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NADPH oxidase gene therapy - Orchard Therapeutics

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: 26 Jun 2019

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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

  • 26 Jun 2019 NADPH oxidase gene therapy is still in phase I/II trials for Chronic granulomatous disease (In adolescents, In adults, In children, In infants) in USA (NCT02234934)
  • 26 Feb 2019 Safety and efficacy data from a phase I/II trial in X-linked chronic granulomatous disease released by Orchard Therapeutics
  • 05 Dec 2018 Efficacy and adverse events data from a proof-of -concept phase I/II trial in X-Linked Chronic Granulomatous presented at the 60th Annual Meeting and Exposition of the American Society of Hematology (ASH-Hem-2018))
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