en.Wedoany.com Reported - The research team has successfully completed the world's first Phase III trial of an in vivo CRISPR therapy, with results showing the treatment effectively reduces the number of attacks in patients with hereditary angioedema. This international double-blind trial, conducted by Amsterdam UMC and other institutions, involved 80 patients. Lead researcher Danny Cohn stated that the results confirm the therapy's efficacy and safety, and these data are key evidence required for regulatory approval of the first in vivo CRISPR gene-editing therapy for market authorization.
The study's findings were recently presented at the annual congress of the European Academy of Allergy and Clinical Immunology in Istanbul and simultaneously published in The New England Journal of Medicine.
The evaluated one-time CRISPR therapy targets hereditary angioedema, a rare disease characterized by recurrent and potentially life-threatening swelling. The study employed a large-scale, double-blind design, with patients randomly assigned to receive the CRISPR therapy, named lonvoguran-ziclumeran, or a placebo. The primary endpoint was the number of attacks from week 5 to week 28 after a single intravenous infusion. Data showed a relative reduction of 87% in attacks in the treatment group, with 62% of patients experiencing no attacks without any maintenance therapy, compared to 11% in the placebo group. Secondary endpoints also showed positive results: a 89% reduction in the need for on-demand treatment, a 91% reduction in moderate-to-severe attacks, and a significant improvement in patient quality-of-life scores compared to the placebo group.
The researchers noted that because trial participants tended to take medication at the earliest signs of potential swelling, some reported swelling may not have been actual attacks. Cohn believes that the number of patients completely free of attacks is expected to increase further once participants are aware they have received the active treatment.
For patients, this means a severe chronic disease could potentially be managed long-term with a single intervention. Patients may no longer require continuous preventive medication, thereby avoiding related side effects, reducing treatment burden and drug dependence, and alleviating anxiety about future attacks. In terms of safety, the therapy was well-tolerated, with common side effects including mild infusion-related reactions, headache, fatigue, and back pain, all of which resolved quickly. No serious adverse events were reported in the treatment group.
Cohn emphasized that data from 37 participants in Phase 1 and Phase 2 trials show the therapy remains effective and safe four years after administration. This progress opens the door for patients with other genetic diseases to receive in vivo CRISPR treatments.
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