Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

Summary

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Timeline

Start

2018-04-16

Primary completion

2023-11-09

Completion

2025-03

Publications

• Wang CY, Chen L, Lin TY, Huang SP. Systematic Identification of Candidate Genes for Inherited Retinal Disease Gene Therapy Integrating Worldwide IRD Cohort and Single-Cell Analysis. J Ophthalmol. 2025 Jun 12;2025:7014745. doi: 10.1155/joph/7014745. eCollection 2025.
• Alibhai AY, De Pretto LR, Yaghy A, Woo KM, Dos Santos Xilau NR, Siddiqui H, Pandiscio CA, Homer A, Curtiss D, Waheed NK. Comparing the Thin Plate Spline and Gaussian Interpolation Methods in Generating Hill of Visions for X-Linked Retinitis Pigmentosa Patients. Transl Vis Sci Technol. 2024 Dec 2;13(12):26. doi: 10.1167/tvst.13.12.26.

Drugs

Indications