Vertex Discontinues AAV Research as Gene Therapy Sector Faces Challenges

Vertex Pharmaceuticals has announced the discontinuation of its adeno-associated virus (AAV) vector research for gene therapies, marking a significant shift in the company's approach to genetic medicine. This move comes amid broader challenges facing the gene therapy sector, with several companies reevaluating their strategies and pipelines.

Vertex's Strategic Shift

A Vertex spokesperson confirmed to Fierce Biotech that the company has decided "not to continue AAV as a delivery mechanism for our genetic therapy programs." Despite this change, Vertex emphasized that its "commitment to cell and genetic therapies remains strong." The company plans to focus on other areas of its pipeline, including:

• The global launch of Casgevy, its gene therapy for sickle cell disease and beta thalassemia
• Continued phase 3 development of zimislecel, an islet cell therapy for type 1 diabetes
• Ongoing research investments in muscular dystrophy and other diseases

This decision follows Vertex's recent exit from an in vivo gene editing collaboration with Verve Therapeutics in February, where it returned the rights to a liver disease gene editing program. The company had also discontinued its pancreatic islet cell therapy for type 1 diabetes in late March.

Industry-wide Challenges in Gene Therapy

Vertex's move reflects a broader trend in the gene therapy space, which has encountered several setbacks in recent months:

1. Workforce Reductions: Companies such as Encoded Therapeutics, Intellia Therapeutics, and Editas have implemented layoffs.

2. Program Discontinuations: Editas was forced to cancel its sickle cell disease gene therapy renizgamglogene autogedtemcel after failing to find a development sponsor.

3. Market Withdrawals: Pfizer announced in February the abandonment of its FDA-approved hemophilia B gene therapy Beqvez across all global markets, citing limited interest from patients and doctors.

4. Partnership Dissolutions: Pfizer ended its development partnership with Sangamo Therapeutics for another hemophilia gene therapy.

5. Reorganizations: Roche recently unveiled a "fundamental reorganization" of its gene therapy unit Spark Therapeutics, resulting in 337 job losses and a $2.4 billion cost to the company.

Regulatory Landscape and Future Outlook

Despite the challenges, there are signs of potential flexibility in the regulatory environment. FDA Commissioner Marty Makary has indicated openness to considering drug approvals based on "plausible mechanism," particularly for rare diseases affecting small patient populations.

As the gene therapy sector navigates these difficulties, companies are reassessing their strategies and focusing on more promising areas of research. The coming months will likely see further adjustments as the industry seeks to overcome current obstacles and advance novel therapeutic approaches.