If you are interested:
TAYSHA GENE THERAPIES PRESENTS NEW PRECLINICAL IN-VITRO DATA ON TSHA-102 IN RETT SYNDROME SUPPORTING MIRARE REGULATION OF MECP2 EXPRESSION AT THE EUROPEAN SOCIETY OF GENE & CELL THERAPY (ESGCT) 30TH ANNUAL CONGRESS
In vitro data demonstrated the miRARE control element downregulates MECP2 transgene and protein expression in response to cellular levels of MeCP2 in cell culture modelshttps://ir.tayshagtx.com/news-relea...pies-presents-new-preclinical-vitro-data-tsha
Data recapitulate in vivo findings in neonatal mice demonstrating TSHA-102 regulated MeCP2 expression in deficient CNS cells and avoided toxic overexpression in cells already expressing MeCP2
Available clinical data from the two adult patients dosed with TSHA-102 in the first cohort (low dose) to be reported in mid-November; dosing of first pediatric Rett syndrome patient expected in the first quarter of 2024
Acadia, page-623
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