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Research programme: CNS and liver disorders gene therapy - uniQure

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Drug Profile

Research programme: CNS and liver disorders gene therapy - uniQure

Alternative Names: AAV capsid variants - uniQure; AAV5 gene therapies - uniQure

Latest Information Update: 28 Jun 2022

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At a glance

  • Originator uniQure
  • Developer 4D Molecular Therapeutics; uniQure
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • No development reported CNS disorders; Liver disorders

Most Recent Events

  • 28 Jun 2022 No recent reports of development identified for preclinical development in CNS-disorders in Netherlands (Parenteral)
  • 28 Jun 2022 No recent reports of development identified for preclinical development in CNS-disorders in USA (Parenteral)
  • 28 Jun 2022 No recent reports of development identified for preclinical development in Liver-disorders in Netherlands (Parenteral)

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