Press Release
• Single intravenous dose of AVB-406 maintained MAPT mRNA knockdown and reduced total tau protein by 84% at six months with no adverse clinical signs
• First preclinical data for AVB-SNCA show up to 80% SNCA knockdown in human neurons, supporting development as a one-time therapy for synucleinopathies
LONDON – October 29, 2026 – AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced new preclinical data for AVB-406, its investigational gene therapy for Alzheimer’s disease and other tauopathies, and AVB-SNCA for the potential treatment of synucleinopathies including Parkinson’s disease, Lewy body disease, and multiple system atrophy. The data will be presented at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress (see below for presentation details).
The presentations highlight advances in AviadoBio’s proprietary vMiX™ RNA interference (RNAi) gene silencing platform, including deep and durable tau protein lowering through six months in mice in the AVB-406 program and dose-dependent silencing of SNCA, the gene encoding alpha-synuclein, in a pilot in vivo study.
The vMiX platform uses an adeno-associated virus (AAV) vector to deliver gene silencing instructions and is designed to enable durable reduction of disease-causing gene expression following a single administration. AVB-406 combines this platform with a blood-brain barrier-crossing capsid and a neuron-selective promoter to silence MAPT, the gene encoding tau, as a potential treatment for Alzheimer’s disease and other tauopathies.
“Our ESGCT presentations reflect both the best-in-class potential of our vMiX platform and the continued progress of AVB-406,” said Alex Bloom, Ph.D., Chief Development Officer of AviadoBio. “In addition to AVB-406, applying vMiX to alpha-synuclein has enabled identification of a lead miRNA for targeted, potent SNCA silencing, an important step toward a potential one-time treatment for Parkinson’s disease and other synucleinopathies, which currently have no disease-modifying therapies available. By combining vMiX with blood-brain barrier crossing capsids and promoters that restrict activity to the CNS, we have the potential to treat many neurodegenerative diseases with high precision after a single IV administration at doses anticipated to be lower than approved systemic AAV gene therapies.”
AVB-406 Data Highlights
- In human neurons, AVB-406 selectively reduced MAPT expression, with no off-target changes in gene expression.
- A single intravenous dose of AVB-406 in mice produced dose-dependent MAPT mRNA knockdown of up to 79% in the brain, with miRNA expression restricted to the central nervous system.
- In a mouse model of tauopathy, a vMiX MAPT-silencing construct reduced total tau protein in the brain by up to 94% and pathological tau in the cortex by up to 86%.
- Silencing was durable: six months after a single dose, AVB-406 maintained MAPT mRNA knockdown and an 84% reduction in endogenous tau protein.
- AVB-406 was well tolerated across all animal studies conducted to date, with no adverse clinical signs. GLP toxicology studies are ongoing.
- AviadoBio plans to advance the program into first-in-human studies for early Alzheimer’s disease in 2027.
AVB-SNCA Data Highlights
- Two miRNA candidates achieved approximately 80% SNCA mRNA silencing in testing that included human iPSC-derived neurons.
- In a pilot in vivo study, the best candidate achieved up to 78% SNCA silencing.
- The data support continued development of AVB-SNCA as a durable, one-time SNCA-lowering gene therapy for Parkinson’s disease and other synucleinopathies.
Presentations Details
AVB-406: An intravenous AAV-miRNA achieving sustained MAPT knockdown for the treatment of Alzheimer’s disease and other tauopathies
- Presentation type: Oral presentation OR054
- Presenter: Deirdre O’Connor, Ph.D.
- Date: Thursday, October 29 at 5:15 PM CET
Silencing of MAPT via AVB-406 in human iPSC-derived neurons: A translational framework for neurodegenerative disease
- Presentation type: Poster P0070
- Presenter: Martina Esposito Soccoio, Ph.D.
- Date: Thursday, October 29 from 2:00–4:00 PM CET
Leveraging the vMiX™ miRNA platform to silence alpha-synuclein (SNCA) as a therapeutic strategy for synucleinopathies
- Presentation type: Poster P0483
- Presenter: Guillaume Lo
- Date: Wednesday, October 28 from 1:30–3:30 PM CET
Evaluation of rationally designed synthetic promoters for expression in neurons and glial cells
- Presentation type: Poster P0012
- Presenter: Dan Cox, Ph.D. with Chromatin Bioscience
- Date: Thursday, October 29 from 2:00–4:00 PM CET
About AVB-406
AVB-406 is an investigational 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 capsids. This approach enables CNS-wide, durable reduction of pathological tau following a single dose. Expression is driven by a neuron-specific promoter to focus activity within neurons.
About AVB-SNCA
AVB-SNCA is being developed as a potential one-time, intravenously administered gene therapy designed to lower SNCA expression in the brain using AviadoBio’s proprietary vMiX™ RNAi gene silencing platform. Parkinson’s disease, Lewy body disease, and multiple system atrophy are progressive neurodegenerative conditions driven by the buildup of the alpha-synuclein protein, which is encoded by the SNCA gene. Collectively, these synucleinopathies affect over 9 million patients worldwide, and there are no disease-modifying treatments currently available.
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 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.
Contact
For AviadoBio Media Inquiries
Farah Speer
SVP, Head of Communications and External Relations
fspeer@aviadobio.com
+1-312-543-2881