UK-based biotechnology company AviadoBio has administered its investigational gene therapy, AVB-101, to the first subject in the Phase I/II ASPIRE-FTD clinical trial.
AVB-101 is designed to halt disease progression by delivering a functional progranulin (GRN) gene.
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It was administered to a patient with frontotemporal dementia (FTD) carrying GRN gene mutations (FTD-GRN) at the Interventional Neurotherapy Center at Mazowiecki Szpital Bródnowski Hospital in Warsaw, Poland.
AviadoBio began the Phase I/II ASPIRE-FTD trial of AVB-101 FTD-GRN patients in October last year.
The open-label, ascending dose, multi-centre trial aims to evaluate the safety and initial efficacy of AVB-101, which is administered via bilateral intrathalamic infusion to individuals with FTD-GRN.
Study sites are operational in Poland, Spain and the Netherlands, with plans to expand the trial to additional countries, including the US.
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By GlobalDataAVB-101 is a one-time therapy designed to restore appropriate progranulin levels in the brain, thereby stopping the progression of the disease.
The gene therapy is offered through a minimally invasive, stereotactic neurosurgical procedure to the thalamus, which is connected to the frontal and temporal lobes of the brain.
This targeted delivery is intended to effectively cross the blood-brain barrier, restoring progranulin levels in the areas most affected while minimising potential systemic exposure.
In November last year, AVB-101 received fast-track designation from the US Food and Drug Administration (FDA).
The drug has also received orphan drug designation from the FDA and the European Commission.
AviadoBio chief medical officer David Cooper said: “There is a critical need for new treatments for people living with FTD-GRN.
“The lack of effective disease-modifying treatments makes this diagnosis particularly difficult for patients and their families who don’t currently have any available treatment options for this devastating and progressive disease.
“Treating the first patient in ASPIRE-FTD marks an important step forward in understanding AVB-101’s potential, which has already shown promise in preclinical studies.”
