Biogen has discontinued all gene therapy programs using adeno-associated virus (AAV) capsids, instead shifting resources to modalities that have the highest likelihood of achieving better treatment outcomes.
The big biotech deprioritized (PDF) early-stage gene therapies back in 2023 as part of an R&D pipeline restructuring. Now, as part of a continued capital allocation approach, the company is washing its hands of AAV tech for good.
“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,” Jane Grogan, Ph.D., Biogen’s head of research, said in a statement to Fierce Biotech. “We remain steadfast in our commitment to rare and genetic diseases and to the most promising science with the greatest potential.”
While most team members who had previously focused on AAV capsids were reassigned to other roles within the organization, a small number of employees were laid off across research titles, including pharmaceutical operations and technology positions, a spokesperson told Fierce.
According to Endpoints News, which first reported the changes, 20 staffers were impacted by the workforce reduction.
Back in January of this year, Grogan announced an undisclosed number of layoffs made to Biogen’s research team, as the company prioritized its preclinical pipeline and shifted resources to external opportunities.
The move was made to “reinvigorate the company's drug discovery capabilities and support long-term sustainable growth,” a spokesperson told Fierce at the time.
Biogen is far from being the first company to abandon AAV capsids, which are used to transport gene therapies into target cells. The technology has been successfully used in approved treatments, such as Novartis, Roche and Spark’s Luxturna for retinal disease, and Novartis’ Zolgensma for spinal muscular atrophy.
Advantages of the science are relative stability and long expression. That being said, AAV capsids are extremely costly to produce. Drugs such as Zolgensma are the 10th most expensive drug in the U.S. this year, with a $2.3 million price tag per dose. The therapies also cannot be re-dosed and are associated with certain safety concerns, such as liver toxicity.
Now, Biogen joins the likes of Roche, Takeda and Vertex Pharmaceuticals, companies that have all shed work in AAV capsid development over the last year. Meanwhile, Pfizer has dumped all gene therapy work entirely.