Biogen and Capsigen announced that they have entered into a strategic research collaboration to engineer novel adeno-associated virus (AAV) capsids that have the potential to deliver transformative gene therapies that address the underlying genetic causes of various CNS and neuromuscular disorders.

As a part of the collaboration, Capsigen’s proprietary TRADE platform and associated technologies will be utilized with the aim to create and identify novel AAV capsids tailored to meet disease-specific transduction profiles. Capsids are the protein coat that protects and facilitates the delivery of the virus’ genetic payload into host cells. The collaboration will leverage Capsigen’s capsid engineering expertise and Biogen’s discovery, development, manufacturing and commercialization capabilities with the goal to accelerate delivery of gene therapies to patients in need.

“Through this collaboration, we aim to solve key technological challenges in the delivery of gene therapies to target tissues. One of our priorities for technology innovation is the discovery of AAV capsids with improved delivery profiles,” said Alfred Sandrock, Jr., M.D., Ph.D., Head of Research and Development at Biogen. “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.”

“At Capsigen, we believe the next revolution in gene therapy will be driven by engineered AAV capsids designed to meet disease-specific transduction profiles,” said John Bial, chief executive officer. “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.”

Under the terms of the agreement, Capsigen will apply its vector engineering approaches to develop novel capsids designed to meet highly customized, disease-specific transduction profiles. Biogen will receive an exclusive license under Capsigen’s proprietary technology for an undisclosed number of CNS and neuromuscular disease targets. Capsigen will receive a US$ 15 million upfront payment and is eligible to receive up to US$ 42 million in potential research milestones and up to an additional US$ 1.25 billion in potential development and commercial payments should the collaboration programs achieve certain developmental milestones and sales thresholds. Capsigen is also eligible to receive royalties on future net sales of products that incorporate capsids resulting from the collaboration.