New gene therapy proving safe and successful in sickle cell patients' treatment

Researchers presenting preliminary data from a clinical trial aimed at discovering a cure for sickle cell disease reveal positive results among its first patients.Sickle cell disease, a genetic blood disorder, is a painful and debilitating condition for which there are few approved therapies.

Researchers involved in the multicenter Ruby Trial presented an update on the safety and effectiveness of a single dose of EDIT-301, an experimental one-time gene editing cell therapy that modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease.

The first four patients, two of whom were treated at Cleveland Clinic Children’s, had their stem cells collected for gene editing. The patients then underwent chemotherapy treatment to destroy their remaining bone marrow, making room for the repaired cells that were later infused back into their body.

This is the first time a novel type of CRISPR gene-editing technology – known as CRISPR/CA12 - is being used in a human study to alter the defective gene. This technology is a highly precise tool to modify blood stem cells genomes to enable robust, healthy blood cell production.

The data showed new white blood cells in all four patients at about four weeks with no severe adverse effects. Patients also achieved a normal level of hemoglobin, which is the most important component of red blood cells that carry oxygen throughout the body. The patients also have been free of sickle cell disease’s associated pain attacks for a period of 11 months and seven months following therapy.

Reference:

Rabi Hanna et al,CLEVELAND CLINIC