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Novel CRISPR therapy is poised to gain first FDA approval to alter genes inside the body
Heidi Ledford reports for Nature from Seattle, Washington.
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The Cas9 protein together with RNA and DNA (orange; artist’s illustration). A new therapy uses the CRISPR-Cas9 gene editing system to treat a rare genetic disease called hereditary angioedema.Credit: Alfred Pasieka/Science Photo Library
A pioneering therapy that edits genes inside a recipient’s body continues to be effective four years after treatment, according to data released by its developer, Intellia Therapeutics.
The therapy, named lonvoguran ziclumeran (lonvo-z), was designed to treat one form of a genetic disorder that causes episodes of painful and potentially dangerous swelling in the throat and other parts of the body. Lonvo-z deploys the CRISPR-Cas9 gene-editing system to disable the gene that produces prekallikrein, a protein made in the liver that promotes blood-vessel leakage.
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Intellia, headquartered in Cambridge, Massachusetts, has applied for approval of lonvo-z to the US Food and Drug Administration (FDA), which is expected to issue a decision by 10 March. If approved, the therapy will become the world’s first CRISPR medicine to be delivered directly inside the body, or in vivo, John Leonard, Intellia’s chief executive, told investors on 12 November. Currently, the only CRISPR therapy approved by the FDA is performed on cells that have first been removed from the body and are subsequently re-infused.
For people with the condition, hereditary angioedema, the gene-editing approach could offer relief from a lifetime of juggling medications to prevent and treat swelling attacks, says Jonathan Bernstein, a hereditary angioedema specialist at the University of Cincinnati in Ohio and an investigator on a lonvo-z trial. “It’s very reassuring,” he says of the four-year data. “That’s what you’re supposed to see.”
The only approved CRISPR therapy is Casgevy, a treatment for two genetic blood disorders, sickle cell anemia and ß-thalassemia. To create a dose of Casgevy, physicians first remove blood stem cells from the recipient, then send them off for gene editing. The edited cells are infused back into the recipient.
The therapy must be made anew for each person, adding time, cost, and complexity. The recipient also has to undergo harsh chemotherapy treatment to make room for the edited cells.