By TONNY ABET

Scientists in Switzerland based medical firms have discovered medication which causes great relief for sickle cell patients.

CRISPR Therapeutics and Vertex Pharmaceuticals, the companies behind the ground-breaking gene-editing technology to treat inherited disorders, last week announced positive data from two patients that underwent pilot treatment.

More than 20,000 babies in Uganda are born with sickle cell disease each year, according to Ministry of Health.

As a parent, the conviction of being unable to rescue your diseased child from unceasing agonising pain due to sickle cell disease is the most draining.

Ms Victoria Gray, 34, a patient with severe sickle cell disease from Mississippi, USA has been relieved of excruciating pain after 30 days of CTX001 treatment.
CTX001 is an investigational therapy that treats inherited disorders of blood such as sickle cell diseases.

“At four months after CTX001 infusion, the patient was free of vaso-occlusive crises (VOCs),” the medical experts revealed in the press statement.

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VOCs is the primary cause of pain in sickle cell patients, according to experts.

VOCs occurs when circulation is obstructed by sickled red blood cells, causing restriction in blood supply to body parts and a shortage of oxygen.

“The patient had a total hemoglobin levels of 11.3 g/dL,” the experts wrote.This is the normal range of hemoglobin in healthy people.

In sickle cell patients, hemoglobin level is often as low as 6g/dL. Hemoglobin level is the measure of blood quality.

In the press release, another patient with beta-thalassemia that previously had to be transfused 16 times a year was also relieved.
“At nine months after CTX001 infusion, the patient was transfusion independent,” the experts revealed.

The patient had also recovered to normal hemoglobin levels of 11.9 g/dL, according to the experts.

“We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous haematopoietic stem cell candidate, CTX001,” said Dr Samarth Kulkarni, the Chief Executive Officer of CRISPR Therapeutics.

“These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention,” he said.

He added: “We continue to enroll these studies as we drive forward to develop CRISPR/Cas9 therapies as a new class of transformative medicines to treat serious diseases.”

“The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia,” said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex.

He added: “While the data are exciting, we are still in the early phase of this clinical program.”

Mr Leiden said they were looking forward to continuing to work with physicians, patients, caregivers and families over the coming months and years to bring forward the best possible therapy for these two serious diseases.

“We shall continue to accelerate our gene-editing programmess for other serious diseases such as Duchenne muscular dystrophy and myotonic dystrophy type 1,” he added.



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