In deal with North Carolina biotech, Vertex takes on Huntingtons disease – The Boston Globe

Vertex Pharmaceuticals, the Boston drug maker best known for its cystic fibrosis treatments, continues to branch out, cutting a deal with a North Carolina-based biotech firm that is targeting several serious illnesses, including the fatal genetic disorder Huntingtons disease.

In an agreement announced Monday, Vertex will pay Ribometrix $20 million up front to discover and develop at least two RNA-targeted medicines, with an option for a third.

RNA or ribonucleic acid is a molecule found in all cells. It acts as a messenger, carrying the DNAs instructions to the cells for making the proteins that are essential for life. Scientists have long believed RNA to be a good therapeutic target in the treatment of illnesses.

The first collaboration that Ribometrix will focus on is Huntingtons disease. That disorder causes the progressive breakdown of nerve cells in the brain. It causes physical and mental abilities to deteriorate, usually in the patients prime working years, and has no cure.

Perhaps the most famous person diagnosed with Huntingtons was Woody Guthrie, the influential folk singer who influenced Bob Dylan and died in 1967 at the age of 55. The faulty gene that causes Huntingtons was identified in 1993.

Targeting RNA is a potentially powerful new approach for treating diseases, said Mark Bunnage, a senior vice president and site head for Boston research at Vertex. He said the partnership adds a novel mechanism to our internal toolkit.

Vertex is largely known for its groundbreaking but costly treatments for cystic fibrosis, another rare disease. But the company has made several business deals and announcements recently that reflect an interest in other maladies, from muscular dystrophy to sickle cell disease to pain.

Michael Solomon, CEO of Ribometrix, said Vertex is a leader in creating transformative medicines through serial innovation and we are proud to partner with them.

If Ribometrix meets certain goals in the collaboration, the Durham-based firm is eligible to receive more than $700 million in total payments from Vertex.

Jonathan Saltzman can be reached at jsaltzman@globe.com

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In deal with North Carolina biotech, Vertex takes on Huntingtons disease - The Boston Globe