Drug Profile
ATP7A gene therapy - Cyprium Therapeutics
Alternative Names: AAV-ATP7A gene therapy; rAAV9-rsATP7A; rsATP7A cDNALatest Information Update: 07 Mar 2024
Price :
$50
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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
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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 Menkes kinky hair syndrome
Most Recent Events
- 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
- 28 Apr 2021 No recent reports of development identified for preclinical development in Menkes-kinky-hair-syndrome in USA (Parenteral)