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Research programme: exon-skipping antisense oligonucleotides - NS Pharma

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Drug Profile

Research programme: exon-skipping antisense oligonucleotides - NS Pharma

Alternative Names: Exon 45 skipping candidate - NS Pharma; Exon 55 skipping candidate - NS Pharma

Latest Information Update: 03 Jul 2025

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At a glance

  • Originator NS Pharma
  • Class Antisense oligonucleotides; Morpholines
  • Mechanism of Action Dystrophin expression stimulants; Gene silencing
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy

Most Recent Events

  • 23 Jun 2025 Exon-skipping antisense oligonucleotides - NS Pharma is available for licensing as of 23 Jun 2025. https://www.nspharma.com/partnership/
  • 23 Jun 2025 Preclinical trials in Duchenne muscular dystrophy in USA (unspecified route) (NS Pharma pipeline, June 2025)

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