Taysha Gene Therapies Inc. (TSHA), a clinical-stage biotechnology company, said preclinical in vitro data on TSHA-102, a scAAV9 gene therapy developed for treating Rett syndrome will be presented at the annual meeting of the American Society of Gene and Cell Therapy (ASGCT).
Rett syndrome is a rare, neuromuscular disorder caused by a mutation in the X-linked methyl CpG-binding protein 2 (MECP2) gene, which regulates neuronal and synaptic function in the brain. Primarily occurring in females, the disease is marked by early onset stagnation of motor and intellectual functioning at 6 to 18 months of age and progressively worsens over time. Rett syndrome affects about 15,000-20,000 individuals in the U.S., EU, and UK, and currently has no approved disease-modifying gene therapies for its treatment.
TSHA-102 is a scAAV gene therapy developed as a one-time treatment for Rett syndrome by addressing the genetic root cause of the disease. The therapy utilizes adeno-associated viral (AAV) vectors to deliver functional forms of MECP2 to cells in the central nervous system (CNS) via an invasive lumbar intrathecal (IT) injection.
The treatment has received a breakthrough therapy, regenerative medicine advanced therapy, fast track, orphan drug, and rare pediatric disease designations from the U.S. Food and Drug Designation (FDA), and orphan drug designation from the European Medicines Agency (EMA).
TSHA-102 was evaluated in preclinical studies on neuronal cell models, and the results are expected to provide mechanistic validation of the drug's construct design. The company plans to report long-term safety and efficacy data from Part A of the Phase 1/2 REVEAL trial later this quarter.
TSHA stock had closed at $6.46, up 1.02% on Monday on the Nasdaq. In pre-market trade today, it was trading at $6.56, 1.55% or 10 cents higher.
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