Choroideremia

2
Pipeline Programs
2
Companies
6
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
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Biogen
BiogenCAMBRIDGE, MA
2 programs
1
BIIB111Phase 33 trials
Natural History of the Progression of Choroideremia StudyN/A1 trial
Active Trials
NCT03359551Completed319Est. Oct 2020
NCT03584165Enrolling By Invitation330Est. Jun 2026
NCT03496012Completed169Est. Dec 2020
+1 more trials
4D Molecular Therapeutics
2 programs
1
4D-110Phase 11 trial
ObservationN/A1 trial
Active Trials
NCT02994368Terminated57Est. Oct 2021
NCT04483440Active Not Recruiting13Est. Aug 2027

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
BiogenBIIB111
BiogenBIIB111
BiogenBIIB111
4D Molecular Therapeutics4D-110
4D Molecular TherapeuticsObservation
BiogenNatural History of the Progression of Choroideremia Study

Clinical Trials (6)

Total enrollment: 954 patients across 6 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
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

1 late-stage (Phase 3) programs — potential near-term approvals
2 companies competing in this space