Biogen Pushes Deeper into Gene Therapy with Capsigen Collaboration


Biogen and Capsigen forged a strategic collaboration to engineer novel adeno-associated virus (AAV) capsids that have the potential to become transformative gene therapies that treat underlying genetic causes of various central nervous system and neuromuscular disorders.

Biogen will harness the power of Washington-based Capsigen’s proprietary TRADE platform and technologies to identify and develop identify novel AAV capsids that are designed to meet disease-specific transduction profiles. Capsids are a protein coat used to protect and enable delivery of the genetic payload into the cellular target.

For Biogen, the collaboration with Capsigen is a continuation of the company’s push into gene therapy. In March, Biogen announced plans to build a new gene therapy manufacturing facility in North Carolina. The plant will support gene therapy manufacturing for Biogen, as well as its partner companies. Earlier this year, Biogen signed a licensing agreement with Germany-based Cevec Pharmaceuticals for its proprietary ELEVECTA Technology for the manufacturing of AAV vectors for gene therapy applications. In January, Biogen signed a gene therapy collaboration with ViGeneron GmbH, also of Germany, to develop AAV therapies for inherited eye diseases.

Alfred Sandrock, Jr., Biogen’s head of Research and Development, said the company hopes this collaboration with Capsigen will enable it to solve key technological challenges associated with the delivery of gene therapies to target tissues. The company did not disclose how many CNS and neuromuscular targets were included in the deal.

“One of our priorities for technology innovation is the discovery of AAV capsids with improved delivery profiles. We are investing for the long-term by building platform capabilities and advanced manufacturing technologies with the goal of accelerating our efforts in gene therapy,” Sandrock said in a statement.


Capsigen Chief Executive Officer John Bial pointed to AAV capsids as the future of gene therapy. He said the next revolution in gene therapy will be driven by engineered AAV capsids that are designed to meet those disease-specific transduction profiles.

“Biogen is a leader in neuroscience, and we are excited for the opportunity to work with them to potentially bring new treatments to patients. This collaboration is consistent with our strategy to work with world-class companies to develop the next generation of gene therapies,” Bial said in a statement.

Under terms of the deal, Capsigen will receive an upfront payment of $15 million and up to $42 million in potential milestones. Should the assets under the deal pass through all clinical hurdles and achieve commercialization, Capsigen could receive up to an additional $1.25 billion. Capsigen is also eligible to receive royalties on future net sales of products that incorporate capsids resulting from the collaboration, according to the deal.


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