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Alnylam Pharma (ALNY) Presents Positive RNAi Platform Data; Plans to Finalize NDC Selection

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Alnylam Presents Clinical and Non-Clinical Data Demonstrating Continued RNAi Platform Optimization and Leadership in the Development of RNA-Based Therapeutics at 12th Annual Meeting of the Oligonucleo

September 28, 2016 4:00 PM EDT

Clinical Data from Phase 1/2 Study of ALN-AAT, an Investigational RNAi Therapeutic Targeting Alpha-1 Antitrypsin (AAT) for the Treatment of AAT Deficiency-Associated Liver Disease, Demonstrate Dose-Dependent and Durable AAT Knockdown

Company to Pursue Follow-On Candidate with Improved Tolerability Profile; Expects to File Clinical Trial Application in 2017

In Addition, Alnylam Presents Non-Clinical Data on RNAi Therapeutics, Including Extensive Review of Toxicology Results of GalNAc Conjugates Demonstrating Wide Therapeutic Index, With Lack of Thrombocytopenia or Pro-Inflammatory Effects

CAMBRIDGE, Mass.--(BUSINESS WIRE)--... More