Research programme: CNS gene therapies - uniQure

Drug Profile

Research programme: CNS gene therapies - uniQure

Alternative Names: AAV 5; AAV5 vector; AAV5-miHTT; AMT-090; AMT-130

Latest Information Update: 02 May 2018

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At a glance

  • Originator Amsterdam Molecular Therapeutics
  • Developer Lund University; uniQure; Universite de Toulouse; University of Cambridge
  • Class Antiparkinsonians; Gene therapies; Nerve growth factors; Small interfering RNA
  • Mechanism of Action DNA-directed RNA polymerase inhibitors; Gene silencing; Gene transference; Glial cell line-derived neurotrophic factor agonists; RNA interference
  • Orphan Drug Status

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

    Yes - Huntington's disease
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Huntington's disease
  • No development reported Hearing loss; Multiple system atrophy; Parkinson's disease

Most Recent Events

  • 25 Apr 2018 Safety and pharmacodynamics data from preclinical trials in Huntington's disease presented at the Annual Meeting of American Academy of Neurology (AAN-2018)
  • 14 Mar 2018 Safety and pharmacodynamics data from preclinical trials in Huntington's disease presented at the 13th Annual Huntington's Disease Therapeutics Conference (HDTC-2018)
  • 22 Jan 2018 AMT 130 is still in preclinical development for Huntington's disease in United Kingdom, France and Netherlands
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