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Biogen and Capsigen to collaborate on novel gene therapies

pharmafile | May 11, 2021 | News story | Manufacturing and Production gene therapy 

Biogen and Capsigen have announced a 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.

Capsigen’s screen technology, the Trade platform, will be used as part of the collaboration to create and identify novel AAV capsids tailored to meet disease-specific transduction profiles. Capsids are the protein coat that protects and facilitates delivery of the virus’ genetic payload into host cells.

In a release the companies said the collaboration will leverage Capsigen’s capsid engineering expertise and Biogen’s discovery, development, manufacturing, and commercialisation capabilities with the goal to accelerate delivery of gene therapies to patients in need.

Alfred Sandrock, Head of Research and Development at Biogen, said: “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.

“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.”

Under the terms of the agreement, Capsigen will apply its vector engineering approaches to develop novel capsids designed to meet highly customised, disease-specific transduction profile and Biogen will receive an exclusive license under Capsigen’s proprietary technology for an undisclosed number of CNS and neuromuscular disease targets.

In a statement, the companies said that Capsigen will receive a $15 million upfront payment and is eligible to receive up to $42 million in potential research milestones, and up to an additional $1.25 billion in potential development and commercial payments, should the collaboration programs achieve certain developmental milestones and sales thresholds.

John Bial, Chief Executive Officer at Capsigen, said: “We believe the next revolution in gene therapy will be driven by engineered AAV capsids designed to meet 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.”

Kat Jenkins

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