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Find Clinical Drug Pipeline Developments & Deals by Affinia Therapeutics
Direct administration of AAV-based Gene Therapies to the central nervous system (CNS) can increase transduction and expression in the CNS. However, even with direct administration, vector "escapes" the CNS and distributes to peripheral organs.
Lonza has worked with Affinia Therapeutics to achieve a four-fold improvement in yield for the company’s lead capsid, Anc80L65. Lonza and Affinia Therapeutics are partnering with the goal of ensuring flexible manufacturing capacity for the next several years.
Proceeds to support continued development of proprietary platform for rationally designed adeno-associated virus vectors (AAV) and advance programs into the clinic.
Affinia Therapeutics’ proprietary AAV vector technology to be used in Vertex’s genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis.
The proceeds will be used to advance the company’s platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.