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ATP7A gene therapy - Cyprium Therapeutics

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Drug Profile

ATP7A gene therapy - Cyprium Therapeutics

Alternative Names: AAV-ATP7A gene therapy; rAAV9-rsATP7A; rsATP7A cDNA

Latest Information Update: 27 Feb 2026

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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

  • 23 Feb 2026 Cyprium Therapeutics plans a clinical trial for Menkes kinky hair syndrome in an undisclosed location
  • 05 Mar 2024 Preclinical development is ongoing in USA
  • 05 Mar 2024 Interim pharmacodynamics data from a preclinical studies in Menkes kirky hair syndrome released by Cyprium Therapeutics

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