The first phase of the trial, which concluded in late October, included five boys aged 6 to 10, who were treated with a single injection of GNT0004. Two patients received the first dose, and three others were treated at the second dose level. The results show good tolerance of GNT0004 combined with transient immunological prophylaxis, as well as evidence of effectiveness, both in terms of microdystrophin expression and functional improvement.
Patients treated at the higher dose showed stabilization of NSAA (North Star Ambulatory Assessment, a measurement scale used in the trials) scores after one to two years of follow-up, compared to a decline in untreated patients in the study. parallel natural history of the disease. For one of the patients, improvement was observed, reaching the maximum score of 34 at 12 months and confirmed at 18 months after treatment.
At the second dose level, the results show that after eight weeks, up to 85% of muscle fibers expressed microdystrophin (average 54%; between 15% to 85% depending on the patient), with reconstitution of the complex of proteins associated with dystrophin. This expression is accompanied bya drop in the CPK level (a biomarker of muscular suffering) between 50% and 87% depending on the patients, a reduction which was maintained up to 18 months of follow-up for the first two patients.
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