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 Part D of the Phase 1 study of zilebesiran, assessing the safety and pharmacology of sequential doses of zilebesiran in patients with Class II or III obesity, were presented during the American Heart Association Hypertension Scientific Sessions 2023.

Taubel, et al. “Safety and Tolerability of Zilebesiran, an RNAi Interference Therapeutic Targeting Hepatic Angiotensinogen Synthesis, in Obese Patients with Hypertension”

New results from a post hoc analysis of the APOLLO-B Phase 3 study of patisiran in patients with ATTR amyloidosis with cardiomyopathy evaluating disease progression following 12 months of treatment with patisiran vs. placebo were presented during the European Society of Cardiology (ESC) Congress 2023.

Damy, et al. “Evaluation of Disease Progression in Patients with ATTR Amyloidosis with Cardiomyopathy Following Treatment with Patisiran: Post hoc Analysis of the APOLLO-B Study”

We presented updated positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA), at the 2023 Alzheimer’s Association International Conference (AAIC).

Cohen et al. “Interim Phase 1 Part A Results for ALN-APP, the First Investigational RNAi Therapeutic in Development for Alzheimer’s Disease”

At the European Association for the Study of the Liver (EASL) Congress 2023, we presented results from a Phase 1/2, 48-month, open-label extension study and a qualitative patient interview study of givosiran.

Sardh, et al. “Final Results from a Phase 1/2, 48-Month, Open-Label Extension Study of Givosiran in Patients with Acute Intermittent Porphyria
Lombardelli, et al. “Patient Experience with Acute Hepatic Porphyria before and after long-term givosiran treatment: a qualitative interview study”

We presented findings from a Phase 1 study of ALN-HSD, an investigational RNAi therapeutic, in healthy adults and patients with nonalcoholic steatohepatitis, at the European Association for the Study of the Liver (EASL) Congress 2023.

Sanyal, et al. “Phase 1 Study of the RNA Interference Therapeutic ALN-HSD in Healthy Adults and Patients with Nonalcoholic Steatohepatitis”

We presented an overview of the preclinical development program for ALN-KHK, an investigational RNAi therapeutic for type 2 diabetes mellitus, at the American Diabetes Association (ADA) 83rd Scientific Sessions.

Noetzli, et al. “Preclinical Development of ALN-KHK, an Investigational RNAi Therapeutic for Type 2 Diabetes Mellitus”

At the European Renal Association (ERA) 2023 Meeting, we presented results from a novel X-ray-based bone oxalosis grading scale for patients with primary hyperoxaluria type 1 (PH1).

Baker, et al. “Development of an X-ray–Based Bone Oxalosis Grading Scale to Assess Oxalate Accumulation in Patients with Primary Hyperoxaluria Type 1”

We presented an overview of the lumasiran clinical development program, including data from the Phase 3 ILLUMINATE-A, ILLUMINATE-B and ILLUMINATE-C studies, in patients with primary hyperoxaluria type 1 (PH1) at the Oxalosis & Hyperoxaluria Foundation (OHF) 14th International Hyperoxaluria Workshop.

Shasha-Lavsky, et al. “Targeting Glycolate Oxidase for the Treatment of Primary Hyperoxaluria Type 1: Development and Clinical Characterization of Lumasiran, an RNAi Therapeutic.”


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