The U.S Food and Drug Administration (FDA) has just cleared a new investigational gene editing therapy for Sickle Cell Disease, called GPH101, for testing in a Phase 1/2 clinical trial.
The trial is known as CEDAR and involves an open-label clinical protocol that tests for the safety and pharmacological properties of GPH101 in adults, as well as the efficacy of it in adults and teens with severe SCD.
The treatment works by harnessing the power of the CRISPR-Cas9 gene-editing tool in conjunction with natural DNA repair, to remove the mutation of beta globin that causes Sickle Cell Disease. This occurs in bone marrow stem cells, which would be removed from the patient, engineered with the correcting gene, and transplanted back into the patient.
The implications of the study are promising, as its success would yield a mechanism for curing the disease through restoring the production of normal hemoglobin while preventing damage in red blood cells.
GPH101 was originally developed by investigators at Stanford University, but has been advanced by the Center for Definitive and Curative Medicine. The therapy’s rights are held exclusively by Graphite Bio, and Co-founder Matthew Porteus, MD, PhD, described the start of the trials as “the culmination of decades of research and scientific investment,” in an interview with Sickle Cell Anemia News.
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