Patients & Caregivers
We strive to be a strong ally to advocacy organizations and the communities they support.
Our Commitment to Patients and Caregivers
At Taysha Gene Therapies, patients, caregivers, and families affected by rare central nervous system disorders are at the heart of everything we do.
We are dedicated to understanding their unique challenges and committed to putting their needs first as we work to deliver potentially life-changing therapies.
Guiding Principles
Our decisions and activities are guided by the following principles:
Transparency
Curiosity
Empathy
Respect
Gene Therapy for Rett Syndrome
Rett syndrome is a monogenic disease. This means it is caused by a change (mutation) in just one gene—the MECP2 gene. This gene gives the body instructions to make a protein called MeCP2, which is important for the brain and nervous system.
When the MECP2 gene doesn’t work properly, the body makes too little—or none—of the MeCP2 protein. Without enough of this protein, the brain and nervous system can’t grow and function the way they should.
Taysha is developing TSHA-102, an investigational gene therapy for Rett syndrome. This potential one-time treatment is designed to address the root cause of Rett syndrome: the MECP2 gene.
Additional education about gene therapy is available from the American Society for Cell and Gene Therapy.
Our Clinical Trials
TSHA-102 is an investigational gene therapy currently being evaluated in our REVEAL clinical trials for the treatment of females with Rett syndrome. The clinical trials are divided into two parts – Part A and Part B.
Part A is designed to evaluate the safety, tolerability and preliminary efficacy of TSHA-102 at two different dose levels (5.7×10¹⁴ vector genomes (vg) and 1×10¹⁵ vg) to determine the dose for the pivotal trial. Part A includes the REVEAL Pediatric Study for females aged 5 to 8 years, and the REVEAL Adolescent & Adult Study for females aged 12 years and older. A total of 12 participants, aged 6 to 21 years, were treated with TSHA-102 in Part A of the studies. Enrollment in Part A is complete.
Part B is the pivotal phase of the study and focuses on evaluating the efficacy and safety of the 1×10¹⁵ vg dose of TSHA-102 in additional participants to support the program’s potential regulatory approval. Part B includes the REVEAL Pivotal Study, which will evaluate the efficacy and safety of TSHA-102 in females with Rett syndrome aged 6 to <22 years. There is also a planned safety-focused study that will evaluate the safety and preliminary efficacy of TSHA-102 in females with Rett syndrome aged 2 to <6 years.
REVEAL Pivotal Study of TSHA-102
The REVEAL Pivotal Study is a single-arm (no placebo group) trial, with each participant serving as their own control. The study will enroll 15 females aged 6 to <22 years with Rett syndrome. The study will evaluate whether participants gain or regain important developmental milestones across the domains of communication, fine motor function and gross motor function, as well as continue to evaluate safety. Taysha has commenced site activation for the REVEAL Pivotal Study after receiving a No Objection Letter from Health Canada and feedback from the U.S. FDA. Enrollment in the REVEAL Pivotal Study is expected to begin in the fourth quarter of 2025.
Additional Resources
Additional information will be available here in the coming weeks.
Community Support and Resources
Information and support are available for families affected by Rett syndrome from patient advocacy organizations such as:
Patient Affairs at Taysha
Taysha’s Patient Affairs team is dedicated to:
- Listening to and learning from the patient and caregiver community to better understand their lived experiences, and integrating these learnings into Taysha’s overall business approach
- Providing transparent information about our research to the community whenever possible and in alignment with industry guidelines and regulations
- Offering educational resources to help families better understand the many facets of clinical research and investigational genetic medicines to support informed conversations and decision-making
- Partnering with patient advocacy organizations on key initiatives that help to raise awareness and progress research for rare diseases
Emily McGinnis
Chief Patient and External Affairs Officer
Chelsea Karbocus
Executive Director, Patient Advocacy
Kristin Phillips
Senior Director, Patient Experience
Karen Wagenbrenner
Study Participant Support Liaison
To connect with a member of our Patient Affairs team, please contact patientaffairs@tayshagtx.com.
