VivoSeq
Ophthalmology

Leber Congenital Amaurosis

2

Pipeline Programs↗

6

Active Trials↗

5

Companies Involved↗

Leber Congenital Amaurosis is an early-stage landscape with 2 programs across 5 companies. The most active players are ProQR Therapeutics, Spark Therapeutics, Inc., Atsena Therapeutics Inc.. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

27/100

Pipeline Velocity

6/100

Mech. Concentration

N/A

Completion Rate

100/100

Attrition Rate

0/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

P

ProQR Therapeutics

1 program

Phase 3S

Spark Therapeutics, Inc.

0 programs

PreclinicalA

Atsena Therapeutics Inc.

0 programs

PreclinicalB

Beacon Therapeutics

0 programs

PreclinicalH

HuidaGene Therapeutics Co., Ltd.

0 programs

Preclinical

Pipeline

Programs by Phase

Preclinical1

Opgx-RDH12

Opus Genetics, Inc

Phase 1
No programs
Phase 2
No programs
Phase 31

Sepofarsen

ProQR Therapeutics

Approved
No programs

Analysis

Trial Status

11

clinical trials

Completed5 · 45%
Active, not recruiting3 · 27%
Recruiting2 · 18%
Not yet recruiting1 · 9%

Clinical Trials

Trial Activity

11 trials

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
Recruiting·HHuidaGene Therapeutics Co., Ltd.·Phase 1/2·NCT05906953·20 enrolled
A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene
Recruiting·LLaboratoires Thea·Phase 3·NCT06891443·32 enrolled
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
Active, not recruiting·AAtsena Therapeutics Inc.·Phase 1/2·NCT03920007·15 enrolled
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Active, not recruiting·SSpark Therapeutics, Inc.·Phase 3·NCT00999609·31 enrolled
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
Active, not recruiting·SSpark Therapeutics, Inc.·Phase 1/2·NCT01208389·12 enrolled
A Phase 1b/2a Open-Label, Dose-Exploration Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-RDH12 Administered in Participants With Leber Congenital Amaurosis With Autosomal-Recessive Retinol Dehydrogenase 12 Mutations
Not yet recruiting·OOpus Genetics, Inc·Preclinical·NCT07681778·10 enrolled
Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
Completed·MMeiraGTx UK II Ltd·N/A·NCT02946879·15 enrolled
Safety Study in Subjects With Leber Congenital Amaurosis
Completed·SSpark Therapeutics, Inc.·Phase 1·NCT00516477·12 enrolled
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
Completed·BBeacon Therapeutics·Phase 1/2·NCT00749957·12 enrolled
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Completed·UUniversity College, London·Phase 1/2·NCT00643747·12 enrolled
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Completed·MMeiraGTx UK II Ltd·Phase 1/2·NCT02781480·15 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)