Leber Congenital Amaurosis 10
2
Pipeline Programs
1
Companies
2
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
0
1
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
1100%
+ 1 programs with unclassified modality
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Thea PharmaMA - Waltham
2 programs1
1
sepofarsenPhase 3RNA Therapeutic1 trial
QR-110Phase 1/21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
Thea Pharmasepofarsen
Thea PharmaQR-110
Clinical Trials (2)
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Start: Jun 2025Est. completion: Oct 2028
Phase 3Recruiting
Extension Study to Study PQ-110-001 (NCT03140969)
Start: May 2019Est. completion: Oct 2022
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
1 companies competing in this space