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UX 053

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Drug Profile

UX 053

Alternative Names: LUNAR-GSD3; LUNAR-GSDIII; UX-053

Latest Information Update: 11 Apr 2023

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At a glance

  • Originator Arcturus Therapeutics
  • Developer Arcturus Therapeutics; Ultragenyx Pharmaceutical
  • Class RNA
  • Mechanism of Action Enzyme replacements
  • Orphan Drug Status

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

    Yes - Glycogen storage disease type III
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I/II Glycogen storage disease type III

Most Recent Events

  • 20 Mar 2023 Ultragenyx Pharmaceutical terminates Phase-I/II clinical trials in Glycogen-storage-disease-type-III in USA, Canada, France, Italy, Spain, United Kingdom (IV) due to sponsor decision not related to safety concerns (NCT04990388)
  • 31 Dec 2022 Ultragenyx Pharmaceutical has patents protection for UX 053
  • 01 Sep 2021 Phase-I/II clinical trials in Glycogen-storage-disease-type-III in USA (Parenteral) (NCT04990388)

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