Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 75/100

Failure Rate

18.2%

4 terminated/withdrawn out of 22 trials

Success Rate

50.0%

-36.5% vs industry average

Late-Stage Pipeline

9%

2 trials in Phase 3/4

Results Transparency

50%

2 of 4 completed trials have results

Key Signals

4 recruiting2 with results

Enrollment Performance

Analytics

Phase 2
6(50.0%)
Phase 1
4(33.3%)
Phase 3
2(16.7%)
12Total
Phase 2(6)
Phase 1(4)
Phase 3(2)

Activity Timeline

Global Presence

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Clinical Trials (22)

Showing 20 of 22 trials
NCT06103487Enrolling By Invitation

Long Term Follow-Up for RGX-111

Role: lead

NCT04704921Phase 2Active Not Recruiting

Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD

Role: collaborator

NCT05407636Phase 3Recruiting

Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD

Role: collaborator

NCT06491927Enrolling By Invitation

Long Term Follow-up for RGX-202

Role: lead

NCT03580083Phase 1Suspended

RGX-111 Gene Therapy in Patients With MPS I

Role: lead

NCT04514653Phase 2Active Not Recruiting

RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD)

Role: collaborator

NCT07236606Phase 3Suspended

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

Role: lead

NCT05693142Phase 2Recruiting

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

Role: lead

NCT05683379Recruiting

AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD

Role: lead

NCT04567550Phase 2Active Not Recruiting

RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME)

Role: collaborator

NCT06942520Phase 2Recruiting

Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME)

Role: collaborator

NCT03566043Phase 2Active Not Recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Role: lead

NCT04571970Phase 1Completed

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Role: lead

NCT02651675Phase 1Terminated

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

Role: lead

NCT03066258Phase 1Completed

Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial

Role: lead

NCT04080050Unknown

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

Role: lead

NCT04591834Withdrawn

Mucopolysaccharidosis Type II Observational

Role: lead

NCT03529786Completed

Mucopolysaccharidosis Type II Natural History

Role: lead

NCT04480476Withdrawn

A Retrospective, Natural History Study in Children With CLN2

Role: lead

NCT04462692Withdrawn

An Observational Study in Children With CLN2 Batten Disease

Role: lead