Drug Profile
AVR RD 04
Alternative Names: AVR-RD-04; CTNS-RD-04Latest Information Update: 25 Aug 2023
Price :
$50
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At a glance
- Originator GenStem Therapeutics
- Developer AVROBIO; University of California, San Diego
- Class Gene therapies; Haematopoietic stem cells therapies; Stem cell therapies
- Mechanism of Action CTNS protein stimulants; CTNS replacements; 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
- Suspended Cystinosis