Contact SCGE




Gene Therapy Trial Report

Summary

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


NCTID NCT06948019 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hereditary Spastic Paraplegia Type 47
Disease Ontology Term DOID:0110799
Compound Name BFB-101
Compound Description AAV9.CBh.AP4B1
Sponsor BlackfinBio Ltd
Funder Type Industry
Recruitment Status
Not yet recruiting
Enrollment Count 5 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant AP4B1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intra-cisterna magna
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-04-22
Completion Date 2032-08
Last Update 2025-04-28

Participation Criteria


Eligible Age 12 Months - 60 Months
Standard Ages Child
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates IND cleared 4/28/25

Resources/Links