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Alnylam welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi toward innovative medicines.

Updated Results from Phase 1/2 Study of ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

December 5, 2016

Updated Results from Phase 1/2 Study of ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

We reported new results from Part C of our Phase 1/2 clinical study of ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.



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

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.





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

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 American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California.



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Interim Results from EXPLORE Natural History Study for Acute Hepatic Porphyrias

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 mean of 9.5 acute attacks reported in the prior year with severe neuropathic pain as the cardinal feature in 100% of attacks, along with other symptoms including nausea or vomiting (>80%), fatigue (77%), and weakness (79%).  Approximately 64% of patients reported experiencing porphyria symptoms between attacks, with about 46% experiencing symptoms daily.  Patients also reported a diminished quality of life and significant healthcare utilization, with a mean of 4.6 overnight hospitalizations per year (range 0-70) and a mean hospital stay duration of 6.6 days (range 1-60).  Of the attacks reported on study, approximately 76% of them required treatment with heme or at a healthcare facility.


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

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.



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

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.


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

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.



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

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.



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2016 RNAi Roundtable Series

July 19, 2016

2016 RNAi Roundtable Series

We are hosting a series of online “RNAi Roundtables” in which Alnylam scientists and key clinical collaborators review recent progress in many of the company’s development-stage pipeline programs and discuss the related disease areas. Click here to access our current and previous presentations.