CRISPR gene therapy seems to cure dangerous inflammatory condition

Nine out of 10 people who received a new version of a CRISPR-based treatment for a potentially life-threatening inflammatory condition seem to have been cured

By Clare Wilson

31 January 2024

A new therapy makes cuts in the gene for kallikrein (shown as graphics), a protein involved in inflammation

BIOSYM TECHNOLOGIES, INC./SCIENCE PHOTO LIBRARY

Nine people with a rare genetic condition that causes life-threatening inflammatory reactions appear to have been cured, after taking part in the first trial of a new version of a CRISPR-based gene therapy.

The condition, called hereditary angioedema, causes people to have sudden episodes of tissue swelling that affects body parts such as the face or throat, similar to aspects of an allergic reaction, although they can’t be treated with anti-allergy medicines.

Ten people who had the one-off gene treatment that is given directly into the body saw their number of “swelling attacks” fall by 95 per cent in the first six months as the therapy took effect. Since then, all but one have had no further episodes for at least a further year, while one person who had the lowest dose of the treatment had one mild attack. “This is potentially a cure,” says Padmalal Gurugama at Cambridge University Hospitals in the UK, who worked on the new approach.

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Hereditary angioedema is caused by mutations in a gene that encodes a protein called C1-inhibitor, which is normally involved in damping down inflammation, part of the immune response.

People with the condition may have sudden episodes of fluid accumulation under their skin several times a month, which are painful and can suffocate them if their throat becomes blocked. The attacks can be triggered by viruses, changing hormone levels or stress.