In a national first, the Food and Drug Administration has given Intellia Therapeutics the go-ahead to begin testing a drug that uses CRISPR gene editing in vivo.
In biology, in vivo means within an organism, rather than in something like a petri dish, and Intellia’s offering is the first time ever that the FDA has approved such testing.
Their drug would prevent swelling attacks in people with a genetic condition called hereditary angioedema.
Typically, treatments and drugs that utilize CRISPR take place outside the body. Cells or tissues are removed and altered ex vivo before being re-introduced inside the patient. In the case of Intellia’s drug, the edited media finds its own way to the liver rather than being injected there.
The advantages are huge if such a drug could be proven to work well—a lack of hospital and laboratory procedures would save a patient thousands, and potentially open up the class of drugs to the lower and middle classes, or to those who are uninsured.
“This is an important milestone for Intellia as it is the first-ever (investigational new drug application) cleared by the FDA for in-vivo gene editing,” RBC Capital Markets analyst Luca Issi said in a report on Inetllia’s stock, which rose following the announcement.
The company plans to file the papers for another such drug later in the year, which would help tamp down on an abnormal protein that builds up in the heart.
Other Western countries have already approved several and even many in vivo CRISPR treatments for testing, among which are New Zealand, The Netherlands, Sweden, the UK, and France.
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