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)

Associated Press

28-04-2025

CHESHIRE, United Kingdom, April 28, 2025 (GLOBE NEWSWIRE) -- BlackfinBio Ltd, a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases, announces today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for a Phase 1/2 clinical trial of its novel adeno-associated virus (AAV) gene therapy, BFB-101, in children with Hereditary Spastic Paraplegia, Type 47 (SPG47).
The trial will be conducted in the U.S. at Boston Children's Hospital and is expected to commence recruiting by the end of 2025. The FDA has granted an orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to BFB-101 for the treatment of SPG47.
SPG47 is a rare, autosomal-recessive, neurological disorder characterised by progressive lower-limb spasticity, developmental delays and intellectual disability in children. It is caused by deleterious changes in the AP4B1 gene. Currently, there is no cure or effective treatment available for this devastating condition. BFB-101 has been designed specifically 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. Preclinical results with BFB-101 have demonstrated promising activity and safety (1).
The Phase 1/2 clinical trial is a single-center open-label study with a primary objective to assess the safety and efficacy of a single injection into the cerebrospinal fluid at the base of the brainstem near the spinal cord, termed an 'ICM' administration. This type of delivery generally allows for rapid and efficient delivery of agents to the central nervous system. BFB-101 will be administered by ICM in up to five children with AP4B1-associated SPG47. The secondary objectives will aim to evaluate the impact of BFB-101 on motor function, development and health-related quality of life.
'The FDA's clearance of the BFB-101 IND is an important milestone for our rare neurological disease program and the company. We look forward to initiating enrollment in the U.S. later this year and are working closely with the investigator team at Boston Children's Hospital to evaluate the therapeutic utility of this gene therapy in children with SPG47,' said Peter Nolan, Founder and Chief Executive Officer of BlackfinBio Ltd.
'We are proud to be leading this important clinical trial at Boston Children's Hospital, bringing forward a precision medicine approach for children affected by SPG47. As clinicians, we see firsthand the impact of this disorder and the urgency for effective treatments. BFB-101 represents a promising gene therapy candidate, and this trial is a critical step toward delivering meaningful change for patients and families affected by AP-4-associated hereditary spastic paraplegia and related conditions,' said Dr. Darius Ebrahimi-Fakhari, M.D., Ph.D., Boston Children's Hospital (Principal Investigator).
'Having overseen this therapeutic innovation from discovery stage, it is a huge source of excitement to reach this important IND milestone. Given the incredibly high unmet need for children with this devastating condition, we are on a mission at BlackfinBio to transform treatment options for these young lives and make a real impact to the lives of their families,' added Professor Mimoun Azzouz, Founder and Chief Scientific Officer of BlackfinBio Ltd.
Reference:
(1): Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47
Disclosure Statement:
Dr. Darius Ebrahimi-Fakhari has consulted for BlackfinBio.
About BFB-101
BFB-101 aims to address the underlying genetic cause of Hereditary Spastic Paraplegia, Type 47 (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 ( https://cureap4.org ), LifeArc ( www.lifearc.org ). This project has also received partial funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 945473 to support AAV9 capsid synthesis for immune response study. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA. The 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, a clinical 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 to treat several rare dopamine deficiency disorders. For more information, please visit https://blackfin.bio and follow us on LinkedIn.
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