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NS 051/NCNP 04

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Drug Profile

NS 051/NCNP 04

Latest Information Update: 21 Feb 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

  • 04 Feb 2025 NS 051/NCNP 04 is available for licensing as of 04 Feb 2025. https://www.nspharma.com/partnership
  • 04 Feb 2025 US FDA grants rare pediatric disease designation status to NS 051/NCNP 04 for Duchenne muscular dystrophy in USA
  • 23 Jan 2025 Preclinical trials in Duchenne muscular dystrophy in Japan (unspecified route)

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