Research programme: CNS gene therapies - uniQure

Drug Profile

Research programme: CNS gene therapies - uniQure

Alternative Names: AAV5; AMT-090; AMT-130

Latest Information Update: 17 Jan 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.

  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • 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

  • 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
  • 22 Mar 2016 uniQure announces intention to submit IND application US FDA
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at so we can help.

Back to top