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A first in human study of AAV-ATP7A

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A first in human study of AAV-ATP7A

Status: Planning
Phase of Trial: Phase I

Latest Information Update: 06 Mar 2024

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At a glance

  • Drugs ATP7A gene therapy Cyprium Therapeutics (Primary)
  • Indications Menkes kinky hair syndrome
  • Focus Adverse reactions; First in man

    • Most Recent Events

    • 06 Mar 2024 New trial record
    • 04 Mar 2024 According to a Cyprium Therapeutics media release, the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) has awarded a three-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children's Hospital and Principal Investigator, Stephen G. Kaler, to fund completion of preclinical studies, manufacturing and preparation of an Investigational New Drug Application for a first-in-human clinical trial.

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