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.

During a late-breaker session at the 18th International Symposium on Amyloidosis, we presented results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

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Maurer, et al. “Primary Results from APOLLO-B, a Phase 3 Study of Patisiran in Patients with Transthyretin-Mediated Amyloidosis with Cardiomyopathy”


During the 18th European Meeting on Complement in Human Disease (EMCHD), taking place August 26-28, 2022, we presented new results from the Phase 2 study of cemdisiran, an investigational RNAi therapeutic targeting the C5 component of the complement pathway that is being developed in collaboration with Regeneron Pharmaceuticals for the treatment of immunoglobulin A nephropathy.

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Barratt, et al. “Results from the Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy”

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.

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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”


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