Genezen to Manufacture UBA5 Gene Therapy for Rare Disease Foundation

UBA5 disorder is a severe, progressive condition affecting fewer than 100 diagnosed children worldwide. Mutations in the UBA5 gene disrupt essential cellular functions, causing symptoms ranging from intractable seizures and spasticity to respiratory distress. Currently, no approved therapies exist for the disorder, leaving families with limited options.

Founded in 2021 by Tommy and Linda Pham after their son Raiden’s diagnosis, the Raiden Science Foundation has worked to bypass traditional research bottlenecks. By collaborating with Genezen, an experienced contract development and manufacturing organization, the foundation intends to apply agile, risk-based frameworks tailored for ultra-rare IND applications. This approach avoids the constraints of conventional manufacturing, which is typically designed for larger patient populations.

Steve Favaloro, CEO of Genezen, noted that the partnership leverages the company's specific expertise in viral vector technology and regulatory navigation. The effort also includes support from Aurelix Bio, which provides the clinical development stewardship necessary to move the therapy from the laboratory toward the clinic. For the Pham family, the transition represents a tangible shift from scientific possibility to a potential treatment pathway for their son and other children facing similar life-threatening diagnoses.