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 data from a post-hoc analysis of the HELIOS-A Phase 3 study comparing efficacy outcomes with vutrisiran versus patisiran in hATTR amyloidosis with polyneuropathy were presented during the 2023 AAN Annual Meeting. Final results from a Phase 4 observational study evaluating the effect of patisiran on polyneuropathy and cardiomyopathy in patients with hATTR amyloidosis with V122I/T60A variants were also presented.

Polydefkis, et al. “Comparison of Efficacy Outcomes with Vutrisiran vs. Patisiran in hATTR Amyloidosis with Polyneuropathy: Post-hoc Analysis of the HELIOS-A Study”

Comenzo, et al. “Effect of Patisiran on Polyneuropathy and Cardiomyopathy in Patients with hATTR Amyloidosis with V122I/T60A Variants: A Phase 4 Observational Study”

During the National Kidney Foundation (NKF) 2023 Spring Clinical Meetings, we presented new 36-month results from the ILLUMINATE-A Phase 3 study of lumasiran in patients with primary hyperoxaluria type 1 (PH1).

Saland, et al. “Efficacy and Safety of Lumasiran in Patients with Primary Hyperoxaluria Type 1: 36-Month Analysis of the ILLUMINATE-A Trial”

Results from nonclinical work investigating the potential utility of combining RNAi-mediated knockdown with REVERSIR-enabled rescue of transgene expression as a rheostat for adeno-associated virus (AAV) -delivered genes in the setting of gene therapy were published in the journal Nature Communications.

Read the Manuscript

We presented post hoc analyses from the Phase 3 ENVISION study of givosiran in patients with acute hepatic porphyria (AHP) through Month 36 at the Hemostasis & Thrombosis Research Society (HTRS) Scientific Symposium, held on March 10-12 in Orlando, Florida.

Deering, et al. “Clinical Outcomes in Patients with Acute Hepatic Porphyria Treated with Givosiran Who Stopped Hemin Prophylaxis at Study Entry: Post hoc Analyses from the Phase 3 ENVISION Study Through Month 36”

We presented non-clinical results on ALN-APP*, our investigational RNAi therapeutic for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy at AD/PD 2023 Advances in Science & Therapy International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders, held on March 28-April 1 in Gothenburg, Sweden.

Brown, et al. – Discovery and Nonclinical Development of ALN-APP, an Investigational RNAi Therapeutic

*The ALN-APP clinical program is being conducted as a partnership between Alnylam Pharmaceuticals and Regeneron Pharmaceuticals, Inc.

On December 15, 2022, we hosted a virtual R&D Day event showcasing Alnylam’s late stage clinical efforts, next wave programs, and platform advances. The event included presentations from Alnylam senior leaders as well as guest speakers.

To view the webcast, click here
To view the R&D Day presentation, click here


During the American Society of Nephrology (ASN) Kidney Week, we presented new 12-month results from the ILLUMINATE-C Phase 3 study of lumasiran in patients of all ages with primary hyperoxaluria type 1 (PH1) and advanced kidney disease. Additional research was presented on the development and validation of a novel assay to determine oxalate concentration in plasma.

Frishberg, et al. “Lumasiran for Patients with Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 12-Month Analysis of the Phase 3 ILLUMINATE-C Trial”
Clausen, et al. “Quantification of Oxalate in Human Plasma by Novel Liquid Chromatography—Tandem Mass Spectrometry: Method Development, Validation, and Application in Lumasiran Clinical Trials”


New 18-month findings evaluating Neurofilament light chain (NfL) as a potential biomarker of treatment response in patients with hATTR amyloidosis with polyneuropathy were presented at the 147th annual meeting of the American Neurological Association (ANA).

Aldinc, et al. “NfL Levels Significantly Decrease in Response to Treatment with Patisiran or Vutrisiran in hATTR Amyloidosis with Polyneuropathy”


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