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An American start-up has successfully treated the first patients using Crispr gene editing therapy directed inside the body towards an internal organ.
The first data from the tests of Intellia Therapeutics, co-founded by the Nobel Prize winner Jennifer Doudna, was an advance for Crispr-based treatments, showing that scientists had overcome challenges that had previously restricted the use of technology to editing cells outside the body or in the eye.
The Boston-based start-up, which works with biotechnology company Regeneron, treated amyloidosis for transthyretin, a devastating disease in which the buildup of a problematic protein hits the heart and nervous system. a patient, reducing life expectancy.
John Leonard, chief executive of Intellia, said he was “very pleased” to see the positive results, which opened the door to treatments beyond the “small subset” of diseases for which Crispr-based treatments have been tried.
“The appeal and promise of Crispr is this notion that you can change any gene, anyway, anywhere in the genome, as long as you can get it. And that last condition is the key,” he said. “This is the first time that Crispr has been introduced into a patient. . . and the first time we have been able to target a gene successfully. ”
Crispr, meaning short, regularly interspersed palindromic repeats, is a system used by bacteria to protect themselves from viruses. In 2012, Doudna and her French partner Emmanuelle Charpentier figured out how to do it use it as a gene editing tool.
Shares of Intellia have risen 233% since it went public in 2016. The company is one of three to have the original patents on the discoveries. The others are Crispr Therapeutics, which treated patients with sickle cell disease, and Editas Medicine, which is in trials to treat a form 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 treat patients in Africa with sickle cell disease.
In their phase 1 trial, a Crispr treatment was inserted into a lipid nanoparticle, which was collected in the blood by the same tissue that takes the cholesterol globules and transported them to the liver. There, single treatment inactivated the TTR gene and reduced the problem protein by 87% in patients with the highest dose. On the 28th there were no serious side effects.
Julian Gillmore, a professor of medicine at University College London who was the lead researcher on the phase 1 trial, has treated patients with the type of amyloidosis for 25 years, but for two decades he was able to do little for them. For the past five years, he has been able to use gene silencers, but these treatments appear to be less effective and require regular infusions.
“From my own personal perspective, having seen these patients get worse for so many years, I have known families for years completely decimated by this disease, it’s amazing to see this revolution,” he said.
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