Patient Receives World's Second Ever CRISPR Gene Therapy

CRISPR has been used to treat beta thalassemia, the first time a gene-editing treatment has been used in the West.
John Loeffler

For the first time in the West, CRISPR has been used to treat human patients using gene-editing therapy.

First Western Treatment Using CRISPR

Switzerland-based CRISPR Therapeutics and Massachusetts-based Vertex have announced progress on the use of the revolutionary gene-editing technology as a treatment for a blood disorder, the first time such a treatment has been used by a western medical clinic.

The first ever use of a CRISPR gene-editing therapy is in China, where a cancer trial using the gene-editing technique for a cancer therapy is underway.


The first patient treated with CTX001, which the company describes as “an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy”, has transfusion-dependent beta thalassemia and has received the therapy as part of an investigational trial into gene-editing therapies in humans.

“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,” said Chief Executive Officer of CRISPR Therapeutics, Samarth Kulkarni.

“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.”


The company also says that they have enrolled the first patient to begin receiving the investigating treatment of CTX001 for severe sickle cell disease and is expected to begin treatment by the middle of 2019.

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“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,” said Vertex’s Executive Vice President and Chief Scientific Officer, David Altshuler.

“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.”

Beta Thalassemia and Sickle Cell Disease

Beta Thalassemia is a genetic blood disorder caused by mutations in the beta-globin gene, which results in low to no beta-globin productions. Beta-globin is an important component of hemoglobin, which is responsible for transporting oxygen through the blood.

Sickle Cell Disease (SCD) is also a genetic blood disorder caused by mutations in the beta-globin gene, which produces abnormal, crescent-shaped hemoglobin. This can cause the blood cells to become rigid and block smaller blood vessels. Severe SCD can lead to acute pain, organ damage, and reduced lifespans.

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