Repeat Therapeutics is a clinical-stage biotechnology company redefining neurodegenerative medicine by targeting the biology of repeat expansion diseases, with an initial focus on C9orf72-associated amyotrophic lateral sclerosis (ALS)
Repeat Therapeutics is a clinical-stage biotechnology company redefining neurodegenerative medicine by targeting the biology of repeat expansion diseases, with an initial focus on C9orf72-associated amyotrophic lateral sclerosis (ALS). The company's oral investigational therapies, RTX-001 and RTX-111, are designed to work with the body's own metabolic pathways to restore cellular balance and protect vulnerable nerve cells, and are currently being evaluated in a Phase 1 study in Australia. Led by CEO and two-time Olympic gold medalist Susan Francia and Nobel Prize-winning RNA pioneer and Chief Scientific Officer Katalin Kariko, Repeat unites world-class RNA, neuroscience, and clinical development expertise to deliver first-in-class, accessible therapies for patients and families affected by ALS and other repeat expansion disorders. Headquartered across Melbourne and San Diego, Repeat Therapeutics is committed to expanding access and affordability while shaping a new era of understanding and treatment in neurodegenerative disease.