Triplet Therapeutics is developing a fundamentally different treatment for repeat expansion disorders such as various spinocerebellar ataxias – Ataxia

Triplet Therapeutics is developing a fundamentally different treatment for repeat expansion disorders such as various spinocerebellar ataxias

Triplet Therapeutics is developing a fundamentally different treatment for repeat expansion disorders such as various spinocerebellar ataxias

Triplet Therapeutics, a biotechnology company based in the US (in Cambridge MA), is harnessing human genetics to develop transformative therapies that treat repeat expansion disorders at their source. Patients with repeat expansion disorders are born with a certain number of repeats in their DNA. Throughout the patient’s life, these repeats expand within the DNA thereby increasing in number resulting in onset and progression of disease. Examples of such repeat expansion disorders include Huntington disease, myotonic dystrophy, SCA1, SCA2, SCA3, SCA6, SCA7 and DRPLA. A significant body of human genetic evidence has identified one central pathway, known as the DNA damage response (DDR) pathway, which drives onset and progression of this group of disorders. Triplet Therapeutics is developing two genetic approaches known as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) to reduce levels of DDR proteins that are directly involved in repeat expansion. Triplet is working to improve the outlook for patients and families affected by this group of more than 40 disorders with an initial focus on Huntington’s disease, spinocerebellar ataxias and myotonic dystrophy

Triplet Therapeutics’ CEO, Nessan Bermingham told Ataxia UK: “The contributions of thousands of patients who participated in genetic research has enabled us to build a fundamentally new understanding of the cause of repeat expansion disorders. We are positioned to rapidly advance our initial development candidates toward the clinic for patients.”

Triplet Therapeutics’ CEO Nessan Bermingham

Posted on 28/01/2020

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