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Leber Congenital Amaurosis

7
Pipeline Programs
6
Companies
7
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
2
5
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
3100%
+ 5 programs with unclassified modality

Competitive Landscape

6 companies ranked by most advanced pipeline stage

HuidaGene Therapeutics
HuidaGene TherapeuticsChina - Shanghai
2 programs
1
1
HG004Phase 1/21 trial
HG004Phase 11 trial
Active Trials
NCT06088992Active Not Recruiting9Est. Oct 2028
NCT05906953Recruiting20Est. Dec 2025
SPARK Biopharma
SPARK BiopharmaKorea - Seoul
2 programs
1
1
voretigene neparvovec-rzylPhase 1/2Gene Therapy1 trial
voretigene neparvovec-rzylPhase 1Gene Therapy1 trial
Active Trials
NCT00516477Completed12Est. Mar 2018
NCT01208389Active Not Recruiting12Est. Oct 2027
Atsena Therapeutics
1 program
1
ATSN-101Phase 1/21 trial
Active Trials
NCT03920007Active Not Recruiting15Est. May 2027
Beacon Therapeutics
Beacon TherapeuticsMA - Cambridge
1 program
1
rAAV2-CB-hRPE65Phase 1/2
Oregon Therapeutics
Oregon TherapeuticsFrance - Paris
1 program
1
rAAV2-CB-hRPE65Phase 1/21 trial
Active Trials
NCT00749957Completed12Est. Sep 2017
MeiraGTx
MeiraGTxNEW YORK, NY
1 program
AAV RPE65PHASE_1_2Gene Therapy1 trial
Active Trials
NCT02781480Completed15Est. Dec 2018

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
HuidaGene TherapeuticsHG004
Atsena TherapeuticsATSN-101
MeiraGTxAAV RPE65
SPARK Biopharmavoretigene neparvovec-rzyl
Oregon TherapeuticsrAAV2-CB-hRPE65
HuidaGene TherapeuticsHG004
SPARK Biopharmavoretigene neparvovec-rzyl

Clinical Trials (7)

Total enrollment: 95 patients across 7 trials

NCT05906953HuidaGene TherapeuticsHG004

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

Start: Oct 2023Est. completion: Dec 202520 patients
Phase 1/2Recruiting
NCT03920007Atsena TherapeuticsATSN-101

Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

Start: Sep 2019Est. completion: May 202715 patients
Phase 1/2Active Not Recruiting
NCT02781480MeiraGTxAAV RPE65

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

Start: Apr 2016Est. completion: Dec 201815 patients
Phase 1/2Completed
NCT01208389SPARK Biopharmavoretigene neparvovec-rzyl

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Start: Nov 2010Est. completion: Oct 202712 patients
Phase 1/2Active Not Recruiting
NCT00749957Oregon TherapeuticsrAAV2-CB-hRPE65

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

Start: Jun 2009Est. completion: Sep 201712 patients
Phase 1/2Completed
NCT06088992HuidaGene TherapeuticsHG004

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

Start: Jan 2023Est. completion: Oct 20289 patients
Phase 1Active Not Recruiting
NCT00516477SPARK Biopharmavoretigene neparvovec-rzyl

Safety Study in Subjects With Leber Congenital Amaurosis

Start: Sep 2007Est. completion: Mar 201812 patients
Phase 1Completed

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 actively recruiting trials targeting 95 patients
6 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.