AviadoBio Presents Positive Preclinical Data for AVB-406 Program in Alzheimer’s Disease and Other Tauopathies at ASGCT 2026

Preclinical AVB-406 data presented in three oral presentations demonstrate the power of the vMiX^TM RNAi platform and support planned clinical initiation for Alzheimer’s disease by the end of 2026

Preclinical data show AVB-406 achieved up to 80% MAPT/tau knockdown in the brain

LONDON--(BUSINESS WIRE)--AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced positive preclinical data for the AVB-406 program in Alzheimer’s disease (AD) and other tauopathies in three oral presentations at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting. The data demonstrate progression of AVB-406 across preclinical validation, platform discovery and scalable manufacturing, and collectively support the company’s plans to advance AVB-406 into clinical trials for AD by the end of 2026.

AVB-406 is being developed as a one-time, intravenously administered gene therapy for AD and other tauopathies. Alzheimer’s disease and other tauopathies are characterized by the pathological accumulation of misfolded, aggregated tau protein which is encoded by the MAPT gene. Developed with the company’s proprietary vMiX™ RNAi gene silencing platform, AVB-406 uses an AAV vector engineered to cross the blood-brain barrier (BBB) via the human Transferrin Receptor 1 (hTfR1). By delivering a three-hairpin artificial miRNA payload to the central nervous system (CNS) via TfR1—one of the most extensively characterized BBB receptors—under a neuron-specific promoter, AVB-406 has the potential to reduce overall MAPT expression in the CNS. AVB-406 has broader potential utility in other primary and secondary tauopathies, including frontotemporal dementia (FTD-MAPT, tau-FTD), primary progressive aphasia (PPA), corticobasal degeneration (CBD), progressive supranuclear palsy (PSP), and chronic traumatic encephalopathy (CTE).

“These data presentations mark an important step forward for AviadoBio, validating both our AVB-406 program and the broader potential of our vMiX platform to enable one-time gene silencing in the central nervous system across a broad range of diseases,” said Alex Bloom, Chief Technology Officer of AviadoBio. “In Alzheimer’s disease and other tauopathies, there are no approved therapies that target tau as a key driver of cognitive decline. These comprehensive program findings reinforce our confidence in AVB-406 as we move toward first-in-human studies later this year.”

Data Highlights:

• AVB-406 was developed using AviadoBio’s proprietary vMiX platform, which enabled systematic screening of more than 1,000 potential miRNA target sites.
• In a series of preclinical studies, including human iPSC models and mice, AVB-406 was evaluated for target engagement, biodistribution, dose-responsive activity, tolerability, and efficacy.
• Notably, AVB-406 sustained dose-dependent MAPT mRNA knockdown in brain cortex tissue in humanized mice, including in key brain regions associated with AD pathology, with in situ hybridization confirming neuronal specificity of miRNA guide expression.
• AVB-406 was well tolerated across all animal studies conducted to date, with no treatment-related adverse findings, including at dose levels achieving up to 80% MAPT mRNA knockdown and corresponding decrease in pathological tau.
• AVB-406 manufacturing at 3L and 10L scales yielded consistent recovery aligned with industry standards, further supported by a 15L toxicology batch achieving >30% recovery.
• CMC development included optimization of upstream and downstream conditions, in addition to formulation development.
• The final product demonstrated consistently high purity, quality, and potency.
• Together, these data demonstrate the power of the vMiX platform to advance AVB-406 as a well-characterized, translationally validated therapeutic candidate with the potential to address a significant unmet need across AD and the broader tauopathy spectrum.

About Alzheimer’s Disease and Tauopathies

Alzheimer’s disease (AD) and other tauopathies are a group of progressive brain disorders characterized by abnormal changes in tau, a protein that normally supports and stabilizes neurons. In these conditions, tau becomes dysfunctional, aggregates together, and disrupts communication between brain cells, ultimately leading to cognitive decline, memory loss, and changes in behavior or movement. Tau aggregation associated with familial MAPT mutations or acquired factors, and resulting neurotoxicity are unifying mechanisms across tauopathies. Preclinical data support the potential therapeutic benefit of MAPT gene silencing for AD, frontotemporal dementia (FTD), and other tauopathies.

About AVB-406

AVB-406 is being developed as a one-time, intravenously administered gene therapy designed to silence MAPT (the tau gene) using AviadoBio’s proprietary vMiX™ RNAi gene silencing platform and blood-brain barrier (BBB)-penetrant AAV capsid. This approach is designed to enable broad CNS exposure and durable reduction of pathological tau following a single dose. AVB-406 is being developed as a potential treatment for Alzheimer's disease and other tauopathies.

About vMiX™

vMiX™ is AviadoBio’s proprietary vectorized RNA interference (RNAi) platform designed to enable one-time, durable gene silencing using AAV delivery. Unlike non-vectorized RNAi approaches that potentially require repeat dosing, vMiX is engineered to provide sustained suppression of disease-causing genes following a single administration. The platform combines potent knockdown, cell-specific expression, and the flexibility to target multiple genes or incorporate knockdown-and-replace strategies, supporting the development of next-generation genetic medicines across a range of diseases.

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 Alzheimer’s disease (AD) and other tauopathies.

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 us on X @AviadoBio and LinkedIn at AviadoBio.