NEW YORK – AviadoBio on Tuesday announced the start of a Phase I/II trial to test its gene therapy AVB-101 in patients with frontotemporal dementia caused by GRN mutations.
With AVB-101, AviadoBio aims to stop disease progression in patients with GRN-associated frontotemporal dementia, an early-onset form of dementia. The drug is designed to deliver a functional copy of the GRN gene to restore levels of the protein it encodes, progranulin, in the brain. The gene therapy is delivered to patients as a one-time treatment into the thalamus through a minimally invasive stereotactic neurosurgical procedure.
"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," AviadoBio Chief Medical Officer David Cooper said in a statement. "Our aim is to use intrathalamic delivery to facilitate biodistribution of the PGRN protein to the areas of the cortex impacted by FTD."
The open-label Phase I/II study, dubbed ASPIRE-FTD, is London-based AviadoBio's first clinical trial since launching in 2019. In the study, researchers will assess the safety and preliminary efficacy of AVB-101 at multiple trial sites in Europe and the US.
AviadoBio's investors include New Enterprise Associates, Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation, the Dementia Discovery Fund, Advent Life Sciences, EQT Lifesciences, and LifeArc. In 2021, the company raised $80 million in a Series A funding round.