As a result, many living with these conditions continue to face ongoing symptoms and debilitating side effects that significantly impact their lives.

For epilepsy, about one-third of patients are unable to achieve seizure control with available medications. For these patients, uncontrolled seizures can affect every aspect of daily life, increasing the risk of injury, cognitive difficulties, social isolation, and sudden unexpected death in epilepsy (SUDEP).

For Parkinson’s disease, most patients eventually develop symptoms that current therapies cannot fully manage, resulting in a gradual loss of mobility, independence, and quality of life.

MicroRNAs (miRNAs) are small regulatory molecules that control how genes are expressed in cells.

They fine tune protein production by regulating other RNA molecules. In the brain, miRNAs play a critical role in coordinating how neurons develop, adapt, and communicate with one another.

Increasing evidence shows that dysregulated miRNA activity is a feature of several neurological disorders.

Unlike most drugs, which act on a single target, each miRNA can regulate entire networks of genes involved in the same biological function. This makes miRNAs particularly relevant in disease driven by multiple converging processes.

In epilepsy, for example, miRNAs regulate networks of genes related to synaptic signaling and plasticity, affecting how nerve cells communicate and respond to activity.

In neurodegenerative diseases like Parkinson’s, miRNAs regulate networks of genes involved in oxidative stress, mitochondrial health, and neuroinflammation. When these processes are disrupted, they can contribute to the progressive loss of brain cells.

Antisense oligonucleotides (ASOs) are short, chemically modified strands of genetic material designed to bind and inhibit specific RNA sequences with high precision.

Our strategy is to selectively inhibit miRNAs that are abnormally overactive in disease, while preserving normal cellular and neuronal function.

This targeted approach is designed to correct dysregulated neuronal networks without broadly suppressing brain activity.

Among the hundreds of miRNAs expressed in the brain, microRNA 134 (miR 134) has emerged as a particularly well characterized regulator of neuronal excitability.

Extensive research has shown that miR-134 is elevated in epileptic brain tissue and that increased miR-134 activity contributes to heightened neuronal excitability.

Since its initial description in 2006, miR 134 has been widely studied in both experimental models and human tissue. Multiple independent studies have demonstrated that inhibiting miR 134 can reduce seizure activity through mechanisms distinct from those of existing anti seizure medications.

This body of evidence makes miR 134 one of the most compelling therapeutic targets in drug resistant epilepsy.