Taysha Gene Therapies’ TSHA-102 Receives the US FDA’s Regenerative Medicine Advanced Therapy Designation for Rett Syndrome
Shots:
- The US FDA granted RMAT designation to TSHA-102 based on safety & efficacy results from the first 3 Rett syndrome patients administered with its low dose (5.7x10^14 total vg) under REVEAL P-I/II adolescent/adult and pediatric trial
- TSHA-102 being evaluated in P-I/II (REVEAL Adult) dose escalation & expansion trial assessing the safety & preliminary efficacy in adult females with Rett syndrome in Canada & US and another P-I/II (REVEAL Pediatric) trial in pediatric females with Rett syndrome in US & cleared in UK
- TSHA-102 is a self-complementary intrathecal AAV9 gene therapy that delivers functional MECP2 to CNS cells, targeting the genetic root cause of the disease
Ref: Taysha Gene Therapies | Image: Taysha Gene Therapies
Related News:- Taysha Gene Therapies’ TSHA-102 Receives the US FDA’s Fast Track Designation for Rett Syndrome
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
