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A private biotech company claims to have associated CRISPR, a gene-derived gene editing tool, with stem cell therapy to treat a human patient, the first time such an advance has been made outside from China.
Vertex Pharmaceuticals and CRISPR Therapeutics announced that they were treating a patient with beta-thalassemia using CRISPR / Cas9 hematopoietic stem cell therapy, called CTX001, according to a Press release.
It is still early for clinical research, which is currently in a phase of evaluating the safety and effectiveness of treatment, but so far, so good.
Researchers also question whether this approach could be used in the treatment of severe sickle cell disease. They have already enrolled a patient in this clinical study, which will be launched in the United States in mid-2019.
"We have made tremendous progress with the CTX001 and are pleased to announce that we have treated the first beta-thalassemic patient in this clinical study," said Dr. Samarth Kulkarni, Chair of the CRISPR Therapeutics Board of Directors. declaration.
"Treating the first patient in this study marks an important scientific and medical milestone and marks the beginning of our efforts to fully realize the promise of CRISPR / Cas9 therapies as a new class of potentially transformative drugs for the treatment of serious diseases."
Thalassemia beta is an inherited blood disorder that prevents people from producing enough hemoglobin, the protein in red blood cells helping to carry oxygen, making them anemic, breathless and tired. As a rule, the disease is treated by regular blood transfusions.
In this treatment, the researchers extract blood samples from the hematopoietic stem cells of a patient and modify them. using CRISPR / Cas9 technology produce higher levels of fetal hemoglobin in red blood cells.
Fetal hemoglobin is a naturally occurring form of hemoglobin at birth, but it is then replaced by the adult form of hemoglobin. It should therefore be noted that no new "foreign" genes are introduced into the cells; he manipulates the genetic information to reactivate the existing fetal gene. In addition, it does not alter any germ cells, such as eggs and sperm, which would result in inherited changes spreading throughout the body's cells.
Blood cells, now endowed with the ability to produce increased levels of fetal hemoglobin and retain more oxygen, are then re-injected into the bloodstream.
"Beta-thalassemia and sickle cell disease are serious, life-threatening diseases. We are evaluating the ex vivo treatment at CTX001 with the goal of creating a potential punctual cure, "added Dr. David Altshuler, Executive Vice President and Scientific Director. at the top. "Our collaboration with CRISPR Therapeutics offers an exciting new therapeutic potential that complements our strategy of using scientific innovation to create transformative drugs for serious diseases."
The actions of CRISPR Therapeutics exploded shortly after the announcement on Monday, as did those of a number of other biotech companies active in the field, Bloomberg reports.
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