Sukumar Nagendran, MD, on Addressing Unmet Needs in Rett Syndrome With Gene Therapy


The president and head of R&D at Taysha Gene Therapies discussed the early clinical evaluation of TSHA-102.

“TSHA-102 is a gene therapy given intrathecally into the lumbar area between L4 and L5. It's a simple procedure done by lumbar puncture. That allows it to be done in multiple sites in many parts of the country, even in an outpatient setting, inpatient setting, or in a hospital, etc. Ease of use is paramount here...”

Historically, the standard of care for Rett syndrome has largely consisted of physical therapy and speech therapy intended to ameliorate symptoms, as no FDA-approved specifically-targeted drugs existed for the condition. Earlier this year, however, Acadia Pharmaceuticals’ trofinetide (marketed as Daybue) was approved by the FDA for the treatment of Rett syndrome.1 Although trofinetide has been shown to have some clinical efficacy and provided an important new option for patients, it does not address the genetic root cause of the condition, and as such there is a need for continued development of new treatments. As a monogenic disorder, gene therapy is an area of potential for Rett syndrome, and multiple gene therapies are currently being developed for the disorder.2 

One of these is Taysha Gene Therapies’ TSHA-102, an investigational adeno-associated virus vector-based gene therapy currently being evaluated in the phase 1/2 REVEAL clinical trial (NCT05606614). Two patients have received the treatment so far at CHU Sainte-Justine, the Université de Montréal Mother and Child University Hospital Center in Montreal, Canada. 

Following the announcement of initial results from the dosing of these first 2 participants in REVEAL, CGTLive™ sat down with Sukumar Nagendran, MD, the president and head of research and development at Taysha, to learn more about the therapy and the trial. Nagendran discussed the currently available options for Rett syndrome and the rapidly evolving landscape of care for this disorder. He also gave an in-depth description of how TSHA-201 functions and an overview of the design of the REVEAL clinical trial. 

1. FDA approves first treatment for Rett Syndrome. News release. FDA. March 13, 2023. Accessed December 7, 2023.
2. Taysha Gene Therapies reports third quarter 2023 financial results and provides corporate and clinical updates. News release. Taysha Gene Therapies, Inc. November 14, 2023. Accessed December 7, 2023.
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