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QR 313

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Drug Profile

QR 313

Alternative Names: PTW-002; QR-313; QRX-313

Latest Information Update: 18 Dec 2023

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At a glance

  • Originator ProQR Therapeutics
  • Developer Phoenicis Therapeutics; ProQR Therapeutics
  • Class Oligonucleotides; Skin disorder therapies; Small interfering RNA
  • Mechanism of Action RNA interference
  • Orphan Drug Status

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

    Yes - Epidermolysis bullosa
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Epidermolysis bullosa

Most Recent Events

  • 06 Sep 2022 Phoenicis Therapeutics plans a phase I/II trial for Epidermolysis bullosa (In children, In adolescent, In adults, In the elderly)in US (Topical, Gel), (NCT05529134)
  • 25 Aug 2021 Phoenicis Therapeutics terminates a phase I/II trial in Epidermolysis bullosa (In adolescents, In adults, In children) in USA, Czech Republic, Germany, France, Spain, United Kingdom (Topical), due to low enrolment (EudraCT2017-004806-17) (NCT03605069)
  • 07 Apr 2021 Phase-I/II development in Epidermolysis bullosa (In adolescents, In adults, In children) is still ongoing in France, Germany and Spain (Topical) (Wings Therapeutics' website, April 2021)

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