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.
On June 25, 2026, we hosted an online RNAi Roundtable to review progress with ALN-6400, an investigational RNAi therapeutic in development to address rare bleeding disorders.
A post-hoc analysis of HELIOS-A demonstrated that vutrisiran and patisiran improved outcomes in patients with hATTR-PN regardless of baseline nerve conduction status, with those treated earlier in their disease course showing the greatest potential for disease reversal. Additionally, results of the NeuroFeeL study showed that neurofilament light chain (NfL) levels correlate with hATTR-PN disease severity, reinforcing NfL’s potential as a biomarker to support earlier diagnosis and clinical management.
A pooled analysis from the KARDIA Phase 2 studies demonstrated zilebesiran’s manageable safety profile as an add-on treatment for patients with hypertension receiving treatment with a background renin-angiotensin-aldosterone system (RAAS) inhibitor. The findings support continued evaluation of zilebesiran in the Phase 3 cardiovascular outcomes (CVOT) trial, ZENITH.
Analyses presented at Heart Failure 2026 reinforce the consistent clinical profile of vutrisiran in patients with the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), including across clinically complex populations with a high disease burden and in the context of concomitant therapies. Additional data further characterize transthyretin (TTR) knockdown and support its relevance in real-world clinical practice.
A pooled safety analysis evaluating vitamin A-related outcomes across clinical trial and real-world datasets shows no evidence of clinically meaningful safety concerns associated with TTR reduction. The design of DemonsTTRate, a global, long-term observational study evaluating real-world use of vutrisiran in ATTR-CM, was also presented.
The design and rationale for the TRITON-PN Phase 3 study evaluating our third-generation investigational transthyretin (TTR) silencer, nucresiran, in patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) was presented at AAN 2026.
Analyses presented at ACC reinforce the totality of data for vutrisiran in patients with ATTR-CM including the impact on cardiovascular outcomes across a range of patient subgroups, including those with most advanced disease and diastolic dysfunction and on health-related quality of life. Real-world evidence also shows high treatment adherence with quarterly dosing.
A pooled safety analysis from the Phase 2 KARDIA studies demonstrate zilebesiran’s acceptable safety profile across a broad hypertension population, supporting continued investigation in the Phase 3 ZENITH cardiovascular outcomes trial.
On March 24, 2026, we hosted a TTR investor webinar to highlight Alnylam’s progress in delivering for patients with ATTR-CM, as well as the long-term growth and durability of the Company’s flagship TTR franchise. The event included presentations from Alnylam’s senior commercial and medical leaders.
To view the webcast, click here
To view the presentation, click here
Data on emerging assays measuring huntingtin (HTT) protein in human cerebrospinal fluid were presented at the CHDI Foundation’s 21st Annual Huntington’s Disease Therapeutics Conference in Palm Springs, CA. Additional presentations on nonclinical data supporting the tolerability of deep and sustained HTT-lowering in wild-type nonhuman primates and an overview of the ongoing Phase 1 study of HTT02 in adult patients with Huntington’s disease were also included. ALN-HTT02 is an investigational RNAi therapeutic targeting exon 1 of HTT for Huntington’s disease.
Bond et al. “Emerging Approaches to Improve the Quantification of Huntingtin Protein in Human Cerebrospinal Fluid”
Cantley et al. “Preclinical Characterization of ALN-HTT02, an Investigational RNAi Therapeutic Targeting Exon 1 of HTT”
Sloan et al. “ALN-HTT02, an Investigational RNAi Therapeutic Targeting Exon 1 of HTT in Phase 1 Development for Huntington’s Disease”
Rates of amyloid-related imaging abnormalities (ARIA) from an interim analysis of the ongoing Phase 1 study of mivelsiran in patients with early-onset Alzheimer’s disease (EOAD) were presented at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference 2025 in San Diego, CA. Mivelsiran is an investigational CNS-targeting RNAi therapeutic designed to reduce amyloid precursor protein (APP) and downstream Aβ peptides.
New analyses highlight that vutrisiran improved measures of heart structure and function and reduced amyloid burden in some patients.
Further findings show vutrisiran preserved kidney function and reduced the risk of death and cardiovascular (CV) events in patients with advanced chronic kidney disease (CKD).
Results from Cohort B of the KARDIA-3 Phase 2 trial evaluating zilebesiran in patients with uncontrolled hypertension with established or at high risk for CV disease on two or more background medications were also presented as a late-breaking abstract.
