Gilmore O’Neill probably couldn’t have picked a worse time to become a biotech CEO. The markets have been tumbling. Layoffs are spreading across the industry. Funding has dried up, and clinical data are dealing crushing blows to promising ideas.

Gilmore O'Neill (Editas)

He’s already getting a crash course in executive leadership in his sixth week as CEO of gene editing biotech Editas Medicine. But, luckily, the former Sarepta Therapeutics chief medical officer did his due diligence when he picked his opportunity.

“I looked at many companies—and while I'd emphasize how excited I was about the opportunity to work with the CRISPR technology—I will say that the other criteria on my wish list was a cash runway,” he told Fierce Biotech in an interview.

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And Editas certainly has that. The company reported more than $566 million in the bank at the end of the first quarter, which had dropped from about $620 million at the end of 2021. This will fund operations for the gene editor into 2024. O’Neill said this figure was a key consideration that tipped the scale toward Editas “given the environment we’re in.” The market turmoil will absolutely drive his decision-making.

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“I'm also very cognizant that … we're seeing things in the biotech indices that really haven't been seen before, so I'm very sensitive to that,” he said.

“I looked at many companies—and while I'd emphasize how excited I was about the opportunity to work with the CRISPR technology—I will say that the other criteria on my wish list was a cash runway." — Gilmore O'Neill, Editas CEO

O’Neill promised that under his watch, Editas will be disciplined in how it spends—but the company will also be “doubling down” on potential partnerships for its assets. He wouldn’t give away too much on what Editas might add to its existing roster of partnerships—which include tie-ups with Bayer units AskBio and BlueRock Therapeutics plus Bristol Myers Squibb—but said the biotech is aware of the potential of its clinical assets.

“We've been very clear about our desire to find partners in a way that expands our bandwidth in an efficient way to maximize the way we deliver the technologies to the general public and to patients,” he said.

Ready to engage on IP

Cash wasn’t the only thing O’Neill looked at when joining Editas. He also saw a biotech on the winning side—at least for now—of a long-running CRISPR patent lawsuit, which has put gene editing companies on one side or the other. He stressed that the intellectual property for Editas’ Cas9 and Cas12a assets is fully and exclusively licensed to the company from the Broad Institute of MIT and Harvard, which won the most recent round in the dispute surrounding how CRISPR can be used for making human medicines. Patents for the Cas9 enzyme technology have been challenged in the courts, while the latter has never been subject to a patent interference suit, O’Neill clarified.

That means that the IP for companies like Intellia Therapeutics, which holds its patents from the University of California, Berkeley (Jennifer Doudna, Ph.D.’s side of the CRISPR universe), have been brought into question. Intellia CEO John Leonard, M.D., however, has dismissed concerns about his company's patents, telling investors earlier this year that the rulings “have no bearing on how we decide what to do or what we need to do.” Leonard did admit that outside of the legal process, there may be other ways to resolve the issues because of the importance of continuing to explore CRISPR for developing human medicines.

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O'Neill is ready to engage, and he disagrees that other gene editing companies can proceed without first clearing their IP with Editas. “I actually think this is a substantial value driver, because I think many of our peers in this space will need to talk to us about this IP as they move forward,” O’Neill said.

Strategy in flight

Since landing at Editas six weeks ago, O’Neill has been “looking under the hood” and is so far happy with his choice to take over from Chairman, President and CEO James Mullen, who transitioned to the executive chairman role. O’Neill inherited a company that had just turfed its chief medical officer, Lisa Michaels. Re-hiring that role became one of O’Neill’s first goals.

Baisong Mei, M.D., Ph.D. (Editas)

The company did just that Monday, bringing on rare disease and blood disorder specialist Baisong Mei, M.D., Ph.D., from Sanofi. O’Neill said he was on the hunt for an expert with a strong translational medicine background, which he says Mei has “in spades,” plus a résumé chock-full of successful approved medicines, clinical development and drug discovery experience. Mei has three global approvals to his name, including hemophilia treatments Alprolix and Eloctate as well as Alnylam’s Onpattro.

In a joint interview in Mei’s seventh hour with the company, the new CMO described the lure of gene editing, Editas’ differentiated technology and the “top notch” drug discovery team as reasons he ultimately accepted the role. He says his previous experience in chemistry, manufacturing and controls and late-stage drug development will lend itself nicely to the early-stage nature of gene editing—after all, all drugs start with discovery.

With that window into what it takes to get a medicine approved, O’Neill said Mei can ask critical questions early on and comes with the experience of knowing what he would do differently for the next therapy.

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Mei is credited with discovering the ultimately approved hemophilia med called Jivi, marketed by Bayer, and Sanofi recently filed for approval of efanesoctocog alfa for the same blood disease, which he worked on.

“Every single day counts, because in clinical development we have so many things to do,” Mei said. A miss on just one day can mean a change in timeline, he noted.

Mei and O’Neill crossed paths earlier in their careers at Biogen, and the CEO said he was impressed by Mei’s technical clinical development and translational leadership skills then. Those skills have only grown, which means O’Neill has confidence that Editas’ drug development will be in capable hands as he navigates being a first-time CEO.

“I am here to be the CEO, and so my key job is to actually run the enterprise,” O’Neill said. Mei, meanwhile, will be laser-focused on moving the therapies EDIT-101 and EDIT-301 through the clinic. “He has an extraordinary sense of urgency and real view of the patients who are waiting for the therapeutics that this technology can give to them.”

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Starting with the vision loss therapy EDIT-101, Editas rolled out a limited data set last year that provided proof-of-concept for the technology. Mei will have the job of further proving the Leber congenital amaurosis 10 therapy works after Editas faced a bit of criticism that just one patient had experienced actual vision improvement. O’Neill said this outcome was not surprising for a phase 1 trial. The point of this early clinical research is to mine the patient population for who might respond the most so as to inform later trials. The disease also has not been well studied, which is the nature of trying to do drug development in rare disease.

“A phase one study in a rare disease for which there's been very little intervention has to carry more and do more,” O’Neill said. More data are expected in the second half of the year.

EDIT-301, meanwhile, is in proof-of-concept development for sickle cell disease and beta thalassemia, both diseases that Mei worked on at Biogen. A top-line readout for the phase 1/2 Ruby trial in sickle cell is expected by the end of the year, while the first patient will be dosed in a phase 1/2 trial for transfusion-dependent beta thalassemia this year. Editas also has therapies in much earlier development for solid tumors and neurological disorders.

With the CMO role filled, O’Neill can turn to other priorities. What those priorities are exactly, he wouldn’t say, of course, but he promised the company would have more to share later this year.

“From a point of view of the future of the company, the strategy as outlined by the company is I think in flight right now,” O’Neill said. “There are elements and assessments that I'm carrying out with the leadership right now and I will be able to talk more about that when I have been here longer.”