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Find Clinical Drug Pipeline Developments & Deals by Capsida
Under the terms of the agreement, Capsida will provide GMP manufacturing of KateTx's gene therapies as the medicines advance through preclinical and clinical development for muscle and heart disease programs.
Under the terms of the collaboration, AbbVie's extensive capabilities will be paired with Capsida's novel adeno-associated virus (AAV) engineering platform and manufacturing capability to identify and advance three programs.
Delivering AAV gene therapy systemically to target the CNS, while limiting exposure to non-target organs (such as the liver), has been a significant challenge in the gene therapy field.
Adeno-Associated Virus Engineering Platform, Platform utilizes combinatorial engineering and automation to screen in high-throughput, generating capsids greatly improved over AAV9 in CNS efficiency and specificity.
CAP-001, a novel engineered capsid packaging a human iduronate-2-sulfatase (IDS) transgene, achieved greater than 25- and 2-fold higher biodistribution than AAV9 delivered IV or intrathecally via intracisternal magna.
Under the agreement, CRISPR Therapeutics will lead R&D of the Friedreich’s ataxia program and perform gene-editing activities for both programs, and Capsida will lead research and development of the ALS program and conduct AAV vectors engineering for both programs.
Capsida also announced a multi-year strategic collaboration and option agreement with AbbVie. The collaboration is aimed at developing best-in-class, targeted gene therapies for three programs in serious neurodegenerative diseases.