Move over, Zolgensma. A new one-dose therapy that could provide a similar mode of treatment for the red-hot therapy area of neurodegenerative disorders has received orphan designation from regulators on both sides of the Atlantic.
AviadoBio’s preclinical drug, AVB-101, is an adeno-associated virus (AAV) gene therapy designed for patients with frontotemporal dementia (FTD) that have mutations of the progranulin gene. FTD is a debilitating form of early-onset dementia that currently has no approved therapies, and the drug has received orphan designation from both the FDA and the European Commission, the company announced today.
