BlackfinBio Plans Gene Therapy Trial

FDA has given BlackfinBio the green light to conduct a Phase 1/2 clinical trial of its novel adeno-associated virus gene therapy, BFB-101, in children with hereditary spastic paraplegia, Type 47 (SPG47). Previously, the agency awarded the company an orphan drug designation and rare pediatric disease designation for BFB-101 and its use in treating SPG47, a rare, autosomal-recessive, neurological disorder characterized by progressive lower-limb spasticity, developmental delays and intellectual disability in children. The condition is caused by deleterious changes in the AP4B1 gene, according to the company.

The clinical trial is intended to evaluate the safety and efficacy of a single injection into the cerebrospinal fluid at the base of the brainstem near the spinal cord. “This type of delivery generally allows for rapid and efficient delivery of agents to the central nervous system,” the company says.