A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

Phase 3 Active not recruiting 4 enrolled Janssen Pharmaceutical K.K.

Non-randomizedParallel-groupQuadruple-blindTreatment

Summary

The purpose of the study is to assess the safety and tolerability of bilateral subretinal delivery of adeno-associated virus vector with a serotype 5 capsid human rhodopsin kinase promoter. retinitis pigmentosa guanosine triphosphatase regulator (AAV5-hRKp.RPGR).

Timeline

Start: 2023-09-12
Primary completion: 2027-06-30
Completion: 2030-11-12

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	AAV5-hRKp.RPGR	Gene therapy (AAV / viral vector)	—	Other

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

Summary

Timeline

Publications

Drugs

Indications