First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

Phase 1/2 Active not recruiting 3 enrolled Dr. Anupam Sehgal GlycoNet · collab Taysha Gene Therapies, Inc. · collab

NaSingle-groupOpen-labelTreatment

Summary

GM2 gangliosidoses are a group of autosomal recessive neurodegenerative diseases characterized by a deficiency of the Hex A enzyme to catabolize GM2, thereby causing GM2 accumulation within cellular lysosomes.Hex A is composed of 2 subunits, α- and β-, coded by the HEXA and HEXB genes, respectively. The primary purpose of the current study is to assess the safety and tolerability of TSHA101 administered via IT injection.

Timeline

Start: 2021-03-12
Primary completion: 2027-03-12
Completion: 2027-03-12

Publications

Ryckman AE, Deschenes NM, Quinville BM, Osmon KJL, Mitchell M, Chen Z, Gray SJ, Walia JS. Intrathecal delivery of a bicistronic AAV9 vector expressing beta-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study. Mol Ther Methods Clin Dev. 2023 Dec 5;32(1):101168. doi: 10.1016/j.omtm.2023.101168. eCollection 2024 Mar 14.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	TSHA-101	Gene therapy (AAV / viral vector)	—	Intrathecal

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

Summary

Timeline

Publications

Drugs

Indications