cag repeat rare disorders
CAG repeat expansions is a genetic mutation causing neurodegenerative disorders.
CAG expansions form hairpin loops particularly with longer repeats, leading to the symptoms of Huntington's Disease (HD), Spinocerebella Ataxia type 1, 2, and 7 (SCA1, SCA2, SCA7), and Spinal and Bulbar Muscular Atrophy (SBMA, Kennedy's disease).
Our Mission & Vision
Our mission is to develop scalable, small-molecule therapies that directly target the RNA-level cause of CAG repeat expansion disorders and enable safe, long-term treatment for patients.
