Drug Profile


Alternative Names: AAV2-GDNF; AMT-140

Latest Information Update: 05 Sep 2017

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At a glance

  • Originator uniQure
  • Developer Lund University; National Institute of Neurological Disorders and Stroke; uniQure; University of California at San Francisco
  • Class Antiparkinsonians; Gene therapies; Neuroprotectants
  • Mechanism of Action Gene transference; Glial cell line-derived neurotrophic factor expression stimulants; Neuron stimulants
  • Orphan Drug Status

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

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

Highest Development Phases

  • Phase I/II Parkinson's disease

Most Recent Events

  • 28 Aug 2017 Protein Sciences Corporation has been acquired by Sanofi
  • 16 Jul 2016 No recent reports of development identified for preclinical development in Parkinson's-disease in Sweden (Intracerebral, Infusion)
  • 30 Sep 2014 The University of California San Francisco and the National Institute of Neurological Disorders and Stroke complete treatment of patients in the first dose cohort in the phase I trial in Parkinson's disease
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