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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: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 06 Nov 2019

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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

    • 06 Nov 2019 According to a Mustang Bio media release, data will be presented at 61st American Society of Hematology Annual Meeting.
    • 22 Aug 2019 According to a Mustang Bio media release, based on the data from this study, MB-107 has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA).
    • 18 Apr 2019 Results published in the New England Journal of Medicine
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