BlackfinBio

BlackfinBio is a biotechnology company developing AAV-based gene therapies for rare neurodegenerative diseases of the central nervous system, with an initial focus on children.

Its lead programme is a one-time gene therapy administered directly to the brain, designed to treat a group of ultra-rare dopamine deficiency disorders for which no treatment currently exists.

The company's pipeline has since expanded to include Hereditary Spastic Paraplegia, with a clinical trial approved by the US FDA.