Novel gene therapy proving safe and successful in sickle cell patients treated at Cleveland Clinic Children’s
Cleveland: 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 ...







