Genezen and Raiden Science Foundation Partner on UBA5 Gene Therapy Trial Manufacturing
Genezen and the Raiden Science Foundation have announced a manufacturing partnership for an AAV9 gene replacement therapy for the UBA5 clinical trial. The collaboration focuses on producing the necessary gene therapy vectors.
Genezen, a contract development and manufacturing organization (CDMO) for gene therapies, and the Raiden Science Foundation (RSF), a parent-led rare disease organization, have established a manufacturing partnership. This collaboration is focused on the AAV9 gene replacement therapy intended for a clinical trial targeting UBA5 disease.
Under the agreement, Genezen will be responsible for the manufacturing of the AAV9 vectors, which are crucial components for the gene therapy. This partnership is expected to advance the clinical development of the UBA5 therapy, aiming to address a rare genetic disorder.
UBA5 disease is a severe condition caused by mutations in the UBA5 gene, often leading to significant neurological impairment in affected individuals, particularly infants and children. The gene therapy seeks to correct this deficiency by replacing or restoring the function of the UBA5 gene.
The Raiden Science Foundation has been actively supporting research and development efforts for UBA5 disease. This manufacturing agreement with Genezen represents a key step in translating research findings into potential clinical treatments for patients suffering from this rare condition.