• AVB-101 is an investigational gene therapy designed to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels in the brain
• AVB-101 is delivered as a one-time treatment into the thalamus to facilitate targeted biodistribution in order to reach broad areas of the brain affected by FTD, while limiting treatment exposure to only the brain itself
• FTD is a leading cause of dementia in people under the age of 65
London, UK; October 10, 2023 — AviadoBio, a pioneering gene therapy company developing and delivering transformative medicines for neurodegenerative disorders, today announced that it has initiated the Phase 1/2 ASPIRE-FTD clinical study of its investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with mutations in the progranulin (GRN) gene.
ASPIRE-FTD is an open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, AVB-101 will be delivered as a one-time treatment into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States.
“FTD is a devastating form of early-onset dementia and there are currently no disease-modifying treatments available,” said Lisa Deschamps, Chief Executive Officer. “The initiation of our company’s first clinical trial demonstrates our commitment to developing and delivering potential transformative gene therapies for those living with neurodegenerative disorders such as FTD.”
“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,” said David Cooper, M.D., Chief Medical Officer. “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.”
Preclinical safety and biodistribution data following intrathalamic delivery to non-human primates demonstrate good tolerability and widespread progranulin expression in brain tissues. These data will be presented at the 30th Annual European Society of Gene & Cell Therapy (ESGCT) meeting in Brussels, Belgium Oct. 24-27, 2023.
People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 has been designed as a potential one-time therapy to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to the affected areas of the brain.
AVB-101 is delivered using a minimally-invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections to other parts of the brain, including the frontal and temporal lobes, which play a critical role in FTD and the symptoms that impact patients and their families. This targeted delivery method aims to limit the treatment to the brain where it is needed most, thereby reducing the dose required and the potential systemic exposure.
More information about the ASPIRE-FTD study can be found at https://clinicaltrials.gov/study/NCT06064890.
