Leukocyte adhesion deficiency type 1 - Healing Genes

Leukocyte adhesion deficiency type 1

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A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene

Phase 1 / 2


Doctors at the University of California, Los Angeles seek patients with severe Leukocyte Adhesion Deficiency-I (LAD-I) to trial a therapeutic gene therapy. This therapy utilizes the patient’s own (autologous) stem cells, which will be gene-modified in the lab to impart a gene for the CD18 immune cell receptor that is lacking in the LAD-I disease. They hope the gene will express the receptor after gene modification and improve the symptoms of the disease. Prior to the modified cells being re-administered via IV, the patient will receive chemotherapy.

Survival and improvement in the patients will be followed for 2 years.


  • Be 3 months of age or older
  • Have confirmed diagnosis of severe LAD-I meeting the study’s clinical criteria
  • Not have a medically-eligible human leukocyte antigen (HLA)-identical sibling donor transplant.


  1. Screening to confirm acceptance to the study.
  2. Blood draw to recover the patient’s own stem cells, which will be gene-modified in the lab (no treatment during that period).
  3. Admission to the study center and a course of chemotherapy.
  4. Single infusion of gene-modified stem cells, with close observation, before the final dose of chemotherapy.
  5. Follow up will continue for 2 years.


The trial will take place at the University of California, Los Angeles. Map.

Donald B Kohn, MD  |  310-794-1964  |  [email protected]

Rocket Pharmaceuticals Inc.

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