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.
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.
On October 9, 2024, we hosted a TTR Investor Day to discuss Alnylam’s potential for market leadership in ATTR amyloidosis. The event included presentations from Alnylam’s senior commercial leaders, as well as a leading expert in ATTR amyloidosis.
To view the webcast, click here
To view the presentation, click here
Additional results from the HELIOS-B Phase 3 study of vutrisiran, an RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), were presented in the Late Breaking Clinical Research Session 1 at the Heart Failure Society of America Annual Scientific Meeting 2024.
New results from a post hoc subgroup analysis of the ENVISION Phase 3 study evaluating long-term outcomes in patients with acute hepatic porphyria (AHP) who were not attack-free after the first six months of GIVLAARI® (givosiran) treatment were presented at The International Congress of Porphyrins and Porphyrias (ICPP 2024). Additional data presented at ICPP 2024 included demographic and clinical characteristics of patients with AHP enrolled in ELEVATE, an international, prospective, observational registry.
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 EHDN & Enroll-HD 2024 Congress in Strasbourg, France. 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.
Sloan, et al. “ALN-HTT02, a Novel C16-siRNA Conjugate for HTT-lowering in the CNS”
Cantley, et al. “Tolerability of HTT-Lowering: Lessons Learned from Nonhuman Primates”
Findings from a subgroup analysis of the KARDIA-2 Phase 2 study of zilebesiran, in development for the treatment of hypertension, and new data from post-hoc analyses of the APOLLO-B Phase 3 and APOLLO-OLE studies of patisiran in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) were presented at the European Society of Cardiology Congress (ESC) 2024.
