Leber Congenital Amaurosis (LCA)

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

Drug Modality Breakdown

Gene Therapy
1100%

Competitive Landscape

1 companies ranked by most advanced pipeline stage

MeiraGTx
MeiraGTxNEW YORK, NY
1 program
AAV OPTIRPE65N/AGene Therapy1 trial
Active Trials
NCT02946879CompletedEst. Jul 2023

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
MeiraGTxAAV OPTIRPE65

Clinical Trials (1)

NCT02946879MeiraGTxAAV OPTIRPE65

Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

Start: Nov 2016Est. completion: Jul 2023
N/ACompleted

Phase Legend

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

Key Insights

1 companies competing in this space