US-based biotechnology firm Biogen and Capsigen have announced a strategic research deal, worth up to $1.3bn, to discover and develop novel adeno-associated virus (AAV) capsids for targeted CNS and neuromuscular disorders.

As per the terms of the agreement, Capsigen’s TRADE platform and related technologies will be used to create and identify novel AAV capsids to meet disease-specific transduction profiles.

Capsids are said to be the protein coat that safeguards and enables the delivery of the genetic payload of the virus into host cells.

The research alliance will use Capsigen’s capsid engineering know-how and Biogen’s discovery, development, manufacturing and commercialisation capabilities to fast-track the delivery of gene therapies to patients who are in need.

Biogen research and development head Alfred Sandrock, Jr. 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.”

As part of the collaboration, Capsigen will use its vector engineering methods to develop novel capsids designed to meet customised, disease-specific transduction profiles.

Biogen will get an exclusive license under Capsigen’s technology for an undisclosed number of CNS and neuromuscular disease targets.

Capsigen will receive an upfront payment of $15m and is qualified to receive up to $42m in potential research milestones as well as up to an additional $1.25bn in potential development and commercial payments.

Also, Capsigen is eligible to receive royalties on future net sales of products that feature capsids as a result of the partnership.

Capsigen CEO John Bial said: “At Capsigen, 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.

“This collaboration is consistent with our strategy to work with world-class companies to develop the next generation of gene therapies.”