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Choroideremia

3
Pipeline Programs
4
Companies
9
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
1
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

4 companies ranked by most advanced pipeline stage

SPARK Biopharma
SPARK BiopharmaKorea - Seoul
1 program
1
AAV2-hCHMPhase 1/21 trial
Active Trials
NCT02341807Completed15Est. Oct 2022
Innovation Pharmaceuticals
1 program
1
rAAV2.REP1 vectorPhase 1/21 trial
Active Trials
NCT02077361Completed6Est. May 2022
4D Molecular Therapeutics
2 programs
1
4D-110Phase 11 trial
ObservationN/A2 trials
Active Trials
NCT04517149Active Not Recruiting21Est. May 2029
NCT02994368Terminated57Est. Oct 2021
NCT04483440Active Not Recruiting13Est. Aug 2027
Biogen
BiogenCAMBRIDGE, MA
2 programs
Natural History of the Progression of Choroideremia StudyN/A1 trial
BIIB111PHASE_33 trials
Active Trials
NCT03359551Completed319Est. Oct 2020
NCT03584165Enrolling By Invitation330Est. Jun 2026
NCT03496012Completed169Est. Dec 2020
+1 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
BiogenBIIB111
BiogenBIIB111
BiogenBIIB111
4D Molecular TherapeuticsObservation
Innovation PharmaceuticalsrAAV2.REP1 vector
SPARK BiopharmaAAV2-hCHM
4D Molecular Therapeutics4D-110
4D Molecular TherapeuticsObservation
BiogenNatural History of the Progression of Choroideremia Study

Clinical Trials (9)

Total enrollment: 996 patients across 9 trials

NCT03584165BiogenBIIB111

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa

Start: Jun 2018Est. completion: Jun 2026330 patients
Phase 3Enrolling By Invitation
NCT03496012BiogenBIIB111

Efficacy and Safety of BIIB111 for the Treatment of Choroideremia

Start: Dec 2017Est. completion: Dec 2020169 patients
Phase 3Completed
NCT03507686BiogenBIIB111

A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111

Start: Nov 2017Est. completion: Jun 202266 patients
Phase 2Completed
NCT045171494D Molecular TherapeuticsObservation

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

Start: Jun 2020Est. completion: May 202921 patients
Phase 1/2Active Not Recruiting
NCT02077361Innovation PharmaceuticalsrAAV2.REP1 vector

An Open Label Clinical Trial of Retinal Gene Therapy for Choroideremia

Start: Apr 2015Est. completion: May 20226 patients
Phase 1/2Completed
NCT02341807SPARK BiopharmaAAV2-hCHM

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

Start: Jan 2015Est. completion: Oct 202215 patients
Phase 1/2Completed
NCT044834404D Molecular Therapeutics4D-110

Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

Start: Jun 2020Est. completion: Aug 202713 patients
Phase 1Active Not Recruiting
NCT029943684D Molecular TherapeuticsObservation

"Natural History" Study of Choroideremia

Start: Dec 2016Est. completion: Oct 202157 patients
N/ATerminated
NCT03359551BiogenNatural History of the Progression of Choroideremia Study

Natural History of the Progression of Choroideremia Study

Start: Jun 2015Est. completion: Oct 2020319 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

4 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.