TRYNGOLZA
olezarsen Nucleic Acid Target: apoC-III, APOC3
- Familial Chylomicronemia Syndrome
- Severe Hypertriglyceridemia
Ionis Pharmaceuticals
Most recent annual filing: 2026-02-26
- Sefaxersen Ph3 topline2026Sefaxersen Ph3 topline, 2026, material
- Ulefnersen Ph3 topline2026Ulefnersen Ph3 topline, 2026, material
- Eplontersen ATTR-CM filing2026Eplontersen ATTR-CM filing, 2026, notable
- Pelacarsen filing2026Pelacarsen filing, 2026, notable
- Ionis corporate updateOct–Nov 2026Ionis corporate update, Oct–Nov 2026, material
- Bepirovirsen PDUFA dateOct 26, 2026Bepirovirsen PDUFA date, Oct 26, 2026, material
- Obudanersen Ph3 topline2027Obudanersen Ph3 topline, 2027, thesis-defining
- Olezarsen TRYNGOLZA filing2027Olezarsen TRYNGOLZA filing, 2027, material
Bar width shows how precise the guidance is — a pin is a stated date, a wide band a six-month window. Deeper blue means more of the thesis rides on the event; it is not a view on the stock.
Pipeline (29 active programs)
DAWNZERA
donidalorsen Nucleic Acid Target: PKK, prekallikrein
WAINUA
eplontersen Nucleic Acid Target: TTR, transthyretin
- Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
- Transthyretin-Mediated Amyloid Cardiomyopathy
- Transthyretin Amyloid Cardiomyopathy
- Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
- Transthyretin Amyloidosis Cardiomyopathy
- Transthyretin Amyloidosis
SPINRAZA
nusinersen Nucleic Acid Target: SMN2
QALSODY
tofersen Nucleic Acid Target: SOD1
- Amyotrophic Lateral Sclerosis
- Presymptomatic SOD1 Mutation
- Presymptomatic SOD1 Amyotrophic Lateral Sclerosis
TEGSEDI
inotersen Nucleic Acid Target: TTR, transthyretin
- Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
- Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
WAYLIVRA
volanesorsen Nucleic Acid Target: apoC-III, APOC3
zilganersen
ION373 Nucleic Acid Target: GFAP
- Alexander Disease
obudanersen
ION582 Nucleic Acid Target: UBE3A-ATS, UBE3A
bepirovirsen
GSK3228836 Nucleic Acid Target: HBV
pelacarsen
TQJ230 Nucleic Acid Target: Apo(a), Lp(a), lipoprotein(a)
- Cardiovascular Disease
sefaxersen
IONIS-FB-LRx · RO7434656 Nucleic Acid Target: complement factor B
ulefnersen
Nucleic Acid Target: FUS
sapablursen
Nucleic Acid Target: TMPRSS6
salanersen
ION306 Nucleic Acid Target: SMN2
ION826
AZD4063 Nucleic Acid Target: transferrin receptor 1
- Undisclosed indication
opemalirsen
tominersen
Nucleic Acid Target: HTT
TEGSEDI
Technology platforms
MsPA backbone chemistry
Mesyl phosphoramidate backbone chemistry designed to improve therapeutic index and durability by increasing metabolic stability, improving potency and reducing non-specific protein interactions; multiple new programs in preclinical development
Bicycle technology · Bicycle Therapeutics
Bicyclic peptide technology licensing collaboration with Bicycle Therapeutics to expand LICA platform for delivery of oligonucleotides to skeletal and cardiac muscle and potentially across the blood-brain barrier via transferrin receptor 1 binding
Targets: transferrin receptor 1
Vect-Horus technology · Vect-Horus
Camelid-derived antibody platform licensed exclusively for systemic delivery of RNA-targeted therapeutics across the blood-brain barrier to CNS targets; first program advanced to preclinical development in 2025
Metagenomi gene editing · Metagenomi
Collaboration leveraging RNA-targeted therapeutics expertise combined with Metagenomi next-generation CRISPR-Cas gene editing systems to discover and develop drugs for validated and novel genetic targets; up to eight targets total
Vect-Horus · Vect-Horus
Systemic delivery platform for RNA-targeted therapeutics using camelid-derived single-domain antibodies to cross the blood-brain barrier and address CNS targets; focus area: central nervous system diseases
Discontinued (1)
- sefaxersen — Geographic Atrophy · insufficient efficacy to advance into Phase 3 development
Biotech Distilled coverage (1)
- 2026-09-10 Ionis Pharmaceuticals (IONS) — Scientific Deep Dive for Olezarsen, Zilganersen and the RNA-Targeted Pipeline 🔒 Paid
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