Genezen and the Raiden Science Foundation have announced a manufacturing partnership for an AAV9 gene replacement therapy for UBA5 disorder, moving the program closer to a first-in-human clinical trial. The agreement marks a major milestone for the parent-led foundation, which has spent four years advancing the therapy from research into clinical manufacturing.
UBA5 disorder is an ultra-rare, progressive neurodevelopmental disease that causes severe disability and seizures and has been diagnosed in fewer than 100 children worldwide. Mutations in the UBA5 gene disrupt critical cellular function and can lead to symptoms including hypotonia, spasticity, dystonia, intractable seizures, developmental delays, gastrointestinal dysfunction, respiratory distress and vision impairment. There are currently no approved therapies for the disorder.
Raiden Science Foundation, founded in 2021 by Tommy and Linda Pham after their son Raiden was diagnosed with UBA5 disorder at 17 months old, said the partnership represents a new phase in its effort to bring the first gene therapy for the disease into the clinic. Tommy Pham said the collaboration gives the foundation confidence because of Genezen’s experience in AAV9 manufacturing for rare and ultra-rare diseases and its ability to adapt to the needs of a parent-led foundation.[
Genezen said the program will benefit from its viral vector technical expertise, in-house analytical methods and experience with agile, risk-based chemistry, manufacturing and controls frameworks tailored to personalized gene therapies. Chief Executive Officer Steve Favaloro said the company is proud to help accelerate the therapy into the clinic for children affected by UBA5 disorder.
Aurelix Bio, a rare disease drug development partner, is also supporting the program through its path-to-clinic model. The foundation said it continues to fundraise and build partnerships to support the regulatory, development and clinical work needed to advance the therapy.