A Cambridge biotech that hopes to develop treatments for devastating rare genetic disorders, including Huntington’s disease, was officially unveiled Tuesday after raising $59 million in venture capital.
Triplet Therapeutics, whose founders include Nessan Bermingham, a serial biotech entrepreneur and venture partner at Atlas Venture, quietly began incubating about a year ago at Alexandria LaunchLabs in Kendall Square. It has tested experimental treatments in rodents and non-human primates.
“This financing gives us a runway of about two years of cash, and we expect that with this financing, we’ll be able to initiate human clinical trials,” Bermingham said.
The venture capital includes a $49 million fund-raising round led by MPM Capital and Pfizer Ventures, the venture capital arm of the drug giant Pfizer. Atlas, which cofounded Triplet and provided $10 million in seed funding, also participated in the fund-raising round, along with Invus, Partners Innovation Fund, and Alexandria Venture Investments.
The startup, which has 29 employees, is targeting “triplet repeat expansion disorders.” These diseases are caused by an unusual genetic change — an expansion of a segment of DNA that contains a repeat of three nucleotides.
Healthy people have triplets in their DNA, according to Bermingham. But more than 40 known inherited conditions, including Huntington’s and myotonic dystrophy, are marked by too many DNA nucleotide repeats.
Several biotechs have tried to keep excessive repeats from causing disease by targeting messenger ribonucleic acid, or mRNA, from the mutated gene. Messenger RNA is a molecule that delivers messages from DNA to make proteins within cells. Those companies, Bermingham said, believe each of those diseases need a tailor-made treatment.
In contrast, Triplet’s researchers think that a common denominator called the “DNA damage response” pathway underlies these genetic diseases and determines when symptoms appear and how fast a disorder progresses.
Triplet is developing two approaches to keep the triplet repeats from expanding and causing diseases.
“There’s a unifying biology that’s driving these diseases,” said Bermingham, a 47-year-old native of Ireland. “Those bespoke therapies are based upon, we believe, an incorrect understanding of the disease.”
Bermingham helped found Intellia Therapeutics, one of several companies that have crafted therapies based on the genome-editing technology CRISPR.
Huntington’s, which Triplet hopes to treat, results in the progressive breakdown of nerve cells in the brain. It causes physical and mental abilities to deteriorate, usually in the patient’s prime working years, and has no cure.
Perhaps the most famous person diagnosed with Huntington’s was Woody Guthrie, the renowned folk singer who influenced Bob Dylan and died in 1967 at the age of 55. The faulty gene that causes Huntington’s was identified in 1993.
Triplet’s management team includes veterans of a number of well-known biotechs, including Wave Life Sciences, Translate Bio, Intellia, and Moderna.
Jonathan Saltzman can be reached at firstname.lastname@example.org