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Sepofarsen - ProQR Therapeutics

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Drug Profile

Sepofarsen - ProQR Therapeutics

Alternative Names: EW-9I3PJC3R; QR-110; QR110 - ProQR Therapeutics

Latest Information Update: 24 Jun 2025

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At a glance

  • Originator ProQR Therapeutics
  • Class Eye disorder therapies; Oligonucleotides
  • Mechanism of Action Cep290 protein modulators; RNA interference
  • Orphan Drug Status

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

    Yes - Leber congenital amaurosis
  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Leber congenital amaurosis

Most Recent Events

  • 11 Jun 2025 Phase-III clinical trials in Leber congenital amaurosis (In adults, In adolescents, In children) in USA (Intravitreous) (NCT06891443)
  • 25 Mar 2025 Laboratoires Thea and Sepul Bio plan a phase III trial for Leber congenital amaurosis (In adolescents, In adults, In children, In the elderly) (Intravitreous, Injection) , (EudraCT2024-518378-14-00) (NCT06891443)
  • 19 Apr 2024 ProQR Therapeutics successfully defended against opposition filed against a key patent for its ADAR-mediated RNA editing platform Axiomer™

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