Our Science

What does it take to address the complexity of neurological diseases?

Broad and deep CNS distribution

Designed for broad CNS exposure, with penetration into deep brain regions where many neurological diseases occur.

What It Enables

Translates best-in-class biodistribution into broad biological effects on disease-relevant genes throughout the brain.

Robust and selective knockdown

Designed to achieve potent target suppression across key CNS regions.

What It Enables

Enables targeting of specific genetic drivers of disabling neurological diseases with a high level of precision.

Durable Effect

Designed for longer duration.

What It Enables

Creates the potential for long-lasting effect in diseases that progress over years, and enables an infrequent clinical dosing regimen.

Neurological diseases can involve different regions, targets, and pathways. Arexera is developing medicines designed to address that complexity with a combination of precision, reach, and durability.

The Arexera Platforms

Platforms designed for best-in-class biodistribution in the CNS, target mRNA reduction and duration of RNAi activity.

di-siRNA

Divalent siRNA architecture deployed against a single gene.

bi-siRNA

Next-generation siRNA architecture for bispecific targeting, with enhanced pharmaceutical properties.

Explore the pipeline

From CNS reach to neurotherapeutic breadth

A platform approach designed to support multiple programs across neurological diseases.

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Multiple regions

Designed to reach disease-relevant CNS regions while maintaining target-specific silencing of the mechanisms implicated in disease progression.

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Multiple targets

Targeted siRNA enables specific silencing across distinct disease-driving or disease-modifying pathways.

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Multiple Programs

Building from Huntington’s disease toward a broader pipeline in neurological disease.

Huntington’s Disease

A therapeutic approach built around HTT, the primary genetic driver of HD, and MSH3, a key modifier of disease onset and progression.

Broader Pipeline

Additional programs extend the platform into other neurological diseases.

Explore the pipeline