Gene Therapy
Total Trials
1
As Lead Sponsor
As Collaborator
0
Total Enrollment
5
NCT06948019
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Phase: Phase 1/2
Role: Lead Sponsor
Start: Aug 31, 2025
Completion: Aug 31, 2032
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