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Leber's Congenital Amaurosis

1
Pipeline Programs
2
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
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Biocorp
BiocorpFrance - Issoire
1 program
1
QR-110Phase 1/21 trial
Active Trials
NCT03140969Completed11Est. Oct 2019
Thea Pharma
Thea PharmaMA - Waltham
1 program
QR-110PHASE_1_2

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
BiocorpQR-110

Clinical Trials (1)

Total enrollment: 11 patients across 1 trials

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Start: Oct 2017Est. completion: Oct 201911 patients
Phase 1/2Completed

Phase Legend

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