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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.

27 Feb, 2025

Posted at 12:00h in Capella

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”

25 Feb, 2025

Posted at 09:01h in Capella

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

(more…)

06 Feb, 2025

Posted at 09:27h in Capella

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”

18 Nov, 2024

Posted at 13:09h in Capella

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”

17 Nov, 2024

Posted at 10:36h in Capella

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”

09 Nov, 2024

Posted at 15:12h in Capella

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”

09 Oct, 2024

Posted at 08:06h in Capella

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

(more…)

29 Sep, 2024

Posted at 09:17h in Capella

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.

Jering, et al. “Effects of Vutrisiran on Echocardiographic Cardiac Structure and Function: The HELIOS-B Trial”

Maurer, et al. “Exploratory Biomarker Analyses from HELIOS-B, a Phase 3 Study of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy”

24 Sep, 2024

Posted at 09:39h in Capella

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.

Ventura, et al. “Long-term clinical outcomes of patients with acute hepatic porphyria who were not attack-free after 6 months of givosiran treatment: a subgroup analysis of the phase 3 ENVISION study”

Sardh, et al. “Patient demographics and clinical characteristics at enrolment in ELEVATE, an international registry of acute hepatic porphyria”

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