Gene Transfer for ADA-SCID Using an Improved Lentiviral Vector (TYF-ADA)

  • End date
    Dec 31, 2021
  • participants needed
  • sponsor
    Shenzhen Geno-Immune Medical Institute
Updated on 23 January 2021


Gene transfer for ADA-SCID using an improved lentiviral vector (TYF-ADA)


This clinical trial will evaluate a safety and efficiency improved lentiviral vector system for delivering a therapeutic gene to patients with severe combined immunodeficiency (SCID) due to a defective adenosine deaminase (ADA) gene. This gene encodes for the adenosine deaminase enzyme, which is essential for the proper growth and function of infection-fighting white blood cells called T and B lymphocytes. Patients who lack this enzyme are vulnerable to frequent and severe infections.

ADA-SCID patients are normally rescued by a bone marrow transplant (BMT) from a matched healthy donor. However, matched donors are difficult to find and donor BMT is associated with high risk. This trial aims to treat ADA-SCID using a safety and efficiency improved self-inactivating lentiviral vector carrying a functional ADA gene to correct the genetic defect. By collecting an individual's stem cells and modifying them with a lentivirus, the gene-corrected cells can be returned to the patient to help produce normal healthy immune cells.The primary objectives are to evaluate the safety of the improved self-inactivating lentiviral vector TYF-ADA, the ex vivo gene transfer clinical protocol and the efficacy of immune reconstitution in patients overcoming frequent infections present at the time of treatment. We will assess the lentiviral gene integration sites and the long-term effect of this gene transfer procedure.

Condition Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Treatment TYF-ADA gene-modified autologous stem cells
Clinical Study IdentifierNCT03645460
SponsorShenzhen Geno-Immune Medical Institute
Last Modified on23 January 2021


Yes No Not Sure

Inclusion Criteria

Diagnosis of classical ADA-SCID based on
A proven defective adenosine deaminase (ADA) gene as defined by direct sequencing of patient DNA
T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells < 300/ul, or less than 50% of normal value for in vitro mitogen stimulation, or absent proliferation in vitro to antigens
With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus or fungus; disseminated BCG infection
No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children
No prior allogeneic stem cell transplantation
Life expectancy 2 months
Negative for HIV infection
Written, informed consent obtained prior to any study-specific procedures

Exclusion Criteria

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