X-Linked Retinitis Pigmentosa

7
Pipeline Programs
4
Companies
8
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
4
2
1
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

4 companies ranked by most advanced pipeline stage

Beacon Therapeutics
Beacon TherapeuticsMA - Cambridge
5 programs
1
2
1
rAAV2tYF-GRK1-hRPGRcoPhase 2/31 trial
AGTC-501Phase 21 trial
rAAV2tYF-GRK1-RPGRPhase 21 trial
rAAV2tYF-GRK1-RPGRPhase 1/21 trial
Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)N/A1 trial
Active Trials
NCT03314207CompletedEst. Feb 2022
NCT03316560Active Not RecruitingEst. Mar 2025
NCT06275620Enrolling By InvitationEst. Dec 2029
+2 more trials
4D Molecular Therapeutics
1 program
1
4D-125 IVT InjectionPhase 1/21 trial
Active Trials
NCT04517149Active Not Recruiting21Est. May 2029
MeiraGTx
MeiraGTxNEW YORK, NY
1 program
1
AAV5-RPGRPhase 1/21 trial
Active Trials
NCT03252847CompletedEst. Nov 2021
Biogen
BiogenCAMBRIDGE, MA
1 program
1
BIIB112Phase 1/21 trial
Active Trials
NCT03116113Completed50Est. Nov 2020

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
Beacon TherapeuticsrAAV2tYF-GRK1-hRPGRco
Beacon TherapeuticsAGTC-501
Beacon TherapeuticsrAAV2tYF-GRK1-RPGR
4D Molecular Therapeutics4D-125 IVT Injection
Beacon TherapeuticsrAAV2tYF-GRK1-RPGR
MeiraGTxAAV5-RPGR
BiogenBIIB112
Beacon TherapeuticsClinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)

Clinical Trials (8)

Total enrollment: 71 patients across 8 trials

NCT04850118Beacon TherapeuticsrAAV2tYF-GRK1-hRPGRco

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

Start: Mar 2024Est. completion: Oct 2029
Phase 2/3Active Not Recruiting
NCT06275620Beacon TherapeuticsAGTC-501

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

Start: Nov 2023Est. completion: Dec 2029
Phase 2Enrolling By Invitation
NCT06333249Beacon TherapeuticsrAAV2tYF-GRK1-RPGR

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

Start: Apr 2021Est. completion: Feb 2027
Phase 2Active Not Recruiting
NCT045171494D Molecular Therapeutics4D-125 IVT Injection

4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)

Start: Jun 2020Est. completion: May 202921 patients
Phase 1/2Active Not Recruiting
NCT03316560Beacon TherapeuticsrAAV2tYF-GRK1-RPGR

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

Start: Apr 2018Est. completion: Mar 2025
Phase 1/2Active Not Recruiting
NCT03252847MeiraGTxAAV5-RPGR

Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)

Start: Jul 2017Est. completion: Nov 2021
Phase 1/2Completed
NCT03116113BiogenBIIB112

A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112

Start: Mar 2017Est. completion: Nov 202050 patients
Phase 1/2Completed
NCT03314207Beacon TherapeuticsClinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)

Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)

Start: Dec 2017Est. completion: Feb 2022
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

4 companies competing in this space