A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

Phase 2 Active not recruiting 14 enrolled Beacon Therapeutics

RandomizedParallel-groupQuadruple-blindTreatment

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: 2021-04-13
Primary completion: 2023-04-11
Completion: 2027-02

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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	AGTC-501	Gene therapy (AAV / viral vector)	—	—

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

Summary

Timeline

Publications

Drugs

Indications