#Spain #San Sebastián #Viralgen #CTNNB1 Syndrome #CTNNB1 Foundation

A Collaborative Effort to Change Lives: Viralgen and the CTNNB1 Foundation Team Up

In a significant step forward for the treatment of rare genetic disorders, Viralgen and the CTNNB1 Foundation have announced a partnership aimed at developing an investigational gene therapy for CTNNB1 Syndrome. This collaboration brings a lot of hope to families affected by this rare neurodevelopmental disorder.

Understanding CTNNB1 Syndrome

CTNNB1 Syndrome arises from mutations in the CTNNB1 gene, which is crucial for brain development. Affected individuals often face various challenges, including developmental delays, motor impairments, and cognitive difficulties. There are about 500 diagnosed cases globally, but actual numbers may be underestimated. Currently, treatments are primarily focused on managing symptoms rather than targeting the underlying genetic issues.

The Partnership in Action

This partnership combines Viralgen's advanced expertise in adeno-associated virus (AAV) manufacturing with the CTNNB1 Foundation's commitment to advancing research in this rare overall spectrum of conditions. According to Špela Miroševič, founder of the CTNNB1 Foundation, this collaboration marks a vital step towards delivering a potentially transformative gene therapy. Their joint effort aims to restore CTNNB1 function, thereby improving the lives of those affected.

Notably, the partnership has garnered support from various organizations, including Asociación CTNNB1, which advocates for affected families in Spain. Their involvement has proven essential in driving research initiatives forward.

Jimmy Vanhove, CEO of Viralgen, shares the excitement and urgency of this project, emphasizing that they are currently in the process of manufacturing a clinically validated vector expected to enter trials next year. This momentum will hopefully yield beneficial outcomes for children and families facing the challenges of CTNNB1 Syndrome.

A Hopeful Future

The initiative aims to make the new therapy available to patients by mid-2025, leveraging the innovative cGMP production technologies at Viralgen. Their proprietary Pro10™ cell line platform will ensure that the treatment is developed at scale, addressing the pressing needs of the CTNNB1 community.

For many, access to this investigational therapy represents a new dawn of hope, shifting the focus from mere symptom alleviation to a genuine potential for improved quality of life.

About the CTNNB1 Foundation and Viralgen

Founded in February 2021, the CTNNB1 Foundation has rapidly expanded its efforts to support all children affected by CTNNB1 Syndrome. The Foundation has engaged over 300 researchers in pursuit of genetic solutions, raising more than 3 million euros through extensive outreach and support campaigns.

On the other hand, Viralgen, inaugurated in 2017, is a subsidiary of AskBio Inc. under the Bayer AG aegis and has developed a reputation for its expertise in the large-scale production of cGMP AAV. Their advanced facilities in San Sebastián, Spain, enable high efficiency in enhancing clinical development timelines, making them an ideal ally in this impactful journey.

Conclusion

As the world stands by, eager for advancements in genetic therapy, the partnership between Viralgen and the CTNNB1 Foundation opens up a pathway to potentially life-changing treatments. Their combined efforts signify a hopeful milestone in a field still emerging but full of promise—truly a leap towards a brighter future for those living with CTNNB1 Syndrome.