Dive Brief:

  • Editas Medicine has regained full rights to a group of experimental CRISPR-based gene editing treatments for eye diseases, after partner AbbVie agreed to rework the terms of a research collaboration.
  • AbbVie inherited the deal through its acquisition last year of Allergan, which in 2017 paid Editas $90 million to gain access to some of the biotech's CRISPR research. The agreement included an option to Editas' lead therapy for a rare form of inherited blindness, which is one of just two experimental CRISPR therapies in company-led clinical trials.
  • Editas is one of the leaders in the high-profile race to use the gene editing technology to develop medicines. Its study is the first to use a CRISPR medicine in an attempt to alter genes inside the body, rather than in cells manipulated in a laboratory. Enrollment was temporarily paused due to the coronavirus pandemic, but should soon restart, the company said Thursday.

Dive Insight:

When Editas launched in 2013, it became the first startup formed to use CRISPR gene editing, which had electrified the world of biological research, to develop human medicines.

Editas was quickly followed by others, among them CRISPR Therapeutics and Intellia Therapeutics, as venture investors, and later on, large pharmaceutical companies were drawn to the technology's potential to fix genetic errors.

One of those drugmakers was Allergan, which took a bet on the first treatment to emerge from Editas' initial research. The treatment targets an ophthalmic disease called Leber congenital amaurosis 10, which is similar to the condition treated by Luxturna, the first gene replacement therapy approved in the U.S.

The deal gave Allergan an option to license that treatment and up to four others, each for different eye diseases. A regulatory filing shows that Editas has since received another $40 million tied to the therapy

While the study of that first drug, called EDIT-101, was opened last summer, the first patient wasn't treated until March of this year. In the intervening months, Allergan agreed to be acquired by AbbVie, leaving the company's existing partnerships subject to the preferences of a new management team.

The Allergan-AbbVie deal closed in May and, three months later, AbbVie returned to Editas rights to EDIT-101 as well as other therapies for other forms of Usher syndrome and retinitis pigmentosa.

Editas CEO Cynthia Collins, on a conference call, wouldn't specify what led to the decision, but implied it was AbbVie's call. "I certainly don’t want to speak on behalf of AbbVie or speculate what the rationale was," she said, "but we were certainly thrilled that they identified and believed that we were the best home for these assets and for this portfolio, given that the technology had originated within Editas."

The two have started a new deal, and while terms won't be disclosed this quarter, it appears to involve a royalty payment Editas will have to pay AbbVie on the returned programs, should any ever get approved. One payment due this quarter "is not material to our cash," said chief financial officer Michelle Robertson.

Soon after Editas and Allergan dosed the first patient in its study, enrollment was paused due to the coronavirus pandemic.

Results are not yet available for that patient, but Editas chief scientific officer Charles Albright said Thursday the patient "remains stable" after three months.

EDIT-101 is designed to work by deleting a genetic mutation that prevents the eye from producing a critical protein. The therapy is injected, working inside the body, unlike another pioneering treatment from CRISPR Therapeutics, which genetically alters stem cells harvested from patients.

Collins said trial sites have been cleared to dose new patients, and Editas hopes to report data on the first patients treated later this year.

By then, Editas also plans to ask the Food and Drug Administration to start a trial for a sickle cell disease therapy that would rival the CRISPR Therapeutics program CTX001, which has shown encouraging results thus far in early testing.



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