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 new long-term results from the global Open-Label Extension (OLE) study of ONPATTRO® (patisiran) at the European Academy of Neurology (EAN) Virtual Congress, held May 23-26, 2020. In addition, results were presented from a Phase 3b trial evaluating treatment with patisiran in patients with hATTR amyloidosis with disease progression after receiving an orthotopic liver transplant (post-OLT). A post-hoc analysis of the ENVISION Phase 3 study of GIVLAARI® (givosiran) was also presented.

Read the press release

View the patisiran global OLE poster

View the patisiran post-OLT poster

View the ENVISION post-hoc analysis

View the presentation on disease burden and healthcare utilization among patients with acute intermittent porphyria

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An abstract describing new 12-month interim data from the ongoing ENVISION open-label extension (OLE) study of givosiran in patients with acute hepatic porphyria (AHP) has been published online by the European Association for the Society of the Liver (EASL) on the International Liver Congress™ (ILC 2020) website.  The data will be presented during the ILC 2020 (London, UK) which was rescheduled to August 25-28, 2020 due to the COVID-19 pandemic.

View the ENVISION OLE abstract

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On November 22nd, 2019, we will be hosting an R&D Day in New York City. The event will showcase Alnylam’s late stage clinical efforts, next wave programs, and platform advances. The Company will also discuss its perspective on its transition toward achieving a self-sustainable financial profile.

To view the webcast, click here. A replay of the webcast will be available at that link on November 23rd.

To view the R&D Day Presentation, click here.

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We presented new positive efficacy results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran, an investigational subcutaneously administered RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1), at the American Society of Nephrology (ASN) 2019 Annual Meeting, held November 5-10 in Washington, DC.

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View the Phase 2 OLE poster

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We presented results on the pediatric cohort of patients (N=16; age range 6-17) from the Phase 1/2 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the International Pediatric Nephrology Association (IPNA) 2019 Annual Meeting, held October 17-21 in Venice, Italy.

Frishberg et al. – Phase 1/2 Study of Lumasiran, Investigational RNAi Therapeutic, in Patients with Primary Hyperoxaluria Type 1

Danese et al. – Understanding the Burden of Primary Hyperoxaluria Type 1 (PH1): A Survey of Physician Experiences with PH1

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On September 16, 2019, we hosted an online RNAi Roundtable to review the progress with patisiran and vutrisiran, RNAi therapeutics in development for the treatment of transthyretin-mediated amyloidosis.

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View the presentation

Read the transcript

ONPATTRO® (patisiran) is approved in the U.S. and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults, in the EU for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy, and in Japan for the treatment of transthyretin (TTR) type familial amyloidosis with polyneuropathy. Vutrisiran is an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.

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We presented new results from an analysis of the UK Biobank demonstrating a significant association of the V122I mutation, a highly prevalent mutation in the transthyretin (TTR) gene, with a clinical diagnosis of polyneuropathy. These results were presented at the Heart Failure Society of America (HFSA) 23rd Annual Scientific Meeting, held September 13-16 in Philadelphia, PA.

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Parker et al. – “The V122I Mutation in Hereditary Transthyretin-Mediated Amyloidosis is Significantly Associated with Polyneuropathy”

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