Welcome to Capella, Alnylam’s destination for updates on our work translating the breakthrough discovery of RNA interference (RNAi) into an innovative new class of medicines. We’ve been pioneering RNAi therapeutics since 2002 and are excited to share our ongoing scientific progress.
On September 14, 2020, we hosted an online RNAi Roundtable to review progress with givosiran, an RNAi therapeutic for the treatment of acute hepatic porphyria.
On September 3, 2020, we hosted an online RNAi Roundtable to review progress with patisiran and vutrisiran, RNAi therapeutics in development for the treatment of transthyretin-mediated amyloidosis. Of note, we announced we have obtained clinical pharmacology data supporting the potential for a biannual subcutaneous dosing regimen option for vutrisiran, providing further product differentiation as a potential best-in-class agent, and discussed new clinical data presented at the European Society of Cardiology 2020 Congress providing further evidence that treatment with patisiran may lead to substantial reduction in cardiac amyloid burden in ATTR amyloidosis.
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During The Digital International Liver Congress™ 2020, we shared new interim data from the Phase 1/2 open-label extension (OLE) study of GIVLAARI® (givosiran) in acute hepatic porphyria (AHP), as well as encore 12-month interim data from the OLE period of the ENVISION Phase 3 study of GIVLAARI.
Stein, et al. – “A Phase 1/2 Open-Label Extension Study of Givosiran, an Investigational RNAi Therapeutic, in Patients with Acute Intermittent Porphyria”
View the 12-month ENVISION OLE results
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.
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Read the paper in Nucleic Acids Research
On August 10, 2020, we hosted an online RNAi Roundtable to review progress with lumasiran, an RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1.
On July 17, 2020, we hosted an online RNAi Roundtable to review progress in our early stage RNAi therapeutics pipeline.
New results and analyses of study data for ONPATTRO® (patisiran) were presented at the 2020 Peripheral Nerve Society (PNS) Annual Meeting, held June 27-30 as a virtual event.
Lin, et al. – “Evaluation of Patisiran with Concomitant or Prior Use of Transthyretin Stabilizers”
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.
On June 23, 2020, we hosted an online RNAi Roundtable to review the progress with ALN-AGT, an RNAi therapeutic in development for the treatment of hypertension.
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.
