Viralgen has partnered with Slovenia-based nonprofit, CTNNB1 Foundation, to develop and manufacture an investigational gene therapy designed to treat CTNNB1 Syndrome, a rare genetic disorder that affects motor and cognitive development in children.
The collaboration will enable development of an investigational therapy using Viralgen’s advanced AAV production processes, including its proprietary Pro10TM cell line platform. The Spain-based CDMO says it is in the process of manufacturing a clinically validated vector that is expected to enter clinical trials this year.
The CTNNB1 Foundation has led the development of the gene therapy program, supported by several organizations, including Asociación CTNNB1, which represents Spanish families affected by the syndrome.
Back in January, Viralgen, a subsidiary of AskBio, announced a partnership with Axovia Therapeutics to advance the development and manufacture of Axovia’s lead program, an AAV9-based gene therapy for patients with Bardet-Biedl Syndrome, a rare disease that can cause vision loss and blindness.
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