Vertex Discontinues AAV Vector Research, Shifts Focus in Gene Therapy Landscape

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 development. This decision comes amid broader changes in the gene therapy field, with several major pharmaceutical companies reevaluating their AAV-based programs.

Vertex's Strategic Pivot

A Vertex spokesperson confirmed to Fierce Biotech that the company has elected "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 several key areas:

• 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 strategic shift follows recent developments in Vertex's pipeline, including the discontinuation of a diabetes candidate that failed to produce sufficient insulin levels in a phase 1/2 trial.

Industry-Wide Reassessment of AAV Vectors

Vertex's decision reflects a broader trend in the pharmaceutical industry, with several companies reconsidering their AAV-based gene therapy programs:

• Takeda ended early-stage AAV work in 2023
• Pfizer recently pulled its AAV-based hemophilia B gene therapy, Beqvez, from the market
• Roche announced a "fundamental reorganization" of its gene therapy unit, Spark Therapeutics, resulting in job cuts and a $2.4 billion cost

These moves indicate a period of intense self-reflection within the gene therapy field, as companies grapple with challenges related to AAV vector technology.

Implications for Gene Therapy Development

The shift away from AAV vectors by major players like Vertex and others raises questions about the future direction of gene therapy research. While AAV has been a popular delivery mechanism for genetic therapies, companies are now exploring alternative approaches to overcome limitations and improve efficacy.

As the industry continues to evolve, it remains to be seen how these strategic changes will impact the development of new genetic treatments and the overall landscape of personalized medicine.