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.
A new subgroup analysis from the KARDIA-1 Phase 2 study of zilebesiran, an investigational RNAi therapeutic in development for the treatment of hypertension, was presented at the 2024 American College of Cardiology (ACC) Annual Scientific Session.
Results from the KARDIA-2 Phase 2 study of zilebesiran, an investigational RNAi therapeutic in development for the treatment of hypertension, when added to standard of care antihypertensive medications, were presented at the 2024 American College of Cardiology (ACC) Annual Scientific Session.
New results from a post-hoc analysis of outpatient heart failure worsening in the APOLLO-B Phase 3 study of patisiran were presented at the 2024 ACC Annual Scientific Session. Additional data were also presented from a meta-regression analysis of the association between change in 6-minute walk distance and survival in transthyretin-mediated amyloidosis with cardiomyopathy.
Fontana, et al. “Outpatient Heart Failure Worsening in Patients with Cardiac Transthyretin Amyloidosis: Results from the APOLLO-B Trial”
Kumar, et al. “Meta-Regression Analysis of the Association Between Change in Six Minute Walk Distance and Survival in Transthyretin-Mediated Amyloidosis With Cardiomyopathy”
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.
