AVB-101 Biomarker Data Readout Expected in 2026
Clinical Milestone Shared at AFTD Annual Caregiver Conference
AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced the completion of the second dose cohort in its Phase 1/2 ASPIRE-FTD clinical trial. ASPIRE-FTD is evaluating multiple doses of AviadoBio’s investigational gene therapy, AVB-101, in people living with frontotemporal dementia (FTD) with GRN gene mutations (FTD-GRN). The company intends to initiate dosing for a third cohort in Q3 2025 and expects to share early biomarker data in 2026. The announcement was made at the Association for Frontotemporal Degeneration (AFTD) annual caregiver conference in Denver.
ASPIRE-FTD is now open and actively recruiting participants at multiple sites across the U.S., Spain, Poland, Sweden and the Netherlands, with additional countries expected to join the trial in the near future. For central nervous system diseases like FTD, the blood-brain barrier presents a challenge to gene therapy delivery. AVB-101, an investigational gene therapy, is administered via a neurosurgical procedure directly to the thalamus—a key hub with widespread projections across the brain, including the frontal and temporal cortex regions most affected in FTD-GRN. This delivery method bypasses the blood-brain barrier and the pial membrane that separates the brain from the cerebrospinal fluid, targeting therapy precisely where needed while potentially reducing required dosage and limiting systemic exposure. Pre-clinical data indicate AVB-101 delivered to the thalamus can increase progranulin levels in cortical brain tissue, potentially restoring physiological function in people with FTD-GRN.
“Dosing our sixth patient with AVB-101 marks an important and encouraging step forward in our research pathway,” said David Cooper, M.D., Chief Medical Officer of AviadoBio. “In the first cohort, we did not see any clinically significant safety findings through follow-up of up to 52 weeks and did not require any immunosuppression prophylactically or reactively.”
“We’re proud to share this important milestone with the FTD community, who continue to inspire our commitment to advancing research for therapies that may slow or stop the progression of this devastating disease,” said Lisa Deschamps, CEO, AviadoBio. “People living with FTD-GRN and their families remain at the heart of everything we do. As we continue this vital work, we look forward to sharing clinical updates.”
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