CRISPR: A clinical trial focusing on somatic gene therapy is about to start in the United States

Publié le 19 Jul, 2016

The Biosafety and Ethics Committees of three universities have approved an initial clinical trial using the CRISPR Cas9 editing technique in humans. This is a somatic gene therapy study, the aim of which is to create genetically modified immune cells using CRISPR to “attack”three types of cancer.


The clinical trial proposed includes 15 patients with multiple myeloma, melanoma and sarcoma. Firstly, the safety of the investigational medicinal treatment must be assessed and the feasibility of producing genetically modified cells by genetic engineering should be tested. Genetically modified immune cells would be injected back into the patient to target the destruction of tumour cells.


Three universities in Texas, California and Pennsylvania will be involved in the trial. The approval of other centres is pending along with FDA[1] authorisation.


[1] Food and Drug Administration.

ASH clinical news (18/07/2016)

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