Taysha Gene Therapies today announced the dosing of the first pediatric patient in their REVEAL Phase 1/2 trial of investigational gene therapy TSHA-102. For Taysha’s full press release, click here.

Key Facts:

• The first female pediatric patient was dosed at RUSH University Medical Center in Chicago, an IRSF-designated Center of Excellence.
• The REVEAL Phase 1/2 pediatric trial in the United States will evaluate the safety and preliminary efficacy of TSHA-102 in female patients in two parts:
  • Part A will enroll six female patients ages 5-8 years old to evaluate two different dosing levels with the first data reported in mid-2024.
  • Part B will enroll up to 21 additional patients and expand to ages 3-8 years old.
• Recruitment for the pediatric trial is active with more information about criteria and study sites available by clicking here.
• Additionally, the MHRA has authorized Taysha’s Clinical Trial Application (CTA) to expand the REVEAL pediatric trial into the United Kingdom.

Today’s announcement marks another exciting milestone for our Rett syndrome community as two companies are now actively dosing adult and pediatric patients in gene therapy clinical trials. We are encouraged by this momentum and remain cautiously optimistic as these clinical trials continue. We at IRSF are committed to continuing to push for more research and more treatments until we find solutions for ALL our loved ones living with Rett syndrome.

Thank you to the patients and families who continue to participate in the clinical trials giving hope to our community; to the scientists and researchers who continue to push exciting treatments and cures forward; and thank you to everyone who supports IRSF’s mission to fund breakthrough research in our pursuit of a world without Rett syndrome.

IRSF is with you every step of the way. For more information about Taysha Gene Therapies and the status of all companies in the Rett research pipeline, click here. Questions? Email us at [email protected].