Original ArticleEfficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials
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Financial Disclosure(s): The author(s) have made the following disclosure(s): A.M.M.: Grants – Spark Therapeutics, Inc, Foundation Fighting Blindness; Nonfinancial support – Center for Advanced Retinal and Ocular Therapeutics, University of Pennsylvania, FM Kirby Foundation; Provisional patent pending and a US patent – Spark Therapeutics, Inc (financial interest waived).1
S.R.: Grants – Spark Therapeutics, Inc (to his institution); Presentations – Spark Therapeutics, Inc.
J.A.W.: Former employee – Spark Therapeutics, Inc (previously with equity/options); Patent pending (primary end point measure) – Spark Therapeutics, Inc.
D.C.C.: Employee – Spark Therapeutics, Inc; Equity/options and patent pending (primary end point measure) – Spark Therapeutics, Inc.
Z.-F.Y., A.T., and J. Wittes: Employees – Statistics Collaborative, Inc, contracted to provide statistical and regulatory consulting to Center for Cellular and Molecular Therapeutics (CCMT)/Children’s Hospital of Philadelphia (CHOP) and Spark Therapeutics, Inc.
J.P. and O.E.: Employees – Westat, Inc (contracted by CCMT/CHOP and Spark Therapeutics, Inc)
K.A.M., D.C., and S.M.: Clinical coordinators and salary support – CCMT/CHOP, Spark Therapeutics, Inc.
S.M.: Patent pending – Spark Therapeutics, Inc.
J.Walshire, T.L.K., and M.D.: Salary support –Spark Therapeutics, Inc; Grants – Carver Center for Macular Degeneration, CHOP.
T.L.K.: Travel support – Spark Therapeutics, Inc.
F.S.: Grants – Regione Campania, CCMT; Serves on boards – Sanofi, Dompe Farmaceutici, Spark Therapeutics, Inc.
B.P.L.: Travel support and funding – Spark Therapeutics, Inc, through CCMT/CHOP for a research study, and Ghent University for consulting.
J.F.W.: Former employee – Spark Therapeutics, Inc; Patent and patent pending – Spark Therapeutics, Inc.
K.A.H.: Employee – Spark Therapeutics, Inc; Equity/options and patent pending (primary end point measure).
J.B.: Grants – Foundation Fighting Blindness, NIH, Spark Therapeutics, Inc; Nonfinancial support – Center for Advanced Retinal and Ocular Therapeutics, University of Pennsylvania, FM Kirby Foundation; Provisional patent pending (primary outcome measure) and US Patent – Spark Therapeutics, Inc (financial interest waived); Co-author on a copyrighted visual function questionnaire used in the phase 3 study.
B.P.L.: Senior Clinical Investigator of the Research Foundation – Flanders (Belgium) (FWO); Support – FWO Flanders Grant OZP 3G004306.
The phase 1 follow-on study and phase 3 study were initially funded by the Center for Cellular and Molecular Therapeutics (CCMT) at The Children's Hospital of Philadelphia (CHOP); sponsorship was then transferred to Spark Therapeutics, Inc (Philadelphia, PA). Funding also provided through the following grants: Foundation Fighting Blindness-sponsored CHOP-Penn Pediatric Center for Retinal Degenerations, National Eye Institute/National Institutes of Health (NIH) (grant no. 8DP1EY023177), Clinical Translational Science Award NIH/National Center for Research Resources (grant no. UL1-RR-024134), NIH/National Center for Advancing Translational Sciences (grant nos. UL1TR000003 and UL1TR001878), Research to Prevent Blindness, the Macula Vision Foundation, the Paul and Evanina Mackall Foundation Trust, the Center for Advanced Retinal and Ocular Therapeutics, the F. M. Kirby Foundation, grants from the National Center for Research Resources, and the Howard Hughes Medical Institute. The sponsors for both the phase 1 follow-on study and the phase 3 study participated in the design of the study, oversight of the study, interpretation of the data, and preparation, review, or approval of the manuscript. In addition to participating in conduct of the trial, the clinical investigators, along with their collaborators and scientists from Spark Therapeutics, Inc., participated in the preparation, review, and approval of the manuscript.
HUMAN SUBJECTS: Human subjects were included in this study. Each of the reported trials received Institutional Review Board/Ethics Committee approval, which adhered to the tenets of the Declaration of Helsinki. All subjects or their parents/legal guardians gave informed consent, and all related work was Health Insurance Portability and Accountability Act compliant.
No animal subjects were used in this study.
Author Contributions:
Conception and design: Maguire, Wellman, Chung, Wittes, Wright, High, Bennett
Data collection: Maguire, Russell, Chung, Wittes, Marshall, McCague, Reichert, Davis, Simonelli, Leroy, Wright, High, Bennett
Analysis and interpretation: Maguire, Russell, Wellman, Chung, Yu, Pappas, Elci, Leroy, Wright, High, Bennett, Wright
Obtained funding: Maguire, Russell, Wellman, Simonelli, High, Bennett
Overall responsibility: Maguire, Russell, Wellman, Chung, Yu, Tillman, Wittes, Pappas, Simonelli, Leroy, Wright, High, Bennett