Research programme: CNS gene therapies - uniQure

Drug Profile

Research programme: CNS gene therapies - uniQure

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

Latest Information Update: 10 Apr 2017

Price : $50

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.

    No
  • 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

  • 04 Apr 2017 Pharmacodynamics data from a preclinical trial in Huntington's disease released by uniQure
  • 09 Jan 2017 uniQure plans a proof-of-concept trial for Huntington’s disease
  • 28 Jun 2016 Uniqure expands its agreement with Protein Sciences to include Uniqure's pipeline of gene therapies
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