CHB HEX N-terminal domain

Taysha Gene Therapies Announces Positive Initial Biomarker Data For TSHA-101, the First Bicistronic Gene Therapy in Clinical Development, Demonstrating Normalization of β-Hexosaminidase A Enzyme Activity in Patients With GM2 Gangliosidosis

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Thursday, January 27, 2022
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TSHA-101 demonstrated expression of both HEXA and HEXB genes in the endogenous ratio, providing the ability to restore and normalize enzyme activity in GM2 gangliosidosis.

Key Points: 
  • TSHA-101 demonstrated expression of both HEXA and HEXB genes in the endogenous ratio, providing the ability to restore and normalize enzyme activity in GM2 gangliosidosis.
  • We expect to provide continued updates on the program, with additional clinical data anticipated by the end of 2022.
  • Based on natural history data, patients with asymptomatic GM2 gangliosidosis have Hex A enzyme levels that are at least 5% of normal activity.
  • TSHA-101 is an investigational gene therapy that delivers both the HEXA and HEXB genes that comprise the -hexosaminidase A enzyme.

Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis

Retrieved on: 
Wednesday, September 29, 2021
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GM2 gangliosidosis is a fatal neurodegenerative disease caused by deficiency in the lysosomal enzyme -hexosaminidase A, also known as Hex A.

Key Points: 
  • GM2 gangliosidosis is a fatal neurodegenerative disease caused by deficiency in the lysosomal enzyme -hexosaminidase A, also known as Hex A.
  • GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder that is part of a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases.
  • There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.
  • Orphan designation in the European Union includes benefits such as protocol assistance, reduced regulatory fees and market exclusivity.