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Leber Congenital Amaurosis 10

4
Pipeline Programs
3
Companies
4
Clinical Trials
1 recruiting
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
2
1
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
4100%
+ 2 programs with unclassified modality

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Biocorp
BiocorpFrance - Issoire
2 programs
1
1
sepofarsenPhase 3RNA Therapeutic1 trial
QR-110Phase 1/21 trial
Active Trials
NCT03913130Terminated9Est. Oct 2022
NCT06891443Recruiting32Est. Oct 2028
ProQR Therapeutics
ProQR TherapeuticsMA - Cambridge
2 programs
2
sepofarsenPhase 2/3RNA Therapeutic1 trial
sepofarsenPhase 2/3RNA Therapeutic1 trial
Active Trials
NCT04855045Unknown15Est. Dec 2023
NCT03913143Active Not Recruiting36Est. Mar 2023
Thea Pharma
Thea PharmaMA - Waltham
2 programs
QR-110PHASE_1_2
sepofarsenPHASE_3RNA Therapeutic

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
Biocorpsepofarsen
ProQR Therapeuticssepofarsen
ProQR Therapeuticssepofarsen
BiocorpQR-110

Clinical Trials (4)

Total enrollment: 92 patients across 4 trials

NCT06891443Biocorpsepofarsen

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Start: Jun 2025Est. completion: Oct 202832 patients
Phase 3Recruiting
NCT04855045ProQR Therapeuticssepofarsen

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

Start: Mar 2021Est. completion: Dec 202315 patients
Phase 2/3Unknown
NCT03913143ProQR Therapeuticssepofarsen

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Start: Apr 2019Est. completion: Mar 202336 patients
Phase 2/3Active Not Recruiting
NCT03913130BiocorpQR-110

Extension Study to Study PQ-110-001 (NCT03140969)

Start: May 2019Est. completion: Oct 20229 patients
Phase 1/2Terminated

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

1 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 92 patients
RNA Therapeutic is the dominant modality (100% of programs)
3 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.