| Recruiting | Donidalorsen Treatment in Children With Hereditary Angioedema Hereditary Angioedema (HAE) | Phase 3 | 2026-07-01 |
| Not Yet Recruiting | ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome Dravet Syndrome | Phase 1 / Phase 2 | 2026-04-01 |
| Recruiting | REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome Angelman Syndrome | Phase 3 | 2025-06-10 |
| Terminated | Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION Alzheimer Disease, Down Syndrome | Phase 1 | 2024-12-20 |
| Recruiting | ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally- Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | Phase 1 / Phase 2 | 2024-10-21 |
| Recruiting | Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administe Pelizaeus-Merzbacher Disease | Phase 1 | 2024-04-10 |
| Recruiting | PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717 Prion Disease | Phase 1 / Phase 2 | 2024-01-04 |
| Terminated | Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | — | 2023-10-03 |
| Active Not Recruiting | The CARDIO-TTRansform Scintigraphy Sub-study Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | — | 2023-04-04 |
| Active Not Recruiting | CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertrig Severe Hypertriglyceridemia | Phase 3 | 2022-12-13 |
| Enrolling By Invitation | An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Car Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | Phase 3 | 2022-11-30 |
| Completed | A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascula Hypertriglyceridemia, Cardiovascular Diseases, Atherosclerosis | Phase 3 | 2022-11-21 |
| Completed | A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels Healthy Participants | Phase 1 | 2022-10-05 |
| Recruiting | Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzba Pelizaeus-Merzbacher Disease | — | 2022-10-03 |
| Completed | A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia Severe Hypertriglyceridemia | Phase 3 | 2022-08-31 |
| Recruiting | Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multipl Multiple System Atrophy | Phase 1 | 2022-07-21 |
| Active Not Recruiting | A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditar Hereditary Angioedema | Phase 3 | 2022-07-13 |
| Completed | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atheroscler Hypertriglyceridemia, Atherosclerotic Cardiovascular Disease, Severe Hypertriglyceridemia | Phase 2 | 2022-05-09 |
| Completed | A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in P Hypertension | Phase 2 | 2022-04-29 |
| Active Not Recruiting | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemi Familial Chylomicronemia Syndrome | Phase 3 | 2022-02-25 |
| Active Not Recruiting | A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LR Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Phase 3 | 2022-01-04 |
| Active Not Recruiting | A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera Phlebotomy Dependent Polycythemia Vera | Phase 2 | 2021-12-30 |
| Recruiting | HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION5 Angelman Syndrome | Phase 1 / Phase 2 | 2021-12-22 |
| Completed | OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Parti Hereditary Angioedema | Phase 3 | 2021-12-03 |
| Active Not Recruiting | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndr Familial Chylomicronemia Syndrome | Phase 3 | 2021-11-18 |
| Completed | A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia Severe Hypertriglyceridemia | Phase 3 | 2021-10-25 |
| Completed | A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224 Steatohepatitis, Nonalcoholic | Phase 2 | 2021-06-17 |
| Active Not Recruiting | FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotroph Amyotrophic Lateral Sclerosis | Phase 3 | 2021-06-14 |
| Completed | A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Fa Chronic Heart Failure With Reduced Ejection Fraction | Phase 2 | 2021-06-08 |
| Active Not Recruiting | A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) Alexander Disease | Phase 3 | 2021-06-01 |
| Completed | A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Unc Hypertension | Phase 2 | 2021-04-28 |
| Active Not Recruiting | CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) | — | 2021-03-31 |
| Completed | A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses Healthy Volunteers | Phase 1 | 2021-03-24 |
| Completed | A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904 Healthy Volunteers | Phase 1 | 2021-02-26 |
| Completed | A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma Relapsed Multiple Myeloma, Refractory Multiple Myeloma | Phase 1 | 2021-01-20 |
| Completed | A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors Advanced Solid Tumors | Phase 1 | 2021-01-05 |
| Completed | A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acrome Acromegaly | Phase 2 | 2021-01-04 |
| Terminated | A Study to Assess the Safety, Tolerability, and Efficacy of ION-827359 in Participants With Mild to Moderate C Chronic Bronchitis, Chronic Obstructive Pulmonary Disease | Phase 2 | 2020-12-22 |
| Completed | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicrone Familial Chylomicronemia Syndrome | Phase 3 | 2020-11-18 |
| Terminated | Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (F Beta Thalassemia Intermedia | Phase 2 | 2020-09-24 |
| Completed | A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Mul Healthy Participants | Phase 1 | 2020-04-23 |
| Completed | An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema Hereditary Angioedema | Phase 2 | 2020-04-01 |
| Active Not Recruiting | CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, I Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | Phase 3 | 2020-03-13 |
| Completed | A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Parti Hereditary Angioedema | Phase 2 | 2020-01-07 |
| Completed | NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IO Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | Phase 3 | 2019-12-11 |
| Completed | A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor Primary IgA Nephropathy | Phase 2 | 2019-12-04 |
| Completed | A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx Hypertension | Phase 2 | 2019-11-13 |
| Completed | A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disea Parkinson's Disease | Phase 1 | 2019-08-12 |
| Completed | Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting S Acromegaly | Phase 2 | 2019-07-25 |
| Completed | GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geo Macular Degeneration, Geographic Atrophy | Phase 2 | 2019-03-04 |
| Completed | Natural History and Functional Status Study of Patients With Lafora Disease Lafora Disease | — | 2019-01-09 |
| Completed | A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx, an Antisense Inhibitor Administered Mild Hypertension | Phase 2 | 2019-01-03 |
| Completed | Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single an Healthy Volunteers, hATTR Amyloidosis | Phase 1 / Phase 2 | 2018-12-21 |
| Completed | A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Mul Healthy Subjects, Cystic Fibrosis | Phase 1 | 2018-12-13 |
| Completed | A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Participants | Phase 1 | 2018-10-15 |
| Completed | Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-ac Acromegaly | Phase 2 | 2018-09-13 |
| Completed | A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Dose Healthy Participants | Phase 1 | 2018-07-09 |
| Withdrawn | Safety and Efficacy of IONIS-FB-Lrx in up to 120 Patients 55 and Older With Geographic Atrophy (GA) Secondary Geographic Atrophy, Age Related Macular Degeneration | Phase 2 | 2018-03-16 |
| Completed | A Study of ISIS 416858 Administered Subcutaneously to Participants With End-Stage Renal Disease (ESRD) on Hemo End-stage Renal Disease (ESRD) | Phase 2 | 2017-12-26 |
| Completed | Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 Diabetes Hepatic Steatosis | Phase 2 | 2017-11-03 |
| Completed | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Mild Alzheimer's Disease | Phase 1 | 2017-10-12 |
| Completed | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subc Healthy Volunteers | Phase 1 | 2017-08-28 |
| Completed | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Hea Thalassemia | Phase 1 | 2017-05-09 |
| Completed | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy Volunteers Healthy Volunteers | Phase 1 | 2017-04-05 |
| Completed | A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers Abnormalities, Cardiovascular | Phase 1 | 2016-09-19 |
| Completed | Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated Tr Elevated Triglycerides (TG) | Phase 1 | 2016-09-01 |
| Completed | Study to Evaluate the Effect of ISIS GCGRRx on Hepatic Lipid and Glycogen Content in Type 2 Diabetes Type 2 Diabetes | Phase 2 | 2016-04-01 |
| Completed | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With E Hypertriglyceridemia, Familial Hypercholesterolemia | Phase 1 | 2015-11-30 |
| Completed | A Study of Safety, PK, & PD of ISIS 416858 Administered Subcutaneously to Patients With End-Stage Renal Diseas End-stage Renal Disease (ESRD) | Phase 2 | 2015-10-01 |
| Completed | Safety, Tolerability and Efficacy of ISIS-GCGRRx in Patients With Type 2 Diabetes Type 2 Diabetes | Phase 2 | 2015-09-01 |
| Completed | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifes Huntington's Disease | Phase 1 / Phase 2 | 2015-08-06 |
| Completed | A Safety, Tolerability, PK, and PD Study of Once Weekly ISIS-FGFR4RX SC in Obese Patients Obesity | Phase 2 | 2015-06-01 |
| Completed | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With El Elevated Lipoprotein(a) | Phase 1 | 2015-04-01 |
| Completed | The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia Hypertriglyceridemia | Phase 3 | 2015-02-05 |
| Completed | The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronem Familial Chylomicronemia Syndrome | Phase 3 | 2014-12-01 |
| Completed | A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 Myotonic Dystrophy Type 1 | Phase 1 / Phase 2 | 2014-12-01 |
| Completed | Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FA FAP, Familial Amyloid Polyneuropathy, TTR | Phase 3 | 2014-06-26 |
| Completed | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipopr Elevated Lipoprotein(a) | Phase 2 | 2014-06-01 |
| Completed | Safety, Tolerability and Efficacy of ISIS-GCCRRx in Type 2 Diabetes Type 2 Diabetes Mellitus | Phase 2 | 2013-10-01 |
| Completed | Safety, Tolerability, and Efficacy of ISIS-PTP1BRx in Type 2 Diabetes Type 2 Diabetes Mellitus, Obese | Phase 2 | 2013-08-01 |
| Completed | Safety, Tolerability and Efficacy of ISIS-GCGRRx in Type 2 Diabetes Type 2 Diabetes Mellitus | Phase 2 | 2013-07-01 |
| Completed | Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy FAP, Familial Amyloid Polyneuropathy, TTR | Phase 2 / Phase 3 | 2013-03-15 |
| Completed | Active Comparator-Controlled Study to Assess Safety and Efficacy of ISIS-FXIRx in Total Knee Arthroplasty Venous Thromboembolism | Phase 2 | 2012-10-01 |
| Completed | Antiarrhythmic and Symptomatic Effect of ISIS CRP Rx Targeting CRP in Paroxysmal Atrial Fibrillation Paroxysmal Atrial Fibrillation | Phase 2 | 2012-10-01 |
| Terminated | Mechanistic Study of ISIS ISIS-APOCIIIRX in Type 2 Diabetes Type 2 Diabetes | Phase 2 | 2012-07-01 |
| Completed | Phase 1/2, Open-label, Dose-escalation Study of IONIS-STAT3Rx, Administered to Patients With Advanced Cancers Advanced Cancers, DLBCL, Lymphoma | Phase 1 / Phase 2 | 2012-02-27 |
| Completed | Safety, Tolerability, and Pharmacokinetic Study of ISIS ApoC-III Rx in Hypertriglyceridemia Hypertriglyceridemia | Phase 2 | 2012-02-01 |
| Completed | Safety, Tolerability, and Pharmacodynamic Study of ISIS CRP Rx in Rheumatoid Arthritis Rheumatoid Arthritis | Phase 2 | 2011-08-01 |
| Completed | Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Carboplatin and Paclitaxel Non-small Cell Lung Cancer | Phase 1 / Phase 2 | 2010-11-01 |
| Completed | Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Docetaxel and Prednisone (CRPC) Castrate-Resistant Prostate Cancer | Phase 1 / Phase 2 | 2010-11-01 |
| Completed | Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Familial Amyotrophic Lateral Sclerosis | Phase 1 | 2010-01-01 |
| Completed | Safety, Tolerability and Activity Study of Multiple Doses of ISIS-SGLT2Rx in Healthy Volunteers Type 2 Diabetes Mellitus | Phase 1 | 2009-01-01 |
| Completed | Safety and Tolerability of Single and Multiple Doses of ISIS 353512 in Healthy Volunteers Inflammatory Diseases | Phase 1 | 2008-07-01 |
| Completed | Safety Study of ISIS 325568 in Healthy Volunteers Type 2 Diabetes Mellitus | Phase 1 | 2007-08-01 |
| Completed | Placebo Controlled, Dose Escalation Study in Subjects With Type 2 Diabetes Mellitus Being Treated With Sulfony Type 2 Diabetes Mellitus | Phase 2 | 2007-02-01 |
| Completed | Examination of Dermatologic Effects From Subcutaneous Injections of ISIS 113715 Type 2 Diabetes Mellitus | Phase 1 | 2006-08-01 |
| Withdrawn | Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Being Treated With S Type 2 Diabetes Mellitus | Phase 2 | 2006-05-01 |
| Terminated | Effects of ISIS 113715 on Insulin Sensitivity, Glucose, and Lipid Metabolism and Energy Expenditure in Type 2 Type 2 Diabetes Mellitus | Phase 2 | 2005-11-01 |
| Completed | Addition of ISIS 14803 to Therapy With Peginterferon and Ribavirin for Chronic Hepatitis C (HCV) Patients Not Hepatitis C, Chronic | Phase 1 / Phase 2 | 2003-06-01 |
| Completed | ISIS 2302-CS27, A 6-Week, Placebo-Controlled Clinical Study to Evaluate the Effectiveness of Alicaforsen (ISIS Ulcerative Colitis | Phase 2 | 2003-04-03 |
| Completed | Safety, Tolerability, and Activity of ISIS 113715 in People With Type 2 Diabetes Mellitus Who Have Not Receive Type 2 Diabetes Mellitus | Phase 2 | 2003-02-01 |
| Completed | ISIS 2302-CS22, A 6-Week, Active-Controlled Clinical Study to Evaluate the Effectiveness of Alicaforsen (ISIS Ulcerative Colitis | Phase 2 | 2002-11-20 |
| Completed | Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease Crohn's Disease | Phase 3 | 2002-05-01 |
| Completed | ISIS 104838, an Inhibitor of Tumor Necrosis Factor, for Active Rheumatoid Arthritis Rheumatoid Arthritis | Phase 2 | 2002-01-01 |
| Completed | ISIS 14803-CS2, Treatment With ISIS 14803, Administered IV in Patients With Chronic Hepatitis C Virus Infectio Hepatitis C, Chronic | Phase 2 | 2001-10-01 |
| Completed | Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease Crohn's Disease | Phase 3 | 2001-01-10 |
| Completed | Carboplatin and Paclitaxel With or Without ISIS 3521 in Treating Patients With Non-Small Cell Lung Cancer Lung Cancer | Phase 3 | 2000-10-13 |
| Approved For Marketing | ATTR Expanded Access Program (EAP) by Ionis Amyloidosis, Hereditary | — | — |
| Completed | A Comparison of ISIS 2922 Used Immediately or Later in Patients With Cytomegalovirus (CMV) of the Eyes Cytomegalovirus Retinitis, HIV Infections | N/A | — |
| Completed | A Study of ISIS 2922 in the Treatment of Advanced Cytomegalovirus Retinitis Cytomegalovirus Retinitis, HIV Infections | N/A | — |
| Approved For Marketing | Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS) Familial Chylomicronemia Syndrome | — | — |
| Available | Zilganersen Expanded Access Program for Individuals With Alexander Disease Alexander Disease | — | — |
| Available | Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema Hereditary Angioedema | — | — |
| Completed | The Safety and Effectiveness of ISIS 2922 in Patients With AIDS Who Have Cytomegalovirus (CMV) of the Eyes Cytomegalovirus Retinitis, HIV Infections | Phase 2 | — |
| Completed | A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patient Cytomegalovirus Retinitis, HIV Infections | Phase 2 | — |