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Clinical trials and research at Ionis Pharmaceuticals, Inc.

• 9Clinical trials
• 14Countries

Newest trials

All recruiting trials (9)

• Obudanersen for Angelman Syndrome in UPD/ID patients

  This study tests obudanersen in children and adults with Angelman syndrome caused by UPD or imprinting defects to see if it improves expressive communication.

  Recruiting Phase 3 Ionis Pharmaceuticals, Inc. · Carrboro, North Carolina · NCT07782827

• Olezarsen for Familial Chylomicronemia Syndrome in kids

  This study tests whether olezarsen given by injection can help children with Familial Chylomicronemia Syndrome.

  Recruiting at 6 sites Phase 3 Ionis Pharmaceuticals, Inc. · Birmingham, Alabama and 5 other locations · NCT07727538

• Ion775 for high triglyceride levels: safety, tolerability, and how well it works

  A drug called ION775 is being tested to see if it can safely lower fasting triglycerides in adults with hypertriglyceridemia or severe hypertriglyceridemia who are already on standard lipid medicines.

  Recruiting at 17 sites Phase 2 Ionis Pharmaceuticals, Inc. · Lincoln, California and 17 other locations · NCT07604974

• ION337 safety and tolerability in children with Dravet syndrome

  This will test whether ION337 is safe and well tolerated in children ages 2 to 12 with Dravet syndrome.

  Recruiting at 7 sites Phase 1/2 Ionis Pharmaceuticals, Inc. · Ann Arbor, Michigan and 6 other locations · NCT07531745

• Donidalorsen for children with hereditary angioedema

  This trial tests if donidalorsen is safe and how it is processed in the body for children aged 2 to under 12 with hereditary angioedema types I or II.

  Recruiting at 18 sites Phase 3 Ionis Pharmaceuticals, Inc. · Santa Monica, California and 17 other locations · NCT07298447

• Evaluating ION440 for treating MECP2 Duplication Syndrome

  This study is testing a new treatment called ION440 to see if it can help children and adults with MECP2 Duplication Syndrome feel better and improve their condition.

  Recruiting at 12 sites Phase 1/2 Ionis Pharmaceuticals, Inc. · San Diego, California and 11 other locations · NCT06430385

• Evaluating a new treatment for Pelizaeus-Merzbacher Disease in children

  This study is testing a new drug called ION356 to see if it is safe and tolerable for children with Pelizaeus-Merzbacher Disease who have a specific genetic change.

  Recruiting at 7 sites Phase 1 Ionis Pharmaceuticals, Inc. · Atlanta, Georgia and 6 other locations · NCT06150716

• Characterizing biomarkers and disease progression in Pelizaeus-Merzbacher Disease

  This study is trying to see how certain markers in the body change over time in people with Pelizaeus-Merzbacher Disease to help develop better treatments in the future.

  Recruiting at 7 sites Ionis Pharmaceuticals, Inc. · Atlanta, Georgia and 9 other locations · NCT05659901

• Evaluating ION464 for Multiple System Atrophy

  This study is testing a new drug called ION464 to see if it is safe and helps adults with multiple system atrophy feel better over a 12-week period and beyond.

  Recruiting at 14 sites Phase 1 Ionis Pharmaceuticals, Inc. · Innsbruck and 14 other locations · NCT04165486

Quick answers

How many clinical trials run by Ionis Pharmaceuticals, Inc. are recruiting right now?

9, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 14 countries. The most sites are in United States (8 trials), France (4 trials), Germany (3 trials), Canada (3 trials), Italy (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 3, Phase 1/2: 2, Phase 1: 2, Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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