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TSHA-101
- Development stage
- Phase 2
- Lead developer
- Taysha Gene Therapies
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intrathecal
01
Overview
TSHA-101 is an investigational gene therapy developed for the treatment of GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases. It utilizes a bicistronic adeno-associated virus serotype 9 (AAV9) vector to deliver both the *HEXA* and *HEXB* genes, encoding the alpha and beta subunits of β-hexosaminidase A enzyme, directly to the central nervous system via intrathecal administration. This approach aims to restore functional Hex A enzyme activity in patients with GM2 gangliosidosis, a fatal neurodegenerative lysosomal storage disorder caused by deficiency in this enzyme. There are currently no approved treatments for this disease. Developed by: Taysha Gene Therapies
02
Targets
HEXB (Beta-hexosaminidase subunit beta)HEXA (Hexosaminidase subunit alpha)
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