Amid a reckoning for the gene therapy field, Vertex Pharmaceuticals has joined a growing list of companies paring back their research efforts around the adeno-associated virus (AAV) vectors used to deliver the personalized medicines.
Vertex has elected "not to continue AAV as a delivery mechanism for our genetic therapy programs,” a company spokesperson told Fierce Biotech on Friday.
Despite the shift away from AAVs, the spokesperson affirmed that Vertex’s “commitment to cell and genetic therapies remains strong.”
The drugmaker will push forward with the global launch of its sickle cell disease and beta thalassemia gene therapy Casgevy, continue the phase 3 development of islet cell therapy zimislecel in Type 1 diabetes and maintain research investments in muscular dystrophy and other diseases, the spokesperson said.
Word of Vertex's AAV pullback was first reported by Endpoints News.
Zimislecel recently became Vertex’s lone clinical-stage islet cell asset, with the biotech dumping another diabetes candidate late last month after it failed to trigger sufficient levels of insulin production in a phase 1/2 trial.
The discontinuation of AAV development isn’t the only change Vertex has made to its gene therapy operations in recent months. The company also pulled out of an in vivo gene editing collaboration with Verve Therapeutics in February, before the partners' liver disease program reached the clinic.
Multiple other personalized medicine players have pivoted away from AAVs as the gene therapy field goes through an intense period of self-reflection. Takeda ended early-stage AAV work in 2023, and, earlier this year, Pfizer pulled the AAV-using hemophilia B gene therapy Beqvez from shelves.
With Beqvez off the market, Pfizer now has no commercial- or clinical-stage gene therapy assets.
Meanwhile, Roche recently unveiled a “fundamental reorganization” of the company’s gene therapy unit Spark Therapeutics, in a move that will cost the Swiss pharma $2.4 billion and lead to 337 employees losing their jobs.
The remaining 310 Spark employees will be folded into parent Roche.