Practical NeurologyFDA Fast Track Designation and IND Clearance Granted to Novel Gene Therapy for GRN-Related Frontotemporal Dementia

The agency forges a path ahead towards genetic interventions in neurodegenerative disorders with the designation.

The FDA has cleared the IND application and granted Fast Track Designation for AVB-101, a gene therapy developed by AviadoBio. This one-time disease-modifying therapy (DMT) aims to replace the mutated GRN gene with a functional copy, potentially restoring progranulin protein levels in the brain, thereby slowing or stopping the progression of FTD-GRN.

Key Points

• AVB-101 is an adeno-associated virus (AAV) gene therapy developed by AviadoBio.
• The FDA has cleared the IND application for AVB-101 and granted it Fast Track Designation.
• AVB-101 aims to replace the mutated GRN gene with a functional copy.
• The therapy is intended to restore progranulin protein levels in the brain, potentially slowing or stopping the progression of FTD-GRN.
• The ASPIRE-FTD clinical trial is currently enrolling participants in Europe, with enrollment expected in the United States in 2024.

Our preclinical program shows robust biodistribution to the brain areas where it is needed to restore levels of progranulin, potentially slowing or stopping the progression of FTD-GRN, with little to no progranulin in the rest of the body where it may have adverse effects.
— David Cooper, MD, Chief Medical Officer of AviadoBio

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