CAMBRIDGE, MASS.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today that it is presenting key scientific data on its Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform. Specifically, the company will be presenting data showing that chemical modifications of siRNA that enhance in vitro stability result in higher liver exposure in vivo and lead to a significantly increased potency and durability of effect in pre-clinical studies. As compared with the standard template chemistry (STC)-GalNAc-conjugate approach used in ALN-TTRsc a subcutaneously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR cardiac amyloidosis ESC-GalNAc-siRNA conjugates demonstrated a 10-fold increased potency in non-human primate (NHP) studies, and a durability of effect that supports once-monthly or potentially even less frequent subcutaneous dosing regimens. The ESC-GalNAc-conjugate technology is being used in a wide range of Alnylam development programs, including ALN-AT3 an RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders which is currently in a Phase 1 clinical study. Our ESC-GalNAc-conjugate delivery platform enables subcutaneous dosing of RNAi therapeutics with increased potency and durability, and a wide therapeutic index. Importantly, we have demonstrated that increased siRNA stability is the key to realizing these potency and durability … Continue reading →

MINSK, Belarus - Name tags aren't required around Team Canada's locker room at the world hockey championship. Maybe that wouldn't be such a bad idea. "We just got together a week ago," winger Joel Ward said. "I barely know some of the trainers' names, to be honest." That would be Mike Burnstein from the Vancouver Canucks and Gerry Townend and Shawn Markwick from the Ottawa Senators. But more to Ward's point is Canada's on-ice chemistry remains a work in progress after just two tournament games. "That's the challenge, I think, every year with this tournament for the North American teams," goaltender Ben Scrivens said. "Obviously I've watched before and that seems to be the running commentary is how quickly teams can gel in, it seems like, every international tournament." This tournament in particular is a challenge for Canada, given that this roster is made up of 19 players whose NHL teams missed the playoffs and four who went seven games in the first round of the Stanley Cup playoffs. Through the shootout loss to France in the opener and Saturday night's victory over Slovakia, coach Dave Tippett can notice the difference between those who had been playing and those who … Continue reading →