Capella

Capella—the Online Voice of Progress in RNAi

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 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.

Jering, et al. “The Relationship Between Cardiac Structure, Function, and Clinical Outcomes and the Impact of Vutrisiran from the HELIOS-B Trial”

Maurer, et al. “Impact of Baseline Heart Failure Severity on Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy in the HELIOS-B Trial: A Subgroup Analysis”

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Sheikh, et al. “Maintenance or Improvement of Functional Capacity, Health Status, and Quality of Life with Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy: Data from the HELIOS-B Study”

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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.

Sloan et al. “ALN-HTT02, an Investigational RNAi Therapeutic Targeting Exon 1 of HTT in Phase 1 Development for Huntington’s Disease”

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

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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.

Brown, et al. “Targeting Cerebral Amyloid Angiopathy at its Source: An Amyloid-beta Precursor Protein-Targeted siRNA Reduced Vascular Amyloid-beta and Hemorrhage in Rodent Models”

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.

Fontana, et al. “Impact of Vutrisiran on Outpatient Worsening Heart Failure in Patients with Transthyretin Amyloidosis with Cardiomyopathy in the HELIOS-B Trial”

Krohn, et al. “Impact of Long-Term Blood Pressure Variability on Adverse Cardiovascular Outcomes in High- and Low-Risk Populations”

Zappe, et al. “Home Blood Pressure Reductions with Zilebesiran in Patients with Mild-to-Moderate Hypertension Are Consistent with Ambulatory and Office Blood Pressure Reductions in the KARDIA-1 Study”

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.

Murad et al. “A Phase 1, Single Ascending Dose Study to Evaluate ALN-TTRsc04, a Next-Generation RNA Interference Therapeutic, in Healthy Participants for Potential Treatment of Transthyretin Amyloidosis”

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”

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

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