RTX-010

RTX-010 is an investigational small molecule therapeutic being developed by Repeat Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS) caused by hexanucleotide repeat expansions in the C9orf72 gene. The drug is designed to selectively inhibit Repeat-Associated Non-AUG (RAN) translation, a non-canonical protein synthesis process that occurs at expanded nucleotide repeats and leads to the production of toxic dipeptide repeat (DPR) proteins. By reducing the accumulation of these proteotoxic DPRs, RTX-010 aims to mitigate neurodegeneration and slow disease progression. It is currently being evaluated in Phase 1 clinical trials (e.g., Part 1C of a multiple dosing study) to assess its safety, tolerability, and pharmacokinetics in patients with C9orf72-mediated ALS.