Allergan and Editas exercise CRISPR option
- Editas Medicine and Allergan Pharmaceuticals have exercised their option for Editas' CRISPR drug. Allergan will develop and commercialize EDIT-101 globally, and Editas will co-develop and share profits and losses in the U.S.
- Allergan has paid an option exercise fee of $15 million, and Editas will pick up a $25 million milestone payment from its big pharma partner upon clearance of an Investigational New Drug (IND) application for EDIT-101.
- Editas has already submitted a data package for human gene transfer clinical protocol registration to the National Institutes of Health for review by the Recombinant DNA Advisory Committee, and plans to file an IND in October 2018.
Allergan and Editas signed their deal in March 2017. Things have moved on apace, with the lead candidate EDIT-101 heading towards the clinic for patients with Leber congenital amaurosis (LCA10), a genetic eye disease that is one of the most common causes of inherited sight loss in children. The disease can also lead to tunnel vision, night blindness, nystagmus and poor pupil reaction. It affects around one in 80,000 people.
The Allergan-Editas deal covers up to five candidates, though, meaning more R&D and commercialization news may be on the horizon. Editas took home $90 million upfront through the transaction, and is eligible for milestone payments that have already started to come in .
"During the second quarter, we continued to drive towards our first IND and to advance our broader pipeline of transformative CRISPR medicines," said Katrine Bosley, president and CEO, Editas Medicine, in a statement releasing second quarter earnings on Monday. "Our lead candidate, EDIT-101 to treat the genetic disease LCA10, is poised to be the first in vivo CRISPR medicine in human trials with an anticipated IND filing in October. Our broader pipeline of ocular and engineered cell medicines is advancing as well."
The CRISPR-Cas9 route to the clinic hasn't been completely straightforward. Editas had a setback in its IND filing in May 2017 as a result of delays at a third-party manufacturer. And a number of CRISPR-related companies, including Editas, saw their stock value tumble in June when a pair of preclinical studies raised concerns about cancer risks associated with the CRISPR-Cas9 system and suppression of the p53 gene.
At the time, an Editas spokesperson said: "[W]e can now achieve much higher targeted integration rates and gene correction without suppressing p53 than the authors of this paper saw in their experiment. Thus, for making medicines, we do not believe p53 suppression is appropriate or needed to achieve high levels of gene correction."
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