Leber Congenital Amaurosis

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

Drug Modality Breakdown

Gene Therapy
1100%
+ 1 programs with unclassified modality

Competitive Landscape

2 companies ranked by most advanced pipeline stage

MeiraGTx
MeiraGTxNEW YORK, NY
1 program
1
AAV RPE65Phase 1/2Gene Therapy1 trial
Active Trials
NCT02781480CompletedEst. Dec 2018
Beacon Therapeutics
Beacon TherapeuticsMA - Cambridge
1 program
1
rAAV2-CB-hRPE65Phase 1/21 trial
Active Trials
NCT00749957CompletedEst. Sep 2017

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
MeiraGTxAAV RPE65
Beacon TherapeuticsrAAV2-CB-hRPE65

Clinical Trials (2)

NCT02781480MeiraGTxAAV RPE65

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

Start: Apr 2016Est. completion: Dec 2018
Phase 1/2Completed
NCT00749957Beacon TherapeuticsrAAV2-CB-hRPE65

Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

Start: Jun 2009Est. completion: Sep 2017
Phase 1/2Completed

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

Gene Therapy is the dominant modality (100% of programs)
2 companies competing in this space