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Alnylam

Archives: Presentations

December 3, 2016

Interim Clinical Results from Fitusiran, an investigational RNAi therapeutic for the Treatment of Hemophilia and Rare Bleeding Disorders

We reported positive interim results from our Phase 1 study of fitusiran in patients with hemophilia with inhibitors as well as from our Phase 2 open-label extension (OLE) study in hemophilia patients without inhibitors at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.

December 3, 2016

Initial Evidence of Clinical Activity Shown with Givosiran (ALN-AS1) in Acute Intermittent Porphyria Patients with Recurrent Attacks

We reported positive initial results from Cohorts 1 and 2 in Part C of our Phase 1 study with givosiran (gi-VOH-si-ran), the International Nonproprietary Name for ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias.  These results were presented at the 58th Annual Meeting of the Read More

November 11, 2016

Interim Results from EXPLORE Natural History Study for Acute Hepatic Porphyrias

We reported interim results from our ongoing EXPLORE study, a multinational, prospective, observational study aimed at characterizing the natural history and clinical management of patients with acute hepatic porphyrias (AHP) who experience recurrent attacks or receive prophylactic treatment to prevent attacks.  Results from 112 patients, of which 104 have acute intermittent porphyria (AIP), showed a Read More

September 28, 2016

Clinical Data from ALN-AAT and Non-Clinical Research Demonstrating Continued RNAi Platform Optimization and Leadership in the Field

We presented clinical and non-clinical data in a series of posters and oral presentations at the 12th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held September 25 – 28, 2016 in Montreal, Quebec.

September 24, 2016

Initial Results from Ongoing Phase 1/2 Study with ALN-GO1, in Development for the Treatment of Primary Hyperoxaluria Type 1

We presented initial clinical data from ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1), at the 17th Congress of the International Pediatric Nephrology Association (IPNA), held September 20 – 24, 2016 in Iguaçu, Brazil.

September 14, 2016

Pre-Clinical Results from ALN-CC5 for the Treatment of Complement-Mediated Diseases

We reported new pre-clinical results in animal models of myasthenia gravis (MG) from ALN-CC5, an investigational RNAi therapeutic in development for the treatment of complement-mediated diseases, at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, held September 14-17 in New Orleans, Louisiana.

September 7, 2016

Positive Interim Results from Ongoing Phase 1 Study with ALN-AS1, in Development for the Treatment of Acute Hepatic Porphyrias

We presented interim results from our ongoing Phase 1 clinical trial with ALN-AS1, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHP), at the 2016 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, held from September 6 – 9, 2016 in Rome, Italy.

July 25, 2016

Interim Data from Ongoing Phase 1 Trial with Fitusiran for the Treatment of Hemophilia and Rare Bleeding Disorders

We reported new data from Parts C and D of our Phase 1 study with fitusiran, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders, at the World Federation of Hemophilia (WFH) World Congress, held July 24-28, 2016 in Orlando, Florida.

July 1, 2016

Updates from Patisiran and Revusiran, in Development for the Treatment of hATTR Amyloidosis

We reported new clinical data from our ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of hereditary TTR-mediated amyloidosis (hATTR amyloidosis). These data were presented at the XV International Symposium on Amyloidosis held July 3 – 7, 2016 in Uppsala, Sweden.

June 11, 2016

Initial Data in Paroxysmal Nocturnal Hemoglobinuria Patients from Ongoing Phase 1/2 Clinical Study with ALN-CC5 Support Potential for Eculizumab Sparing and Improved Disease Control for Eculizumab Inadequate Responders

We reported data from 6 patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) enrolled in Part C of our ongoing Phase 1/2 clinical study with ALN-CC5, an investigational RNAi therapeutic targeting complement component 5 (C5) for the treatment of complement-mediated diseases. These data were presented at the 21st Congress of the European Hematology Association (EHA) Meeting in Read More