According to foreign media reports, Biogen has discontinued all gene therapy programs based on adeno-associated virus (AAV) capsids.
In fact, Biogen had already deprioritized gene therapies back in 2023. Now, as part of its ongoing capital allocation strategy, the company is completely abandoning AAV technology.
Biogen, a biotechnology company with a strong foundation in rare and genetic diseases, stated that it remains committed to this field and will focus its resources on advanced therapies and medicines with greater potential to deliver better outcomes for patients.
While most team members previously responsible for AAV vector development have been reassigned to other roles within the company, a small number of employees faced layoffs, affecting research, operations, and technical positions.
Biogen is not the first company to abandon AAV technology.
AAV (adeno-associated virus) is a commonly used viral vector for delivering gene therapies, with advantages including relative stability and long-term expression.
To date, several gene therapies based on this technology have been approved for market use, such as Roche's Spark Therapeutics' Luxturna for retinal diseases and Novartis' Zolgensma for spinal muscular atrophy.
However, AAV gene therapies also face significant challenges in cost, immunogenicity, and safety. The "sky-high" treatment prices of $1–3 million present a stark contradiction to their uncertain clinical benefits. This has led to a "rational adjustment period" in the field, with some companies choosing to scale back or exit, shifting resources to other areas with greater certainty.
Takeda Pharmaceuticals ended its early-stage AAV research in 2023.
Roche also implemented a "fundamental restructuring" of its gene therapy unit, Spark Therapeutics, writing down $2.4 billion in goodwill.
Earlier this year, Pfizer withdrew its AAV gene therapy Beqvez for hemophilia B from the market. The product, which had only received FDA approval in April 2024 for treating hemophilia B with a price tag of $3.5 million, was abandoned by Pfizer less than a year after its launch.
Similarly, Vertex announced this year that it would discontinue research using AAV as a gene therapy vector.
Now, Biogen has joined the ranks, becoming another major pharmaceutical company to exit AAV technology.
The once "star" vector is facing a winter, with giants collectively shifting direction. However, this does not negate the future of gene therapy but may instead encourage the industry to invest capital in more promising new technologies, fostering the emergence and growth of next-generation gene therapies.
Reference :https://www.fiercebiotech.com/biotech/biogen-officially-ends-all-aav-gene-therapy-work-prompting-team-restructure
