TSHA-120

TSHA-120 is an investigational gene therapy developed for the treatment of giant axonal neuropathy (GAN), a rare and progressive neurodegenerative disorder affecting both the peripheral and central nervous systems. The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the gigaxonin gene via intrathecal administration. It incorporates a novel miRNA-responsive auto-regulatory element (miRARE) platform to regulate transgene expression. Clinical studies have shown that TSHA-120 can promote nerve fiber regeneration, slow disease progression, and improve motor function in GAN patients, with long-term safety demonstrated over several years of follow-up[1][4][6]. Despite promising results, development was discontinued by Taysha Gene Therapies after FDA feedback indicated significant challenges with study design feasibility for regulatory approval in this ultra-rare disease[1][2][5].

Other names

AAV9-GANAAV-9-GANAAV 9-GANscAAV9-GANscAAV-9-GANscAAV 9-GAN