FDA moves closer to sickle cell cure that uses gene editing

The Food and Drug Administration may be one step closer toward what could be the first approval of a drug that uses the groundbreaking gene-editing tool CRISPR. The drug, called exa-cel, treats sickle cell disease, an inherited blood disorder that affects an estimated 100,000 people in the U.S., most of whom are Black, according to the Centers for Disease Control and Prevention. The illness causes the body’s red blood cells, usually disk-shaped, to take on a crescent or sickle shape.

It’s unclear what effects an off-target edit would have on a patient — it entirely depends on where it happens in the DNA. “Off-target editing does not necessarily mean that there’s going to be a bad outcome,” said committee member Scot Wolfe, a professor of molecular, cell and cancer biology at UMass Chan Medical School.

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FDA takes first step to protect children from medications that look and taste like candyBerkeley Lovelace Jr. is a health and medical reporter for NBC News. He covers the Food and Drug Administration, with a special focus on Covid vaccines, prescription drug pricing and health care. He previously covered the biotech and pharmaceutical industry with CNBC.
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