Sarah McCague, MS

female silhouette icon
Sarah McCague, MS, is the Administrative Manager of Clinical In Vivo Gene Therapy at Children's Hospital of Philadelphia.

Appointments and Referrals: 1-800-TRY-CHOP (1-800-879-2467)

Education and Training

Undergraduate Degree

BA - Middlebury College, Middlebury, VT

Graduate Degree

MS in Clinical Research Organization and Management - Drexel University College of Medicine, Philadelphia, PA

Titles and Academic Titles

Administrative Manager

Departments and Services




Chung, D.C., Bertelsen, M., Lorenz, B., Pennesi, M.E., Leroy, B.P., Hamel, C.P., Pierce, E., Sallum, J., Larsen, M., Stieger, K., Preising, M., Weleber, R., Yang, P., Place, E., Liu, E., Schaefer, G., DiStefano-Pappas, J., Elci, O.U., McCague, S., Wellman, J.A., High K.A., Reape K.Z. (2019) The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene. Am J Ophthalmol. 199, 58-70.

Maguire, A.M., Russell, S., Wellman, J.A., Chung, D.C., Yu, Z.F., Tillman, A., Wittes, J., Pappas, J., Elci, O., Marshall, K.A., McCague, S.,… Bennett, J. (2019) Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials. Ophthalmology, 126(9), 1273-1285.


Chung, D.C., McCague, S., Yu, Z-F., Thill, S., DiStefano-Pappas, J., Bennett, J., … High, K.A. (2017) Novel mobility test to assess functional vision in patients with inherited retinal dystrophies. Clinical & Experimental Ophthalmology, 2017 August 31.

Russell, S., Bennett, J., Wellman, J.A., Chung, D.C., Yu, Z-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., McCague, S., … Maguire, A.M. (2017). Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: A randomised, controlled, open-label, phase 3 trial. The Lancet, 390(10097), 849-860.


Bennett, J., Wellman, J., Marshall, K.A., McCague, S., Ashtari, M., DiStefano-Pappas, J., … Maguire, A.M. (2016). 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. The Lancet, 388(10045), 661-672.


McCague, S., Wellman, J., Yu, F., Thill, S., DiStefano-Pappas, J., Bennett, J., … Chung, D.C. Mobility testing validation study – Using a novel, standardized mobility test to evaluate functional vision in patients with inherited retinal degeneration. Poster presented at: Association for Research in Vision and Ophthalmology Annual Meeting; 2015 May 3-7; Denver, CO.


Bennett, J., Ashtari, M., Wellman, J., Marshall, K.A., Cyckowski, L.L., Chung, D.C., McCague, S., … Maguire, A.M. (2012). AAV2 gene therapy readministration in three adults with congenital blindness. Science Translational Medicine, 4(120), 120ra15.

Awards and Honors

2018, Academic Excellence Award, Drexel University College of Medicine