Genezen and Raiden Science Foundation Partner to Advance First AAV9 Gene Replacement Therapy for UBA5 Disorder Toward Clinical Trial
核心洞察
Genezen (搜索) and the Raiden Science Foundation (搜索) announced a manufacturing partnership for an AAV9 gene replacement therapy (搜索) targeting UBA5 disorder (搜索), an ultra-rare neurodevelopmental condition with no approved treatments.
UBA5 disorder (搜索) affects fewer than 100 children worldwide, causing severe disability, intractable seizures, and life-threatening complications beginning in infancy.
The partnership transitions the program from four years of R&D into clinical manufacturing, bringing it closer to a first-in-human clinical trial.
INDIANAPOLIS and BEAVERTON, Ore. — Genezen (搜索), a gene therapy contract development and manufacturing organization (CDMO), and the Raiden Science Foundation (搜索) (RSF), a parent-led rare disease organization, have entered into a manufacturing partnership to produce an AAV serotype 9 gene replacement therapy for UBA5 disorder (搜索), the parties announced August 3, 2026. The collaboration moves RSF's gene therapy program from four years of research and development into clinical manufacturing, marking a decisive step toward the first-in-human clinical trial for this devastating, ultra-rare condition.
UBA5 disorder (搜索) is an ultra-rare, progressive neurodevelopmental disorder with fewer than 100 children diagnosed worldwide. The UBA5 (搜索) gene encodes instructions critical for cellular breakdown and function; mutations in this gene result in protein malfunction, posing life-threatening risks and reducing quality of life as early as infancy. Symptoms include hypotonia, spasticity, dystonia, intractable seizures, developmental delays, gastrointestinal dysfunction, respiratory distress, and vision impairment. Currently, no approved therapies exist for UBA5 disorder.
A Parent-Led Mission Gains Momentum
RSF was founded in 2021 by Tommy and Linda Pham, whose son Raiden was diagnosed with UBA5 disorder (搜索) at 17 months of age on August 2, 2021. The foundation aims to overcome traditional research barriers to advance treatments and accelerate medical breakthroughs for children suffering from UBA5 disorder and other rare diseases. Working alongside scientific, regulatory, manufacturing, and clinical partners, RSF has built a pathway to bring the first gene therapy for UBA5 disorder into clinical trial.
"When we started RSF, we said we wanted to create more than hope, and today, we are doing exactly that," said Tommy Pham, Co-Founder and President of Raiden Science Foundation (搜索). "Collaborating with Genezen (搜索) gives us great confidence. Their experience with AAV9 manufacturing for rare and ultra-rare diseases is going to help us accelerate our program to trial, and just as important, they truly understand the needs of a parent-led foundation and have found ways to adapt to the unique requirements and challenges of manufacturing a gene therapy for such an ultra-rare disorder."
Pham added: "Something that felt impossible five years ago after Raiden's diagnosis, is now so much closer to reality. Every milestone we have reached has been possible because our community, donors and partners believed in our mission."
Tailored Manufacturing for Ultra-Rare Disease
Gene therapies for ultra-rare diseases offer a pragmatic pathway to deliver potentially life-saving treatments where no standard of care exists. However, conventional manufacturing approaches — designed for scalability and larger patient population-based trials — are poorly suited to the timelines, economics, and personalization required for ultra-rare investigational new drug (IND) applications. The partnership with Genezen (搜索) intends to provide an accelerated path for production of the UBA5 (搜索) therapy by utilizing the CDMO's deep viral vector technical expertise, a complete complement of in-house custom and platform analytical vector-specific methods, and specialty in ultra-rare therapy manufacturing.
Genezen (搜索) brings over 12 years of experience and proven capability with agile, risk-based CMC frameworks tailored to enable rapid development and clinical delivery of personalized gene therapies.
"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 (搜索)," said Steve Favaloro, Chairman and Chief Executive Officer at Genezen (搜索). "By combining our ultra-rare manufacturing and regulatory experience, Genezen is uniquely positioned to serve customers like the Raiden Science Foundation (搜索) and is proud to help accelerate this program into the clinic for Raiden and other children facing this disease."
Industry Collaboration Driving Progress
The program has brought together partners from across the industry, including Aurelix Bio (搜索), a rare disease drug development partner that helps promising therapies move from scientific potential to the clinic with alignment, discipline, and momentum.
"Even while providing round-the-clock care for a child with a life-altering diagnosis, the Pham family has remained unwavering in its commitment to advancing this therapy and helping other families along the way," said Brandon M. Henry, MD, Chief Executive and Medical Officer at Aurelix Bio (搜索), who also serves as Chief Drug Development Officer for Raiden Science Foundation (搜索). "We are proud to support this program through Aurelix's integrated path-to-clinic model and to bring the same disciplined program stewardship and coordinated execution to each of our parent-foundation partnerships."
The partnership underscores Genezen (搜索)'s mission to support parent-led foundations and develop efficient, scalable pathways for ultra-rare disease programs, backed by proven end-to-end capabilities from concept through commercialization.