Capella

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 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.



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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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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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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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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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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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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.






 

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