Pre-Clinical Data Presented with ALN-AT3 at ASH

Pre-Clinical Data Presented with ALN-AT3 at ASH

Today we are presenting new data from our ALN-AT3 program for the treatment of hemophilia and other bleeding disorders at the 54th American Society of Hematology (ASH) Annual Meeting in Atlanta. ALN-AT3 comprises part of our “Alnylam 5×15™” strategy, which is aimed at bringing innovative medicines to patients, with a focus on RNAi therapeutics toward genetically defined targets for diseases with very high unmet medical need.

The new research with ALN-AT3, an RNAi therapeutic for the treatment of hemophilia, shows potent and durable knockdown of antithrombin (AT) in non-human primates, with corresponding increases in thrombin generation up to four-fold. ALN-AT3 utilizes our proprietary GalNAc conjugate delivery platform, which enables subcutaneous dose administration. We are excited by these new data, and see the potential for a once-weekly or twice-monthly dosing regimen at doses as low as 0.15-0.3 mg/kg and at a volume of injection for human administration less than 0.5 mL.

Also, don’t forget to register for our upcoming RNAi Roundtable on conjugate delivery, where we will discuss the latest progress from our ALN-AT3 program, as well as our ALN-TTRsc program for the treatment of transthyretin-mediated amyloidosis (ATTR). The event will take place this Friday, December 14 at 11:00 a.m. ET, and will be available as a webcast here on Capella.



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