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Capella

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

Measurement Properties of the Rasch-built Overall Disability Scale (R-ODS) in patients with hATTR Amyloidosis with Polyneuropathy

May 24, 2017

Measurement Properties of the Rasch-built Overall Disability Scale (R-ODS) in patients with hATTR Amyloidosis with Polyneuropathy

We presented data evaluating the measurement properties of the Rasch-built Overall Disability Scale (R-ODS) in patients with symptomatic hereditary ATTR (hATTR) amyloidosis with polyneuropathy.  R-ODS is a 24-item patient-reported outcome instrument that assesses activity and social participation limitation. R-ODS assessments were collected from two trials investigating patisiran.  These data were presented at the International Society for Pharmacoeconomics and Outcomes Research annual meeting in Boston, Massachusetts.


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Nonclinical Studies Investigating Mechanisms of Hepatotoxicity for GalNAc-siRNA Conjugates

May 3, 2017

Nonclinical Studies Investigating Mechanisms of Hepatotoxicity for GalNAc-siRNA Conjugates

We presented nonclinical data on the safety profile and mechanisms of hepatotoxicity for GalNAc-siRNA conjugates at the TIDES 2017 meeting in San Diego, California.


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Cardiac Subgroup Results from Phase 2 OLE Study of Patisiran, in Development for hATTR Amyloidosis

April 29, 2017

Cardiac Subgroup Results from Phase 2 OLE Study of Patisiran, in Development for hATTR Amyloidosis

We reported final 24-month results from the subgroup of hereditary ATTR (hATTR) amyloidosis patients with cardiac involvement in the Phase 2 open-label extension (OLE) study of patisiran at the European Society of Cardiology Heart Failure (ESC-HF) Congress in Paris, France.


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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.


Final Clinical Results from ORION-1 Phase 2 Study of Inclisiran, in Development for Hypercholesterolemia

March 17, 2017

Final Clinical Results from ORION-1 Phase 2 Study of Inclisiran, in Development for Hypercholesterolemia

Alnylam and The Medicines Company reported final results from the ORION-1 Phase 2 study of inclisiran, an investigational RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia, at the American College of Cardiology (ACC) Scientific Session 2017 in Washington DC.



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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.