Both the second and first patients dosed in the study received the lower of the 2 doses of the gene therapy that Taysha Gene Therapies is evaluating in the phase 1/2 REVEAL trial.
Charlie Trippodo turned 8 years old last month. That’s another year trapped in a body that won’t let her walk or talk. But recent trial studies in gene therapy have
Shares of Taysha Gene Therapies, Inc. (TSHA) are up 132% on Monday after the company announced positive clinical results for TSHA-102 and $150 million in private placement financing.
April 12, 2021 Taysha Gene Therapies has acquired exclusive worldwide rights to a clinical-stage adeno-associated virus 9 (AAV9) gene therapy program (TSHA-120) for the treatment of giant axonal neuropathy.
TSHA-120 is currently being evaluated in an ongoing open-label, nonrandomized, dose-escalation clinical trial conducted by the U.S. National Institute of Neurological Disorders and Stroke (NINDS) division of the National Institutes of Health (NIH). The primary end point is safety, and a primary measure of TSHA-120 s clinical efficacy is the Motor Function Measure 32 (MFM32) score. TSHA-120 has demonstrated the potential to improve MFM32 scores in patients. Additional data from the study are expected later this year.