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.
We published results from the EXPLORE natural history study of more than 100 acute hepatic porphyria (AHP) patients with recurrent attacks in Hepatology in a paper titled, “EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks.”
We presented results on the pediatric cohort of patients (N=16; age range 6-17) from the Phase 1/2 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the International Pediatric Nephrology Association (IPNA) 2019 Annual Meeting, held October 17-21 in Venice, Italy.
On October 10, 2019, we hosted an online RNAi Roundtable to review the progress with lumasiran, an RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1.
On October 7, 2019, we hosted an online RNAi Roundtable to review the progress with givosiran, an RNAi therapeutic in development for the treatment of acute hepatic porphyria.
On September 16, 2019, we hosted an online RNAi Roundtable to review the progress with patisiran and vutrisiran, RNAi therapeutics in development for the treatment of transthyretin-mediated amyloidosis.
ONPATTRO® (patisiran) is approved in the U.S. and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults, in the EU for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy, and in Japan for the treatment of transthyretin (TTR) type familial amyloidosis with polyneuropathy. Vutrisiran is an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
We presented new results from an analysis of the UK Biobank demonstrating a significant association of the V122I mutation, a highly prevalent mutation in the transthyretin (TTR) gene, with a clinical diagnosis of polyneuropathy. These results were presented at the Heart Failure Society of America (HFSA) 23rd Annual Scientific Meeting, held September 13-16 in Philadelphia, PA.
We presented additional results from the ENVISION Phase 3 study and the Phase 1/2 open-label extension (OLE) study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP), at the 2019 International Congress on Porphyrins and Porphyrias (ICPP), held September 8-11 in Milan, Italy.
We are hosting a series of online “RNAi Roundtables” at which Alnylam scientists and program leaders, clinical collaborators, and patients or patient advocates review recent progress in many of our late-stage pipeline programs and discuss the related disease areas.
We presented results from a proteome-wide biomarker analysis of samples from the APOLLO Phase 3 study of ONPATTRO® (patisiran), as well as results from an analysis of the UK Biobank on clinical outcomes and medical history of individuals with the non-pathogenic transthyretin (TTR) ‘stabilizing’ T119M variant, at the Second European Meeting of ATTR Amyloidosis (EU-ATTR) for Doctors and Patients, held September 2-3 in Berlin, Germany.
Our partner The Medicines Company and collaborators presented positive complete results from the ORION-11 Phase 3 study of inclisiran, an RNAi therapeutic in development for the treatment of hypercholesterolemia, at the European Society of Cardiology’s ESC Congress 2019, held August 31 to September 4, 2019 in Paris, France.
