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Rugonersen - Oak Hill Bio

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Drug Profile

Rugonersen - Oak Hill Bio

Alternative Names: OHB-724; RG 6091; RO 7248824; UBE3A-LNA

Latest Information Update: 28 Jun 2026

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At a glance

  • Originator Roche
  • Class Antisense oligonucleotides
  • Mechanism of Action Gene silencing; UBE3A 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

  • No development reported Angelman syndrome

Most Recent Events

  • 28 Jun 2026 No recent reports of development identified for phase-I development in Angelman-syndrome(In children, In infants) in Italy (Intrathecal, Injection)
  • 28 Jun 2026 No recent reports of development identified for phase-I development in Angelman-syndrome(In children, In infants) in Netherlands (Intrathecal, Injection)
  • 28 Jun 2026 No recent reports of development identified for phase-I development in Angelman-syndrome(In children, In infants) in Spain (Intrathecal, Injection)

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