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.
Presentations demonstrated the potential of RNAi therapeutics to transform the treatment of cardiovascular disease.
Human genetics and proteomics data from UK Biobank as well as data from preclinical models relevant to ALN-6400—an investigational RNAi therapeutic targeting plasminogen for bleeding disorders—were presented at the 2025 European Hematology Association (EHA) Congress, June 12-15, Milan, Italy.
Detailed results from the KARDIA-2 Phase 2 study supporting the safety and efficacy of zilebesiran, an investigational RNAi therapeutic for the treatment of hypertension, when used in combination with one of three standard classes of antihypertensive medications were published in The Journal of the American Medical Association.
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.
