Gene Therapy Shifts: Vertex and BMS Adjust Strategies

Vertex Pharmaceuticals has become the latest company to scale back its research efforts focused on adeno-associated virus (AAV) vectors, a common delivery mechanism for gene therapies. A Vertex spokesperson confirmed on Friday that the company has decided “not to continue AAV as a delivery mechanism for our genetic therapy programs.”

Despite this shift away from AAVs, Vertex emphasized its ongoing commitment to cell and genetic therapies. The company will proceed with the global launch of its sickle cell disease and beta thalassemia gene therapy, Casgevy, continue the Phase 3 development of its islet cell therapy, zimislecel, for Type 1 diabetes, and maintain research investments in muscular dystrophy and other diseases. The news of Vertex’s AAV pullback was initially reported by Endpoints News. This decision follows Vertex’s recent discontinuation of another diabetes candidate and its withdrawal from an in vivo gene editing collaboration with Verve Therapeutics.

In related news, Bristol Myers Squibb (BMS) is also recalibrating its gene therapy manufacturing strategy. Roughly two years after acquiring a former Novartis facility in Libertyville, Illinois, to support the production of viral vectors for its CAR-T cancer treatments, Breyanzi and Abecma, BMS has announced plans to cease vector production at the site. The Libertyville facility, previously utilized by Novartis for the gene therapy Zolgensma, was acquired by BMS in early 2023 to enhance its viral vector capacity for its commercial cell therapy portfolio.

A BMS spokesperson stated that internalizing viral vector production remains a key priority for the company, which has continued to expand in this area over the past year. Consequently, BMS intends to consolidate its vector manufacturing efforts from the Libertyville site to its other cell therapy plant located in Devens, Massachusetts. The spokesperson acknowledged the “difficult decision” to cease vector manufacturing in Libertyville, a site that had operated at a high standard for BMS Cell Therapy. The decision has impacted some employees, though specific layoff numbers were not disclosed. BMS is currently “evaluating options” for the Libertyville plant and is in discussions with its landlord regarding lease plans. The Libertyville location is no longer listed on BMS’ online sitemap.

BMS’s Devens facility, a 700,000-square-foot plant, handles both process development and commercial manufacturing. An FDA clearance in 2023 enabled the Devens campus to begin producing commercial cell therapies. BMS also has cell therapy manufacturing facilities in New Jersey and Washington. This production pivot aligns with a broader cost-cutting initiative by BMS, which aims to save $2 billion by the end of 2027, adding to a previous $1.5 billion savings plan announced in April 2024. The earlier initiative involved over 2,000 layoffs, and the more recent effort is expected to result in further job reductions.

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