the commercial side, we have not done much work—that’s one of the areas I’ll spend a bit of my time over the next number of months, because in thinking forward about what we need to begin putting in place, as you consider commercializing these kinds of products, this can be a complex space. We haven’t done much of that.

But I’ve built virtually every one of Biogen’s commercial organizations outside the U.S., so I’ve got a fair bit of experience thinking about how to commercialize products in different markets around the world.

Previous articleFast and Efficient Plastic-Degrading Enzyme Developed Using AI
Next articleFDA Lifts Clinical Hold on Pfizer DMD Gene Therapy Linked to Patient Death