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NS 050/NCNP 03

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Drug Profile

NS 050/NCNP 03

Alternative Names: Exon 50 skipping antisense oligonucleotide; NS-050/NCNP-03

Latest Information Update: 25 Dec 2024

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At a glance

  • Originator Nippon Shinyaku
  • Developer 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

  • Phase I/II Duchenne muscular dystrophy

Most Recent Events

  • 18 Sep 2024 Phase-I/II clinical trials in Duchenne muscular dystrophy (In children, In adolescents) in Japan, USA (IV) (NCT06053814)
  • 13 Sep 2024 NS Pharma plans phase-I/II trial for Duchenne muscular dystrophy in Japan(Parenteral)
  • 10 Sep 2024 US FDA grants rare pediatric disease designation status to NS 050/NCNP 03 for Duchenne muscular dystrophy in USA

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