Genezen and the Raiden Science Foundation Announce Partnership for AAV9 Gene Replacement Therapy for UBA5 Clinical Trial

INDIANAPOLIS and BEAVERTON, Ore., Aug. 3, 2026 /PRNewswire/ -- Genezen, a best-in-class gene therapy contract development and manufacturing organization (CDMO), and the Raiden Science Foundation (RSF), a parent-led rare disease organization, today announced a manufacturing partnership for an AAV Serotype 9 gene replacement therapy for UBA5 disorder. This partnership marks a major milestone for RSF, moving its UBA5 gene therapy program from four years of R&D into clinical manufacturing and one step closer to the first-in-human clinical trial for this devastating disorder.

UBA5 disorder is an ultra-rare, progressive neurodevelopmental disorder that leads to severe disability and seizures, with fewer than 100 children diagnosed worldwide. UBA5 itself is a gene that encodes instructions critical for cellular breakdown and function. Mutations in the UBA5 gene result in protein malfunction, posing life-threatening risks and a reduction in quality of life as early as infancy. Symptoms include hypotonia, spasticity, dystonia, intractable seizures, developmental delays, gastrointestinal dysfunction, respiratory distress, and vision impairment. There are currently no approved therapies for UBA5 disorder, underscoring the urgency for an effective treatment option.

Founded in 2021, RSF aims to overcome traditional research barriers to advance treatments and accelerate medical breakthroughs to help children around the world who are suffering from UBA5 disorder and other rare diseases. Founded by Tommy and Linda Pham, the foundation is named for their son Raiden who was diagnosed with UBA5 disorder at 17 months of age on August 2, 2021. Working alongside scientific, regulatory, manufacturing and clinical partners, RSF is building a pathway to bring the first gene therapy for UBA5 disorder into clinical trial. This partnership with Genezen represents a significant milestone for RSF and marks a new phase in the organization's efforts, as they continue to fundraise and bring together industry and philanthropic partners to support the development, regulatory and clinical work needed.

"We are incredibly grateful to have reached this stage in our journey, moving from bench toward bedside for Raiden and other children affected by UBA5 disorder. When we started RSF, we said we wanted to create more than hope, and today, we are doing exactly that. Collaborating with Genezen 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," said Tommy Pham, Co-Founder and President of Raiden Science Foundation. "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."

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, such as in the case for RSF's UBA5 therapy. The partnership with Genezen intends to provide an accelerated path for production of the UBA5 therapy 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 has proven experience 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," said Steve Favaloro, Chairman and Chief Executive Officer at Genezen. "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. We are truly honored to partner with the Raiden Science Foundation and other early parent-founded biotechs to help cure rare and ultra-rare diseases."

The program brought together partners from across the industry, including Aurelix Bio, a rare disease drug development partner helping 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, who also serves as Chief Drug Development Officer for Raiden Science Foundation. "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'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.

About Genezen
Genezen is a best-in-class gene therapy CDMO with over 12 years of experience and state-of-the-art viral vector manufacturing facilities. From concept to commercial scale, Genezen partners with innovators to deliver life-saving gene and cell therapies worldwide. Learn more at genezen.com or follow Genezen on LinkedIn.

About Raiden Science Foundation
The mission of RSF is to harness the power of community to develop cures for rare diseases, starting with UBA5 disorder. Our vision is to create a future where there are paths forward for treating rare diseases and empowering families to make a difference. For more information and to support RSF, please visit www.raidenscience.org/

About Aurelix Bio
Aurelix Bio is a clinical development platform built to accelerate rare disease therapies from concept to clinic. Aurelix partners with patient advocacy organizations, early-stage biotech companies, and academic teams navigating critical translational inflection points, bringing disciplined program stewardship, strategic decision-making, and predictable progress to areas where resources are constrained but urgency is high. For more information, please visit https://aurelixbio.com/

SOURCE Genezen