AviadoBio Gene Therapy Drug AVB-101 Gets Orphan Drug Tags in U.S. and EU

The FDA and the European Commission have granted AviadoBio Orphan Drug designations for its investigational gene therapy, AVB-101, for the treatment of patients with frontotemporal dementia (FTD).

AVB-101 is an investigational, adeno-associated virus (AAV) gene therapy for FTD patients with mutations in the progranulin (GRN) gene.

The one-dose therapy is designed to restore GRN to normal levels throughout the central nervous system and slow or arrest disease progression.

UK-based AviadoBio said it plans to start clinical trials of the gene therapy later this year.