PHARMA/BIOTECH UPDATE:NEW SUBCUTANEOUS THERAPY TARGETING INHIBITORS PRESENTED AT ASH

A new subcutaneous therapy being tested could provide patients with hemophilia and rare bleeding disorders suffering with an inhibitor, a welcomed alternative to current available treatment methods.

News Medical reports Alnylam Pharmaceuticals, Inc. presented new pre-clinical data with ALN-AT3 recently at the 2013 American Society of Hematology (ASH). ALN-AT3 is a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders.

Executive Vice President and Chief Medical Officer of Alnylam, Askshay Vaishaw, M.D., Ph.D. said, “ALN-AT3 is aimed at correcting these bleeding disorders by knockdown of AT – an endogenous anticoagulant – thus, increasing thrombin generation and improving hemostasis. These new data presented at ASH demonstrate that repeat administration of ALN-AT3 is well tolerated in animal models of hemophilia and suggest that our RNAi therapeutic has the potential for a wide therapeutic index in subjects with hemophilia.”

Phase I clinical trial with ALN-AT3 is expected to start in early 2014.  Click the link to keep up to date with the new developments coming out of Alnylam Pharmaceuticals.

Photo credit-Alnylam Pharmaceuticals, Inc.

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