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AX 3875

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Drug Profile

AX 3875

Alternative Names: AX-3875

Latest Information Update: 30 May 2025

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At a glance

  • Originator ProQR Therapeutics
  • Class Oligonucleotides
  • Mechanism of Action Gene expression modulators
  • 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

  • Research CNS disorders; Metabolic disorders

Most Recent Events

  • 20 Mar 2025 Early research in CNS disorders in Netherlands (Parenteral) (ProQR Therapeutics pipeline, March 2025)
  • 20 Mar 2025 Early research in Metabolic disorders in Netherlands (Parenteral) (ProQR Therapeutics pipeline, March 2025)
  • 13 Mar 2025 ProQR Therapeutics has patent pending for Axiomer® program in multiple countries

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