Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Bennett, Jean; Wellman, Jennifer; Marshall, Kathleen A; Mccague, Sarah; Ashtari, Manzar; DiStefano Pappas, Julie; Elci, Okan U; Chung, Daniel C; Sun, Junwei; Wright, J. Fraser; Cross, Dominique R; Aravand, Puya; Cyckowski, Laura L; Bennicelli, Jeannette L; Mingozzi, Federico; Auricchio, Alberto; Pierce, Eric A; Ruggiero, Jason; Leroy, Bart P; Simonelli, Francesca; High, Katherine A; Maguire, Albert M.

doi:10.1016/S0140-6736(16)30371-3

Safety and efficacy have been shown in a phase 1 dose-escalation study involving a unilateral subretinal injection of a recombinant adeno-associated virus (AAV) vector containing the RPE65 gene (AAV2-hRPE65v2) in individuals with inherited retinal dystrophy caused by RPE65 mutations. This finding, along with the bilateral nature of the disease and intended use in treatment, prompted us to determine the safety of administration of AAV2-hRPE65v2 to the contralateral eye in patients enrolled in the phase 1 study.

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial / Bennett, Jean; Wellman, Jennifer; Marshall, Kathleen A; Mccague, Sarah; Ashtari, Manzar; DiStefano Pappas, Julie; Elci, Okan U; Chung, Daniel C; Sun, Junwei; Wright, J. Fraser; Cross, Dominique R; Aravand, Puya; Cyckowski, Laura L; Bennicelli, Jeannette L; Mingozzi, Federico; Auricchio, Alberto; Pierce, Eric A; Ruggiero, Jason; Leroy, Bart P; Simonelli, Francesca; High, Katherine A; Maguire, Albert M.. - In: THE LANCET. - ISSN 0140-6736. - 388:10045(2016), pp. 661-72-672. [10.1016/S0140-6736(16)30371-3]