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SRP-4053 — Competitive Intelligence Brief
phase 3
Antisense oligonucleotide
Dystrophin pre-mRNA (exon 53)
Neuromuscular/Genetic
Small molecule
Live · refreshed every 30 min
Target snapshot
SRP-4053 (SRP-4053) — Sarepta Therapeutics, Inc.. 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.
Comparator set (6 drugs)
| Drug | Generic | Sponsor | Phase | Class | Target | Approval / PDUFA |
|---|---|---|---|---|---|---|
| SRP-4053 TARGET | SRP-4053 | Sarepta Therapeutics, Inc. | phase 3 | Antisense oligonucleotide | Dystrophin pre-mRNA (exon 53) | |
| 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). SRP-4053 — Competitive Intelligence Brief. https://druglandscape.com/ci/srp-4053. Accessed 2026-05-13.
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