Today, Taysha Gene Therapies made several exciting announcements about the progress of their REVEAL Phase 1/2 trial in Canada and the future study of their gene therapy, TSHA-102. To read their full press release, click here.
- Updates on the first adult patient dosed in the REVEAL Phase 1/2 trial in Canada:
- TSHA-102 was well-tolerated by the first patient and there were no treatment-emergent serious adverse events (SAEs) reported at the six-week assessment.
- The patient showed clinical improvement in key efficacy measures including the CGI-I and RSBQ at four weeks post-treatment that included autonomic function (sleep and breathing), vocalization, and gross and fine motor skills.
- The U.S. FDA cleared Taysha’s Investigational New Drug (IND) application for TSHA-102 in pediatric patients with Rett syndrome.
- The first U.S. clinical trial will enroll females with Rett aged 5-8 years old, with plans to expand to ages 3-8 years old in future parts of the study.
- Additional details about the clinical trial, criteria, and study sites will be shared when available.
- Taysha submitted a Clinical Trial Application (CTA) to the United Kingdom MHRA.
- Taysha secured an additional $150 million in funding to support their continued operations including the Rett syndrome program.
Taysha’s announcements mark another milestone for Rett syndrome. We are grateful to the patients and families who are participating in this clinical trial. You are giving hope to our community, and we cannot thank you enough.
We are grateful to the scientists and researchers who continue to push exciting treatments including cures forward so that our loved ones can someday do more and struggle less.
And we are grateful to all the donors who support our mission and allow us to fund breakthrough research in our pursuit of a world without Rett syndrome. This potential gene therapy uses discoveries made by Dr. Steven Gray, the recipient of IRSF funding to establish his independent lab and study gene therapies to treat Rett. We promise to continue to invest broadly and smartly in the most promising research to find solutions for ALL our loved ones with Rett syndrome.
IRSF is with you every step of the way. We promise to continue to relentlessly pursue treatments and curative approaches to create a world without Rett syndrome. Questions? Email us at [email protected].