Biogen Discontinues AAV Gene Therapy Programs, Shifts Focus to High-Potential Modalities

Biogen, a leading biotechnology company, has announced the discontinuation of all gene therapy programs utilizing adeno-associated virus (AAV) capsids. This strategic move, part of the company's ongoing efforts to restructure its R&D pipeline and allocate capital more efficiently, marks a significant shift in Biogen's approach to rare and genetic diseases.

Restructuring and Resource Reallocation

The decision to end AAV gene therapy work has prompted a team restructure within Biogen. While most team members previously focused on AAV capsids have been reassigned to other roles within the organization, a small number of employees across various research titles, including pharmaceutical operations and technology positions, have been laid off. According to reports, approximately 20 staffers were affected by this workforce reduction.

Jane Grogan, Ph.D., Biogen's head of research, emphasized the company's commitment to a disciplined approach in capital allocation. "In building the New Biogen, we are taking a disciplined approach to capital allocation, directing our resources to pioneer modalities and medicines that have the highest probability of delivering better outcomes for patients," Grogan stated. She further added, "We remain steadfast in our commitment to rare and genetic diseases and to the most promising science with the greatest potential."

Industry Trend: Moving Away from AAV Capsids

Biogen's decision to abandon AAV capsid technology aligns with a broader industry trend. Several other pharmaceutical giants, including Roche, Takeda, and Vertex Pharmaceuticals, have also recently discontinued their work on AAV capsid development. Pfizer has gone even further, ceasing all gene therapy work entirely.

While AAV capsids have been successfully used in approved treatments such as Novartis, Roche, and Spark's Luxturna for retinal disease and Novartis' Zolgensma for spinal muscular atrophy, the technology presents significant challenges. AAV-based therapies are extremely costly to produce, with drugs like Zolgensma ranking among the most expensive in the United States, carrying a price tag of $2.3 million per dose. Additionally, these therapies cannot be re-dosed and are associated with certain safety concerns, including liver toxicity.

Despite these drawbacks, AAV capsids offer advantages such as relative stability and long expression. However, the high production costs and associated risks have led many companies to explore alternative approaches in the field of gene therapy and rare disease treatment.