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.
San Sebastián, Spain and Ljubljana, Slovenia, February 5th, 2025 — 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 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 this 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 Pro10TM cell line platform, ensuring the therapy is developed at scale to meet the needs of the CTNNB1 community.
With this partnership, Viralgen and the CTNNB1 Foundation are poised to bring hope to families affected by CTNNB1 Syndrome.
About CTNNB1 Syndrome
CTNNB1 Syndrome is a rare neurodevelopmental disorder caused by loss-of-function mutations in the CTNNB1 gene, essential for brain development. With around 500 diagnosed cases worldwide, the true number may be higher due to misdiagnosis as cerebral palsy. The estimated incidence is 2.6 to 3.2 per 100,000 births, affecting approximately 243,000 people globally.
The syndrome leads to various developmental issues, including hypotonia, spasticity, cognitive deficits, familial exudative vitreoretinopathy (which can cause blindness), microcephaly, and behavioral challenges. Unlike cerebral palsy, CTNNB1 Syndrome is progressive and results in significant lifelong morbidity. Discovered in 2012, it has limited preclinical models, and treatments mainly focus on symptom management and physiotherapy.
About CTNNB1 Foundation
Founded by Špela Miroševič in Slovenia in February 2021 after her son Urban's diagnosis, the CTNNB1 Foundation quickly expanded to support all children affected by CTNNB1 Syndrome. After contacting over 300 researchers, the Foundation formed a team of experts to accelerate gene replacement therapy development. Through extensive media outreach, a four-year fundraising campaign, and community support, it raised 3 million euros for this initiative. The Foundation is dedicated to global inclusion, conducting key studies and pioneering research in gene therapy. The development of Urbagen, a gene therapy for CTNNB1 Syndrome, is progressing toward clinical trials, with strong collaboration from global research institutions. Successful outcomes could greatly enhance patient quality of life and benefit other rare disease therapies.
About Viralgen
Viralgen, founded in 2017 as a subsidiary of AskBio Inc. within the Bayer AG group, is a Contract Development and Manufacturing Organization (CDMO) specializing in the large-scale production of cGMP adeno-associated virus (AAV) for cell and gene therapies. Utilizing the Pro10™ suspension manufacturing platform, Viralgen achieves high titers for all AAV serotypes, enhancing clinical development and commercialization efficiency. Located in San Sebastian, Spain, its state-of-the-art facility features three cGMP suites with over 2,000L capacities, certified by the AEMPS. The company also offers advanced services for transitioning from clinical to commercial manufacturing and has recently expanded its development services to include construct and process optimization.
For more information, visit viralgen.com.
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