Capella—the Online Voice of Progress in RNAi

Alnylam welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi into innovative medicines. For Alnylam, Capella is our online voice for communicating the scientific progress we are making as we work to develop innovative medicines for patients.

We reported interim results from our ongoing Phase 1 study with givosiran at the 2017 International Congress on Porphyrins and Porphyrias (ICPP), held June 25 – 28, 2017 in Bordeaux, France. Data presented were from the first three unblinded cohorts from Part C, in patients with acute intermittent porphyria (AIP) that experience recurrent attacks and initial data from the open-label extension (OLE) study.

Read our press release
View the complete Phase 1 interim and OLE data presentation
View the results from the EXPLORE natural history study
View the poster on healthcare utilization and costs
View the poster on disease burden in patients with AIP and recurrent attacks

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.


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.


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.

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 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.


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.

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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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