Target the Gene, Silence the Disease

RNAi technology makes it possible to target the genes that cause disease.

Treating diseases with significant unmet needs demands a unique approach at a fast pace, that’s exactly where we thrive. Our targeted RNAi molecule, TRiM™, platform leverages a natural pathway to “silence” genes causing disease. With drug candidates in a growing number of disease areas, TRiM™ has shown it can target genes wherever they are.

What We Do Best

Acceleration

Every day we can save in our development timeline is one day earlier a person may benefit from our future therapies.

Collaboration

We go farther when we work together to reimagine how therapeutics are developed and delivered.

Growth

We don’t let uncertainty keep us from tackling big questions and finding meaningful solutions.

Above & Beyond

Our goal is to have 20 individual products, either partnered or wholly owned, in clinical trials or on the market by 2025. See how we’re already on the way to achieving our ambitious vision.

Discover Our Science

Our Pipeline^*

There is no shortage of needs that we can endeavor to serve. As scientific explorers, we use our discoveries to develop solutions for the people who need them most.

Plozasiran

Severe HypertriglyceridemiaLiverPhase 3

Plozasiran (formerly ARO-APOC3) is designed to reduce the production of the protein Apolipoprotein-CIII (ApoC3) through the natural RNA interference (RNAi) mechanism.  ApoC3 is a protein that is produced in liver cells and inhibits the formation and clearance of various lipids and lipoproteins, including triglycerides.

Clinical Trials

ActiveID: NCT06347003

This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.

ActiveID: NCT06347016

This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After Month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.

CompleteID: NCT03783377

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).

CompleteID: NCT04720534

The purpose of AROAPOC3-2001 is to evaluate the efficacy and safety of ARO-APOC3 in participants with severe hypertriglyceridemia. Participants will receive 2 subcutaneous injections of ARO-APOC3.

Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults with Severe Hypertriglyceridemia (SHASTA-3)

Severe Hypertriglyceridemia Active ID: NCT06347003

View on ClinicalTrials.gov

Inclusion Criteria

Established diagnosis of severe hypertriglyceridemia (SHTG) and prior documented evidence (medical history) of fasting TG levels of ≥ 500 mg/dL (≥5.65mmol/L)
Mean fasting TG level ≥500 mg/dL (≥5.65 mmol/L) collected at 2 separate and consecutive visits at least 7 days apart and no more than 17 days apart during the screening period
Fasting low density lipoprotein-cholesterol (LDL-C) ≤130 mg/dL (≤3.37 mmol/L) at screening
Screening HbA1C ≤8.5%
Willing to follow diet counseling and maintain a stable low-fat diet
Must be on standard of care lipid-lowering medications per local guidelines (unless documented as intolerant as determined by the Investigator)

Timeline

April 4, 2024

Study First Posted
May 2024

Study Start Date
October 2026

Estimated Completion Date

Trial Details

Start date:

May 2024

End date:

October 2026

Participants:

405

Eligibility criteria:

18 Years or Older, All Sexes, No Healthy Volunteers

Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults with Severe Hypertriglyceridemia (SHASTA-4)

Severe Hypertriglyceridemia Active ID: NCT06347016

This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After Month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.

View on ClinicalTrials.gov

Inclusion Criteria

Established diagnosis of severe hypertriglyceridemia (SHTG) and prior documented evidence (medical history) of fasting TG levels of ≥500 mg/dL (≥5.65 mmol/L)
Mean fasting TG level ≥500 mg/dL (≥5.65 mmol/L) collected at 2 separate and consecutive visits at least 7 days apart and no more than 17 days apart during the screening period
Fasting low density lipoprotein-cholesterol (LDL-C) ≤130 mg/dL (≤3.37 mmol/L) at screening
Screening HbA1C ≤8.5%
Must be on standard of care lipid-lowering medications per local guidelines (unless documented as intolerant as determined by the Investigator)

Timeline

April 4, 2024

Study First Posted
May 2024

Study Start Date
October 2026

Estimated Completion Date

Trial Details

Start date:

May 2024

End date:

October 2026

Participants:

300

Eligibility criteria:

18 Years or Older, All Sexes, No Healthy Volunteers

A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-APOC3 in Adult Healthy Volunteers as Well as in Severely Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome

Familial Chylomicronemia SyndromeSevere Hypertriglyceridemia Complete ID: NCT03783377

View on ClinicalTrials.gov

Inclusion Criteria

Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
Willing to provide written informed consent and to comply with study requirements
Normal electrocardiogram (ECG) at screening
Hypertriglyceridemic patients must have a history of fasting serum triglycerides of at least 300 mg/dL (3.38 mmol/L) at screening or verifiable diagnosis of FCS

Timeline

December 2018

Study First Posted
March 2019

Study Start Date
February 2021

Actual Study Completion Date

Trial Details

Start date:

March 2019

End date:

February 2021

Locations:

Australia, Canada, New Zealand

Participants:

112

Eligibility criteria:

18 Years to 70 Years (Adult, Older Adult), All Sexes, Accepts Healthy Volunteers

A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Severe Hypertriglyceridemia (SHASTA-2)

Severe Hypertriglyceridemia Complete ID: NCT04720534

The purpose of AROAPOC3-2001 is to evaluate the efficacy and safety of ARO-APOC3 in participants with severe hypertriglyceridemia. Participants will receive 2 subcutaneous injections of ARO-APOC3.

View on ClinicalTrials.gov

Inclusion Criteria

Based on medical history, evidence of TG ≥ 500 mg/dL and ≤ 4000 mg/dL at Screening
Fasting TG ≥ 500 mg/dL at Screening
Willing to follow diet counseling per Investigator judgment based on local standard of care
Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
Willing to provide written informed consent and to comply with study requirements

Timeline

January 2021

Study First Posted
May 2021

Study Start Date
March 2023

Actual study Completion Date

Trial Details

Start date:

May 2021

End date:

March 2023

Locations:

Australia, Canada, Germany, Hungary, Netherlands, New Zealand, Poland, United States

Participants:

229

Eligibility criteria:

18 Years and older (Adult, Older Adult), All Sexes, No Healthy Volunteers

Zodasiran

Homozygous Familial HypercholesterolemiaLiverPhase 3

Zodasiran, formally ARO-ANG3, is designed to reduce production of angiopoietin-like protein 3 (ANGPTL3), a liver synthesized inhibitor of lipoprotein lipase and endothelial lipase.

ANGPTL3 inhibition has been shown to lower serum LDL, serum and liver triglyceride and has genetic validation as a novel target for cardiovascular disease.

Clinical Trials

RecruitingID: NCT07037771

This multicenter, randomized, placebo-controlled study will evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous familial hypercholesterolemia (HoFH). After completion of the double blind (DB) treatment period subjects will be eligible to continue in the optional open-label extension (OLE) period of the study. All placebo subjects who opt to continue will transition to active drug during the OLE Period.

ActiveID: NCT05217667

Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks. Participants who complete the first 36 week treatment period may opt to continue in an additional 24-month extension period during which they will receive up to 8 doses open-label doses of ARO-ANG3.

Phase 3 Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

Homozygous Familial Hypercholesterolemia Recruiting ID: NCT07037771

View on ClinicalTrials.gov

Inclusion Criteria

Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study
Body weight ≥35 kg at Screening as patients could theoretically be <35 kg as the study continues.
HoFH based on a supportive genetic test or a clinical diagnosis (total cholesterol >500 mg/dL OR treated LDL-C concentration of ≥ AND both parents with documented total cholesterol >250 mg/dL OR cutaneous or tendinous xanthoma before 10 years of age)
LDL-C ≥70 mg/dL (1.8 mmol/L)
Hemoglobin A1c (HbA1c) ≤9.5%
Total bilirubin <2xULN, unless in previously confirmed cases of Gilbert's syndrome
Alanine aminotransferase or aspartate aminotransferase <3×ULN
On standard of care, maximally tolerated lipid-lowering therapy

Timeline

June 25, 2025

Study First Posted
June 17, 2025

Study Start Date
August 20, 2027

Estimated Completion Date

Trial Details

Start date:

June 17, 2025

End date:

August 20, 2027

Locations:

United States

Participants:

Eligibility criteria:

12 Years or Older, All Sexes, No Healthy Volunteers

Phase 2 Study to Evaluate the Safety and Efficacy of ARO-ANG3 in Subjects With Homozygous Familial Hypercholesterolemia (HOFH)(Gateway)

Homozygous Familial Hypercholesterolemia Active ID: NCT05217667

View on ClinicalTrials.gov

Inclusion Criteria

Fasting LDL-C >100 mg/dL at Screening
Weight of ≥ 40 kg and body mass index ≥ 18.5 and ≤ 40 kg/m2
Diagnosis of HoFH based on a supportive genetic test or clinical diagnosis
On stable maximally tolerated lipid lowering therapy
Willing to abide by stable low-fat, low-cholesterol, heart-healthy diet for at least 4 weeks prior to Day 1
Participants of childbearing potential (males & females) must agree to use highly-effective contraception during the study and for at least 24 weeks from the last dose of study medication.
Women of childbearing potential must have a negative pregnancy test and cannot be breastfeeding
Women of childbearing potential on hormonal contraceptives must be stable on the medications for > 2 menstrual cycles prior to Day 1
Willing to provide written informed consent and to comply with study requirements

Timeline

February 2022

Study First Posted
April 2022

Study Start Date
July 2023

Estimated Study Completion Date

Trial Details

Start date:

April 2022

End date:

July 2023

Locations:

Australia, Canada, South Africa, United States

Participants:

Eligibility criteria:

16 Years and older (Child, Adult, Older Adult), All Sexes, No Healthy Volunteers

ARO-RAGE

Inflammatory Pulmonary DiseasesLungPhase 2

ARO-RAGE is designed to reduce production of the Receptor for Advanced Glycation End products (RAGE) as a potential treatment for various muco-obstructive and inflammatory pulmonary diseases.

Clinical Trials

ActiveID: NCT07241546

The purpose of this study is to evaluate the impact of inhaled ARO-RAGE on the late asthmatic response (LAR) following an inhaled allergen challenge in participants with mild atopic asthma.

CompleteID: NCT05276570

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of ARO-RAGE in normal healthy volunteers (NHVs) and in participants with inflammatory lung disease. In Part 1 of the study, NHVs will receive a single dose of ARO-RAGE or placebo. In Part 2 of the study, adult participants with inflammatory lung disease will receive 2 doses of ARO-RAGE or placebo. Additional NHVs may be randomized to receive 1 or 2 doses of ARO-RAGE or placebo at Sponsor discretion. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.

CompleteID: NCT05533294

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-RAGE Injection in normal healthy volunteers.

Study of Inhaled ARO-RAGE in Allergen-induced Mild Asthma

Inflammatory Pulmonary Diseases Active ID: NCT07241546

The purpose of this study is to evaluate the impact of inhaled ARO-RAGE on the late asthmatic response (LAR) following an inhaled allergen challenge in participants with mild atopic asthma.

View on ClinicalTrials.gov

Inclusion Criteria

Clinically stable, mild atopic asthma (FEV1 ≥70% predicted)
Established allergy confirmed by positive skin prick test at screening
Willing and able to perform lung function tests and other study-related procedures
Participants of childbearing potential must consent to use a method of highly-effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last investigational product administration, whichever is later

Timeline

November 21, 2025

Study First Posted
December 2025

Study Start Date
April 2028

Estimated Completion Date

Trial Details

Start date:

December 2025

End date:

April 2028

Participants:

Eligibility criteria:

18 to 65 Years Old, All Sexes, No Healthy Volunteers

A Phase 1/2a Study Evaluating the Effects of ARO-RAGE in Healthy Subjects and Patients With Inflammatory Lung Disease

Inflammatory Pulmonary Diseases Complete ID: NCT05276570

View on ClinicalTrials.gov

Inclusion Criteria

Normal pulmonary function tests at Screening (NHVs only)
Confirmed diagnosis of asthma based on source verifiable medical record (asthma patients only)
No abnormal finding of clinical relevance at Screening (other than asthma for asthma patients)
Stable dose of asthma controller medications for at least 4 weeks prior to Screening (asthma patients only)
Non-smoking
Able to produce an induced sputum sample at Screening
Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception. Males must not donate sperm during the study and for at least 12 weeks following the last dose of study drug
Willing to provide written informed consent and to comply with study requirements

Timeline

March 2022

Study First Posted
June 2022

Study Start Date
February 2024

Estimated Study Completion Date

Trial Details

Start date:

June 2022

End date:

February 2024

Locations:

Australia, New Zealand

Participants:

121

Eligibility criteria:

18 Years to 65 Years, All Sexes, Accepts Healthy Volunteers

A Phase 1 Study Evaluating the Effects of ARO-RAGE Injection for Subcutaneous Administration in Healthy Subjects

AsthmaInflammatory Pulmonary Diseases Complete ID: NCT05533294

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-RAGE Injection in normal healthy volunteers.

View on ClinicalTrials.gov

Inclusion Criteria

Normal pulmonary function tests at Screening prior to sputum induction
Normal 12-lead electrocardiogram (ECG) at Screening
Non-smoking
Able to produce an induced sputum sample at Screening
Participants of child-bearing potential (male and female) must use highly effective contraception and cannot donate sperm or eggs during the study or for at least 12 weeks following the end of the study or last dose of study drug, whichever is later. Women must have a negative pregnancy test and cannot be breastfeeding
Willing to provide written informed consent and to comply with study requirements