Rare Disease Biotech Triplet Quietly Shuts Down

Less than three years after launching with $59 million in a series A funding round to find treatments for repeat expansion disorders, Triplet Therapeutics quietly shut down, first reported by STAT news and then later confirmed by Triplet CEO Nessan Bermingham’s post on LinkedIn.

Triplet was formed to develop antisense therapies for repeat expansion disorders, including Huntington’s disease, myotonic dystrophy, spinocerebellar ataxias, and potentially up to 40 additional repeat expansion disorders.

But repeat failures of other companies’ Huntington’s disease trials and a slowdown in biotech financing left the company struggled to attract investors for capital to continue its operations.

Triplet was founded by Bermingham, Atlas Venture, and Andrew Fraley, to pursue a transformative approach to developing treatments for repeat expansion disorders, a group of more than 40 known genetic diseases associated with expanded DNA nucleotide repeats. The company targeted one central pathway, known as the DNA damage response (DDR) pathway, which evidence had shown drives onset and progression of this group of disorders, which include Huntington’s disease, myotonic dystrophy and various spinocerebellar ataxias.

By developing antisense oligonucleotide and small interfering RNA candidates to precisely knock down key components of the DDR pathway that drive repeat expansion, Triplet planned to target the fundamental driver of these diseases and halt onset and progression across a wide range of repeat expansion disorders.

Author: Rare Daily Staff