The president and head of R&D at Taysha Gene Therapies discussed his expectation that younger patients my see even better results with the company’s gene therapy than the adult patients treated so far.
This is the third part of an interview with Sukumar Nagendran, MD. For the first part, click here.
"What actually excites me and my team is that given that we have such a good clinical response in adults, we think the clinical response in the pediatric population could be even better. This now gives us the gamut of the age range from pediatrics to adults to treat in a very difficult to treat central nervous system disorder.”
Taysha Gene Therapies’ TSHA-102, an investigational adeno-associated virus (AAV) vector-based gene therapy for Rett syndrome, is administered intrathecally and is intended to deliver a copy of miniMECP2, a truncated version of the disease-targeted MECP2 gene, via an AAV9 vector.1,2 The gene therapy product also incorporates use of the company's novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform, which is intended to enable regulation of cellular expression for both endogenous and exogenous MECP2. In mid-2023, Taysha Gene Therapies dosed the first 2 patients in the first-in-human phase 1/2 REVEAL clinical trial (NCT05606614) evaluating TSHA-102.3,4 At the time, the study was only open to adult patients, although its eligibility criteria was later expanded to include patients as young as years old.5 Furthermore, following the announcement of promising early efficacy and safety results from the first 2 adult patients treated in REVEAL, Taysha announced in January 2024 that the first child had been treated in the separate phase 1/2 REVEAL Pediatric Study clinical trial (NCT06152237).6 The REVEAL Pediatric Study is initially treating female patients aged 5 to 8 years who have stage 3 Rett syndrome.
In light of the ongoing developments for TSHA-102, CGTLive™ sat down with Sukumar Nagendran, MD, the president and head of research and development at Taysha, in December 2023 to discuss his future expectations for the gene therapy product. Nagendran emphasized his view that based on the surprisingly robust efficacy results seen in adult patients, TSHA-102 will likely have even greater efficacy in very young patients. He also noted the importance of newborn screening for Rett syndrome and pointed out how intervening with a disease-modifying treatment at younger ages could help alleviate the burden of care and associated economic cost of the disease.