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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
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nPreclinical 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\n\nTSHA-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\n\nSignificant 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\n\nThese 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 \n\nData support an IND/CTA filing for TSHA-102 in Rett syndrome in the second half of 2021, with initiation of a Phase 1/2 trial anticipated by year-end\n\nEstimated 25,000 patients in U.S. and Europe represent multi-billion-dollar commercial opportunity\n\n DALLAS--(BUSINESS WIRE)--\nTaysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the publication of new preclinical data for TSHA-102 in Rett syndrome. The data were published online and will be included in the May edition of Brain, a highly esteemed neurological science peer-reviewed journal.\n\n“Effective gene therapy targeting MECP2 for the treatment of Rett syndrome has been elusive due to the inability to properly regulate transgene expression,” said Steven Gray, Ph.D., Chief Scientific Advisor at Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. “The built-in self-regulatory feedback loop mechanism in TSHA-102, work that was initiated in my laboratory in 2007, is a completely novel approach that allows for regulated expression of MECP2 on a cell-by-cell basis. These results published today are highly encouraging and allow us to conceive additional novel approaches using miRARE in conditions with dose-sensitive genes.”\n\n“The complexities...