BlackfinBio Limited, a spinout company from the University of Sheffield, has received approval from the US Food and Drug Administration (FDA) to trial a novel therapy for a rare genetic neurological disease
The company received approval for its Investigational New Drug (IND) application to begin Phase 1/2 clinical trial for its novel adeno-associated virus (AAV) gene therapy targeting Hereditary Spastic Paraplegia, Type 47 (SPG47).
The trial will be conducted at Boston Children’s Hospital in the United States. It will assess the safety, tolerability, and preliminary efficacy of its AAV gene therapy in patients with Hereditary Spastic Paraplegia, Type 47 (SPG47).
“We are proud to lead 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).
Hereditary Spastic Paraplegia: A rare neurological disorder caused by genetic mutation
Hereditary Spastic Paraplegia, Type 47 (SPG47) is a slowly-progressing rare disease that causes malformation of the brain. It is caused by a specific mutation in the SPG47 gene that creates a deficiency of the AP4B1 protein, which transports typically other molecules around inside the cells of the nervous system. The condition causes muscle weakness, developmental delays, and impaired speech in children, and there is currently no cure or effective treatment.
The researchers will conduct a phase 1/2 clinical trial testing the safety and effectiveness of a gene therapy called BFB-101 in up to five children with a rare genetic disorder called AP4B1-associated SPG47. The treatment is delivered by injecting the fluid around the brain and spinal cord (ICM administration) to reach the central nervous system quickly.
BlackfinBIO aims to progress treatment
To advance treatment for this disease, BlackfinBio raised £2.75m in seed investment. The company’s AAV-based gene therapy aims to address the underlying genetic cause of Hereditary Spastic Paraplegia, by delivering a functional copy of the AP4B1 gene, to reverse or stop disease progression.
Blackfinbio is built on research from leading gene therapy expert Professor Mimoun Azzouz, Chair of Translational Neuroscience at the internationally renowned Sheffield Institute for Translational Neuroscience at the University of Sheffield.
Professor Mimoun Azzouz, Scientific Founder of BlackfinBio, said: “We are thrilled to take this important step forward in our mission to develop transformative therapies for patients with rare neurological diseases. This marks not just a major scientific advancement, but offers hope for the families of children living with the relentless challenges of Hereditary Spastic Paraplegia.
“The milestone underscores our unwavering commitment to translate groundbreaking research into meaningful impact for those who need it most. We look forward to working closely with the FDA and the clinical team at Boston Children’s Hospital as we advance this programme.”
“Having overseen this therapeutic innovation from the 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.
Peter Nolan, CEO of BlackfinBio Limited, said: “The FDA’s clearance of the BFB-101 IND is an important milestone for our rare neurological disease programme and the company. We look forward to initiating enrollment in the US later this year. We 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.”
