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."
About ASPIRE-FTD
ASPIRE-FTD is a Phase 1/2 open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, eligible patients receive a one-time administration of AVB-101 delivered as a set of MRI-guided infusions into the thalamus during a minimally invasive stereotactic neurosurgical procedure at an expert neurosurgical center in the U.S. or EU.
More information about the ASPIRE-FTD study can be found at www.aspire-ftd.com or https://clinicaltrials.gov/study/NCT06064890.
About AVB-101
An investigational gene therapy, AVB-101 contains a correct (non-mutated) version of the GRN gene. It is designed to restore levels of progranulin in the brain, potentially slowing or stopping the progression of FTD-GRN. AVB-101 is delivered as a one-time infusion directly into the brain via a minimally invasive neurosurgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center. AVB-101 has been granted orphan designation by the U.S. Food and Drug Administration (FDA) and European Commission. AviadoBio and Astellas Pharma Inc. have entered into an option agreement for a worldwide exclusive license for the development and commercial rights to AVB-101 in FTD-GRN and other indications.
About AviadoBio
At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with neurological conditions. With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King's College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients.
AviadoBio's investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson Johnson Innovation JJDC, Inc., SV Health Investor's Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), LifeArc Ventures, and Astellas Pharma.
For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.
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Farah Speer
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fspeer@aviadobio.com
+1-312-543-2881
