Editas Medicine has delayed the target date for filing an IND for its lead candidate. The setback to the Allergan-partnered CRISPR program stems from delays at a third-party manufacturer working on Leber congenital amaurosis treatment LCA10.
Cambridge, Massachusetts-based Editas had planned to file an IND for LCA10 by the end of the year. Now Editas has delayed that major moment in its short history and that of the broader CRISPR field until the middle of next year. The delay stems from a misstep in the production of a material used in the manufacture of the adeno-associated viral (AAV) vectors Editas will use to deliver its gene editing payloads.
“AAV manufacturing requires several steps to happen in perfect sequence for things to all come together. And we’re using several external contractors to perform these steps. We have to produce the input material that all comes together to then create the AAV in a cell culture systems,” Editas CTO Vic Meyer told investors.
“In our case, one of the input materials failed a quality specification and we needed to go back and remake that material. That delay in remaking the material caused us to miss the manufacturing slot with the AAV CMO and that combined with the remaking material pushed out the timeline.”
The delay will potentially see Editas fall behind CRISPR Therapeutics and Intellia Therapeutics in the race to bring a CRISPR asset to the clinic. CRISPR expects to file for clearance to test its lead beta-thalassemia candidate in Europe by the end of the year. And Intellia is on track to generate the preclinical package it needs to support a FDA nod for a study of its transthyretin therapeutic by early 2018.
Shares in Editas slipped 6% in after-hours trading following the release of news of the delay. But management is seeking to spin the setback as hiding a silver lining for the longer-term prospects of the program.
“It does create a window of opportunity to incorporate elements of Allergan’s ophthalmology preclinical development and manufacturing expertise into the program,” Editas CEO Katrine Bosley said on a conference call with investors to discuss the company's first quarter results.
Editas brought Allergan on board in March, in part to tap into the ophthalmology expertise of the larger company. Allergan paid $90 million to secure an option on five programs, including the lead LCA10 candidate.