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.
The following presentations include the latest data from the HELIOS-B Phase 3 study and results from across the flagship transthyretin amyloidosis (TTR) franchise presented at Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology.
An Analysis of US Insurance Claims Data examining real-world treatment persistency among patients receiving tafamidis was presented at the American College of Cardiology Scientific Session 2025.
Patel, et al. “Real-World Persistency on Tafamidis: An Analysis of US Insurance Claims Data”
The following analyses from the Phase 3 HELIOS-B study which evaluated vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy were presented at the American College of Cardiology Scientific Session 2025. Two analyses were also published in the Journal of American College of Cardiology.
The design of an ongoing Phase 1 study of ALN-HTT02—an investigational RNAi therapeutic targeting exon 1 of huntingtin (HTT) for Huntington’s disease—was presented at the CHDI Foundation’s 20th Annual Huntington’s Disease Therapeutics Conference in Palm Springs, CA. Nonclinical data supporting the tolerability of deep and sustained HTT-lowering in wild-type nonhuman primates after single and repeated intrathecal administration of ALN-HTT02—which employs Alnylam’s C16-siRNA CNS delivery platform—were also shared.
On February 25, 2025, we hosted an 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 a leading expert in Huntington’s disease.
To view the webcast, click here
To view the presentation, click here
Therapeutic rationale for APP-lowering siRNA and preclinical results from rodent models of CAA were presented at the International Stroke Conference (ISC), February 5-7, 2025, Los Angeles, United States.
The following studies were presented at the American Heart Association (AHA) Scientific Sessions 2024: a new analysis of the Phase 3 HELIOS-B study of vutrisiran, an investigational RNAi therapeutic for the treatment of ATTR cardiomyopathy, and a new analysis of the Phase 2 KARDIA-1 study of zilebesiran, an investigational RNAi therapeutic for the treatment of hypertension.
Results from the interim Phase 1 study of nucresiran (ALN-TTRsc04), an investigational next-generation RNAi therapeutic for the treatment of transthyretin amyloidosis, were presented at the American Heart Association (AHA) Scientific Sessions 2024.
The design of a Phase 1 study of ALN-HTT02—an investigational RNAi therapeutic targeting huntingtin (HTT) for Huntington’s disease—was presented at the 31st Annual Meeting of the Huntington Study Group (HSG) in Cincinnati, Ohio.
Sloan, et al. “ALN-HTT02, a Novel C16-siRNA Conjugate for HTT-lowering in the CNS”
Trial design and rationale for the global Phase 2 cAPPricorn-1 study of mivelsiran (ALN-APP) in patients with cerebral amyloid angiopathy (CAA), were presented at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference 2024.
