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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.

Final 24-Month Results from Phase 2 OLE Study of Patisiran, in Development for hATTR Amyloidosis

April 26, 2017

Final 24-Month Results from Phase 2 OLE Study of Patisiran, in Development for hATTR Amyloidosis

We reported final 24-month results from our Phase 2 open-label extension (OLE) study of patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR (hATTR) amyloidosis. These data were presented at the American Academy of Neurology (AAN) 2017 Annual Meeting in Boston, Massachusetts.



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Pre-Clinical Data with a Combinatorial RNAi/Vaccination Therapy as a Potential Functional Cure for Chronic Hepatitis B Virus Infection

April 20, 2017

Pre-Clinical Data with a Combinatorial RNAi/Vaccination Therapy as a Potential Functional Cure for Chronic Hepatitis B Virus Infection

We presented pre-clinical data with a combinatorial RNAi/vaccination therapy for Hepatitis B Virus (HBV) infection at The EASL International Liver Congress, held April 19-23 in Amsterdam, The Netherlands. Treatment with the combination therapy led to an HBV-specific immune response and sustained loss of HBsAg in a mouse model of chronic HBV infection. We believe this approach holds potential promise for use in a clinical setting to treat patients with hepatitis B.


Results of Survey Assessing Utility and User Experience with Alnylam Act Reported at Annual Clinical Genetics Meeting

March 23, 2017

Results of Survey Assessing Utility and User Experience with Alnylam Act Reported at Annual Clinical Genetics Meeting

We presented results from a survey evaluating the utility and user experience of an independent genetic testing and counseling service for hereditary ATTR (hATTR) amyloidosis that is supported by Alnylam – Alnylam Act – at the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, held March 21 – 25 in Phoenix, Arizona. In May 2016, a 20-question survey was sent to 142 healthcare professionals who registered for an account to use Alnylam Act. The results of the survey demonstrated the free third-party services are useful in diagnosing or ruling out hATTR amyloidosis in individuals at risk based on symptomology or family history. The survey also showed that the program is helping individuals overcome barriers to genetic testing, such as lack of – or inadequate – insurance coverage.


Alnylam Act

February 27, 2017

Alnylam Act

Alnylam is sponsoring free, third-party genetic testing and counseling for individuals who may carry a gene mutation known to be associated with hereditary ATTR (hATTR) amyloidosis. The Alnylam Act™ (formerly known as Alnylam Assist) program was created to potentially increase diagnosis rates and to provide genetic counseling to help people make more informed decisions about their health. Learn More.

New Exploratory Analysis Results from Fitusiran, an Investigational RNAi Therapeutic for the Treatment of Hemophilia and Rare Bleeding Disorders

February 2, 2017

New Exploratory Analysis Results from Fitusiran, an Investigational RNAi Therapeutic for the Treatment of Hemophilia and Rare Bleeding Disorders

We reported new results from an exploratory analysis of our Phase 1 study with fitusiran, an investigational RNAi therapeutic, in patients with hemophilia A or B with or without inhibitors at the 10th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD) held February 1 – 3, 2017 in Paris, France.




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R&D Day 2016

December 16, 2016

R&D Day 2016

On December 16, 2016, we hosted our fourth consecutive R&D Day in New York City. Alnylam management and key opinion leaders discussed our most advanced clinical programs, reviewing all the latest data for patisiran, fitusiran and givosiran.



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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