Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)

NCT05747261 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

RECRUITING

Status

40

Enrollment

INDUSTRY

Sponsor class

Conditions

Spinal Muscular Atrophy (SMA)

Interventions

GENETIC: ANB-004, dose 1
GENETIC: ANB-004, dose 2
GENETIC: ANB-004, dose 3

Sponsor

Biocad