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Ultevursen - ProQR Therapeutics

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Drug Profile

Ultevursen - ProQR Therapeutics

Alternative Names: QR-421; QR-421a; QRX-421

Latest Information Update: 24 Apr 2025

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At a glance

  • Originator ProQR Therapeutics
  • Developer Laboratoire Thea; ProQR Therapeutics
  • Class Antisense RNA; Eye disorder therapies; Oligonucleotides
  • Mechanism of Action RNA interference; USH2A protein modulators
  • Orphan Drug Status

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

    Yes - Usher syndromes
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II/III Retinitis pigmentosa
  • Phase II Usher syndromes

Most Recent Events

  • 11 Dec 2024 Phase-II clinical trials in Retinitis pigmentosa (In adolescents, In adults, In children, In the elderly) in USA (Intravitreous) (NCT06627179)
  • 04 Oct 2024 Laboratoires Thea plans to initiated a phase II trial for Retinitis Pigmentosa (In children, In adolescents, In adults, In the elderly) (Intravitreal) (NCT06627179)
  • 08 Dec 2023 ProQR completes the divestment of sepofarsen and ultevursen to Laboratoires Théa to continue the development of these programs for patients with LCA10 and Usher syndrome

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