Triplet Therapeutics is leveraging insights from human genetics to develop therapies targeting the underlying cause of repeat expansion disorders (REDs), a group of more than 50 known genetic diseases including spinocerebellar ataxias (SCAs) as well as Huntington’s disease (HD), myotonic dystrophy type 1 (DM1) and others.
Triplet seeks to stop the progression of REDs, which are each characterized by short repeating DNA sequences (“repeats”) in a single, different gene. For decades, the biomedical community thought each of these disorders would require its own unique therapeutic approach. But recent human genetic studies have revealed a potential unifying driver across the disorders: the DNA Damage Response (DDR) pathway. DDR genes have been found to lengthen the DNA repeats in these disorders, leading to increasing toxic effects of the expanding disease gene. Triplet hopes to prevent the repeats from lengthening – thereby stopping or delaying disease onset and halting or slowing progression before significant damage can be done – by using antisense oligonucleotides and small interfering RNA development candidates to precisely knock down key components of the DDR pathway that drive repeat expansion. This approach operates upstream of current approaches in development, which instead target the downstream products of expansion.
The company has raised $59 million in financing and assembled an experienced team and board as well a world-class scientific advisory board. It has selected its lead candidate, and clinical trials are set to start in 2nd half of 2021.