TSHA-118

TSHA-118 is an investigational AAV9-based gene therapy designed to treat CLN1 disease, also known as infantile Batten disease. Developed by Taysha Gene Therapies, the therapy utilizes a self-complementary AAV9 viral vector to deliver a functional copy of the CLN1 gene, which encodes the lysosomal enzyme palmitoyl-protein thioesterase-1 (PPT1). By restoring PPT1 enzyme production, TSHA-118 aims to address the underlying genetic cause of this neurodegenerative lysosomal storage disorder. The therapy is administered intrathecally. Although Taysha deprioritized the program in 2022 to focus on other candidates, it has received several regulatory designations, including Orphan Drug and Fast Track from the FDA, and has been used in investigator-initiated clinical settings.