Triplet Therapeutics

Triplet Therapeutics is leveraging insights of human genetics to develop therapies targeting the underlying cause of repeat expansion disorders, a group of more than 40 known genetic diseases including Huntington’s disease, myotonic dystrophy and spinocerebellar ataxias. Triplet is developing 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 current 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