ZilganersenION373 / GFAP / RNA-Targeted Therapeutic / Neurology Alexander DiseasePhase 1/3ActiveGlobal
ION582ION582 / UBE3A-ATS / RNA-Targeted Therapeutic / Neurology Angelman SyndromeClinicalActive
ION269ION269 / APP / RNA-Targeted Therapeutic / Neurology Alzheimer's DiseaseClinicalActive
ION717ION717 / PRNP / RNA-Targeted Therapeutic / Neurology Prion DiseaseClinicalActive
ION356ION356 / PLP1 / RNA-Targeted Therapeutic / Neurology Pelizaeus-Merzbacher DiseaseClinicalActive
ION859ION859 / LRRK2 / RNA-Targeted Therapeutic / Neurology Parkinson's DiseaseClinicalActive
ION464ION464 / SNCA / RNA-Targeted Therapeutic / Neurology Multiple System AtrophyClinicalActiveParkinson's DiseaseClinicalActive
ION440ION440 / MECP2 / RNA-Targeted Therapeutic / Neurology MECP2 Duplication SyndromeClinicalActive
OlezarsenApoC-III / RNA-Targeted Therapeutic / Cardiology Severe HypertriglyceridemiaClinicalActive
Hereditary AngioedemaClinicalActive
SapablursenTMPRSS6 / RNA-Targeted Therapeutic / Specialty/Rare Polycythemia VeraClinicalActive
UlefnersenFUS / RNA-Targeted Therapeutic / Neurology Amyotrophic Lateral SclerosisClinicalActive
TofersenSOD1 / RNA-Targeted Therapeutic / Neurology Amyotrophic Lateral Sclerosis (Presymptomatic SOD1)ClinicalActive
IONIS-MAPTRxBIIB080 / TAU / RNA-Targeted Therapeutic / Neurology Alzheimer's DiseaseClinicalActive
TominersenIONIS-HTTRx / HTT / RNA-Targeted Therapeutic / Neurology Huntington's DiseasePhase 2Active
ION306ION306 / SMN2 / RNA-Targeted Therapeutic / Neurology Spinal Muscular AtrophyClinicalActive
Transthyretin-Mediated Amyloid CardiomyopathyPhase 3Active
ION904ION904 / Angiotensinogen / RNA-Targeted Therapeutic / Cardiology Treatment-Resistant HypertensionClinicalActive
BepirovirsenHepatitis B Virus / RNA-Targeted Therapeutic / Other Medicines Hepatitis B Virus InfectionPhase 3Active