Research programme: AAV-based gene therapies - Bristol-Myers Squibb/uniQure

Drug Profile

Research programme: AAV-based gene therapies - Bristol-Myers Squibb/uniQure

Alternative Names: AAV-S100A1; AMT 120; AMT-126; S100A1

Latest Information Update: 14 Apr 2016

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At a glance

  • Originator Bristol-Myers Squibb; uniQure
  • Class Calcium binding proteins; Cardiovascular therapies; Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Cardiovascular disorders

Most Recent Events

  • 04 Apr 2016 UniQure plans to file an IND application for Cardivascular disorders
  • 06 Apr 2015 uniQure and Bristol-Myers Squibb enter into a collaboration agreement to develop AAV based gene therapies for cardiovascular disorders
  • 11 Aug 2014 Pharmacodynamics data from a preclinical trial in Cardiovascular disorders released by uniQure
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