Next Huge Milestone for Rett

The last 12 months have been an unprecedented time for Rett syndrome. In December 2021, Acadia Pharmaceuticals reported that the first (and only) Phase 3 clinical trial in Rett syndrome had positive results. As hopefully many of you know, Acadia submitted a New Drug Application to the FDA for approval of trofinetide for Rett syndrome.

Now we have the next huge milestone: Taysha Gene Therapies has begun recruitment for the first-ever gene replacement therapy treatment for Rett syndrome. The Phase 1/2 REVEAL Adult Study, an open-label, dose escalation study of TSHA-102 gene therapy, will enroll participants in Canada.

This study is years in the making. IRSF first invested in understanding if gene therapy would be a possible treatment for Rett by funding Dr. Steven Gray in 2007. Fast forward to today, and Taysha is using Dr. Gray’s discoveries, putting them to test in adult women with Rett syndrome.

Watch this video that Taysha has produced for our community to learn about this clinical trial and the 2 additional clinical trials they plan to conduct in pediatric girls and in males with Rett syndrome.

None of this would be possible without you. Your donations to IRSF allow us to continue to fund critical work. Your support of IRSF allowed us to create the IRSF Clinical Trial Committee which met with the Taysha Gene Therapies team to improve the clinical trial protocol that is being used. And your willingness to be Active Research Participants, donating your time to share your voice with companies like Taysha, has allowed their team to build a protocol based on what is most important to you. We are all in this together. We are ALL IN for a Cure.