Published: Feb 03, 2021
- First potentially disease-modifying gene therapy for GM2 gangliosidosis to enter clinical studies
- Expect to continue patient identification, screening, and enrollment in Stage 1 of the study throughout 2021
NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 2021 (GLOBE NEWSWIRE) Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2, an investigational gene therapy for the treatment of GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease.
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Massachusetts
United-states
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