The gene therapy is intended for the treatment of frontotemporal dementia with progranulin mutations. Credit: Robert Kneschke/Shutterstock.com.

AviadoBio has commenced the Phase I/II ASPIRE-FTD clinical study evaluating the AVB-101 gene therapy in people with frontotemporal dementia (FTD) with progranulin (GRN) mutations.

The multi-centre, open-label study intends to assess the preliminary efficacy and safety of the investigational therapy.

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In the study, AVB-101 will be delivered into the patient’s thalamus, a part of the brain, through a stereotactic neurosurgical procedure.

This procedure will be carried out at expert neurosurgical centres throughout the US and Europe.

Patients with FTD-GRN and GRN mutations are claimed to produce a reduced amount of progranulin protein.

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AviadoBio chief medical officer David Cooper said: “While it is known that progranulin protein supplementation is possible using gene therapy, effective brain distribution remains challenging, primarily because of the brain’s anatomy.

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“Our aim is to use intrathalamic delivery to facilitate biodistribution of the PGRN protein to the areas of the cortex impacted by FTD.

“AVB-101 has already shown great promise in preclinical studies and we now look forward to this important next step in its clinical development.”

In non-human primates, intrathalamic delivery of the gene therapy demonstrated good tolerability and progranulin expression in tissues of the brain.

AVB-101 gene therapy delivers a functional copy of the GRN gene and restores appropriate progranulin levels in the brain.

In February of this year, AviadoBio signed an exclusive licence and collaboration agreement with Neurgain Technologies on an investigational gene therapy spinal delivery technology.

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