Ionis Pharmaceuticals
Pioneer in antisense oligonucleotide therapeutics with partnered and wholly owned programs.
Therapeutic areas
Selected products and programs
Linked curated drug profiles
Selected active clinical trials
Phase 3 · Recruiting · Angelman Syndrome
Donidalorsen Treatment in Children With Hereditary AngioedemaPhase 3 · Recruiting · Hereditary Angioedema (HAE)
A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric ParticipantsPhase 3 · Recruiting · Familial Chylomicronemia Syndrome
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)Phase 1 · Recruiting · Multiple System Atrophy
A Study to Assess the Safety, Tolerability and Efficacy of ION775Phase 2 · Recruiting · Hypertriglyceridemia
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)Phase 1 · Recruiting · Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Selected patent records
inotersen sodium · Expires 2031-04-29
US 9061044inotersen sodium · Expires 2031-04-29
US 9399774inotersen sodium · Expires 2031-04-29
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