AviadoBio gene therapy candidate receives orphan designation approval

AviadoBio announced that both the U.S. Food and Drug Administration (FDA) and the European Commission (EC) have granted orphan designation to AVB-101, a one-time gene therapy for the treatment of frontotemporal dementia (FTD).

AVB-101 is an investigational, adeno-associated virus (AAV) gene therapy designed for FTD patients with mutations in the Progranulin (GRN) gene. AVB-101 is designed to slow or arrest disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore normal progranulin levels. FTD is a debilitating form of early onset dementia that currently has no approved therapies.

“Achieving orphan designations in both the US and EU represents an important milestone in bringing AVB-101 to FTD patients who are living with a debilitating disease without treatment options. We are committed to advancing our proprietary gene therapy platform and working with regulatory authorities and the FTD community to bring a desperately needed option to patients. We look forward to initiating clinical trials for AVB-101 later in 2022,” said Lisa Deschamps, CEO, AviadoBio.

“FTD is a devastating condition, for patients, their families and friends, for which there is no effective treatment. A one-time treatment of AVB-101 has the potential to supplement the missing gene on a long-term basis, thereby stopping FTD in its tracks. As a clinician who has been treating this patient group for many years, I am looking forward to seeing how this therapeutic candidate performs in clinical studies,” said Prof. Christopher Shaw, co-founder and chief scientific and clinical advisor, AviadoBio.

Orphan drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition that affects fewer than 200,000 individuals in the U.S. The EC grants orphan designation to encourage the development of medicines intended for the treatment of a seriously debilitating or life-threatening rare condition, defined as one that affects fewer than five in 10,000 people in the EU.