$PLASMATECH BIOPHARMACEUTICAL(PTBI)$ • PlasmaTech Biopharmaceuticals (NASDAQ:PTBI) adds an adeno-associated virus (AAV) gene therapy candidate for the treatment of juvenile Batten disease to its pipeline via a license from UNeMed, the technology transfer and commercialization office for the University of Nebraska Medical Center. The company is also developing AAV gene therapies for the treatment of Sanfilippo syndromes.
• Juvenile Batten disease, a rare inherited disorder caused primarily by mutations in the CLN3 gene, principally affects the nervous system. After a few years of normal development, children with the condition develop progressive vision loss, intellectual and motor disability, difficulties in speech and seizures. It is inherited via an autosomal recessive pattern, that is, each parent carries one copy of the mutated gene. They typically do not exhibit symptoms of the disorder. The condition manifests itself when both copies are present. It affects 1 in 100K people worldwide, but is more prevalent in Finland. There are no approved therapies for the