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Monday, 08/14/2023 3:57:56 PM

Monday, August 14, 2023 3:57:56 PM

Post# of 463446
Taysha gene therapy P1/2 for Rett Syndrome.
Taysha Gene Therapies Reports Initial Clinical Data from First Adult Rett Syndrome Patient Dosed in REVEAL Phase 1/2 Trial
https://finance.yahoo.com/news/taysha-gene-therapies-reports-initial-110200128.html

Data from first adult patient dosed in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent serious adverse events (SAEs) as of six-week assessment and improvement in key efficacy measures, including Clinical Global Impression – Improvement (CGI-I), Clinical Global Impression – Severity (CGI-S) and Rett Syndrome Behavior Questionnaire (RSBQ), four weeks post-treatment

Principal Investigator (PI) observed clinical improvement in multiple domains, including autonomic function (sleep and breathing), vocalization, as well as gross motor skills (gained ability to sit unassisted for three minutes) and fine motor skills (gained ability to hold objects), supported by initial clinical data and video evidence

United States (U.S.) Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for TSHA-102 in pediatric patients with Rett syndrome



I don't recall anyone posting this. I'd never heard of the company.

Seems to me that the FDA is permitting Taysha to trial children with less preliminary evidence than it required from Anavex. Looks like a potential competitor. Comments?

Also today:
Taysha raises $150M PIPE, shares single patient data on Rett syndrome gene therapy
https://endpts.com/taysha-raises-150m-pipe-shares-single-patient-data-on-rett-syndrome-gene-therapy/

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