12
Participants
Start Date
September 30, 2007
Primary Completion Date
March 20, 2018
Study Completion Date
March 20, 2018
voretigene neparvovec-rzyl
Subjects will be dosed unilaterally (one eye) beginning with the lowest dose. Subjects will be injected with AAV2-hRPE65v2 by means of a subretinal injection. Dose escalation to the next cohort will be dependent on assessment of the safety data by the DSMB out to at least 4 weeks following the injection. Because there is a delay between time of delivery of AAV2 and the peak transgene expression there will be a delay of six weeks between all subjects.
The Children's Hospital of Philadelphia, Philadelphia
Spark Therapeutics, Inc.
INDUSTRY