Tauopathies are a group of neurodegenerative diseases caused by the abnormal accumulation of the protein tau inside neurons. Tau normally stabilizes microtubules—structures essential for neuron health and communication—but when it misfolds and aggregates, it disrupts cellular function, leading to neuronal death and brain atrophy.

Primary tauopathies include conditions in which tau aggregation is the main driver of disease, such as:

* Progressive Supranuclear Palsy (PSP)
* Frontotemporal Dementia (FTD, including Pick’s disease)
* Corticobasal Degeneration (CBD)

Secondary tauopathies, like Alzheimer’s Disease, involve tau pathology alongside other factors such as amyloid-β accumulation.

Together, tauopathies affect over 55 million people worldwide, including approximately 40 million with Alzheimer’s disease, 10 million with Frontotemporal Dementia, and up to 3 million with Progressive Supranuclear Palsy, Corticobasal Degeneration, and related disorders.

Depending on the brain regions affected by tau accumulation, these conditions lead to progressive cognitive impairment, memory loss, behavioral and personality changes, language difficulties, and motor dysfunction, severely impacting patients’ quality of life. Despite this heavy burden, no disease-modifying therapies currently exist.

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At miRNOVA Therapeutics, we believe the best way to treat tauopathies is not by suppressing tau across the entire brain, but by selectively reducing its accumulation in the regions truly affected by the disease.

Our approach is based on the principle that tau has essential physiological functions — global reduction can disrupt healthy neuronal activity and cause unwanted side effects.

Using our engineered microRNA platform, we achieve precise, localized tau reduction, restoring neuronal health where pathology occurs while preserving normal brain function elsewhere.

At miRNOVA Therapeutics, we harness the power of engineered microRNAs (miRNAs) to precisely and safely silence disease-driving genes in the brain.

Our artificial microRNAs are inspired by naturally occurring miRNAs, but rationally redesigned to selectively bind and reduce the messenger RNA (mRNA) of MAPT, the gene that encodes the tau protein.

To achieve spatial precision, these therapeutic miRNAs are expressed under cell-specific promoters and delivered through vectors that can be directed to affected brain regions, ensuring local efficacy while preserving healthy areas.

This approach enables fine-tuned, regionally targeted tau reduction—a new paradigm for treating tauopathies with high efficacy and minimal risk.

This regionally targeted therapy represents a paradigm shift: high efficacy with minimal risk.
 
 

We have demonstrated clear target engagement of our compound, confirming its direct interaction with the intended molecular pathway. Importantly, treatment led to up to 70% reduction in pathological tau, a critical hallmark of neurodegeneration. In addition, our compound achieved phenotypic rescue in mouse models, improving both cognitive and motor deficits, highlighting its potential functional impact.

We create value by developing novel, disease-modifying therapies with strong preclinical evidence of efficacy, targeting key mechanisms in neurodegeneration. Our compounds demonstrate robust target engagement, significant pathological tau reduction, and functional rescue in relevant models, setting us apart in a competitive landscape.

We protect this value through astrong intellectual property strategy, including patents covering composition of matter, methods of use, and proprietary technologies. This, combined with ourdeep scientific know-howand ongoing innovation, ensures sustainable competitive advantage and maximizes potential for partnerships and future commercialization.

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