Taysha Gene Therapies, a clinical-stage gene therapy company focused on developing treatments for central nervous system diseases, provided clinical updates for its investigational programs TSHA-120 in Giant Axonal Neuropathy (GAN) and TSHA-102 in Rett Syndrome at their recent R&D Day. The company discussed the results of comprehensive data analysis for TSHA-120, addressing feedback from the U.S. Food and Drug Administration (FDA) regarding the primary endpoint and heterogeneity of GAN. They also shared encouraging initial clinical observations for TSHA-102 in Rett syndrome.
In regards to TSHA-120, Taysha conducted a thorough analysis of available data and developed a disease progression model that demonstrates predictable and homogenous disease progression in classic GAN. They also identified objective measurements that show a clinically meaningful treatment effect. Taysha plans to discuss these findings with the FDA in a formal meeting expected in Q3 2023.
As for TSHA-102, the first adult patient with Rett syndrome has been dosed in the REVEAL Phase 1/2 trial, and initial clinical observations