Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 35/100

Failure Rate

0.0%

0 terminated/withdrawn out of 14 trials

Success Rate

100.0%

+13.5% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

57%

4 of 7 completed trials have results

Key Signals

1 recruiting4 with results

Enrollment Performance

Analytics

Phase 1
6(54.5%)
Phase 2
5(45.5%)
11Total
Phase 1(6)
Phase 2(5)

Activity Timeline

Global Presence

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Clinical Trials (14)

Showing 14 of 14 trials
NCT07174726Phase 2Recruiting

A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa

Role: lead

NCT04850118Phase 2Active Not Recruiting

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

Role: lead

NCT06275620Phase 2Enrolling By Invitation

A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)

Role: lead

NCT06333249Phase 2Active Not Recruiting

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

Role: lead

NCT03316560Phase 1Active Not Recruiting

Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

Role: lead

NCT02416622Phase 1Completed

Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)

Role: lead

NCT03314207Completed

Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)

Role: lead

NCT02599922Phase 1Active Not Recruiting

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)

Role: lead

NCT02935517Phase 1Active Not Recruiting

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)

Role: lead

NCT01054339Phase 2Completed

Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency

Role: lead

NCT00749957Phase 1Completed

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

Role: lead

NCT02331173Completed

Clinical Evaluation of Patients With X-linked Retinoschisis

Role: lead

NCT01846052Completed

Clinical and Genetic Characterization of Individuals With Achromatopsia

Role: lead

NCT00430768Phase 1Completed

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency

Role: collaborator

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