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NADPH oxidase gene therapy - Orchard Therapeutics

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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 102

Latest Information Update: 05 Jan 2025

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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
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Chronic granulomatous disease
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Chronic granulomatous disease

Most Recent Events

  • 01 Dec 2024 University of California at Los Angeles and Genethon completes a phase I/II trial in Chronic granulomatous disease (in infants, In children, In adolescents, In adults) in USA (NCT02234934)
  • 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

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