Pioneering a New Path for Rare CNS Disorders

Zy Therapeutics is developing ZY-001, the first potential disease-modifying therapy to directly target brain calcifications — an overlooked driver of seizures and cognitive decline.

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Our Focus

Targeting the Root Cause, Not Just the Symptoms

At Zy Therapeutics, we are a rare disease biotech company focused on a critical unmet need: intracranial calcifications. These “brain stones” are calcium-phosphate deposits that disrupt normal neuronal signaling, leading to seizures, developmental challenges, and cognitive decline in devastating rare CNS disorders.

Our mission is to transform brain calcification from an untreatable symptom into a treatable target.

Our Solution

Introducing ZY-001: A First-in-Class Approach

We are advancing ZY-001 (disodium etidronate), a novel, CNS-targeted formulation of a proven molecule. By re-engineering this compound for enhanced absorption and brain delivery, ZY-001 is designed to directly chelate excess calcium and normalize tissue mineral balance, offering the first potential disease-modifying treatment for conditions driven by pathological calcium buildup.

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Who We Serve

Addressing Devastating Rare Diseases

Our initial focus is on rare neurological disorders where brain calcification is a primary driver of disease burden.

Sturge-Weber Syndrome (SWS)

Targeting calcifications linked to seizures and cognitive decline.

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Tuberous Sclerosis Complex (TSC)

Addressing calcified tubers that contribute to drug-resistant epilepsy.

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Neurocysticercosis

Neutralizing calcified lesions that remain a source of seizures.

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Fahr’s Disease (PFBC)

Aiming to slow or reverse progressive movement and cognitive symptoms.

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Our Commitment

Science with Purpose, Medicine with Meaning

Our team brings over 100 years of combined expertise in rare CNS disorders, clinical epilepsy research, and translational neuroscience. As scientists and clinicians, we are driven by a single goal:  to turn scientific insight into life-changing therapies for those who currently have none.

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Advancing Our Science and Engaging Our Community

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