Science 3 min read

Scientists Successfully use CRISPR Gene Therapy on Patient

Liya Graphics /

Liya Graphics /

Researchers from U.S.-based company Vertex and Switzerland-based CRISPR Therapeutics have reportedly used CRISPR gene therapy to treat the blood disorder of a volunteer patient. This is the second time ever in history that the controversial treatment has been used on a human and the first attempt in the West.

The first ever use of CRISPR gene-editing treatment was conducted by a Chinese group of researchers from the Sichuan University in Chengdu, China. The team reportedly injected a person with cells containing edited genes to treat lung cancer.

This time, Western scientists treated their patient with an “investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy” called CTX001. This CRISPR treatment contains transfusion-dependent beta-thalassemia and is part of the company’s human trial on gene-editing therapy.

In a statement regarding the trial, CRISPR Therapeutics CEO, Samarth Kulkarni, said:

“We have made tremendous progress with CTX001 and are pleased to announce that we’ve treated the first patient with beta thalassemia in this clinical study.

Treating the first patient in this study marks an important scientific and medical milestone and the beginning of our efforts to fully realize the promise of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases.”

CRISPR Gene Therapy Human Trial

The CRISPR gene therapy CTX001 is an ex vivo treatment that involves the extraction of hematopoietic stem cells from human blood cells, editing the genes, and the reinfusion of the cells through stem cell transplant.

According to Vertex, the procedure is basically a bone marrow transplant where they “introduce a new part.” Aside from the beta-thalassemia patient, Vertex said that they enrolled another patient to receive CTX001 treatment for severe sickle cell disease. The patient will begin CRISPR gene therapy by the middle of this year.

Vertex’s Executive Vice President and CSO, David Altshuler, explained:

“Beta-thalassemia and sickle cell disease are serious, life-threatening diseases, and we are evaluating ex vivo treatment with CTX001 with the goal of creating a one-time potential curative therapy.

Our collaboration with CRISPR Therapeutics offers an exciting new potential therapeutic approach that complements our strategy of using scientific innovation to create transformative medicines for serious diseases.”

Both beta-thalassemia and sickle cell disease (SCD) are genetic blood disorders caused by mutations in the beta-globin gene. Although a great first step in gene therapy, this could only be the beginning of treatments involving genetic editing.

Read More: The Latest Breakthroughs Of Gene Editing Tech, CRISPR-Cas9

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Chelle is the Product Management Lead at INK. She's an experienced SEO professional as well as UX researcher and designer. She enjoys traveling and spending time anywhere near the sea with her family and friends.

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