Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Low Risk

Score: 15/100

Failure Rate

0.0%

0 terminated/withdrawn out of 10 trials

Success Rate

100.0%

+13.5% vs industry average

Late-Stage Pipeline

40%

4 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 3

4(57.1%)

Phase 1

2(28.6%)

Phase 2

1(14.3%)

7Total

Phase 3(4)

Phase 1(2)

Phase 2(1)

Activity Timeline

Global Presence

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Clinical Trials (10)

Showing 10 of 10 trials

NCT03293524Phase 3Completed

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

Role: lead

NCT02064569Phase 1Completed

Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients

Role: lead

NCT03672968Unknown

EAP_GS010_single Patient

Role: lead

NCT03295071Completed

REALITY LHON Registry

Role: lead

NCT02652780Phase 3Completed

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)

Role: lead

NCT07303296Phase 2Recruiting

Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients

Role: lead

NCT03326336Phase 1Active Not Recruiting

Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

Role: lead

NCT03406104Phase 3Completed

RESCUE and REVERSE Long-term Follow-up

Role: lead

NCT02652767Phase 3Completed

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

Role: lead

NCT05555784Completed

Evaluation of Impact of Disease and Visual Disability on Quality of Life and Loss of Independence of Patients Living in France With Leber's Hereditary Optic Neuropathy (LHON) Through Qualitative and Quantitative Data Collection

Role: collaborator

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