Achromatopsia
4
Pipeline Programs
2
Companies
5
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
4
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Gene Therapy
3100%
+ 4 programs with unclassified modality
Competitive Landscape
2 companies ranked by most advanced pipeline stage
MeiraGTxNEW YORK, NY
4 programs2
AAV - CNGB3Phase 1/2Gene Therapy
adeno-associated virus vector AAV- CNGA3Phase 1/2Gene Therapy
Ocular assessmentsN/A1 trial
Prior exposure to AAV-CNGA3 or AAV-CNGB3N/AGene Therapy1 trial
Active Trials
Beacon TherapeuticsMA - Cambridge
3 programs2
AGTC-402Phase 1/21 trial
rAAV2tYF-PR1.7-hCNGB3Phase 1/21 trial
Clinical and Genetic Characterization of Individuals With AchromatopsiaN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Beacon TherapeuticsAGTC-402
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 (5)
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)
Start: Aug 2017Est. completion: Aug 2026
Phase 1/2Active Not Recruiting
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)
Start: Apr 2016Est. completion: Jul 2026
Phase 1/2Active Not Recruiting
Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
Start: Jun 2017Est. completion: Apr 2024
N/ATerminated
NCT01846052Beacon TherapeuticsClinical and Genetic Characterization of Individuals With Achromatopsia
Clinical and Genetic Characterization of Individuals With Achromatopsia
Start: Jun 2013Est. completion: Apr 2017
N/ACompleted
Natural History Study for Achromatopsia
Start: Sep 2011Est. completion: May 2023
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