BlackfinBio Ltd Announces Submission of IND Application to the U.S. FDA for Novel AAV Gene Therapy to Treat Hereditary Spastic Paraplegia, Type 47 (SPG47)

Cheshire, UK – March 18, 2025 – BlackfinBio Ltd, a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases, announced today the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its novel adeno-associated virus (AAV) gene therapy, BFB-101, targeting Hereditary Spastic Paraplegia, Type 47 (SPG47).

SPG47 is a rare, autosomal recessive neurodegenerative disorder characterized by progressive lower-limb spasticity, intellectual disability and developmental delays. It is caused by changes (pathogenic variants) in the AP4B1 gene. Currently, there is no cure or no effective treatment available for this devastating condition.

“The IND submission marks a significant milestone for BlackfinBio and the SPG47 community.” said Peter Nolan, CEO of BlackfinBio Limited. “This achievement is the culmination of years of dedicated research and collaboration. We acknowledge theimportant contribution of LifeArc and Cure AP-4, who helped fund the discovery of this program at the University of Sheffield, and our private investors, whose support has been instrumental in advancing this program to the clinic.”

Pending regulatory clearance, BlackfinBio plans to initiate a Phase 1/2 clinical trial in the United States to assess the safety, tolerability, and preliminary efficacy of BFB-101 in patients with SPG47.

“We are thrilled to take this important step forward in our mission to develop transformative therapies for patients with rare neurological diseases,” added Professor Mimoun Azzouz, Founder and Chief Scientific Officer of BlackfinBio. “We look forward to working closely with the FDA and the broader medical community as we advance this program.”

About BFB-101
BFB-101 aims to address the underlying genetic cause of SPG47 by delivering a functional copy of the AP4B1 gene, with the goal of halting or reversing disease progression. 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. BFB-101 was originally developed by Professor Mimoun Azzouz at the University of Sheffield with the support of Cure AP-4 and LifeArc. The U.S. FDA has granted an orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to BFB-101 for the treatment of SPG47.

About BlackfinBio Ltd
BlackfinBio is a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases. The pipeline comprises BFB-101, an IND stage AP4B1 replacement AAV gene therapy for spastic paraplegia 47 – an ultra-rare genetic neurological disease for which no treatment currently exists. BFB-201 is a preclinical stage gene therapy approach using three gene fusion (AADC-TH-CH) to treat several rare dopamine deficiency disorders.

About LifeArc:
LifeArc is a not-for-profit medical research organisation that turns promising scientific research into impact for people living with rare diseases and global infectious diseases.

We form partnerships, and provide scientific expertise and funding to help break down the barriers preventing scientific breakthroughs from becoming life-transforming treatments and cures. We have been doing this for more than 30 years and our work has resulted in five licensed medicines, including cancer drug pembrolizumab and lecanemab for Alzheimer’s disease.

Our goal is a world where no one with a rare disease or a global infectious disease misses out on life-changing innovation because of complexity, cost or risk.

LifeArc is a company limited by guarantee (registered in England and Wales under no. 2698321) and a charity (registered in England and Wales under no. 1015243 and in Scotland under no. SC037861).

About Cure AP-4
Cure AP-4 is a non-profit organization founded in 2016 by families of two of the known SPG47 patients. It was originally founded as Cure SPG47, but the mission has since expanded to include all four AP-4 related disorders due to shared natural history, goals and patient/family needs. For more information, please visit https://cureap4.org

For media enquiries, please contact:
Peter Nolan, Chief Executive Officer