AAV9-TECPR2

AAV9-TECPR2 is an investigational adeno-associated virus (AAV) serotype 9-based gene therapy designed to treat TECPR2-related disorder, also known as hereditary sensory and autonomic neuropathy type IX (HSAN IX). This rare, autosomal recessive neurodegenerative condition is caused by mutations in the TECPR2 gene, which is essential for autophagy and vesicular trafficking. The therapy utilizes a single-stranded AAV9 vector to deliver a codon-optimized human TECPR2 transgene under the control of a synthetic promoter (JeT). Preclinical studies have demonstrated that intrathecal administration can reduce axonal degeneration, restore autophagic homeostasis, and improve motor and sensory functions in mouse models. The program is a collaborative effort involving the TECPR2 Research Foundation, the Fierce Pierce Foundation, UT Southwestern, and Boston Children's Hospital, with manufacturing support from Forge Biologics.

Other names

AAV9-mediated TECPR2 gene replacement therapyAAV-9-mediated TECPR2 gene replacement therapyAAV 9-mediated TECPR2 gene replacement therapy