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.
Non-clinical data for APP-targeting siRNA showing reduced beta-CTF and corrected endosomal abnormalities in cell models of Alzheimer’s Disease were presented at AD/PD 2024, in Lisbon, Portugal.
New preclinical data for ALN-HTT02—an RNAi therapeutic development candidate targeting huntingtin (HTT) for Huntington’s disease (HD)—were presented during the CHDI Foundation’s 19th Annual Huntington’s Disease Therapeutics Conference in Palm Springs, CA . Additional data were presented on the emerging tolerability profile of the underlying C16-siRNA central nervous system delivery platform, highlighting potential differences with antisense oligonucleotides (ASOs) in development for HD.
Cantley, et al. “A New Approach to HTT-Lowering Using C16-siRNA Conjugates”
De Angelis, et al. “Emerging Tolerability Profiles of C16-siRNA Conjugates for CNS Delivery”
Results from the Phase 2 KARDIA-1 study of zilebesiran, an investigational RNAi therapeutic targeting hepatic synthesis of angiotensinogen (AGT) in development for the treatment of hypertension, were published in the Journal of the American Medical Association (JAMA).
Results from a subgroup analysis of the Phase 3 ENVISION study examining the long-term (up to 36 months) efficacy and safety of givosiran in Taiwanese patients with acute hepatic porphyria (AHP) were published in the Journal of the Formosan Medical Association.
Results from a real-world retrospective cohort study on the clinical features of acute hepatic porphyria (AHP) in Japan were published in the Orphanet Journal of Rare Diseases.
On December 13, 2023, 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 presentation, click here
24-month results, including data on the odds of disease progression as assessed by NYHA class and disease stage, from an interim analysis of the open-label extension period of the APOLLO-B Phase 3 study of investigational patisiran in patients with the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis were presented during the American Heart Association (AHA) Scientific Sessions 2023.
New results from the KARDIA-1 Phase 2 study of zilebesiran, an investigational RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of patients with hypertension and high cardiovascular risk, were presented at the American Heart Association (AHA) Scientific Sessions 2023.
New 24-month results from the ILLUMINATE-C Phase 3 study of lumasiran in patients with advanced primary hyperoxaluria type 1 (PH1) were presented at the American Society of Nephrology (ASN) Kidney Week 2023 Meeting. Additional findings from a retrospective study of the natural history of advanced PH1 were also presented.
Lieske, et al. “Lumasiran for Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 24-Month Analysis of the Phase 3 ILLUMINATE-C Trial”
Lieske, et al. “Natural History of Advanced Primary Hyperoxaluria Type 1: A Retrospective Study”
Results from the APOLLO-B Phase 3 study of investigational patisiran in patients with the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis were published in the New England Journal of Medicine.
