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X-Linked Combined Immunodeficiency Diseases

Forms of combined immunodeficiency caused by mutations in the gene for INTERLEUKIN RECEPTOR COMMON GAMMA SUBUNIT. Both severe and non-severe subtypes of the disease have been identified.

Drugs by status

Phase 3 trials (1)

  • immune globulin (human)

Phase 2 trials (19)

  • plerixafor
  • busulfan
  • cl20-4i-ef/a-hyc-opt
  • mecasermin
  • Filgrastim
  • palifermin
  • magnesium sulfate
  • cyclophosphamide
  • stem cells
  • alemtuzumab
  • autologous cd34+ cells
  • autologous cd34+ cells transduced withpsrs11.efs.il2rg.pre
  • cytarabine
  • autologous cd34+ cell transduced with g2scid vector
  • threonate
  • autologous cd34+ hematopoietic stem cells transduced ex-vivo with a gamma-globin lentiviral vector
  • cd34+ hscs transduced with the lentivirus vector, vsv-g pseudotyped cl20-4i-ef1α-hγc-opt
  • tyf-il-2rg gene-modified autologous stem cells
  • base-edited hematopoietic stem and progenitor cells

Phase 1 trials (3)

  • cyclosporine
  • mycophenolate
  • il2rg gene into the blood forming stem cells (cd34+ cells)

Organizations

Organization Involved with Phase 2 Indications (9)

  • National Heart, Lung, and Blood Institute (NHLBI)
  • National Institute of Allergy and Infectious Diseases (NIAID)
  • University of London
  • Harvard University
  • Shenzhen Geno-Immune Medical Institute
  • Capital Medical University
  • University of California, Los Angeles
  • Assisi Foundation
  • St. Jude Children's Hospital

Organization Involved with Phase 1 Indications (2)

  • National Cancer Institute (NCI)
  • University of Washington

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