Get ready for 'Prime Editing' — gene-editing tech that can rewrite your DNA with a single injection

A biotechnology company in Cambridge, Massachusetts, says it is engineering cells inside a living human body in hopes of one day ending genetic diseases.

The company boasted that recent trials were performed on DNA inside of a person rather than the typical method of extracting the DNA first.

'A major milestone in gene editing.'

Clustered Regularly Interspaced Short Palindromic Repeats is technology used to selectively modify the DNA of living organisms. A company called Prime Medicine believes it has a more efficient way of accurately editing DNA than the CRISPR method — called Prime Editing — working directly in the body.

The company says the first disorder it is tackling with live trials is an illness called Wilson disease.

Wilson disease is a rare disorder where excess copper is abnormally stored in different places in the body, most commonly causing damage to the liver, brain, or eyes.

Prime Medicine's aim is to "rewrite faulty DNA responsible for Wilson disease that can cause severe liver and brain damage," wrote Max Marchione, founder of DNA testing company Superpower.

Marchione called it a "major milestone in gene editing."

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Delivered intravenously, the medicine uses PM577, an editing product using lipid nanoparticles to "correct pathogenetic ... mutations through a single" injection.

Studies explain that lipid nanoparticles "are not only limited to small molecular drugs but also include biomacromolecules such as messenger RNA (mRNA), small interfering RNA (siRNA), and antibodies."

PM577a is what targets the mutations and has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration for the treatment of Wilson disease, Prime Medicine said in a press release.

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Du Yu/Xinhua/Getty Images

"I never dreamed that in my lifetime I would see this applied to my rare disease," Rhonda Rowland, president of the Wilson Disease Association in Atlanta, Georgia, told Nature. "It's going to be a really exciting year," she added.

Prime Medicine said it intends to develop more products to address "the majority of pathogenic variants" around the world, and it is considering targeting another gene mutation, R778L, the most common in East Asian populations.

Prime Medicine is a publicly traded company currently trading around $3.65 per share at the time of this publication.

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