Taysha Gene Therapies Touts Encouraging Preclinical Data From Rett Syndrome Candidate

Taysha Gene Therapies Inc TSHA presented preclinical data from neonatal mouse models on TSHA-102 for Rett syndrome, including new data in wild-type mice, at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting.
In wild-type mice treated with TSHA-102, new data showed no harmful impact on survival, neurobehavioral functions, and overall health, suggesting miRARE regulated expression of MeCP2 with the below results from the study:
- No toxicity relative to vehicle treatment.
- No reduction in survival over 36 weeks.
- No treatment effect on Bird Score (a measure of Rett syndrome-like behaviors and pathologies) analysis relative to vehicle treatment
- No impact on overall growth throughout the study
In Mecp2–/Y knockout mice (mouse model recapitulating developmental, physiological, and behavioral features of human Rett syndrome) treated with TSHA-102 with the below results from the study:
- 47% survived the 36-week study vs. a median survival of 8.1 weeks with vehicle‐treated knockout mice, representing a significant (p<0.0001) >4-fold extension of their lifespan
- Restoration of normal and faster-than-normal growth.
- Aggregate Bird Score was significantly improved at several time points, with a significant delay in the onset of severe Rett syndrome-like phenotypes.
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