August 2020

20 Aug Nonclinical Research on Pharmacodynamic Durability of GalNAc-siRNA Conjugates Published in “Nucleic Acids Research”

We published nonclinical research describing the durability of gene silencing by GalNAc-conjugated siRNAs. The article, titled, “Investigating the Pharmacodynamic Durability of GalNAc-siRNA Conjugates,” was selected as a Breakthrough Paper by Nucleic Acids Research. Read the press release Read the paper in Nucleic Acids Research

30 Jun New 12-Month Interim Data From the ENVISION Phase 3 Study of Givosiran in Acute Hepatic Porphyria

Interim results from the open-label extension (OLE) period of the ENVISION Phase 3 study of GIVLAARI^® (givosiran) in acute hepatic porphyria (AHP) were presented by study investigator Eliane Sardh, M.D., Ph.D. during a webinar hosted by Alnylam, demonstrating sustained efficacy and acceptable safety through 12 months of treatment, with evidence for potentially improved efficacy over time. Read our press release View the presentation View the webinar

19 Jun New Positive Results for Fitusiran Presented at WFH

New positive results from an interim analysis of the Phase 2 open-label extension (OLE) study of fitusiran, in development for the treatment of hemophilia A or B, with or without inhibitors, were presented at the World Federation of Hemophilia (WFH) Virtual Summit, held June 14-19, 2020. Read the press release View the presentation

10 Jun ENVISION Phase 3 Results for Givosiran Published in the “New England Journal of Medicine”

The New England Journal of Medicine (NEJM) published pivotal results from the ENVISION Phase 3 study of givosiran, our RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria, in a manuscript titled “Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.” Read our press release Read the publication in the New England Journal of Medicine

07 Jun ILLUMINATE-A Phase 3 Results for Lumasiran Presented at ERA-EDTA

Positive results from the ILLUMINATE-A Phase 3 study of lumasiran, an investigational RNAi therapeutic in development for the treatment of Primary Hyperoxaluria Type 1 (PH1), were presented by study investigators at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress, held June 6-9, 2020 as a virtual event. Read the press release View the presentation