The cell and gene therapy space in recent months has hit several speedbumps, including layoffs, dropped drugs and discontinued partnerships.
Vertex Pharmaceuticals will no longer work on the adeno-associated virus vector technology commonly used to package and deliver gene therapies, according to a Friday report from Endpoints News.
BioSpace has reached out to Vertex for independent confirmation of the news and will update this article accordingly.
The AAV pullback comes after Vertex in February turned its back on a deal it had signed with Verve Therapeutics, handing back the rights to an in vivo liver disease gene editing program. The 2022 deal cost Vertex $60 million upfront, though the partners never unveiled their specific disease target. In late March, Vertex also discontinued its pancreatic islet cell therapy, packaged in a proprietary immunoprotective device, which it was proposing as a therapy for type 1 diabetes.
Terminating work on the AAV platform could also imperil other Vertex partnerships, including its 2020 contract with Affinia Therapeutics to develop novel AAVs for gene therapies—though Vertex has yet to confirm whether that deal would indeed be affected. According to its press announcement at the time, Vertex is using Affinia’s AAV vector technology to package and deliver its gene therapies for a variety of indications, including Duchenne muscular dystrophy and myotonic dystrophy type 1.
Vertex’s decision to do away with its AAV work continues what has been a rough few months for the cell and gene therapy space.
Several notable players have reduced their workforces in recent months, including Encoded Therapeutics, Intellia Therapeutics and Editas. Editas, in particular, was also forced to can its sickle cell disease gene therapy renizgamglogene autogedtemcel after a fruitless search for a development sponsor.
Pfizer, one of the heaviest hitters in the arena, announced in February that it would abandon its FDA-approved hemophilia B gene therapy Beqvez across all global markets, pointing to “the limited interest patients and their doctors have demonstrated in hemophilia gene therapies.” A month earlier, Pfizer turned its back on development partner Sangamo Therapeutics, with which it was advancing another hemophilia gene therapy.
Still, there are some positive signs on the horizon for gene therapies, not least of which is the apparent openness of new FDA Commissioner Marty Makary to some regulatory flexibility for gene therapies, particularly for rare diseases. In an interview last month with Megyn Kelly, Makary said the FDA could consider drug approvals based on a “plausible mechanism,” particularly for diseases that affect “a small number of people.”
