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A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells

Trial Profile

A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells

Status: Suspended
Phase of Trial: Phase I/II

Latest Information Update: 24 Apr 2023

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At a glance

  • Drugs MB 107 (Primary) ; Busulfan
  • Indications Immunodeficiency disorders; X-linked genetic disorders
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Acronyms LVXSCID-ND
  • Most Recent Events

    • 14 Apr 2023 Status changed from recruiting to suspended.
    • 19 May 2022 According to a Mustang Bio media release, UCSF Benioff Children's Hospital in San Francisco is one of the clinical site of the study.
    • 19 May 2022 According to a Mustang Bio media release, interim data from this trial were presented at the 25th American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting.
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