CRISPR-Based Gene Therapy Shows Promise in Curing Hereditary Angioedema

Nine out of ten patients in a trial experienced a significant reduction in swelling attacks, marking a potential breakthrough in the treatment of this life-threatening condition.

• CRISPR-based gene therapy has shown promising results in curing hereditary angioedema, a potentially life-threatening inflammatory condition, with nine out of ten patients in a trial experiencing a significant reduction in swelling attacks.
• The therapy, developed by Intellia Therapeutics, targets the kallikrein gene, which is responsible for producing a protein involved in inflammation. By editing this gene, the therapy effectively prevents angioedema attacks.
• The treatment was administered directly into the body, a method known as 'in vivo' delivery, which is simpler and quicker than other gene therapies that require cells to be taken out of the body, modified in the lab, and then reinfused.
• Patients from the UK, New Zealand, and the Netherlands who participated in the trial reported a significant improvement in their physical and mental wellbeing, with many able to return to normal life and come off long-term medication.
• Further trials are planned to assess the long-term safety and effectiveness of the therapy, and while the treatment has shown remarkable results, it is not expected to be available soon due to its high cost.

3 Articles

The Guardian New Scientist Evening Standard