SPY421.97-0.14 -0.03%
DIA338.65-2.11 -0.62%
IXIC14,161.35+121.67 0.87%

Taysha Gene Therapies Announces Publication of Preclinical Data for TSHA-102 in Rett Syndrome in Brain, a Highly Esteemed Neurological Science Peer-Reviewed Journal

Preclinical data provide quantitative evidence of miRARE's ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions in both wild type and knockout mouse models of Rett

05/10/2021 07:04

Preclinical data provide quantitative evidence of miRARE's ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions in both wild type and knockout mouse models of Rett syndrome

TSHA-102 resulted in a statistically significant survival extension by 56% in 4-5-week-old knockout Rett mice with meaningful accumulated disease whereas unregulated constructs did not extend survival significantly in the validated MECP2 knockout Rett mouse model

Significant survival benefit in 4-5-week-old TSHA-102-treated knockout Rett mice with meaningful accumulated disease a more translatable model of the disorder in humans

These quantitative data, for the first time, demonstrated miRARE's ability to regulate gene expression on a cell-by-cell basis, highlighting its potential application in numerous dose-sensitive diseases.