Research programme: CNS and liver disorders gene therapy - uniQure/4D Molecular Therapeutics

Drug Profile

Research programme: CNS and liver disorders gene therapy - uniQure/4D Molecular Therapeutics

Alternative Names: AAV capsid variants - uniQure

Latest Information Update: 23 Apr 2015

Price : $50

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

  • Research CNS disorders; Liver disorders

Most Recent Events

  • 19 Jan 2014 Early research in CNS disorders in Netherlands (Parenteral)
  • 19 Jan 2014 Early research in CNS disorders in USA (Parenteral)
  • 19 Jan 2014 Early research in Liver disorders in Netherlands (Parenteral)
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