The trial will primarily evaluate the safety and efficacy of BFB-101, while also assessing its impact on motor function, development, and quality of life for the participants.

BlackfinBio, based in Sheffield, UK, focuses on developing treatments for rare neurological diseases and was founded by Nolan and Professor Mimoun Azzouz.

Professor Mimoun Azzouz, BlackfinBio’s Chief Scientific Officer, highlighted the high unmet need for children affected by SPG47 and the importance of this clinical trial.

BlackfinBio Ltd has received FDA clearance for its Investigational New Drug (IND) application to conduct a Phase 1/2 clinical trial of its gene therapy BFB-101 for Hereditary Spastic Paraplegia, Type 47 (SPG47).

The upcoming clinical trial will be an open-label study assessing the safety and efficacy of BFB-101, administered via intrathecal cerebrospinal fluid injection to five children diagnosed with AP4B1-associated SPG47.

This trial will take place at Boston Children’s Hospital, with participant recruitment expected to begin by the end of 2025.

Dr. Darius Ebrahimi-Fakhari from Boston Children’s Hospital emphasized the urgency for effective treatments for SPG47, highlighting BFB-101 as a promising candidate.

SPG47 is a rare neurological disorder characterized by progressive lower-limb spasticity, developmental delays, and intellectual disability due to mutations in the AP4B1 gene.

Peter Nolan, CEO of BlackfinBio, emphasized the significance of this IND clearance as a major step for the company’s neurological disease program.

The therapy has also received orphan drug and rare pediatric disease designations from the FDA, highlighting its potential significance.

Currently, there are no effective treatments for SPG47, making BFB-101 a potential breakthrough by delivering a functional copy of the AP4B1 gene to halt or reverse disease progression.