One of the companies working on the cutting edge of the field of gene silencing is Pasadena, California-based Arrowhead Pharmaceuticals, which expectS 2020 to be a real inflection point.
Alnylam’s gene-silencing therapy for a rare kidney disorder met the main goal of a late-stage study, bringing the company a step closer to marketing the first approved treatment for the condition.
Alnylam Pharmaceuticals Inc. priced the company’s gene silencing drug to treat patients with a rare genetic disorder that can cause severe pain at $575,000 per year after receiving an early U.S. approval.
Regeneron Pharmaceuticals is investing $800 million in Alnylam Pharmaceuticals to develop treatments for eye and central nervous system diseases using gene-silencing RNA interference technology.
Alnylam and Sanofi announced a strategic restructuring of their RNAi therapeutics alliance to streamline and optimize development and commercialization of certain products for rare genetic diseases.
