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Taysha Gene Therapies' TSHA-101 Receives the US FDA's Orphan Drug Designation and Rare Pediatric Disease Designation for GM2 Gangliosidosis

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Taysha Gene Therapies' TSHA-101 Receives the US FDA's Orphan Drug Designation and Rare Pediatric Disease Designation for GM2 Gangliosidosis

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  • The US FDA has granted ODD and RPD to the Taysha Gene Therapies’ TSHA-101 for GM2 Gangliosidosis. The company expects the therapy to enter the clinic by the end of 2020
  • The US FDA’s two designations demonstrated the strength of the translational data package supporting TSHA-101 for GM2 Gangliosidosis
  • TSHA-101 is an AAV9-based gene therapy- currently under development for rare- neurodegenerative disease that causes a progressive dysfunction of the CNS

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: Nordic Life Science


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