Prime editing, a mightier version of CRISPR/Cas9 technology, has been part of rigorous research and development in recent years. Now, U.S. regulators have greenlit the first-ever clinical trial for this technology.
Massachusetts-based Prime Medicine received the go-ahead from the U.S. Food and Drug Administration (FDA) after preclinical data showed that its candidate was able to correct mutations in chronic granulomatous disease (CGD).
CGD is a rare condition and affects around one in 200,000 people worldwide. It is caused by mutations in any of the six genes that code for the molecule nicotinamide adenine dinucleotide phosphate (NADPH), which is responsible for carrying electrons within cells. White blood cells called phagocytes don’t function properly, and as a result, they fail to protect the body from bacterial and fungal infections.
Leave a reply