ATP7A gene therapy - Cyprium Therapeutics

Drug Profile

ATP7A gene therapy - Cyprium Therapeutics

Alternative Names: AAV-ATP7A gene therapy; rsATP7A cDNA

Latest Information Update: 06 Apr 2017

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At a glance

  • Originator National Institute of Child Health and Human Development
  • Developer Cyprium Therapeutics; National Institute of Child Health and Human Development
  • Class 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.

    Yes - Menkes kinky hair syndrome
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Menkes kinky hair syndrome

Most Recent Events

  • 14 Mar 2017 ATP7A gene therapy licensed to Cyprium Therapeutics worldwide
  • 14 Mar 2017 Preclinical trials in Menkes kinky hair syndrome in USA (Parenteral)
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