- Genezen and the Raiden Science Foundation have entered a manufacturing partnership to advance an AAV9 gene replacement therapy for UBA5 disorder into clinical manufacturing.
- The partnership moves RSF’s UBA5 gene therapy program from four years of research and development toward a first-in-human clinical trial.
Genezen has entered a manufacturing partnership with the Raiden Science Foundation (RSF) to support clinical manufacturing of an AAV Serotype 9 (AAV9) gene replacement therapy for UBA5 disorder. The collaboration advances RSF’s gene therapy program from four years of research and development into clinical manufacturing, with the aim of supporting a first-in-human clinical trial. The agreement expands Genezen’s role as a gene therapy CDMO providing contract manufacturing services for ultra-rare disease programs.
UBA5 disorder is an ultra-rare progressive neurodevelopmental disorder affecting fewer than 100 diagnosed children worldwide, according to the companies. The disorder has no approved therapies. Genezen said it will provide manufacturing support using its viral vector expertise, in-house analytical methods, and manufacturing capabilities for ultra-rare therapies to help accelerate production of the investigational therapy.
“We are inspired by the Pham family’s resilience and dedication to develop a therapy for their son and other children diagnosed with UBA5 disorder. By combining our ultra-rare manufacturing and regulatory experience, Genezen is uniquely positioned to serve customers like the Raiden Science Foundation and is proud to help accelerate this program into the clinic for Raiden and other children facing this disease.”
Steve Favaloro, Chairman and Chief Executive Officer at Genezen
The Raiden Science Foundation, established in 2021 by Tommy and Linda Pham after their son Raiden was diagnosed with UBA5 disorder, is working with scientific, regulatory, manufacturing, and clinical partners to advance the therapy into clinical testing. The organization said the partnership represents a new phase in its efforts to support the program’s development, regulatory activities, and clinical work.
The program also includes support from Aurelix Bio, which is providing drug development services through its path-to-clinic model. Genezen said the collaboration reflects its strategy to support parent-led foundations developing therapies for rare and ultra-rare diseases through end-to-end manufacturing capabilities.
