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Capella

Capella—the Online Voice of Progress in RNAi

Welcome to Capella, Alnylam’s destination for updates on our work translating the breakthrough discovery of RNA interference (RNAi) into an innovative new class of medicines. We’ve been pioneering RNAi therapeutics since 2002 and are excited to share our ongoing scientific progress.

29 Aug, 2020

Posted at 11:25h in Capella

During The Digital International Liver Congress™ 2020, we shared new interim data from the Phase 1/2 open-label extension (OLE) study of GIVLAARI® (givosiran) in acute hepatic porphyria (AHP), as well as encore 12-month interim data from the OLE period of the ENVISION Phase 3 study of GIVLAARI.

Stein, et al. – “A Phase 1/2 Open-Label Extension Study of Givosiran, an Investigational RNAi Therapeutic, in Patients with Acute Intermittent Porphyria”
View the 12-month ENVISION OLE results

(more…)

20 Aug, 2020

Posted at 09:45h in Capella

We published nonclinical research describing the durability of gene silencing by GalNAc-conjugated siRNAs. The article, titled, “Investigating the Pharmacodynamic Durability of GalNAc-siRNA Conjugates,” was selected as a Breakthrough Paper by Nucleic Acids Research.

Read the press release
Read the paper in Nucleic Acids Research

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10 Jul, 2020

Posted at 10:56h in Capella

New results and analyses of study data for ONPATTRO® (patisiran) were presented at the 2020 Peripheral Nerve Society (PNS) Annual Meeting, held June 27-30 as a virtual event.

Adams, et al. – “Global Open-Label Extension: 24-Month Data of Patisiran in Patients with hATTR Amyloidosis”

Lin, et al. – “Evaluation of Patisiran with Concomitant or Prior Use of Transthyretin Stabilizers”

Peltier, et al. – “Impact of Patisiran on Activities of Daily Living and Functional Status in hATTR Amyloidosis”

Coelho, et al. – “Open-Label Study of Patisiran in Patients with hATTR Amyloidosis Post-Orthotopic Liver Transplant”

Ticau, et al. – “Neurofilament Light Chain May Serve as a Biomarker in Hereditary Transthyretin-Mediated Amyloidosis”

30 Jun, 2020

Posted at 09:00h in Capella

Interim results from the open-label extension (OLE) period of the ENVISION Phase 3 study of GIVLAARI® (givosiran) in acute hepatic porphyria (AHP) were presented by study investigator Eliane Sardh, M.D., Ph.D. during a webinar hosted by Alnylam, demonstrating sustained efficacy and acceptable safety through 12 months of treatment, with evidence for potentially improved efficacy over time.

Read our press release

View the presentation

View the webinar

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18 Jun, 2020

Posted at 09:26h in Capella

We are hosting a series of online “RNAi Roundtables” at which Alnylam scientists and program leaders, as well as clinical collaborators, will review recent progress in many of our pipeline programs and platform.

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10 Jun, 2020

Posted at 17:07h in Capella

The New England Journal of Medicine (NEJM) published pivotal results from the ENVISION Phase 3 study of givosiran, our RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria, in a manuscript titled “Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.”

Read our press release

Read the publication in the New England Journal of Medicine

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