Gene Therapy for Frontotemporal Dementia Granted Orphan Drug Status


The one-time therapy is meant to address familial early-onset dementia linked to the GRN gene.

Both the US FDA and European Commission have granted orphan drug designation to AviadoBio’s AVB-101, a one-time gene therapy intended to treat frontotemporal dementia.1

The investigational, AAV-based gene therapy delivers a functional copy of the progranulin GRN gene, helping to restore normal progranulin levels in patients who have an autosomal dominant mutation in the gene, which is one of 3 linked to familiar cases of the neurodegenerative disease, for which there are currently no approved treatments.

“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, in a statement.

The therapy, which is delivered directly into the thalamus, is currently in preclinical trials.

AviadoBio is also exploring the application of their gene therapy technology to other neurodegenerative diseases, namely amyotrophic lateral sclerosis (ALS). Frontotemporal dementia and ALS share some genetic drivers, disease mechanisms, and pathologies, suggesting that there may be a place for AviadoBio’s approach, which focuses on precision dosing to neural networks in order to promote broad expression throughout the central nervous system.

“While neurodegenerative conditions are focal at onset, the pathology eventually spreads throughout the nervous system. We have seen that modifying gene expression can be curative, but achieving widespread distribution is the greatest challenge. We have shown that precision micro dosing to neural networks will deliver broad CNS expression, providing safe and effective treatments,” said Christopher Shaw, cofounder and chief scientific and clinical advisor of AviadoBio.2 “We are directly exploiting insights into the causes of diseases to design therapies that have the potential to cure patients for whom there are no effective treatments. I believe that AviadoBio has the potential to move neurodegeneration from palliation to prevention.”

1. AviadoBio’s Gene Therapy Candidate Receives Orphan Designation from Food and Drug Administration and European Commission in Frontotemporal Dementia. News release. AviadoBio. April 27, 2022.
2. AviadoBio™ Raises $80 million in Series A Financing to Advance Neurodegenerative Gene Therapy Platform. News release. AviadoBio. December 2, 2021.
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