First Phase 3 Trial of In Vivo CRISPR Therapy Completes
This is the first completed Phase 3 trial of an in vivo CRISPR therapy, providing the efficacy and safety data needed for regulatory review and potentially opening the door to treating other genetic diseases with direct gene editing.
Reporting from 1 sources: GIGAZINE.
A research team led by Amsterdam University Medical Center announced results from the first completed Phase 3 trial of an in vivo CRISPR therapy, targeting hereditary angioedema. The therapy, lonvoguran ziclumeran (lonvo-z), reduced swelling attack frequency by 87% compared to placebo in 80 patients. The findings were presented at the EAACI annual meeting and published in the New England Journal of Medicine.
The trial randomized 80 patients to receive either lonvo-z or placebo. From week 5 to week 28 after a single intravenous infusion, the lonvo-z group saw an 87% lower frequency of swelling attacks compared to placebo. The need for on-demand medication dropped by 89%, moderate to severe attacks fell by 91%, and quality of life measures improved. During the six-month evaluation, 62% of patients on lonvo-z had no attacks and required no continuous preventive treatment, versus 11% in the placebo group.
Dr. Danny Cohn of Amsterdam University Medical Center noted that patients tended to use medication early when they felt swelling, so it is uncertain whether all reported swelling was due to hereditary angioedema attacks. He added that if patients knew they received the active drug, they might feel more confident to withhold medication, potentially increasing the number judged attack-free. Common side effects included mild infusion-related reactions, headache, fatigue, and back pain. No serious health issues were reported after treatment. Data from earlier Phase 1 and 2 trials showed the effect and safety of lonvo-z were maintained up to four years after dosing.
Lonvo-z has not yet received regulatory approval. Dr. Cohn stated that these results provide the necessary data for regulators to decide whether to approve lonvo-z as a drug, and that the trial opens the door for applying in vivo CRISPR therapy to other genetic diseases.
Synthesized by Yomimono from the 1 cited source below, including Japanese-language reporting where cited, then editorially reviewed before publishing.
Sources
- GIGAZINE 体内で遺伝子を書き換えるCRISPR治療が初の第3相試験を完了