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Stargardt Disease

12
Pipeline Programs
10
Companies
16
Clinical Trials
6 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
7
2
1
2
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
250%
Small Molecule
250%
+ 9 programs with unclassified modality

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Kubota Vision
Kubota VisionJapan - Tokyo
2 programs
1
1
EmixustatPhase 3Small Molecule1 trial
EmixustatPhase 2Small Molecule1 trial
Active Trials
NCT03033108Completed23Est. Dec 2017
NCT03772665Completed194Est. Jun 2022
Alkeus Pharmaceuticals
1 program
1
ALK-001Phase 35 trials
Active Trials
NCT07419334Recruiting230Est. Dec 2030
NCT04239625Active Not Recruiting200Est. Dec 2026
NCT03845582Completed200Est. Jun 2024
+2 more trials
Ocugen
OcugenPA - Malvern
1 program
1
OCU410STPhase 2/31 trial
Active Trials
NCT05956626Active Not Recruiting51Est. Sep 2026
Nanoscope Therapeutics
2 programs
1
Gene Therapy-vMCO-010Phase 2Gene Therapy1 trial
Gene Therapy Product-MCO-010N/AGene Therapy1 trial
Active Trials
NCT06048185Active Not Recruiting6Est. Jul 2027
NCT05417126Completed6Est. Sep 2023
Biocorp
BiocorpFrance - Issoire
3 programs
3
AAVB-039Phase 1/2
SB-007Phase 1/21 trial
tinlarebantPhase 1/2
Active Trials
NCT06942572Recruiting57Est. Dec 2028
AAVantgarde Bio
AAVantgarde BioItaly - Milan
1 program
1
AAVB-039Phase 1/21 trial
Active Trials
NCT07161544Recruiting75Est. Jul 2032
Ascidian Therapeutics
1 program
1
ACDN-01Phase 1/22 trials
Active Trials
NCT06445322Recruiting50Est. Aug 2030
NCT06467344Recruiting15Est. Dec 2030
Ray Therapeutics
Ray TherapeuticsCA - Berkeley
1 program
1
RTx-021Phase 1/21 trial
Active Trials
NCT07439887Recruiting18Est. Dec 2030
Belite Bio
Belite BioSAN DIEGO, CA
1 program
1
tinlarebantPhase 1/21 trial
Active Trials
NCT05266014Completed13Est. Aug 2023

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Alkeus PharmaceuticalsALK-001
Kubota VisionEmixustat
OcugenOCU410ST
Alkeus PharmaceuticalsALK-001
Nanoscope TherapeuticsGene Therapy-vMCO-010
Alkeus PharmaceuticalsALK-001
Kubota VisionEmixustat
Alkeus PharmaceuticalsALK-001
Ray TherapeuticsRTx-021
AAVantgarde BioAAVB-039
BiocorpSB-007
Ascidian TherapeuticsACDN-01
Belite Biotinlarebant
Alkeus PharmaceuticalsALK-001
Ascidian TherapeuticsACDN-01

Showing 15 of 16 trials with date data

Clinical Trials (16)

Total enrollment: 1,338 patients across 16 trials

Study of ALK-001 on the Progression of Stargardt Disease

Start: Apr 2026Est. completion: Dec 2030230 patients
Phase 3Recruiting

Safety and Efficacy of Emixustat in Stargardt Disease

Start: Jan 2019Est. completion: Jun 2022194 patients
Phase 3Completed

A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease

Start: Aug 2023Est. completion: Sep 202651 patients
Phase 2/3Active Not Recruiting

Phase 2/3 Study of ALK-001 in Geographic Atrophy

Start: May 2019Est. completion: Jun 2024200 patients
Phase 2/3Completed

Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease

Start: Jul 2022Est. completion: Sep 20236 patients
Phase 2Completed

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

Start: Dec 2019Est. completion: Dec 2026200 patients
Phase 2Active Not Recruiting

Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease

Start: Jan 2017Est. completion: Dec 201723 patients
Phase 2Completed

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease

Start: Aug 2015Est. completion: Nov 2026160 patients
Phase 2Enrolling By Invitation

Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease

Start: Jan 2026Est. completion: Dec 203018 patients
Phase 1/2Recruiting

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

Start: Sep 2025Est. completion: Jul 203275 patients
Phase 1/2Recruiting

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

Start: Feb 2025Est. completion: Dec 202857 patients
Phase 1/2Recruiting

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

Start: Jun 2024Est. completion: Dec 203015 patients
Phase 1/2Recruiting

This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease

Start: Mar 2021Est. completion: Aug 202313 patients
Phase 1/2Completed

Phase 1 Safety Study of ALK-001 in Healthy Volunteers

Start: Apr 2014Est. completion: Feb 201540 patients
Phase 1Completed

Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)

Start: Jun 2024Est. completion: Aug 203050 patients
N/ARecruiting
NCT06048185Nanoscope TherapeuticsGene Therapy Product-MCO-010

Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study

Start: Oct 2023Est. completion: Jul 20276 patients
N/AActive Not Recruiting

Related Jobs

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
6 actively recruiting trials targeting 1,338 patients
10 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.