BlackfinBio Receives FDA Clearance to Initiate Phase 1/2 Clinical Trial of Novel Gene Therapy BFB-101 for Rare Neurological Disorder SPG47
CHESHIRE, UK – April 28, 2025 – BlackfinBio Ltd, a clinical-stage gene therapy company, today announced FDA clearance of its IND application for a Phase 1/2 clinical trial of BFB-101, a novel AAV gene therapy for children with Hereditary Spastic Paraplegia, Type 47 (SPG47).
The U.S. trial at Boston Children’s Hospital is expected to begin enrollment by the end of 2025. The FDA has granted BFB-101 orphan drug (ODD) and rare pediatric disease (RPDD) designations for SPG47.
SPG47, caused by AP4B1 gene mutations, is a rare neurological disorder with no current cure. BFB-101 aims to deliver a functional AP4B1 gene to halt or reverse disease progression. Promising preclinical safety and activity have been shown (1).
The single-center, open-label Phase 1/2 study will assess safety and efficacy of a single ICM injection of BFB-101 in up to five children with SPG47. Secondary objectives include evaluating impact on motor function, development, and quality of life. More information is on ClinicalTrials.gov. “The FDA’s clearance is a key milestone. We look forward to enrolling patients in the U.S. and evaluating BFB-101’s therapeutic potential for children with SPG47,” said Peter Nolan, CEO of BlackfinBio Ltd.
Dr. Darius Ebrahimi-Fakhari, Principal Investigator at Boston Children’s Hospital, stated, “We are proud to lead this trial, bringing a precision medicine approach to children with SPG47, and BFB-101 represents a promising candidate.”
Professor Mimoun Azzouz, CSO of BlackfinBio Ltd., added, “Reaching this IND milestone is a huge step. We are committed to transforming treatment options for children with this devastating condition.”
About BFB-101
BFB-101 is an AAV gene therapy delivering a functional AP4B1 gene to address the underlying cause of SPG47. It has shown potential in preclinical studies. BFB-101 was developed by Professor Mimoun Azzouz with support from Cure AP-4 and LifeArc, and received partial funding from the Innovative Medicines Initiative 2 Joint Undertaking. BFB-101 has ODD and RPDD for SPG47.
Source:
https://blackfin.bio/blackfinbio-announces-fda-clearance-to-initiate-phase-1-2-clinical-trial-for-novel-aav-gene-therapy-bfb-101-to-treat-the-rare-neurological-disease-hereditary-spastic-paraplegia-type-47-spg47/
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