DELIVERING THE RIGHT DRUG TO THE RIGHT PLACE
Our investigational gene therapy, AVB-101, is administered using a minimally invasive neurosurgical procedure directly to the thalamus. The thalamus is an “information relay hub” for receiving sensory and motor information from the body, which it then distributes using billions of pathways throughout the brain. It is also one of the brain areas found to have the highest expression of progranulin. Gene therapy aims to deliver functional gene copies to deficient cells.
When AVB-101 is administered into the thalamus, it delivers a working copy of the GRN gene to thalamic neurons. This will provide the neurons with the instructions for progranulin production, thereby increasing levels of progranulin throughout the brain. By increasing levels of progranulin in cortical brain tissue, AviadoBio hopes to restore physiological function to patients with FTD due to progranulin mutations (who are relatively deficient in progranulin).