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

Phase 2 Enrolling by invitation 24 enrolled Beacon Therapeutics

Non-randomizedSequentialOpen-labelTreatment

Summary

This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.

Timeline

Start: 2023-11-14
Primary completion: 2025-11
Completion: 2029-12

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)	—	Other

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

Summary

Timeline

Publications

Drugs

Indications