Biogen will partner with Capsigen to engineer novel adeno-associated virus (AAV) capsids for gene therapies designed to treat CNS and neuromuscular disorders, through a strategic research collaboration that could generate more than $1.3 billion for Capsigen.

The companies said they will create and identify novel AAV capsids tailored to meet disease-specific transduction profiles through their collaboration, designed to combine Capsigen’s Transcription-Dependent Directed Evolution (TRADE™) platform and associated technologies with Biogen’s discovery, development, manufacturing, and commercialization capabilities.

Capsigen’s technology is designed to produce dose optimized, fit for purpose vectors that may have applicability across Biogen’s gene therapy pipeline.

“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,” Alfred Sandrock, Jr., MD, PhD, Biogen’s head of research and development, said yesterday in a statement. “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.”

Capsigen has agreed to apply its vector engineering approaches to develop novel capsids designed to meet highly customized, disease-specific transduction profiles. Capsigen also agreed to issue Biogen an exclusive license to apply Capsigen’s technology for an undisclosed number of CNS and neuromuscular disease targets.

In return, Biogen agreed to pay Capsigen $15 million upfront, up to $42 million in potential research milestone payments, and up to an additional $1.25 billion in potential development and commercial payments tied to the collaboration programs achieving development and sales milestones.

Capsigen is also eligible to receive royalties on future net sales of products that incorporate capsids resulting from the collaboration.

By partnering with Capsigen, Biogen becomes the latest biopharma giant to join the scramble to develop improved AAV capsids for gene therapy.

Just last week, Dyno Therapeutics—which has inked partnerships totaling up-to-$4 billion with Novartis, Sarepta Therapeutics, and Roche and its Spark Therapeutics subsidiary—completed a $100-million Series A financing. The proceeds are intended to fund development of enhanced AI-based capsids designed through Dyno’s CapsidMap™ platform, a delivery platform developed in the lab of George M. Church, PhD, of Harvard Medical School, one of Dyno’s co-founders and the chair of its scientific advisory board.

Last month, LogicBio Therapeutics granted CANbridge Pharmaceuticals a worldwide license for AAV sL65, the first capsid based on LogicBio’s sAAVy™ platform, to support development of CANbridge gene therapy programs for Fabry disease and Pompe disease, plus options for two additional indications. In return, CANbridge agreed to pay LogicBio $10 million upfront, up to $581 million in options payments plus clinical, regulatory, and commercial milestone payments; plus up to double-digit royalties on net sales.

In February, Munich-based Sirion Biotech launched a collaboration of undisclosed value with Sanofi to develop new and modified AAV capsids for gene therapies targeting multiple unspecified “life-threatening disorders” affecting major human organs. Two months earlier, 4D Molecular Therapeutics went public through an initial public offering that raised $204.7 million in net proceeds.

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

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