Capella

22 Oct, 2020 ILLUMINATE-B Phase 3 Results for Lumasiran Presented at ASN

Positive results from the six-month primary analysis of the ongoing ILLUMINATE-B Phase 3 open-label pediatric study of lumasiran, an investigational RNAi therapeutic in development for the treatment of adults and children with Primary Hyperoxaluria Type 1 (PH1), were presented at the American Society of Nephrology (ASN) Kidney Week 2020, held October 22-25 as a virtual event. Additionally, new 12-month results from the ILLUMINATE-A pivotal Phase 3 study and the ongoing Phase 2 open-label extension (OLE) study were also presented.

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View the ILLUMINATE-A presentation
View the Phase 2 OLE presentation
View the Chart Review presentation

30 Sep, 2020 RNAi Therapeutics Platform Progress at OTS 2020 Annual Meeting

We presented progress on our RNAi therapeutics platform efforts at the Oligonucleotide Therapeutics Society (OTS) 2020 Annual Meeting.

Akinc et al. – “ALN-COV: An Investigational RNAi Therapeutic for COVID-19”
Brown et al. – “Evidence for an Intracellular Depot that Contributes to the Extended Duration of Activity of GalNAc-siRNA Conjugates”
Jadhav et al. – “Human Translation of GalNAc-siRNA Conjugates with Improved Specificity”
Janas et al. – “Advancing the Safety and Reach of RNAi Therapeutics”
MacLauchlin et al. – “The ADME of siRNA GalNAc Conjugates”

14 Sep, 2020 RNAi Roundtable: Givosiran, for the Treatment of Acute Hepatic Porphyria

On September 14, 2020, we hosted an online RNAi Roundtable to review progress with givosiran, an RNAi therapeutic for the treatment of acute hepatic porphyria.

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03 Sep, 2020 RNAi Roundtable: Patisiran & Vutrisiran, in Development for the Treatment of Transthyretin-Mediated Amyloidosis

On September 3, 2020, we hosted an online RNAi Roundtable to review progress with patisiran and vutrisiran, RNAi therapeutics in development for the treatment of transthyretin-mediated amyloidosis. Of note, we announced we have obtained clinical pharmacology data supporting the potential for a biannual subcutaneous dosing regimen option for vutrisiran, providing further product differentiation as a potential best-in-class agent, and discussed new clinical data presented at the European Society of Cardiology 2020 Congress providing further evidence that treatment with patisiran may lead to substantial reduction in cardiac amyloid burden in ATTR amyloidosis.

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29 Aug, 2020 New Results for GIVLAARI® (givosiran) at The Digital International Liver Congress™ 2020

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

20 Aug, 2020 Nonclinical Research on Pharmacodynamic Durability of GalNAc-siRNA Conjugates Published in “Nucleic Acids Research”

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.

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Read the paper in Nucleic Acids Research

10 Aug, 2020 RNAi Roundtable: Lumasiran, in Development for the Treatment of Primary Hyperoxaluria Type 1

On August 10, 2020, we hosted an online RNAi Roundtable to review progress with lumasiran, an RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1.

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17 Jul, 2020 RNAi Roundtable: Early Stage RNAi Therapeutics Pipeline

On July 17, 2020, we hosted an online RNAi Roundtable to review progress in our early stage RNAi therapeutics pipeline.

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10 Jul, 2020 New Results for ONPATTRO (patisiran) at 2020 PNS Annual Meeting

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 New 12-Month Interim Data From the ENVISION Phase 3 Study of Givosiran in Acute Hepatic Porphyria

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.

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