The “revolution” of Crispr gene editing treats the internal organ for the first time

A new US company has successfully treated the first patients using a Crispr gene-editing therapy aimed at an internal organ in the body.

The Nobel Prize was won by Intellia Therapeutics ’first trial data Jennifer Doudna, marked a breakthrough Crispr-based treatments, showing that scientists have overcome the challenges that limited the use of technology to edit cells outside the body or outside the eye.

The Boston-based start-up, working with biotechnology company Regeneron, treated transthyretin amyloidosis, a devastating disease whose accumulation of problematic protein affects the patient’s heart and nervous system, reducing their life expectancy.

John Leonard, CEO of Intellia, said he was “very pleased” to see positive results, which opened the door for him. beyond treatments A “small subset” of patients who have tried Crispr-based treatments.

“Crispr’s appeal and promise in any genus, however, is the idea that you can change anywhere in the genome until you get there. And that last condition is key,” he said. “It’s the first time Crispr has been given an infusion to the patient. . . and the first time we were able to successfully target a gene. ”

Crispr – which includes intermittent short palindromic repeats – is a system used by bacteria to protect against viruses. In 2012, Doudna and her French colleague Emmanuelle Charpentier found out how they did it use it as a tool for editing genes.

Shares of Intellia have risen 233% since its launch in 2016. The company is one of the cities with original patents on discoveries. They are others Crispr Therapeutics, the patient was treated for sickle cell disease, and Editas Medicine, which is in trials to treat a type of inherited blindness.

Intellia wants to edit bone marrow to treat blood-based diseases without transplanting cells, including working with the Bill & Melinda Gates Foundation to care for patients with African-American cells.

In the phase 1 trial, Crispr treatment was embedded in a lipid nanoparticle to pick up the same tissue that takes up cholesterol globules in the blood and transport it to the liver. There, the only treatment was inactivating the TTR gene and reducing the problem protein by 87 percent in patients with the highest dose. By day 28 there were no serious side effects.

Julian Gillmore, a professor of medicine at University College London, who was the lead researcher in the phase 1 trial, has treated patients with amyloidosis for 25 years, but could do little for them in two decades. For the past five years, he has been able to use gene silencers, but these treatments appear to be more effective and require regular infusions.

“From my personal point of view, having seen these patients get worse for so many years, I have known for years that this disease has completely destroyed families, it is horrible to see this revolution,” he said.

FT Health

Sign up for the monthly FT Health newsletter – essential information on global health for decision makers.

Register here with a click