GM1 gangliosidosis (GM1) Archives

Taysha’s Promising Gene Therapy Results Marred by Patient’s Death

Business, Clinical Trials, Gene Therapy, GM1 gangliosidosis (GM1), Interim Data, Methicillin-Resistant Staphylococcus Aureus (MRSA), Patient Deaths, R&D, Rare Genetic Diseases, Shares, Tay-Sachs Disease

Shares of Taysha Gene Therapies were down more than 10 percent after a patient’s death marred positive interim data for the company’s experimental gene therapy treatment for Sandhoff and Tay-Sachs diseases, which are two forms of GM2 gangliosidosis.

January 28, 2022/by BioSpace

Passage Bio’s Gene Therapy for GM1 Gangliosidosis Hit with Clinical Hold

Adeno-Associated Virus (AAV), Clinical Hold, FDA, FDA/Regulatory, Gene Therapy, GM1 gangliosidosis (GM1), R&D, Rare Pediatric Disease Priority Review Voucher, University of Pennsylvania (Penn)

Passage Bio’s first attempt to develop a GM1 gangliosidosis gene therapy treatment ran into a roadblock in the form of a clinical hold.

August 13, 2020/by Andrew Humphreys