Taysha: Initial Clinical Data from First Adult Rett Syndrome Phase 1/2 Trial

 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

Clinical Trial Application (CTA) submitted to the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) for TSHA-102 in pediatric patients with Rett syndrome

Private placement financing (“PIPE”) is expected to result in gross proceeds of approximately $150 million from new and existing investors and, net proceeds from PIPE, along with existing cash and cash equivalents, extends cash runway into the third quarter of 2025

Conference call and live webcast today at 8:30 AM Eastern Time

Taysha management will hold a conference call and webcast today at 8:30 a.m. ET to review its financial and operating results and to provide a corporate update. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13740092. The live webcast can be accessed here: https://viavid.webcasts.com/starthere.jsp?ei=1624983&tp_key=25b742b70a. An archived version of the webcast will be available for 30 days and can be accessed by visiting Taysha’s website at https://ir.tayshagtx.com/news-events/events-presentations.

https://finance.yahoo.com/news/taysha-gene-therapies-reports-initial-110200128.html