Viralgen and CTNNB1 Foundation partner to manufacture a new gene therapy for CTNNB1 Syndrome | PR Newswire [Video]

Patients with CTNNB1 Syndrome will benefit from this joint effort to develop and manufacture a new AAV investigational gene therapy, which aims to restore gene function and may improve quality of life.

, /PRNewswire/ — Viralgen and the CTNNB1 Foundation have come together to manufacture an investigational gene therapy designed to treat CTNNB1 Syndrome, a rare genetic disorder that affects motor and cognitive development in children. This partnership will bring hope to affected individuals by providing access to an investigational therapy that may have the potential to significantly improve patient outcomes.

CTNNB1 Syndrome is a rare neurodevelopmental disorder caused by mutations in the CTNNB1 gene, which plays a critical role in brain development and function. Symptoms include developmental delays, motor impairment, speech challenges, and intellectual disabilities. This collaboration will leverage Viralgen’s cutting-edge expertise in AAV manufacturing and the CTNNB1 Foundation’s commitment to advancing research in this rare disease. Together, the …