The world’s first complete gene editing phase I clinical trial completed in China: safe and effective

2020-05-05 16:22

Nature Medicine just published the world’s first gene editing phase I clinical trial results online: safe and effective. 


This is a dose-escalation study of ex-vivo knocked-out, expanded, and selected PD-1 knockout-T cells from autologous origin. The researchers isolated the immune cells from patients with refractory non-small-cell lung cancer, armed it through gene editing, and then mass-cultivated them into the patient to fight against cancer cells. The primary endpoint of this Phase 1 clinical trial is safety and feasibility, and the secondary endpoint is efficacy, for a total of 22 patients with advanced lung cancer were recruited for testing.


Of the 22 recruited patients, 17 patients had gene-edited immune cells sufficient for reinfusion therapy, and 12 of them were finally able to receive treatment. After receiving gene-edited immune cell reinfusion therapy, all treatment-related adverse events were grade 1/2.


Edited immune cells can be detected in peripheral blood after infusion. The median progression-free survival was 7.7 weeks, and the median overall survival was 42.6 weeks. Through next-generation sequencing, the median mutation frequency of off-target events was only 0.05% among the 18 candidate sites. From this, the researchers concluded that the clinical application of CRISPR/Cas9 gene-edited T cells is usually safe and feasible, and future trials should use improved gene editing methods to improve the therapeutic effect.

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