C-7DLXRNAi therapeutics, gene silencing, siRNA delivery
0-100 chain-logic scale · 15 dimensions · scored on public evidence
Evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of ALN-6222 in adults with obesity.
First-in-human randomized, double-blind, placebo-controlled single ascending dose study with subcutaneous ALN-6222.
Phase 1 clinical trial record published on ClinicalTrials.gov.
Evaluate efficacy, pharmacodynamics, and safety in adults with mild to moderate hypertension pretreated with zilebesiran.
Clinical study of ALN-AGT01 RVR in participants previously treated with zilebesiran.
Clinical trial record published on ClinicalTrials.gov.
Evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of ALN-2232 in people with obesity, including use with tirzepatide.
Clinical study of single ascending doses, multiple doses, and multiple doses co-initiated with tirzepatide.
Clinical trial record published on ClinicalTrials.gov.
Evaluate safety, tolerability, pharmacodynamics, and pharmacokinetics of ALN-4915 in adult healthy volunteers.
Clinical study in adult healthy volunteers assessing PD and PK effects.
Clinical trial record published on ClinicalTrials.gov.
Evaluate safety and tolerability of single ascending doses and characterize single-dose pharmacokinetics in healthy volunteers.
Single ascending dose clinical study in adult healthy volunteers.
Clinical trial record published on ClinicalTrials.gov.
Treat hATTR-PN by improving neurologic impairment, quality of life, nutritional status, disability, and gait speed, and demonstrate superior serum TTR reduction versus in-study vutrisiran.
TRITON-PN clinical study comparing nucresiran with in-study vutrisiran on clinical and serum transthyretin outcomes.
TRITON-PN clinical trial record published on ClinicalTrials.gov.
Evaluate safety, tolerability, pharmacodynamic effects, and pharmacokinetic effects in patients with Alzheimer's disease.
Clinical study of intrathecal ALN-5288 after dose administration.
Clinical trial record published on ClinicalTrials.gov.
Evaluate whether zilebesiran reduces cardiovascular death, nonfatal myocardial infarction, nonfatal stroke, or heart failure events in patients with inadequately controlled hypertension and established or high-risk cardiovascular disease.
Event-driven clinical outcomes study comparing zilebesiran versus placebo.
Clinical trial record published on ClinicalTrials.gov.
Evaluate efficacy in ATTR-CM by reducing all-cause mortality, cardiovascular events, and improving patient-reported health status and quality of life.
TRITON-CM clinical study comparing nucresiran with placebo on mortality, cardiovascular events, and quality-of-life endpoints.
TRITON-CM clinical trial record published on ClinicalTrials.gov.
Develop an RNAi prophylactic approach with a high resistance barrier against SARS-CoV-2 variants and potential future coronavirus pandemics.
Preclinical testing of two lipophilic siRNA conjugates targeting conserved SARS-CoV-2 regions, including resistance passaging studies and intranasal prophylaxis in Syrian hamsters.
Preclinical study published in Nucleic Acids Research.
The two-siRNA combination achieved at least 3-log viral reduction, prevented emergence of resistance mutations seen with single siRNA treatment, and protected hamsters from weight loss and lung pathology when administered prophylactically.
Treat ATTR-CM by inhibiting hepatic transthyretin production and reducing death and cardiovascular events while preserving functional capacity and quality of life.
Double-blind randomized trial of subcutaneous vutrisiran 25 mg versus placebo every 12 weeks for up to 36 months in 655 patients with ATTR-CM.
HELIOS-B results published in The New England Journal of Medicine.
Vutrisiran lowered the risk of death from any cause and recurrent cardiovascular events versus placebo, preserved 6-minute walk distance and KCCQ-OS score, and had a similar adverse-event incidence to placebo.
Evaluate safety, tolerability, pharmacokinetics, efficacy, and pharmacodynamics in overweight to obese healthy volunteers and overweight to obese patients with type 2 diabetes.
Clinical study of single ascending doses in healthy volunteers and multiple doses in patients with type 2 diabetes.
Clinical trial record published on ClinicalTrials.gov.
Develop medicines that selectively silence genes causing or contributing to disease by preventing specific proteins from being made.
RNA interference therapeutics using gene silencing and siRNA delivery across rare and prevalent diseases.
Company site describes an investigational pipeline spanning rare and prevalent diseases.