November 19, 2019 07:06 AM ESTUpdated 10:59 AM

In a landmark first glimpse of human data from Vertex, CRISPR/Cas9 gene therapy signals early benefit

Natalie Grover

Reporter

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

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In a landmark first glimpse of human data from Vertex, CRISPR/Cas9 gene therapy signals early benefit

Natalie Grover

Reporter

Unlock this article instantly by becoming a free subscriber.

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In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Natalie Grover

Reporter

Unlock this article instantly by becoming a free subscriber.

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