A new idea in Huntington's
Clinical trials in Huntington's disease are testing treatments that aim to reduce the production of a mutant form of the protein huntingtin, which kills neurons and drives the array of symptoms that mark the disease. A new company launching Tuesday is taking a different approach — one that, if successful, could be used to treat other diseases that, like Huntington's, are caused by segments of a gene repeating over and over.
Triplet Therapeutics believes it can treat these "repeat expansion disorders" by restricting the expansion of those repeats of DNA in the first place. The company envisions designing antisense oligonucleotides and small interfering RNA to halt some of the errant activity of the so-called DNA damage response pathway, which could in turn prevent the onset and progression of the inherited diseases.
Triplet was started by Atlas Venture and partner Nessan Bermingham, who's taken on the CEO role. It is launching with a $49 million Series A round led by MPM Capital and Pfizer Ventures. Other repeat expansion disorders include myotonic dystrophy and spinocerebellar ataxias.
Triplet Therapeutics believes it can treat these "repeat expansion disorders" by restricting the expansion of those repeats of DNA in the first place. The company envisions designing antisense oligonucleotides and small interfering RNA to halt some of the errant activity of the so-called DNA damage response pathway, which could in turn prevent the onset and progression of the inherited diseases.
Triplet was started by Atlas Venture and partner Nessan Bermingham, who's taken on the CEO role. It is launching with a $49 million Series A round led by MPM Capital and Pfizer Ventures. Other repeat expansion disorders include myotonic dystrophy and spinocerebellar ataxias.
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