tidagixagene derxeparvovec

tidagixagene derxeparvovec (formerly TSHA-120) is an investigational self-complementary adeno-associated virus serotype 9 (scAAV9) gene therapy designed to treat giant axonal neuropathy (GAN), a rare, progressive, and fatal autosomal recessive neurodegenerative disorder. GAN is caused by loss-of-function mutations in the GAN gene, which encodes gigaxonin, a cytoskeletal regulatory protein essential for intermediate filament turnover. Without functional gigaxonin, intermediate filaments accumulate, leading to axonal swelling and degeneration in both the central and peripheral nervous systems. tidagixagene derxeparvovec delivers a codon-optimized human GAN transgene under the control of a minimal synthetic JeT promoter to restore functional gigaxonin expression. Originally developed at the University of Texas Southwestern Medical Center, the therapy was licensed by Taysha Gene Therapies, which partnered with Astellas Pharma. However, following FDA feedback requiring a randomized, placebo-controlled trial, Taysha discontinued clinical development of the program in September 2023. The National Institutes of Health (NIH) continues to evaluate the therapy in clinical trials.

Other names

scAAV9-JeT-GANoptscAAV-9-JeT-GANoptscAAV 9-JeT-GANoptscAAV9/JeT-GAN