SAN DIEGO, CA, Dec 2025 — RepeatTherapeutics, a biotechnology company developing potential treatments forneurodegenerative diseases with a current focus on amyotrophic lateralsclerosis (ALS), today announced that thefirst participant has been dosed in its Phase 1 clinical study evaluatingRTX-101, the company’s lead investigational candidate for ALS associated withthe C9orf72 hexanucleotide repeat expansion.
C9orf72-associated ALS is the most common known genetic cause of familial ALS. RTX-101and RTX-111 are orally administered investigational small-molecule combination therapiesdesigned to restore cellular balance and reduce neurotoxic stress in motorneurons. It is hypothesized that the investigational therapies modulate keybiochemical processes involved in oxidative injury and cellular stress inC9orf72-mediated ALS with potential broader application to other neurodegenerativediseases.
The Phase 1 study is designed to evaluate the safety, tolerability, andpharmacokinetics of RTX-101, RTX-111 in healthy volunteers, with plannedexpansion into individuals with C9orf72-associated ALS. The study is beingconducted in Australia under the Therapeutic Goods Administration (TGA)Clinical Trial Notification (CTN) pathway, with Human Research Ethics Committee(HREC) approval.
“Dosing the first participant marks an important early milestone for Repeat,” saidSusan Francia, co-founder of Repeat Therapeutics. “ALS remains a devastatingdisease with limited treatment options, and our team is committed to advancingthis research thoughtfully and responsibly. These early studies are designed tohelp us better understand the safety of our investigational approaches as wework toward addressing an area of unmet need.”
Repeat Therapeutics is advancing a focused pipeline of investigationalapproaches for repeat expansion disorders, with an emphasis on understandingand addressing metabolic and cellular stress pathways.
.png)
.png)
