Startup companies in the US Successfully treating the first patient group using Crispr gene-editing therapies directed in vivo to internal organs.
Early experimental data from Intellia Therapeutics, founded by Nobel Prize winner Jennifer Doudna. It’s a major breakthrough for Crispr-based therapies, demonstrating that scientists have overcome challenges that previously restricted the use of technology to modify cells outside the body or in the eye.
Boston startups Working with biotechnology company Regeneron, it treats transthyretin amyloidosis, a serious disease in which problematic protein buildup affects the patient̵7;s heart and nervous system. shorten life expectancy
Intellia Chief Executive John Leonard said he was “extremely pleased” to see the positive results. which opens the door to treatment that goes beyond “Small subgroups” of diseases for which Crispr treatment has been tried.
“The allure and promise of Crispr is the idea that you can change any gene, anywhere in the genome. As long as you can get there And that last requirement is an important one,” he said. “This is the first time Crispr has been infected into a patient . . . and the first time we have been able to successfully target the gene.”
Crispr – which stands for cluster short repetitive palindromic – is a system used by bacteria to protect themselves from viruses. In 2012, Doudna and her French colleague Emmanuelle Charpentier discovered how to use the tool as an editor. gene
Shares in Intellia are up 233% since going public in 2016. The company is one of only three with original discovery patents, others include Crispr Therapeutics, which treats sickle cell disease patients, and Editas Medicine, which is in trials to treat sickle cell disease. Treating Inherited Blindness
Intellia is looking to fix bone marrow to cure blood diseases without cell transplants. including working with the Bill & Melinda Gates Foundation to treat patients in Africa with sickle cell disease.
In a phase I trial, Crispr therapy was inserted into lipid nanoparticles. which is collected in the blood by the same tissue that clots cholesterol and transports it to the liver. One-time treatment inactivated the TTR gene and reduced the problem protein by 87 percent in patients receiving the highest dose. There were no serious side effects on day 28.
Julian Gillmore, a professor of medicine at University College London who was lead investigator in the Phase I trial, has treated patients with amyloidosis for 25 years, but for the past two decades he has done little to them. only in the last five years He could use Gene’s silencer. But these treatments appear to be less effective and require regular dosing.
“From my own personal perspective Years of watching these patients worsened. I have known families who have been completely ravaged by this disease for years. It is incredible to see this revolution,” he said.
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