Early data from first human CRISPR trials reveals promising results
Category: #health  By Pankaj Singh  Date: 2019-11-21
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Early data from first human CRISPR trials reveals promising results

Vertex Pharmaceuticals and CRISPR Therapeutics have recently revealed early data from their joint trials to test CRISPR treatment in humans. The data was obtained from two patients suffering from serious blood disorders. Trial data indicates towards a promising breakthrough in what could be a one-time-only treatment for patients with chronic ailments.

The treatment, dubbed CTX001, is a dual-phase study, designed to study effects of the medication in transfusion dependent beta thalassemia patients and sickle cell disease patients. The data obtained from this trial comes from one patient in each trial.

Two years prior to the trials, the patient with beta thalassemia required an average of 16.5 blood transfusions each year. During the same period, the sickle cell patient suffered from vaso-occlusive crises every year. Vaso-occlusive crises involves a complication of the condition which causes blood cells to stick together and block blood vessel. The conditional is painful and can cause significant tissue and organ damage.

Nine months post the CTX001 treatment, the beta thalassemia patient showed almost-normal hemoglobin levels and required no further blood transfusions. The sickle cell patient, four months post treatment no longer suffered from vaso-occlusive crises and displayed near-normal hemoglobin levels as well.

Red blood cell count with fetal hemoglobin in both patients was adequately high – over 99% in the former and 94% in the latter. These positive outcomes indicate that the CRISPR treatment successfully carried out its objective.

Beta thalassemia and sickle cell disease are both caused by beta-globin gene mutation. This mutation leads to defective or missing hemoglobin, which is the red blood cell component responsible for carrying oxygen. The CTX001 was developed on the basis of fetal hemoglobin’s ability to display protective tendencies in adults with blood disorders. Fetal hemoglobin is a component found in newborns which is later replaced by adult hemoglobin.

Vertex CEO Jeffery Leiden, M.D., Ph.D. has reportedly stated that while data yields promising results, the clinical study is still in its early stages. He expressed his anticipation towards working with various healthcare experts in the years ahead to develop effective treatments for these two severe blood ailments. Dr. Leiden also stated that studies will be undertaken to expedite gene-editing treatments for other ailments including myotonic dystrophy type 1 and Duchenne muscular dystrophy.
 

Source Credit: https://www.fiercebiotech.com/biotech/vertex-crispr-s-gene-editing-treatment-for-blood-disorders-shows-promise-early-data



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Pankaj Singh

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Pankaj Singh

Pankaj Singh Develops content for Market Size Forecasters, Algosonline, and a couple of other platforms. A Post Graduate in Management by qualification, he worked as an underwriter in the UK insurance domain before deciding to switch his field of profession. With exp...

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