Triplet Therapeutics

Triplet Therapeutics is developing transformational treatments for patients with repeat expansion disorders (REDs) – a group of more than 50 known genetic diseases including Huntington’s disease, myotonic dystrophy type 1, spinocerebellar ataxias, fragile X syndrome, and familial amyotrophic lateral sclerosis (ALS) – leveraging insights from the human genetics of REDs. Triplet designs and develops antisense oligonucleotide and small interfering RNA development candidates to precisely knock down key components of the DNA damage response (DDR) pathway that drive repeat expansion. This approach operates upstream of the leading approaches in development, which instead target the downstream products of expansion. By precisely reducing activity of select DDR mechanisms, Triplet’s approach is designed to halt onset and progression across a wide range of diseases.

Headquarters Cambridge, MA
Pipeline Preclinical
Twitter @TripletThera