Viralgen and the CTNNB1 Foundation have partnered 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 teams will focus on developing a treatment that may have the potential to enhance motor function, communication, and overall quality of life for patients with CTNNB1 Syndrome.
Špela Miroševič, founder of the CTNNB1 Foundation highlighted the importance 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. Viralgen's understanding of the urgency we feel to help CTNNB1 patients has been evident from day one, and I am honored to be working with such an outstanding team."
This progress was also supported by several organizations, including Asociación CTNNB1, which represents Spanish families affected by the Syndrome, and whose efforts have been instrumental in advancing research. Fundación Columbus also played a role in establishing key connections that made this partnership possible.
"We are happy to collaborate with the CTNNB1 Foundation," mentioned Jimmy Vanhove, CEO of Viralgen. "We are in the process of manufacturing a clinically validated vector that is expected to enter clinical trials next year. This is an important project that will bring hope to many children and families affected by CTNNB1 Syndrome."
This collaboration will enable development of this new investigational therapy, ensuring that patients may receive its benefits by mid-2025. The CTNNB1 Foundation has led the development of this gene therapy program, which will be manufactured leveraging Viralgen's innovative technologies for cGMP production. Viralgen will contribute by using its advanced AAV production processes, including its proprietary Pro10™ cell line platform, ensuring the therapy is developed at scale to meet the needs of the CTNNB1 community.