Viralgen and CTNNB1 Foundation Partner to Develop AAV Gene Therapy for CTNNB1 Syndrome

• Viralgen and the CTNNB1 Foundation have partnered to develop and manufacture an AAV-based gene therapy for CTNNB1 Syndrome.

Viralgen and the CTNNB1 Foundation have announced a collaboration to develop an investigational gene therapy for CTNNB1 Syndrome, a rare neurodevelopmental disorder that affects motor and cognitive function. The therapy, produced using Viralgen’s AAV manufacturing platform, is expected to advance toward clinical trials next year.

CTNNB1 Syndrome is caused by mutations in the CTNNB1 gene, leading to developmental delays, motor impairment, and intellectual disabilities. The partnership will combine Viralgen’s expertise in contract manufacturing with the CTNNB1 Foundation’s research efforts to bring a potential therapy to affected patients. The therapy aims to restore gene function and may help improve motor skills, communication, and overall patient well-being.

Spela Mirosevic, founder of the CTNNB1 Foundation, emphasized the significance of the collaboration: “This partnership brings us closer to delivering a potentially life-changing gene therapy that may restore CTNNB1 function and improve the lives of those affected by this rare condition.”

The initiative has received support from organizations including Asociación CTNNB1, which represents Spanish families affected by the disorder, and Fundación Columbus, which played a role in facilitating the collaboration. Viralgen CEO Jimmy Vanhove confirmed that the company is manufacturing a clinically validated vector, with patient access anticipated by mid-2025.