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.

New results from the six-month primary analysis of the ILLUMINATE-C Phase 3 open-label study of lumasiran in patients of all ages with advanced primary hyperoxaluria type 1 (PH1) were presented at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress (May 19-22, 2022).

Findings from a separate pooled pharmacokinetic and pharmacodynamic analysis of data from four clinical trials of lumasiran were also presented.

Read the press release
Groothoff, et al. “Lumasiran for Patients With Primary Hyperoxaluria Type 1 With Impaired Kidney Function: Data From the 6-Month Analysis of the Phase 3 ILLUMINATE-C Trial”
Gansner, et al. “Pharmacokinetics and Pharmacodynamics of Lumasiran: Analysis of Four Clinical Studies”

During the annual Heart Failure congress of the Heart Failure Association of the European Society of Cardiology, taking place May 21-24, 2022, we presented new 18-month results from the exploratory cardiac endpoints in the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis.

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Garcia-Pavia, et al. “HELIOS-A: 18-Month Exploratory Cardiac Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis”

New results from the HELIOS-A study of vutrisiran, an investigational RNAi therapeutic, in hereditary transthyretin-mediated (hATTR) amyloidosis patients with polyneuropathy, were presented during the Peripheral Nerve Society (PNS) 2022 Annual Meeting, taking place May 14-17, 2022.

Ajroud-Driss, et al. “HELIOS-A: Impact of Vutrisiran on Quality of Life and Functional Status in Hereditary Transthyretin-Mediated Amyloidosis”

New results on Alnylam’s platform advancements enabling delivery of therapeutic short interfering RNAs (siRNAs) to the central nervous system (CNS) were presented at the TIDES USA, Oligonucleotide & Peptide Therapeutics 2022 Meeting being held in Boston on May 9-12. Additional preclinical data were presented on Alnylam’s GEMINI platform for simultaneous silencing of two transcripts within the CNS with a single siRNA entity.

Thiel CS., “Recent Advancements for the Delivery of siRNAs to the Central Nervous System”

New results from the 12-month analysis of the ongoing ILLUMINATE-B Phase 3 open-label pediatric study of OXLUMO® (lumasiran) in patients less than six years of age with primary hyperoxaluria type 1 (PH1) were presented at the Pediatric Academic Societies (PAS) 2022 Meeting (April 21-25, 2022).

Michael, et al. “Efficacy and Safety of Lumasiran for Infants and Young Children With Primary Hyperoxaluria Type 1: 12-Month Analysis of the Phase 3 ILLUMINATE-B Trial”

New findings from a 24-month analysis of the ongoing ILLUMINATE-A Phase 3 study of OXLUMO® (lumasiran) in patients with primary hyperoxaluria type 1 (PH1) were presented during the National Kidney Foundation (NKF) Spring Clinical Meetings (April 6-10, 2022).

Lieske, et al. “Efficacy and Safety of Lumasiran in Patients with Primary Hyperoxaluria Type 1: 24-Month Analysis of the ILLUMINATE-A Trial”

Sas, et al. “Impact of Baseline Urinary Oxalate on Response to Lumasiran in Patients with Primary Hyperoxaluria Type 1”

Additional findings from a post hoc analysis of the impact of baseline urinary oxalate on response to lumasiran in patients with PH1 in the ILLUMINATE-A study were presented.

New results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic, in hereditary transthyretin-mediated (hATTR) amyloidosis patients with polyneuropathy, were presented during a poster presentation at the 71st Annual Meeting of the American College of Cardiology (ACC) held April 2–4, 2022. The data help further our understanding of the safety and efficacy of vutrisiran in patients with hATTR amyloidosis with polyneuropathy who have variants that have previously been associated with predominant cardiomyopathy.

González-Duarte, et al. “HELIOS-A: Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy”

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