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AviadoBio Doses First Patient in Phase I/II Trial of Frontotemporal Dementia Gene Therapy

NEW YORK – AviadoBio on Monday said the first patient with GRN-mutated frontotemporal dementia (FTD) has received its investigational gene therapy AVB-101 in a Phase I/II trial.

FTD is a form of early-onset dementia that occurs in people between the age of 45 and 68, and patients can die within three to 10 years of diagnosis. In a third of patients, FTD is caused by autosomal dominant mutations in three genes, including GRN. Patients with GRN-mutated FTD produce lower amounts of progranulin, a protein involved in lysosomal function, neuronal survival, and inflammation. AVB-101 is designed to deliver a functional copy of the GRN gene directly to the brain to slow or halt disease progression by restoring levels of progranulin in the frontal and temporal cortex.

In the recently started open-label dose-escalation ASPIRE-FTD trial, patients receive a single dose of AVB-101, which is delivered via an infusion to the thalamus at a neurosurgical center. London-based AviadoBio is evaluating the safety and preliminary efficacy of AVB-101 in the Phase I/II trial. 

The firm has opened clinical trial sites in Poland, Spain, and the Netherlands and plans to expand into the US. The first patient was treated at Mazowiecki Szpital Bródnowski Hospital's Interventional Neurotherapy Center in Poland. 

"The lack of effective disease-modifying treatments makes this diagnosis particularly difficult for patients and their families," AviadoBio Chief Medical Officer David Cooper said in a statement. "Treating the first patient in ASPIRE-FTD marks an important step forward in understanding AVB-101's potential."