A recent release published in BioSpace announced that a gene therapy company is vigorously pursuing the study of AVB-101 to treat patients with frontotemporal dementia (FTD), including FTD with GRN mutations, which is the principal cause of dementia in adults under 65 years of age.
AviadoBio, along with its pioneering research team focused on transformative medicines to treat neurodegenerative disorders, initiated the ASPIRE-FTD Phase 1/2 study of AVB-101 to treat people with frontotemporal dementia with GRN gene mutations. Mutations in the GRN gene have been established as a cause of FTD, but it is uncertain whether the same holds true for certain GRN variants.
About ASPIRE-FTD
The ASPIRE-FTD trial was designed to study the preliminary efficacy and safety of AVB-101 in treating FTD-GRN patients. For this study, AVB-101 is administered as a one-time neurosurgical procedure, using coordinates from medical imaging to direct the tip of a delicate instrument into the thalamus. The procedures will be conducted at various expert neurosurgical facilities in the United States and Europe.
About the Thalamus
The temporal and frontal lobes play a critical role in FTD, and the thalamus connects to various areas in the brain, including the temporal and frontal lobes.
CEO Lisa Deschamps explained that no currently available treatments are capable of modifying this devastating disease. Dr. David Cooper, CMO of AviadoBio, added that the company’s focus is now on assisting the biodistribution of the GRN gene to the cortex areas affected by FTD.
People diagnosed with FTD with GRN mutations experience a reduction in progranulin protein. AVB-101, as a one-time targeted therapy, delivers a copy of the GRN gene that restores proper levels of the gene to the brain’s affected areas.
Due to its targeted delivery, AVB-101 limits the therapy to areas within the brain, reducing the required treatment dose.
About FTD with FTD/GRN Mutations
FTD, or early-onset dementia, generally leads to death seven to thirteen years after the onset of symptoms. This devastating form of dementia causes behavioral and personality changes, apathy, and a reduction in mobility.
Compared to Alzheimer’s, FTD has a greater impact on patients’ work and family, as a larger percentage of patients are under the age of 65. One-third of all FTD cases are genetic (inherited), with approximately 2,200 new cases reported each year and an estimated eleven thousand individuals in the UK and the US reported as having FTD-GRN.
