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.
TSHA-102 is currently being evaluated in two ongoing REVEAL Phase 1/2 clinical trials being conducted in the U.S. and Canada. The study is divided into two parts – Part A and Part B. Part A focuses on evaluating the safety, tolerability and efficacy of two different dose levels (5.7×10¹⁴ vg and 1×10¹⁵ vg) of the investigational gene therapy. Taysha has completed dosing of the 12 patients in Part A of the study.
The goal of Part B, which will enroll additional patients, is to collect further safety and efficacy data to support potential regulatory approval.
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.
