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GTX-102 — Competitive Intelligence Brief
phase 3
Antisense oligonucleotide
FUS mRNA
Neurology
Small molecule
Live · refreshed every 30 min
Target snapshot
GTX-102 (GTX-102) — Ultragenyx Pharmaceutical Inc. GTX-102 is an antisense oligonucleotide that reduces production of FUS protein by targeting FUS mRNA in patients with FUS-associated neurological disease.
Comparator set (6 drugs)
| Drug | Generic | Sponsor | Phase | Class | Target | Approval / PDUFA |
|---|---|---|---|---|---|---|
| GTX-102 TARGET | GTX-102 | Ultragenyx Pharmaceutical Inc | phase 3 | Antisense oligonucleotide | FUS mRNA | |
| Qalsody | TOFERSEN | Biogen Ma | marketed | Antisense Oligonucleotide [EPC] | Superoxide dismutase 1 (SOD1) mRNA | 2023-01-01 |
| Amondys 45 | CASIMERSEN | Sarepta | marketed | Antisense Oligonucleotide [EPC] | exon 53 of dystrophin pre-mRNA | 2021-01-01 |
| Vyondys 53 | GOLODIRSEN | Sarepta | marketed | Antisense Oligonucleotide [EPC] | exon 53 of dystrophin pre-mRNA | 2019-01-01 |
| Exondys 51 | ETEPLIRSEN | Sarepta | marketed | Antisense Oligonucleotide [EPC] | exon 51 of dystrophin pre-mRNA | 2016-01-01 |
| Kynamro | MIPOMERSEN | Kastle Theraps Llc | marketed | Antisense Oligonucleotide | 2013-01-01 | |
| PF-07852352 Influenza saRNA 1 | pf-07852352-influenza-sarna-1 | Pfizer | marketed | antisense oligonucleotide | influenza virus |
Recent regulatory actions (last 90 days)
Upcoming PDUFA dates (next 180 days)
Patent timeline (forward window)
Sponsor landscape (Antisense oligonucleotide class)
- Ionis Pharmaceuticals, Inc. · 3 drugs in this class
- Sarepta Therapeutics, Inc. · 2 drugs in this class
- Isarna Therapeutics GmbH · 1 drug in this class
- Leadiant Biosciences, Inc. · 1 drug in this class
- NYU Langone Health · 1 drug in this class
- Avidity Biosciences, Inc. · 1 drug in this class
- Ultragenyx Pharmaceutical Inc · 1 drug in this class
- European Organisation for Research and Treatment of Cancer - EORTC · 1 drug in this class
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Cite this brief
Drug Landscape (2026). GTX-102 — Competitive Intelligence Brief. https://druglandscape.com/ci/gtx-102. Accessed 2026-06-10.
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