Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

​ Conditions: HSP; Hereditary Spastic Paraplegia; Hereditary Spastic Paraparesis; Hereditary Spastic Paraplegia Type 50; Hereditary Spastic Paraplegia Type 47; Hereditary Spastic Paraplegia Type 51; Hereditary Spastic Paraplegia Type 52; SPG47; AP4B1; Neurogenetic Disorders; Neurodevelopmental Conditions; Movement Disorders; Gene Therapy

Interventions: Biological: BFB-101 (AAV9-CBh-AP4B1)

Sponsors: BlackfinBio Ltd; Boston Children’s Hospital

Not yet recruiting Conditions: HSP; Hereditary Spastic Paraplegia; Hereditary Spastic Paraparesis; Hereditary Spastic Paraplegia Type 50; Hereditary Spastic Paraplegia Type 47; Hereditary Spastic Paraplegia Type 51; Hereditary Spastic Paraplegia Type 52; SPG47; AP4B1; Neurogenetic Disorders; Neurodevelopmental Conditions; Movement Disorders; Gene Therapy
Interventions: Biological: BFB-101 (AAV9-CBh-AP4B1)
Sponsors: BlackfinBio Ltd; Boston Children’s Hospital
Not yet recruiting