Our innovative research consists of a growing portfolio of highly promising programs offering treatments and life-changing opportunities for patients worldwide.

SPG47 is an ultra-rare form of AP-4 Hereditary Spastic Paraplegia (AP-4 HSP) with an estimated prevalence in excess of 5,000 patients worldwide. It’s a progressive neurodevelopmental and neurodegenerative disease characterised by early childhood spasticity, microencephaly and seizures.

The AP4B1 gene helps to move things inside our cells, specifically from the trans-Golgi network to the endosomal compartment. When this gene does not work correctly, we call it a loss-of-function mutation, which causes SPG47.

BFB-101 is a patented AAV vector that expresses the AP4B1 gene, developed by Professor Mimoun Azzouz at the University of Sheffield with the support of Cure AP-4 and LifeArc.

It is administered via the brain as a single lifetime dose and has shown potential in restoring AP-4 function in vitro and improving motor function in AP4B1 mutant mice.

As a late-stage pre-clinical project, we anticipate filing an IND in the U.S. for a phase I/II clinical trial in late 2023. In early/mid-2024, we will be ready to begin the clinical trial of BFB-101 and are in the process of securing further funding to embark on this exciting new phase of the project.

“Very young children will benefit the most; this treatment could alter the trajectory of their lives. This is our legacy project.”

Founder and Chief Business Officer