LETI-101 / ElevateBio - LARVOL DELTA

ElevateBio Announces Compelling Preclinical Data Demonstrating the Potential of LETI-101 as a Treatment for Huntington’s Disease (GlobeNewswire) - "ElevateBio...announced the presentation of new preclinical data from LETI-101, the Huntington's disease (HD) program developed by its gene editing and R&D technology business, ElevateBio Life Edit...Dose-dependent editing of the targeted mHTT exon 50 site, leading to potentially therapeutically relevant reductions of mHTT protein in the striatum, cortex, and thalamus at clinically relevant dose levels evaluated in BACHD transgenic mice; Bilateral intrastriatal administration of LETI-101 in NHPs was well-tolerated with a NOAEL obtained for the highest evaluated dose; Therapeutically relevant vector copy number and nuclease and guide expression were observed across brain regions that are critically vulnerable in HD; No change in immune response to the nuclease and minimal systemic vector distribution were observed."

Preclinical • Huntington's Disease