First Human CRISPR Trials
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First Human CRISPR Trials

 CRISPR Gene Editing Technology Cures two blood disorders in Human Trials

Results from First Human CRISPR Trials were released indicating that two patients, one with beta-thalassemia and one with sickle cell disease, have potentially been cured of their conditions.

Vertex Pharmaceuticals and CRISPR Therapeutics jointly conducted the two trials.

Jeffery Leiden, CEO of Vertex, said that this is the first clinical evidence to demonstrate that Crispr/Cas9 can be used to cure or potentially cure serious genetic illnesses. Mr. Leiden termed it as a ‘remarkable scientific milestone.’

First Human CRISPR Trials
Vertex Pharmaceuticals CEO Jeffery Leiden. Credits: Forbes

CRISPR- Cas9 is a gene-editing system famous for its ability to repair or insert genes into DNA. The therapies tested in the clinical trials work by extracting bone marrow stem cells from the patients, editing these stem cells to fix the genetic mutations that cause the diseases, and then infusing the cells back into the patients.

First Human CRISPR Trials – Detailed Process

The patient’s body then takes over and can produce new, healthy cells. The engineering of the cells is done ex vivo. This allows scientists to make sure the correct changes are made, and the

re are no improper edits to the genome.

It has been nine months since the patient with beta-thalassemia received the one-time-only treatment and over four months for the patient with sickle cell disease. Leiden says that both of their conditions have improved significantly.

The patient with beta-thalassemia, who used to undergo more than 16 blood transfusions each year, hasn’t needed an infusion since the treatment.

The patient with sickle cell disease experienced an average of seven excruciating health crises per year before the treatment. Since the procedure, the patient has not experienced any intense health crisis.

Samarth Kulkarni, CEO of CRISPR Therapeutics, termed the First Human CRISPR Trials as dramatic.

First Human CRISPR Trials
CRISPR Therapeutics CEO Sam Kulkarni. Credits: Forbes

Both patients suffered side effects during the treatment, but doctors concluded they were caused by the bone marrow preparation, not the Crispr treatment.

Mr. Kulkarni added that it was too early to contemplate any pricing discussions for the CRISPR treatment.

First Human CRISPR Trials- The Way Forward

The applications of CRISPR seem limitless, but the field has encountered several ethical controversies. Last year, Chinese scientist He Jiankui shocked the medical community by announcing that he had altered the genes of two human children.

But germline editing was not a concern in First Human CRISPR Trials, where only somatic, or non-reproductive cells, were altered.

In addition to following these initial patients for the next two years to see if their diseases reoccur, Leiden says they’re enrolling multiple patients with both illnesses for the next phase of the clinical trial and will be starting treatments for those patients soon.

Rahul Mishra is a Science enthusiast and eager to learn something new each day. He has a degree in Microbiology and has joined forces with Biotecnika in 2019 due to his passion for writing and science.