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Antisense oligonucleotide - Insmed

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Drug Profile

Antisense oligonucleotide - Insmed

Latest Information Update: 24 Apr 2026

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At a glance

  • Originator Insmed
  • Class Antisense oligonucleotides
  • Mechanism of Action APTX protein expression stimulants
  • 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

Highest Development Phases

  • Preclinical Ataxia

Most Recent Events

  • 21 Apr 2026 Preclinical trials in Ataxia in USA (unspecified route) prior to April 2026 (Insmed pipeline, April 2026)

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