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Results by Cambridge-based Intellia and Regeneron show how CRISPR could transform treatment

Cambridge-based Intellia and Regeneron said they have positive results from a study involving CRISPR research.

(Bloomberg) -- In a milestone for gene therapy, drugmakers Intellia Therapeutics Inc. and Regeneron Pharmaceuticals Inc. reported results from the first clinical trial using Nobel Prize-winning Crispr technology to treat disease inside the human body.

The study of six patients showed significant reductions in levels of a harmful liver protein associated with a genetic disease, the companies said Saturday. While small, the early-stage trial fuels hopes that the promise of Crispr may one day come true: to treat and perhaps cure genetic diseases by precisely editing human DNA.

“There’s a feeling like we’re walking through a door here into all kinds of new possibilities,” said John Leonard, Intellia’s chief executive officer, in a briefing before the results were published in the New England Journal of Medicine. “And there’s not many moments in medicine where you get to experience that.”


The results are an exciting sign of where Crispr therapies are headed, said Eric Topol, a geneticist and director of Scripps Research Translational Institute,

“We’re moving far faster with Crispr than anyone thought we would,” he said.

Discovered in bacteria in the early 1990s, Crispr is a mechanism for cutting and pasting specific sequences of DNA that won the Nobel Prize for two of its innovators last year. While it’s long been predicted to bring about a revolution in treatment, few Crispr-based therapies have made it into humans.

So far, most gene-editing therapies have involved removing cells from the body, manipulating the genes, and then replacing the cells. Some experimental therapies for blood disorders have corrected genes in patients’ cells and then transplanted them back into the body.

Other gene therapies, such as Spark Therapeutics Inc.’s Luxturna for eye disease, don’t edit genes; instead, they deliver a correct copy of the flawed DNA to cells, sometimes packaged in viruses. Such therapies have been marked by high prices and in some cases slow adoption. Editas Medicine Inc. is developing a Crispr-based therapy for a form of blindness that would work directly on genes in the body.


Still other scientists have experimented with Crispr in living people, but under ethically suspect circumstances. Chinese scientist He Jiankui used the technology to edit the genomes of twin female embryos in 2018, leading to a public outcry and beefed-up restrictions.

Delivered intravenously into the body, the Intellia/Regeneron therapy, called NTLA-2001, is aimed at using Crispr to inactivate a gene linked to a hereditary liver disease. The companies have designed the treatment to address a form of transthyretin amyloidosis, a rare and often fatal disorder that occurs when a gene called TTR causes abnormal proteins to build up in the body’s organs and tissues.

NTLA-2001 is designed to be a single-dose treatment that stops the formation of those protein deposits. In the companies’ study, protein levels dropped significantly -- by as much as 96% -- within a matter of weeks. No adverse events were observed in the first four weeks of the trial.

“There are things that we and others are doing with Crispr as a medicine that are much more likely to work because we’ve seen this work,” said David Lebwohl, Intellia’s chief medical officer.

While it’s too early to say anything conclusive about the treatment, reducing the level of disease-causing proteins translates to results for patients, said Julian Gillmore, a professor of medicine at the National Amyloidosis Centre at University College London and an investigator in the trial.


“The more you knock it down, the better the outcome,” he said in the briefing. “There is potential for very real clinical benefit for patients.”