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: 10 Oct 2017

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At a glance

  • Originator Amsterdam Molecular Therapeutics
  • Developer Lund University; uniQure; Universite de Toulouse; University of Cambridge
  • Class 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 Hearing loss; Huntington's disease; Multiple system atrophy; Parkinson's disease

Most Recent Events

  • 06 Oct 2017 AMT-130 receives Orphan Drug status for Huntington's disease in USA
  • 28 Aug 2017 Protein Sciences Corporation has been acquired by Sanofi
  • 26 Apr 2017 uniQure announces intention to submit IND application for AMT 130 in Huntington's disease in 2018
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