Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 55/100

Failure Rate

16.9%

15 terminated/withdrawn out of 89 trials

Success Rate

73.2%

-13.3% vs industry average

Late-Stage Pipeline

18%

16 trials in Phase 3/4

Results Transparency

61%

25 of 41 completed trials have results

Key Signals

7 recruiting25 with results13 terminated

Enrollment Performance

Analytics

Phase 2

29(48.3%)

Phase 3

15(25.0%)

Phase 1

13(21.7%)

Early Phase 1

2(3.3%)

Phase 4

1(1.7%)

60Total

Phase 2(29)

Phase 3(15)

Phase 1(13)

Early Phase 1(2)

+1 more

Activity Timeline

Global Presence

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Clinical Trials (89)

Showing 20 of 89 trials

NCT06617429Phase 3Active Not Recruiting

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

Role: lead

NCT07447648Recruiting

Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

Role: collaborator

NCT06415344Phase 3Enrolling By Invitation

Long-term Extension of GTX-102 in Angelman Syndrome

Role: lead

NCT07157254Phase 2Recruiting

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

Role: lead

NCT04360265Phase 3Enrolling By Invitation

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Role: lead

NCT02716246Phase 2Recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Role: lead

NCT06636383Recruiting

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Role: lead

NCT06067802Phase 2Suspended

Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Role: collaborator

NCT03604835Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

Role: lead

NCT03773770Unknown

Expanded Access to Triheptanoin

Role: lead

NCT05139316Phase 3Completed

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Role: lead

NCT03970278Completed

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

Role: lead

NCT06636071Phase 3Active Not Recruiting

Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta

Role: lead

NCT04884815Phase 1Active Not Recruiting

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

Role: lead

NCT04632953Active Not Recruiting

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program