CRISPR-Infused Stem Cell Therapy Used To Treat Patient With Rare Blood Disorder

View of a short piece of a DNA double helix. Peter Artymiuk/Wellcome Collection CC BY

A private biotech venture claims to have combined CRISPR, a bacteria-derived gene-editing tool, with stem cell therapy to treat a human patient, the first time such a breakthrough has been made outside of China.

Vertex Pharmaceuticals and CRISPR Therapeutics have announced they are in the process of treating a patient with beta thalassemia using a CRISPR/Cas9 hematopoietic stem cell therapy, known as CTX001, according to a press release.

It's still early days for the clinical research, which is currently in a phase that's assessing the safety and efficacy of the treatment, however, so far, so good.

Off the back of this, the researchers are also investigating whether the approach could be used to treat severe sickle cell disease. They have already enrolled a patient in this clinical study, which will be launched in the US sometime in mid-2019.

“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,” Dr Samarth Kulkarni, Chief Executive Officer of CRISPR Therapeutics, said in a statement.

“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.”

Beta thalassemia is an inherited blood disorder that results in people not producing enough hemoglobin, the protein in red blood cells that helps to carry oxygen, leaving them anemic, short of breath, and tired. Typically, the condition is treated using regular blood transfusions.

In this treatment, researchers obtain a patient’s hematopoietic stem cells from blood samples and tweak them using CRISPR/Cas9 technology to produce higher levels of fetal hemoglobin in red blood cells.

Fetal hemoglobin is a form of hemoglobin that is naturally present at birth, but is then replaced by the adult form of hemoglobin. So, it’s worth noting that no new “outsider” genes are introduced into the cells, it’s manipulating the genetic information to turn the existing fetal gene back on. It’s also not editing any germline cells, such as eggs and sperm, which would result in inheritable changes propagating through every cell in the body.

The blood cells, now endowed with the ability to produce boosted levels of fetal hemoglobin and hold more oxygen, are then reinfused into the bloodstream. 

“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,” added Dr David Altshuler, Executive Vice President and Chief Scientific Officer at Vertex. “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.” 

Shares of CRISPR Therapeutics skyrocketed shortly after the announcement on Monday, as did those of a number of other biotech companies working in the field, Bloomberg reports.

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