Alnylam earns $7m milestone payment from Genzyme for work on rare disease drug

Alnylam Pharmaceuticals Inc., a Cambridge biopharmaceutical company specializing in RNAi gene technology, said Monday that it has earned a $7 million milestone payment from Genzyme for its work on patisiran, a potential treatment for a rare disease called ATTR.

Genzyme is the Cambridge-based division of the French drug maker Sanofi SA. (In 2011, Sanofi bought Genzyme for $20.1 billion.)

John Maraganore. File photo. Ruby Arguilla Tull/Bloomberg News.

“We believe patisiran holds considerable promise to become a breakthrough therapy for the treatment of ATTR, a progressive and debilitating orphan disease. As the lead program in our ‘Alnylam 5x15’ product strategy, this program is also a key part of building Alnylam for the future and driving towards commercialization,” Alnylam chief executive John Maraganore said in a statement. “We have made tremendous progress with this program in recent months, including reporting positive Phase II results, and initiating both an open-label Phase II extension study as well as our Phase III APOLLO study. We look forward to continued progress with Genzyme as our collaborator for the development and commercialization of our TTR program in the Japanese and Asian markets, and we are pleased to achieve this milestone based on successful completion of our Phase II study.”

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ATTR is an inherited, progressively debilitating, and fatal disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier for retinol binding protein. Mutations in TTR cause abnormal amyloid proteins to accumulate and damage body organs and tissue, Alnylam said in its press release.