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Achromatopsia

3
Pipeline Programs
2
Companies
7
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
3
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
3100%
+ 5 programs with unclassified modality

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Beacon Therapeutics
Beacon TherapeuticsMA - Cambridge
4 programs
3
AGTC-402Phase 1/21 trial
rAAV2tYF-CB-hCNGA3Phase 1/2
rAAV2tYF-PR1.7-hCNGB3Phase 1/21 trial
Clinical and Genetic Characterization of Individuals With AchromatopsiaN/A1 trial
Active Trials
NCT01846052Completed56Est. Apr 2017
NCT02935517Active Not Recruiting24Est. Aug 2026
NCT02599922Active Not Recruiting32Est. Jul 2026
MeiraGTx
MeiraGTxNEW YORK, NY
4 programs
Ocular assessmentsN/A1 trial
Prior exposure to AAV-CNGA3 or AAV-CNGB3N/AGene Therapy1 trial
AAV - CNGB3PHASE_1_2Gene Therapy1 trial
adeno-associated virus vector AAV- CNGA3PHASE_1_2Gene Therapy1 trial
Active Trials
NCT04124185Completed85Est. May 2023
NCT03278873Terminated34Est. Apr 2024
NCT03001310Completed23Est. Oct 2019
+1 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
MeiraGTxadeno-associated virus vector AAV- CNGA3
Beacon TherapeuticsAGTC-402
MeiraGTxAAV - CNGB3
Beacon TherapeuticsrAAV2tYF-PR1.7-hCNGB3
MeiraGTxPrior exposure to AAV-CNGA3 or AAV-CNGB3
Beacon TherapeuticsClinical and Genetic Characterization of Individuals With Achromatopsia
MeiraGTxOcular assessments

Clinical Trials (7)

Total enrollment: 265 patients across 7 trials

NCT03758404MeiraGTxadeno-associated virus vector AAV- CNGA3

Gene Therapy for Achromatopsia (CNGA3)

Start: Aug 2019Est. completion: Jun 202111 patients
Phase 1/2Completed
NCT02935517Beacon TherapeuticsAGTC-402

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)

Start: Aug 2017Est. completion: Aug 202624 patients
Phase 1/2Active Not Recruiting
NCT03001310MeiraGTxAAV - CNGB3

Gene Therapy for Achromatopsia (CNGB3)

Start: Jan 2017Est. completion: Oct 201923 patients
Phase 1/2Completed
NCT02599922Beacon TherapeuticsrAAV2tYF-PR1.7-hCNGB3

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)

Start: Apr 2016Est. completion: Jul 202632 patients
Phase 1/2Active Not Recruiting
NCT03278873MeiraGTxPrior exposure to AAV-CNGA3 or AAV-CNGB3

Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3

Start: Jun 2017Est. completion: Apr 202434 patients
N/ATerminated
NCT01846052Beacon TherapeuticsClinical and Genetic Characterization of Individuals With Achromatopsia

Clinical and Genetic Characterization of Individuals With Achromatopsia

Start: Jun 2013Est. completion: Apr 201756 patients
N/ACompleted
NCT04124185MeiraGTxOcular assessments

Natural History Study for Achromatopsia

Start: Sep 2011Est. completion: May 202385 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

2 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.