FDA grants regenerative medicine advanced therapy designation to Rett syndrome treatment

The FDA has granted regenerative medicine advanced therapy designation to an intrathecally delivered AAV9 gene transfer therapy to treat younger individuals with Rett syndrome, according to the manufacturer.

The FDA’s decision was based on a review of available safety and efficacy data from the first three enrollees with Rett syndrome given a low dose of TSHA-102 (5.7x1014 total vg) in the phase 1/2 REVEAL clinical trials in pediatric, adolescent and adult populations, Taysha Gene Therapies Inc. said in a release.

Rett is characterized by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MECP2), which is essential for regulating neuronal and synaptic function in the brain. Symptoms, which can progress rapidly, include loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disability and shortened life span.

Regenerative medicine advanced therapy (RMAT) designation aims to expedite the development and review of regenerative medicine therapies that treat, modify, reverse or cure a serious condition, along with preliminary clinical evidence pointing to the potential to address unmet medical needs for the condition.

“We believe receiving RMAT designation reinforces the high unmet medical need in Rett syndrome and the therapeutic potential of TSHA-102 to change the treatment paradigm,” Sukumar Nagendran, MD, president and head of research and development at Taysha, said in the release. “We believe this important recognition from the FDA further supports the potential of our gene therapy candidate to bring meaningful change to patients and families living with Rett syndrome.”