Crispr’s Gene-editing ‘revolution’ treats the internal organ for the first time

A U.S. start-up has successfully treated the first patients using Crispr gene modification therapy directed at the body to an internal organ.

The first test data from Intellia Therapeutics, co-founded by the Nobel Prize winner Jennifer Doudna, marked an advance for Crispr-based treatments, showing scientists had overcome challenges that had previously limited the use of technology to edit cells outside the body or in the eye.

The Boston-based start-up, working with biotech company Regeneron, has treated transthyretin amyloidosis, a devastating disease in which an accumulation of a problematic protein affects a patient’s heart and nervous system, cutting off his hopes of life.

John Leonard, chief executive of Intellia, said he was “super gratified” to see the positive results, which opened the door to treatments beyond the “small subset” of diseases for which Crispr-based treatments have been tested.

“The attraction and promise of Crispr is this notion that you can change any gene, however, anywhere in the genome, as long as you can get there. And the last condition is the key,” he said. “This is the first time Crispr has ever been infused into a patient. . . and the first time we were able to target a gene successfully. ”

Crispr – which means agglomerates regularly spaced short palindromic repeats – is a system used by bacteria to protect themselves from viruses. In 2012, Doudna and her French colleague Emmanuelle Charpentier discovered how to do it use it as a gene editing tool.

Shares in Intellia have increased 233 percent since it became public in 2016. The company is one of three with original patents on the shares. The others are Crispr Therapeutics, which has treated patients for sickle cell disease, and Editas Medicine, which is in trials to treat a form of inherited blindness.

Intellia seeks to modify bone marrow to treat blood-based diseases without transplanting cells, including working with the Bill & Melinda Gates Foundation to treat patients in Africa with sickle cell disease.

In its phase 1 trial, a Crispr treatment was inserted into a lipid nanoparticle, which was taken up in the blood from the same tissue that picks up cholesterol cells, and transported to the liver. Here, single treatment inactivated the TTR gene and reduced problem protein by 87 percent in patients with the highest dose. There were no serious side effects for day 28.

Julian Gillmore, professor of medicine at University College London who was the lead researcher of the phase 1 trial, has treated patients with the type of amyloidosis for 25 years, but for two decades there was not much to do for it. them. For the past five years, she has been able to use genetic silencers – but these treatments seem less effective and require regular infusions.

“From my personal perspective, having seen these patients worsen for so many years, I know families for years all decimated by this disease, it’s incredible to see this revolution,” he said.

FT Health

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