A US startup has successfully treated early patients with Crispr gene-editing therapy directed in the body to an internal organ.
Early trial data from Intellia Therapeutics, co-founded by the Nobel laureate Jennifer Doudna, represents a penetration of CRISPR-based therapies, showing that scientists have overcome challenges that previously restricted the use of technology to modifying cells outside the body or in the eye.
The Boston-based startup, working with biotech company Regeneron, has treated transtretin amyloidosis, a devastating disease in which a buildup of a protein causes problems with a patient’s heart and nervous system, reducing life expectancy.
Intellia CEO John Leonard said he was “extremely pleased” to see the positive results, which opened the door to الباب Post-treatments The ‘small subset’ of diseases for which CRISPR-based therapies have been tried.
“The allure and promise of Crispr is the idea that you can alter any gene, anyway, anywhere in the genome, as long as you can get it there. And that last condition is the prerequisite.” “This is the first time CRISPR has been injected into a patient . . . and the first time we have been able to successfully target a gene.”
Crispr — which stands for symmetrically spaced short repeats — is a system that bacteria use to protect themselves from viruses. In 2012, Doudna and fellow Frenchwoman Emmanuel Charpentier discovered how to do it Use it as a tool for gene editing.
Shares in Intellia are up 233 percent since it went public in 2016. The company is one of three to hold original patents on discoveries. the others CRISPR treatments, which has treated patients with sickle cell anemia, and Editas Medicine, which is conducting trials to treat a form of genetic blindness.
Intellia is looking to modify bone marrow to treat blood-based diseases without cell transplants, including working with the Bill & Melinda Gates Foundation to treat patients in Africa with sickle cell disease.
In the first phase of the experiment, Crispr treatment was inserted into a lipid nanoparticle, which was captured in the blood by the same tissue that holds the cholesterol globules, and transported to the liver. There, the one-time treatment inactivated the TTR gene and reduced the problematic protein by 87 percent in patients receiving the highest dose. There were no serious side effects by day 28.
Julian Gilmore, a professor of medicine at University College London who was the lead researcher in the first phase of the trial, treated patients with amyloidosis for 25 years, but over two decades there was little he could do for them. In the past five years, he’s been able to use genetic silencers – but these treatments have appeared to be less effective and require regular injections.
“From my personal perspective, watching these patients get worse for so many years, I’ve known for years that families are completely decimated by this disease, it’s amazing to see this revolution,” he said.
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