NEW YORK – The US Food and Drug Administration has granted BlackfinBio permission to begin a Phase I/II trial of BFB-101, a gene therapy it is developing for children with hereditary spastic paraplegia type 47 (SPG47), a rare neurological disease, the company said Monday.
Following the FDA's clearance of its investigational new drug (IND) application, UK-based BlackfinBio said it will start recruiting patients for the study at Boston Children's Hospital by year-end.
"We look forward to initiating enrollment in the US 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," BlackfinBio Founder and CEO Peter Nolan said in a statement.
SPG47 is caused by a mutation in the AP4B1 gene that results in progressive lower-limb spasticity, developmental delays, and intellectual disability in children. BFB-101 aims to halt or potentially reverse disease progression in children with the condition by delivering a functional copy of the AP4B1 gene.
In the open-label Phase I/II trial, investigators will assess the safety and efficacy of a single injection of BFB-101 into the cerebrospinal fluid at the base of the brainstem near the spinal cord in up to five children with this condition. Investigators will also evaluate the effect of the gene therapy on motor function, development, and health-related quality of life.