Antisense Oligonucleotide

This page covers all Antisense Oligonucleotide drugs tracked by Drug Landscape: marketed products and active clinical-stage compounds, targeting influenza virus, APOC3 mRNA, SOD1 gene.

Targets

influenza virus · APOC3 mRNA · SOD1 gene · Dystrophin pre-mRNA (exon 53) · DMPK mRNA · SMN2 gene · GABA_A receptor alpha 1 subunit · Apolipoprotein(a) mRNA · FUS mRNA · Protein kinase C-alpha (PKC-alpha) mRNA

Marketed (2)

• Kynamro · Kastle Theraps Llc · Metabolic
• PF-07852352 Influenza saRNA 1 · Pfizer · Infectious Disease

Phase 3 pipeline (11)

• Del-desiran (AOC 1001) · Avidity Biosciences, Inc. · Cardiovascular
  Del-desiran is an antisense oligonucleotide that reduces production of apolipoprotein C-III (apoC-III) to lower triglycerides and improve lipid metabolism.
• Tivanisiran sodium ophthalmic solution · Sylentis, S.A. · Ophthalmology
  Tivanisiran sodium ophthalmic solution is an antisense oligonucleotide that targets the SOD1 gene to reduce the production of superoxide dismutase 1 protein.
• EZN-2279 · Leadiant Biosciences, Inc. · Cardiovascular
  EZN-2279 is an antisense oligonucleotide that inhibits apolipoprotein C-III (apoC-III) expression to reduce triglycerides and improve lipid metabolism.
• ION363 · Ionis Pharmaceuticals, Inc. · Cardiovascular
  ION363 is an antisense oligonucleotide that reduces production of apolipoprotein C-III (apoC-III), a protein that impairs triglyceride clearance and increases cardiovascular risk.
• SRP-4053 · Sarepta Therapeutics, Inc. · Neuromuscular/Genetic
  SRP-4053 is an antisense oligonucleotide that modulates dystrophin production by promoting exon 53 skipping in Duchenne muscular dystrophy patients with mutations amenable to this approach.
• SRP-9003 · Sarepta Therapeutics, Inc. · Neuromuscular/Genetic Disorders
  SRP-9003 is an antisense oligonucleotide that reduces production of DMPK protein by targeting mutant DMPK mRNA in myotonic dystrophy type 1.
• RO7234292 (RG6042) · Hoffmann-La Roche · Neurology
  RO7234292 is a small molecule antisense oligonucleotide that targets the SMN2 gene to increase production of the survival motor neuron protein.
• SAGE-547 · Supernus Pharmaceuticals, Inc. · Neurology
  SAGE-547 is an intravenous formulation of an antisense oligonucleotide that targets the GABA_A receptor alpha 1 subunit.
• AT-001 · NYU Langone Health · Cardiovascular
  AT-001 is an antisense oligonucleotide that reduces production of lipoprotein(a) by targeting hepatic apolipoprotein(a) synthesis.
• GTX-102 · Ultragenyx Pharmaceutical Inc · Neurology
  GTX-102 is an antisense oligonucleotide that reduces production of FUS protein by targeting FUS mRNA in patients with FUS-associated neurological disease.
• ISIS 3521 · European Organisation for Research and Treatment of Cancer - EORTC · Oncology
  ISIS 3521 is an antisense oligonucleotide that inhibits protein kinase C-alpha (PKC-alpha) expression to suppress cancer cell proliferation and survival.

Phase 2 pipeline (6)

• ISIS apoC-III Rx · Ionis Pharmaceuticals, Inc. · Cardiovascular
  Antisense oligonucleotide that targets apolipoprotein C-III
• ISIS-FGFR4RX · Ionis Pharmaceuticals, Inc. · Oncology
  Antisense oligonucleotide targeting fibroblast growth factor receptor 4
• AOC 1044 · Avidity Biosciences, Inc. · Immunology
  AOC 1044 is an investigational antisense oligonucleotide.
• AZD9150 · Acerta Pharma BV · Oncology
  AZD9150 is an antisense oligonucleotide that targets the RNA of the MYC oncogene, leading to reduced expression of MYC protein.
• AP 12009 10 µM · Isarna Therapeutics GmbH · Oncology
  AP 12009 is an antisense oligonucleotide that inhibits transforming growth factor-beta 2 (TGF-β2) expression to reduce immunosuppression and promote anti-tumor immunity.
• IONIS GHR-LRx · Ionis Pharmaceuticals, Inc. · Endocrinology
  IONIS GHR-LRx is an antisense oligonucleotide that inhibits growth hormone receptor (GHR) expression to reduce growth hormone signaling.