First Gene Therapy Trial for Rett Syndrome - based on Bird Lab research

On 29th March 2022 a clinical trial was announced by the California-based Company “Taysha Gene Therapies”. This is the first gene therapy trial for Rett syndrome and is based on a “mini-MeCP2” construct that Prof Adrian Bird’s laboratory published in Nature in 2017 (Tillotson et al, 2017) and patented via Edinburgh University. Taysha sub-licensed the IP and will conduct a phase 1 trial in Montreal in the coming months. The evidence that Rett syndrome might be treatable at all stems for the Bird group’s work published 15 years ago in Science (Guy et al, 2007) showing that Rett syndrome in mice can be reversed.

Taysha press release

Abstract from Taysha website referring to mini-MeCP2 construct

TSHA-102 is being developed for the treatment of Rett syndrome, one of the most common genetic causes of severe intellectual disability. TSHA-102 is constructed from a neuronal specific promoter, MeP426, coupled with the miniMECP2 transgene, a truncated version of MECP2, and miRNA-Responsive Auto-Regulatory Element, or miRARE, our novel miRNA target panel, packaged in self-complementary AAV9.

Related links

Professor Bird's lab