Key Highlights

Risk & Performance

Based on historical performance

High Risk

Score: 65/100

Failure Rate

20.0%

1 terminated/withdrawn out of 5 trials

Success Rate

0.0%

-86.5% vs industry average

Late-Stage Pipeline

40%

2 trials in Phase 3/4

Results Transparency

0 of 0 completed trials have results

Key Signals

1 recruiting

Phase 3

2(50.0%)

Phase 1

2(50.0%)

4Total

Phase 3(2)

Phase 1(2)

Loading network data...

Showing 5 of 5 trials

Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

Role: lead

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

Role: lead

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

Role: lead

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

Role: collaborator

A Study of the Natural Course of SURF1 Deficiency

Role: lead

All 5 trials loaded