Our Clinical Studies
Receiving a devastating diagnosis can be overwhelming, especially when there are no approved therapies available and limited information to be found. It is important to know that you are not alone.
Hereditary Angioedema
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
Amyotrophic Lateral Sclerosis
Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Severe Hypertriglyceridemia
A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
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Therapeutic Conditions
Cardio-Renal
Neurological
Cancer
Upcoming Clinical Studies
Hypertriglyceridemia, Cardiovascular Diseases, Atherosclerosis
A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia
Healthy Participants
A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants
Hereditary Angioedema
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
Severe Hypertriglyceridemia, Hypertriglyceridemia, Atherosclerotic Cardiovascular Disease
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), and/or With Severe Hypertriglyceridemia
Hypertension
A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled Hypertension
Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
Phlebotomy Dependent Polycythemia Vera
A Study to Evaluate IONIS-TMPRSS6-LRx (ISIS 702843) in Patients With Polycythemia Vera
Hereditary Angioedema
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
Angelman Syndrome
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Severe Hypertriglyceridemia
A Study of ISIS 678354 Administered to Participants With Severe Hypertriglyceridemia
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
A Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Steatohepatitis, Nonalcoholic
A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224
Working with our Community
Through our collaborations with patient advocacy organizations and communities worldwide we work to identify and understand the most urgent needs of people living with severe diseases and their caregivers. As experts in living with their disease, the patient and caregiver perspectives inform our approach to research.
