Ionis
Ionis Pharmaceuticals, based in Carlsbad, California, develops antisense oligonucleotide (ASO) drugs — short synthetic strands of DNA-like molecules that bind to a target RNA and prevent the cell from producing a disease-driving protein. The portfolio includes SPINRAZA for SMA (partnered with Biogen), WAYLIVRA for familial chylomicronemia, and TRYNGOLZA for severe hypertriglyceridemia, plus late-stage programs across cardiovascular, neurological, and rare diseases.
- Lead asset
- olezarsen · Filed · Familial Chylomicronemia
- oligonucleotide · ApoCIII antisense oligonucleotide
- Pipeline
- 1 drug · 1 program
- 1 Cardio-Renal
- Modalities
- oligonucleotide×1
Peers in this space
Pipeline & catalysts
- Preclin0
- Ph10
- Ph20
- Ph30
- Filed1
- Approved0
Past catalyst impact
Insider activity
Congressional trading
Top ETF holders
KOL network
Federal lobbying
- FDA user fee reauthorization (PDUFA / BsUFA)$595K
- NIH appropriations · NCI funding$104K
- Prescription Drug User Fee programs$104K
- Drug Supply Chain Security Act$90K
- BIOSIM Act / Biosimilars$90K