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    LHON therapy: Excellent safety and potential visual benefits


    Dr. Sahel, who is also director of the Institut de la Vision, and chairman of the department of ophthalmology, Centre Hospitalier National d’Ophtalmologie des XV-XX Paris, presented the preliminary results of the Phase Ib/IIa.

    In the dose-escalation trial performed in one center, patients with chronic LHON were given one intravitreal injection in the eye with the worse visual acuity. Then they were followed for 48 weeks followed by an extension study lasting more than 4 years to evaluate the safety. The VA in all patients was worse than 20/200 in both eyes. The primary objective of the study was to determine the safety and tolerability of the vector and the bio-dissemination and immune responses. The secondary objective was to determine the effect on the VA and other visual function.

    Four doses were tested in five cohorts that included three patients in each cohort: 9E9, 3E10, 9E10 and 1,8E11 vg/eye, and the third dose was used during the extension cohort. Data safety monitoring was performed before each dose escalation.

    The mean patient age was 49.5 years; 86.6% were men. The mean age at disease onset was 42.3 years (range, 32-53 years); the average disease duration at treatment was 5.9 years (range, 0.62-22.2 years).

    At the time of this report, nine patients completed the follow-up evaluation at 48 weeks, according to Dr. Sahel.

    VA improvement

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