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Research programme: CNS gene therapies - uniQure

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Drug Profile

Research programme: CNS gene therapies - uniQure

Alternative Names: AAV 5; AAV5 vector; AAV5-miC; AMT-090

Latest Information Update: 28 Mar 2023

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At a glance

  • Originator Amsterdam Molecular Therapeutics
  • Developer Lund University; uniQure; Universite de Toulouse; University of Cambridge
  • Class Antidementias; Antiparkinsonians; Gene therapies; Neuroprotectants
  • Mechanism of Action Gene silencing; Gene transference; Glial cell line-derived neurotrophic factor agonists
  • 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
  • Available For Licensing Yes

Highest Development Phases

  • No development reported Amyotrophic lateral sclerosis; Dementia; Hearing loss; Multiple system atrophy; Parkinson's disease

Most Recent Events

  • 28 Mar 2023 No recent reports of development identified for preclinical development in Amyotrophic-lateral-sclerosis in Netherlands (Parenteral)
  • 28 Mar 2023 No recent reports of development identified for preclinical development in Dementia in Netherlands (Parenteral)
  • 15 Feb 2019 Preclinical trials in Amyotrophic lateral sclerosis in Netherlands (Parenteral) before February 2019
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