AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, will present preliminary data at the 15th International Conference on Frontotemporal Dementias showing early signs of dose-dependent increases in cerebrospinal fluid (CSF) progranulin across four ascending doses in its ongoing Phase 1/2 ASPIRE-FTD clinical trial evaluating AVB-101, its investigational gene therapy being developed for people living with frontotemporal dementia with GRN mutations (FTD-GRN).

To date, the ASPIRE-FTD trial has dosed 17 patients from nine countries with 20 active trial sites across the United States, United Kingdom, Canada and in six European countries. Enrollment in the fourth cohort is ongoing.

Patients with FTD-GRN have a significant deficiency of progranulin, which negatively impacts brain cell function. AVB-101 is designed to restore progranulin levels in the brain. Cerebrospinal fluid (CSF) progranulin serves as a primary biomarker to assess target engagement of AVB-101 and potential resulting increases in progranulin in the brain. Across each of the four cohorts, dose-dependent increases have been observed in the average CSF progranulin, with 12-week data showing 36% and 90% increases over baseline in the third and fourth dose cohorts, respectively to date.1

“AVB-101’s targeted brain delivery represents a novel approach to increase progranulin exclusively in neurons, thereby limiting any potential systemic exposure,” said Professor Rik Vandenberghe, Director of the Memory Clinic at University Hospitals Leuven. “The early signs of dose-dependent increase in CSF progranulin are consistent with production by the neurons from the thalamus in the brain, suggesting AVB-101 is reaching its target in patients who are significantly deficient in this necessary protein. We are encouraged by these early biomarker data and their potential to impact clinical disease progression over time.”

AVB-101 is a differentiated and precise approach to restoring progranulin in the brain of FTD-GRN patients. 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 safely and effectively bypass the blood-brain barrier, delivering targeted treatment directly to the brain to restore progranulin levels in the frontal and temporal cortex where it is needed most, while at the same time minimizing the dose required.

“Following the standardization of AVB-101 intrathalamic delivery and expansion to five expert neurosurgical centers2,3, we are pleased that AVB-101 has been well tolerated across four ascending dose cohorts, with no serious adverse events related to treatment, including in the highest dose cohort,” said Dr. David Cooper, Chief Medical Officer at AviadoBio. “In addition, there has been no need for prophylactic or reactive immunosuppression.”

ASPIRE-FTD is also exploring biomarkers of neurodegeneration, including neurofilament light chain (NfL). While natural history data and recent randomized placebo trials suggest NfL should increase at least 13-14% per year, initial data from early low-dose cohorts in the ASPIRE-FTD trial suggest average serum NfL at 52 weeks is at or below baseline.

“The one-year biomarker data on NfL are encouraging. We await the long-term NfL results from the higher dose cohorts, where we observed the important increase in CSF progranulin,” said Professor James Rowe, Director of Cambridge Centre for Frontotemporal Dementia.

  1. Data cutoff: September 30, 2026.
  2. Lonser RR, Rabon M, Damante M, Elder JB, Zabek M, Cooper DL. Direct image-guided convective perfusion of the bilateral thalami for gene therapy in frontotemporal dementia: technical note. J Neurosurg. 2025 Dec 19;144(3):682-689.
  3. Lonser RR, Elder JB, Damante MA, Gray W, Zabek M, Rabon M, Cooper DL. Prescriptive Approach to Convective Perfusion of the Bilateral Thalami for Gene Therapy. Oper Neurosurg. 2026 Jun 3.

About AviadoBio

AviadoBio is relentlessly chasing cures by developing and translating groundbreaking science and precision delivery into potentially life-changing medicines across neurodegenerative diseases. Powered by a precision approach built on three integrated levels — payload, target, and delivery — the company is advancing a pipeline of targeted, one-time AAV gene therapies across frontotemporal dementia with GRN mutations (FTD-GRN), retinitis pigmentosa (RP) and other retinal dystrophies, as well as a novel intravenous treatment for Alzheimer’s disease (AD) and related tauopathies, including FTD-MAPT.

Founded in 2019 from pioneering research at King’s College London and the UK Dementia Research Institute, AviadoBio combines deep neuroscience expertise with proven biopharma leadership. Its capabilities include gene supplementation, optogenetics, and its proprietary vMiX™ RNAi gene-silencing platform, enabling durable, disease-modifying approaches across multiple degenerative conditions. Headquartered in London, the company operates across the UK and the U.S. and is backed by leading global life sciences investors and strategic partners.

For more information, please visit www.aviadobio.com and follow AviadoBio on X @AviadoBio and LinkedIn at AviadoBio.

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. The ASPIRE-FTD clinical trial is currently open at trial sites in Belgium, Canada, Italy, the Netherlands, Poland, Spain, Sweden, the United Kingdom, and the United States.

More information about the ASPIRE-FTD study can be found at www.aspire-ftd.com or https://clinicaltrials.gov/study/NCT06064890.

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