Drug Profile
NADPH oxidase gene therapy - Orchard Therapeutics
Alternative Names: Autologous CD34+ cells transduced with lentiviral vector containing GP91PHOX gene - Genethon; Autologous CD34+cells transduced with the G1XCGD viral vector - Genethon; Chronic granulomatous disease gene therapy - Genethon; g1xcgd; G1XCGD transduced CD34+ cells; Lentiviral G1XCGD Gene Therapy - Genethon; Lentiviral vector transduced CD34+ cells; NADPH oxidase gene therapy - Genethon; OTL 102Latest Information Update: 02 Feb 2024
Price :
$50
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At a glance
- Originator Genethon
- Developer Genethon; University of California at Los Angeles
- Class Gene therapies
- Mechanism of Action Gene transference; NADPH oxidase stimulants
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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
- Phase I/II Chronic granulomatous disease
Most Recent Events
- 24 Jan 2024 Orchard Therapeutics has been acquired by Kyowa Kirin
- 29 Jan 2020 NADPH oxidase gene therapy receives Orphan Drug status for Chronic granulomatous disease in USA
- 29 Jan 2020 Orchard Therapeutics plans a registrational study in Chronic granulomatous disease (In adolescents, In adults, In children, In infants) in USA (IV)