Alpha-1 antitrypsin gene therapy

Drug Profile

Alpha-1 antitrypsin gene therapy

Alternative Names: Alpha-antitrypsin gene therapy

Latest Information Update: 14 May 2001

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At a glance

  • Originator Vanderbilt University
  • Developer Valentis; Vanderbilt University
  • Class Gene therapies
  • Mechanism of Action Trypsin inhibitors
  • Orphan Drug Status

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


Highest Development Phases

  • Discontinued Cystic fibrosis; Emphysema; Respiratory syncytial virus infections

Most Recent Events

  • 14 May 2001 Discontinued-I for Emphysema in USA (Inhalation)
  • 14 May 2001 Discontinued-Preclinical for Respiratory syncytial virus infections in USA (Unknown route)
  • 14 May 2001 Discontinued-Unspecified Phase in Cystic fibrosis in USA (Inhalation)
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