Viralgen Vector Core, Elpida Partner on SPG50 and CMT4J Clinical Trials

Viralgen Vector Core and Elpida Therapeutics have joined forces to manufacture gene therapies for use in clinical trials sponsored by Elpida Tx involving patients living with Spastic Paraplegia 50 (SPG50) or Charcot-Marie-Tooth disease type 4J (CMT4J). The trials will take place at various sites in North America and Europe and will explore potential safety and efficacy of the new treatments to be manufactured, as well as potential quality of life improvements.

SPG50 is a rare disease caused by mutations in the adaptor protein complex-4 mu 1 subunit (AP4M1). The disease symptoms of children with SPG50 begin with low muscle tone, microcephaly (small head) and epilepsy, then progress to affect the entire body, resulting in intellectual disabilities and movement/neurological abnormalities. 

CMT4J is an autosomal recessive, potentially severe form of CMT disease caused by mutations of the lipid phosphatase FIG4, a protein involved in the regulation of phosphoinositide lipids and membrane trafficking within cells. The same CMT4J genotype can present symptoms ranging from mild clinical signs to severe disability.

The partnership aims to enable the efficient manufacture of the adeno-associated vector serotype 9 (AAV9) based therapies for use in Elpida Tx’s anticipated multi-site clinical trials. Viralgen will manufacture these therapies using its production process with the Pro10 cell line, which is exclusively licensed from AskBio and has been found to increase scalability, performance, and yield of AAV therapies.

Terry Pirovolakis, founder, and CEO of Elpida Tx, said, “Viralgen is truly an amazing partner, demonstrating their genuine commitment to advance potential therapies for impacted patients. We feel so grateful and honored to be working with such an incredible partner.”