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The US initiative has successfully treated first-time patients using a Crispr gene that directs the body’s internal organs to internal organs.
The first experiment from Intellia Therapeutics, developed by Nobel Prize winner Jennifer Doudna, shows the success of Crispr, showing scientists how to deal with problems that previously prevented the use of technology to transform cells outside the body or in the eyes.
Starting from Boston, working with technology company Regeneron, he cured transthyretin amyloidosis, a debilitating disease in which a combination of complex proteins beats a patient’s heart and nervous system, cutting back on their life expectancy.
John Leonard, head of Intellia, said he was “very excited” to see the positive results, which opened the door ongoing care the “micro-virus” of the disease for which Crispr’s drug was tested.
“The attraction is Crispr’s promise and the idea that you can change any color, anytime, anywhere in the genome, as long as you get there. And this last thought is the key, ”he said. “This is the first time Crispr has been introduced into a patient. . . and first to be able to fight genes successfully. ”
Crispr – which refers to the repeated recurrence of palindromic – is a method used by bacteria to protect themselves from viruses. In 2012, Doudna and her French colleague Emmanuelle Charpentier figured out how to do it use it as a genetic modification tool.
Shares in Intellia have increased by 233% since their first disclosure in 2016. The company is one of only three to receive initial certificates from its findings. The others are Crispr Therapeutics, which treats patients with sickle cell disease, and Editas Medicine, which are experimenting with other types of congenital skin.
Intellia is looking to replace bone marrow transplants without the use of blood transfusions, in addition to working with the Bill & Melinda Gates Foundation to treat patients in Africa with cholera.
In his phase 1 trial, Crispr treatment was injected into the lipid nanoparticle, which was taken from the blood by the same muscle that holds cholesterol globules, and carried to the liver. There, only one treatment resulted in the TTR gene reducing protein deficiency 87% in patients at high doses. There were no serious side effects on the 28th.
Julian Gillmore, a professor of medicine at University College London who was a senior researcher in the Phase 1 exam, has treated patients with amyloidosis for 25 years, but for 20 years there was little he could do for them. In the last five years, they have been able to use gene blockers – but the drugs do not seem to work and need to be injected frequently.
“In my opinion, seeing these patients get worse for many years, I’ve known families for years after being wiped out by the disease, it’s amazing to see this change,” he said.
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