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X-Linked Retinitis Pigmentosa

9
Pipeline Programs
6
Companies
12
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

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

Drug Modality Breakdown

Gene Therapy
1100%
+ 12 programs with unclassified modality

Competitive Landscape

6 companies ranked by most advanced pipeline stage

MeiraGTx
MeiraGTxNEW YORK, NY
4 programs
1
1
Genetic: AAV5-hRKp.RPGRPhase 31 trial
AAV5-hRKp.RPGRPhase 21 trial
AAV5-hRKp.RPGRN/A2 trials
AAV5-RPGRPHASE_1_21 trial
Active Trials
NCT04794101Active Not Recruiting97Est. Dec 2029
NCT04312672Completed42Est. Sep 2025
NCT03252847Completed49Est. Nov 2021
+2 more trials
Beacon Therapeutics
Beacon TherapeuticsMA - Cambridge
5 programs
2
2
rAAV2tYF-GRK1-RPGRPhase 2/31 trial
rAAV2tYF-GRK1-hRPGRcoPhase 2/31 trial
AGTC-501Phase 22 trials
Adeno-associated virus vector expressing human RPGR genePhase 2Gene Therapy
Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)N/A1 trial
Active Trials
NCT03314207Completed14Est. Feb 2022
NCT06275620Enrolling By Invitation24Est. Dec 2029
NCT06333249Active Not Recruiting14Est. Feb 2027
+2 more trials
4D Molecular Therapeutics
1 program
1
4D-125Phase 1/2
Frontera Therapeutics
Frontera TherapeuticsChina - Suzhou
1 program
1
FT-002Phase 11 trial
Active Trials
NCT05874310Recruiting18Est. Nov 2027
UNION therapeutics
UNION therapeuticsDenmark - Hellerup
1 program
1
FT-002Phase 1
Biogen
BiogenCAMBRIDGE, MA
1 program
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
2030
2031
MeiraGTxAAV5-hRKp.RPGR
MeiraGTxGenetic: AAV5-hRKp.RPGR
Beacon TherapeuticsrAAV2tYF-GRK1-hRPGRco
MeiraGTxAAV5-hRKp.RPGR
Beacon TherapeuticsAGTC-501
Beacon TherapeuticsAGTC-501
Beacon TherapeuticsrAAV2tYF-GRK1-RPGR
MeiraGTxAAV5-RPGR
BiogenBIIB112
Frontera TherapeuticsFT-002
Beacon TherapeuticsClinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
MeiraGTxAAV5-hRKp.RPGR

Clinical Trials (12)

Total enrollment: 551 patients across 12 trials

NCT04794101MeiraGTxAAV5-hRKp.RPGR

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Start: Dec 2020Est. completion: Dec 202997 patients
Phase 3Active Not Recruiting
NCT04671433MeiraGTxGenetic: AAV5-hRKp.RPGR

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Start: Dec 2020Est. completion: Sep 2024105 patients
Phase 3Completed
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 203185 patients
Phase 2/3Active Not Recruiting
NCT06646289MeiraGTxAAV5-hRKp.RPGR

A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)

Start: Oct 2024Est. completion: Oct 203024 patients
Phase 2Active 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 202924 patients
Phase 2Enrolling By Invitation
NCT06333249Beacon TherapeuticsAGTC-501

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 202714 patients
Phase 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 202529 patients
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 202149 patients
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
NCT05874310Frontera TherapeuticsFT-002

Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa

Start: Feb 2023Est. completion: Nov 202718 patients
Phase 1Recruiting
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 202214 patients
N/ACompleted
NCT04312672MeiraGTxAAV5-hRKp.RPGR

Long-term Follow-up Gene Therapy Study for RPGR- XLRP

Start: Jul 2017Est. completion: Sep 202542 patients
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

1 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 551 patients
6 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.