LONDON, May 14, 2026
AviadoBio presented new preclinical data at the American Society of Gene and Cell Therapy (ASGCT) 2026 Annual Meeting demonstrating the potential of its investigational AVB-406 gene therapy for Alzheimer’s disease and other tauopathies. The company reported that AVB-406 achieved up to 80% MAPT/tau knockdown in the brain, supporting plans to initiate first-in-human clinical trials by the end of 2026.
AVB-406 is being developed as a one-time intravenous gene therapy designed to silence the MAPT gene, which produces tau protein — a major driver of neurodegeneration in Alzheimer’s disease and related disorders. Using AviadoBio’s proprietary vMiX™ RNAi platform, the therapy combines a blood-brain barrier-penetrant AAV vector with a neuron-specific artificial miRNA payload to achieve targeted and durable tau suppression throughout the central nervous system.
AVB-406 Shows Strong Tau Reduction and Brain Targeting
According to AviadoBio, preclinical studies in human iPSC models and humanized mice demonstrated strong dose-dependent reduction of MAPT mRNA in critical brain regions associated with Alzheimer’s pathology. Researchers confirmed that the therapy achieved broad neuronal delivery while maintaining high specificity through in situ hybridization analysis.
The company stated that AVB-406 was well tolerated across all animal studies, with no treatment-related safety findings observed even at dose levels producing up to 80% tau knockdown and significant reductions in pathological tau accumulation. The therapy uses an AAV vector engineered to cross the blood-brain barrier (BBB) through the human Transferrin Receptor 1 (hTfR1), one of the most widely studied CNS delivery targets in neurodegenerative drug development.
AviadoBio believes the approach could provide durable disease modification following a single administration, potentially overcoming limitations associated with repeat-dose RNA interference therapies.
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vMiX Platform Expands One-Time CNS Gene Silencing Potential
The company also highlighted the broader capabilities of its vMiX™ vectorized RNAi platform, which enabled screening of more than 1,000 potential miRNA target sites during development of AVB-406. Unlike traditional non-vectorized RNAi approaches, vMiX is designed to achieve long-term gene silencing after a single dose, creating opportunities for treating chronic neurodegenerative diseases.
Manufacturing and CMC data presented at ASGCT showed successful scale-up across 3L, 10L, and 15L production batches, with consistently high purity, potency, and recovery rates aligned with industry manufacturing standards. AviadoBio stated that the scalable manufacturing process strengthens readiness for planned clinical development and future commercialization.
Beyond Alzheimer’s disease, AVB-406 may also have potential utility across multiple primary and secondary tauopathies, including frontotemporal dementia (FTD-MAPT), corticobasal degeneration (CBD), progressive supranuclear palsy (PSP), primary progressive aphasia (PPA), and chronic traumatic encephalopathy (CTE).
Dr. Alex Bloom, Chief Technology Officer at AviadoBio, stated that the ASGCT presentations validate both the AVB-406 program and the broader vMiX platform as a strategy for achieving one-time gene silencing in the CNS across a wide range of neurological diseases.
Source: AviadoBio press release
