A gene therapy for patients with X-linked severe combined immunodeficiency (X-SCID) has passed pre-clinical safety and efficacy tests in China – paving the way for trials in the country.
Professor Adrian Thrasher from UCL Great Ormond Street Institute of Child Health, UK, has been working with Chinese researchers on the project.
China is facing a rising incidence of X-SCID and reports 300 new cases a year. The researchers say there is an “urgent” need to develop gene therapy tailored to Chinese patients.
In a study of nine Chinese patients, the researchers identified six novel mutations in the IL2RG gene. Among the nine patients, two adolescents with atypical disease were also identified.
The researchers tested two self-inactivating lentiviral vectors (SIN-LV), provided by Prof Thrasher, in the laboratory. They decided to move forward with a codon-optimised gene after finding it seemed more effective than the “wild type” version.
Animal studies of this SIN-EFS-IL2RG.co vector transduced CD34+ cell treatment showed it did not have cancer-causing properties.
The findings have been reported in the journal Genes & Diseases.
The researchers write: “Overall, our studies have demonstrated the efficiency and safety of SIN-IL2RG-LV, which paves the way for conducting X-SCID gene therapy clinical trials in China in the near future.”
Source:
Hu M, Xu Q, Zhang F, Buckland KF, Gao Y, Du W, Ding Y, Zhou L, Sun X, Ma L, Zhang Z, Tang X, Zhao X, Thrasher AJ, An Y (2025) “Preclinical ex vivo IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency disease.” Genes & Diseases, May 2025, doi: 10.1016/j.gendis.2024.101445
Link: https://www.sciencedirect.com/science/article/pii/S2352304224002423
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