Ionis Pharmaceuticals, Inc.

This study tests a drug called sapablursen in about 50 adults with polycythemia vera, a rare blood cancer that causes too many red blood cells. Participants currently need regular blood removal (phlebotomy) to manage their condition. The goal is to see if the drug can reduce how …

This study looks at whether the drug eplontersen can reduce abnormal protein buildup in the hearts of people with a rare condition called ATTR-CM. About 150 participants from a larger study will get heart scans after 140 weeks of treatment. The goal is to see if the drug clears t…

This study tests an experimental drug called zilganersen (ION373) in 54 people with Alexander disease, a rare brain disorder. The goal is to see if the drug can improve or stabilize walking and other motor functions. Participants receive the drug or a placebo via spinal injection…

This study tests a drug called olezarsen in people with familial chylomicronemia syndrome (FCS), a rare genetic condition that causes extremely high blood fat levels. The goal is to see if the drug can safely lower triglycerides over the long term. About 60 participants who compl…

This study looks at the long-term safety of a drug called eplontersen for people with a rare, inherited nerve disease (hATTR-PN). About 151 participants who completed earlier studies will receive the drug and be monitored for side effects and changes in nerve function. The goal i…

This study looks at the long-term safety of a drug called olezarsen for people with severe hypertriglyceridemia (very high blood fats). About 885 adults who completed a prior olezarsen study will receive the drug for up to 3 years while staying on their usual cholesterol-lowering…

This study looks at the long-term safety of a drug called eplontersen in people with ATTR-CM, a condition where abnormal proteins build up in the heart and cause it to weaken. About 1,400 adults who have already completed a related study or have a confirmed diagnosis will receive…

This study tests the safety of a new drug called olezarsen in 24 people with familial chylomicronemia syndrome (FCS), a rare condition that causes extremely high blood fats. Participants have already been treated with a similar drug, volanesorsen. The study monitors for side effe…

This study tests a medicine called donidalorsen for people with hereditary angioedema (HAE), a condition that causes sudden, painful swelling. The goal is to see if the drug is safe and effective over the long term at preventing these attacks and improving quality of life. About …

This study tests an experimental drug called ION363 in people with a rare, inherited form of ALS caused by FUS gene mutations. The goal is to see if the drug can slow the disease and help people live longer. About 89 participants will receive the drug via spinal injection, and re…

This study uses MRI scans to measure amyloid protein buildup in the hearts of up to 150 people with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Participants are already enrolled in a larger treatment study. The goal is to see how the amyloid burden changes over time,…