#USA #Indianapolis #Genezen #AAV9 Gene Therapy #Raiden Science Foundation

Genezen Collaborates with Raiden Science Foundation for UBA5 Gene Therapy

In a groundbreaking development in the field of gene therapy, Genezen, a leading contract development and manufacturing organization (CDMO), has officially partnered with the Raiden Science Foundation (RSF) to manufacture an AAV Serotype 9 gene replacement therapy targeting UBA5 disorder. This collaboration represents a significant leap forward for RSF, transitioning its UBA5 gene therapy program from four years of research and development directly into clinical manufacturing, setting the stage for a pioneering human clinical trial against this rare and severe disorder.

Understanding UBA5 Disorder

UBA5 disorder is an extremely rare and progressive neurodevelopmental condition, affecting fewer than 100 children globally. It leads to life-altering symptoms such as severe disability and seizures due to mutations in the UBA5 gene, which plays a critical role in cellular breakdown and function. Children diagnosed with this disorder face challenges from early infancy, including hypotonia, spasticity, dystonia, intractable seizures, and significant developmental delays. Currently, there are no approved therapies to treat UBA5 disorder, highlighting an urgent need for effective treatments.

Founded in 2021, the Raiden Science Foundation aims to dismantle the conventional barriers to research in order to expedite medical advances for children suffering from rare diseases like UBA5. Named after the Pham family's son Raiden, who was diagnosed with UBA5 disorder at 17 months, the foundation is committed to providing hope and real solutions to families affected by this debilitating condition. The partnership with Genezen marks a turning point in their journey to bring the first gene therapy for this disorder to clinical trials.

The Significance of the Partnership

Tommy Pham, Co-Founder and President of RSF, expressed his gratitude at reaching this milestone, stating, "When we started RSF, we aspired to do more than just create hope—we aimed to turn that hope into reality. Collaborating with Genezen instills confidence in us; their expertise in manufacturing AAV9 for ultra-rare diseases is invaluable in accelerating our program towards clinical trial. Importantly, they understand the unique demands of a parent-led initiative for such a rare disorder." His sentiment reflects the untold dedication shown by families impacted by UBA5 and their relentless pursuit of viable treatment options.

Gene therapies present a viable pathway for delivering potentially life-saving treatments where no standard therapies are available. However, standard manufacturing practices, often designed for larger-scale trials, fall short in addressing the personalized needs required for ultra-rare diseases such as UBA5. Genezen aims to streamline this process, leveraging its extensive experience and specialized capabilities to enhance the manufacturing of the UBA5 therapy. Their focus on ultra-rare therapies and use of agile risk-based frameworks sets them apart in the industry, allowing faster development and delivery of personalized gene treatments.

Steve Favaloro, Genezen's Chairman and CEO, noted, "The Pham family's resilience fuels our commitment to this partnership. We are proud to leverage our specialized skills to aid RSF in advancing the program, with hopes that it will soon help Raiden and others living with UBA5. This collaboration marks a critical step toward fulfilling the needs of families striving for solutions in the face of rare diseases."

The project has attracted significant industry support, including alignment with Aurelix Bio, which aids the transition from scientific discovery to clinical application. Brandon M. Henry, MD, Chief Executive and Medical Officer at Aurelix Bio, complemented the initiative's dedication: "Even amid the challenges of caring for a child with UBA5 disorder, the Pham family remains committed to advancing this therapy and offering support to other families in similar circumstances."

Looking Ahead

This partnership is a beacon of hope for families touched by UBA5 disorder. As both organizations gear up for the upcoming phases, including clinical trials, their collaborative efforts underscore the urgency and necessity of finding innovative treatments for ultra-rare diseases. By combining their strengths, Genezen and RSF not only aim to develop groundbreaking therapies but also to build a supportive community that empowers affected families to champion their cause.

For further information about their initiatives, visit Genezen or explore the Raiden Science Foundation's mission at RSF.