RTX-001

RTX-001 is an orally bioavailable small molecule developed by Repeat Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) associated with hexanucleotide repeat expansions in the *C9orf72* gene. The expansion leads to the production of toxic dipeptide repeat (DPR) proteins and RNA foci through repeat-associated non-AUG (RAN) translation. RTX-001 is designed to selectively inhibit the production of these toxic species, thereby addressing the underlying molecular pathology of the disease. The compound is currently being evaluated in a Phase 1 first-in-human clinical trial (NCT06141330) to assess its safety, tolerability, and pharmacokinetics in healthy adult volunteers.