We are committed to providing healthcare professionals with the information and programs that will support their efforts to treat rare, monogenic diseases of the central nervous system.
The TSHA-102 Program
TSHA-102 is a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome that aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the central nervous system. The TSHA-102 clinical development program is designed to support the potential future approval for a broad population of patients aged 2 years and older with Rett syndrome through an efficient and rigorously designed pathway.
In close collaboration with the U.S. Food and Drug Administration, Taysha designed the TSHA-102 pivotal program so that efficacy data from the REVEAL Pivotal Trial in participants aged 6 to <22 years who have reached developmental plateau can be used to represent patients aged 2 to <6 years, while safety data in younger children are generated through the separate ASPIRE Trial to support a broad label for TSHA-102.
REVEAL Pivotal Trial (Part B)
The REVEAL Pivotal Trial is a single-arm, open-label study evaluating the efficacy and safety of TSHA-102 in females with Rett syndrome, with each patient serving as their own control. Each participant will receive a single administration of the high dose (1×10¹⁵ total vector genomes (vg)) of TSHA-102 delivered by lumbar intrathecal injection.
The study is enrolling 15 females aged 6 to <22 years in the developmental plateau population of Rett syndrome. The primary endpoint will assess response rate, defined as the percentage of patients who gain or regain one or more defined developmental milestone from a list of 28 across the core functional domains of communication, fine motor and gross motor, following dosing with TSHA-102. The 28 selected milestones are clinically meaningful and have a 0% to <6.7% likelihood of spontaneous gain/regain in the untreated Rett syndrome population aged ≥6 years based on an extensive review of natural history data (N = ~1100 females with Rett syndrome, with up to 14 years follow-up)18. Enrollment in the REVEAL Pivotal Trial is underway.
(ClinicalTrials.gov Identifier: NCT05606614)
ASPIRE Trial
The ASPIRE Trial will evaluate the safety and preliminary efficacy of TSHA-102 in females aged 2 to <4 years with Rett syndrome. The study will enroll three females. Each participant will receive a single administration of the 1×10¹⁵ total vg dose of TSHA-102, scaled to account for the lower brain volume in 2 to <4‑year‑olds, delivered by lumbar intrathecal injection.
REVEAL Phase 1/2 Adolescent and Adult Trial (Part A)
The REVEAL Phase 1/2 Adolescent and Adult Trial is a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in females aged 12 and older with Rett syndrome.
A total of six participants aged 15 to 21 years were treated with TSHA-102 in this study. Two of the participants received the low dose (5.7×10¹⁴ vg) of TSHA-102 and four of the participants received the high dose (1×10¹⁵ vg) of TSHA-102. These participants will continue to be monitored throughout the study. Enrollment for this trial is complete.
REVEAL Phase 1/2 Pediatric Trial (Part A)
The REVEAL Phase 1/2 Pediatric Trial is a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in females aged 5 to 8 years with Rett syndrome.
A total of six participants aged 6 to 8 years were treated with TSHA-102 in this study. Two of the participants received the low dose (5.7×10¹⁴ vg) of TSHA-102 and four of the participants received the high dose (1×10¹⁵ vg) of TSHA-102. These participants will continue to be monitored throughout the study. Enrollment for this trial is complete.
If you are a healthcare provider and have questions or would like additional information on the trials, please contact medinfo@tayshagtx.com.
For additional information for patients and caregivers, please visit our Patient & Caregivers page.
At Taysha, we are focused on running well-designed clinical trials to assess the safety and efficacy of investigational therapies and provide the highest probability of bringing an approved therapy to the broader patient community. We are collaborating closely with patients, caregivers, patient organizations and medical experts to conduct clinical trials.
Expanded Access, sometimes called Managed Access, Compassionate Use, Named Patient or Early Access, is a potential pathway for a patient with an immediately life-threatening condition, serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Taysha evaluates physician requests for Expanded Access outside of a clinical study on a case-by-case basis when certain conditions are met. All Expanded Access requests must be submitted by the patient’s treating physician.
Patients or caregivers who are interested in learning more should speak with their physician. Physicians may submit questions about clinical trials and Taysha’s Expanded Access Policy to medinfo@tayshagtx.com.