We are a a biotechnology company focused on developing targeted therapies for orphan genetic diseases.

Valerion Therapeutics is utilizing a proprietary antibody-mediated delivery platform to target therapies to specific tissues via a well-known and broadly studied transport pathway.This platform is capable of enhanced intracellular delivery of a variety of active therapeutic payloads via a transport mechanism that is present in muscle and neurons and elevated in certain types of cancer. Pipeline candidates include therapeutic agents aimed at addressing a host of orphan genetic disorders with limited or no current therapies.

What is Pompe Disease?

Pompe disease is a rare multisystem genetic disorder that is characterized by absence or deficiency of the lysosomal enzyme alpha-glucosidase (GAA). This enzyme is required to breakdown (metabolize) the complex carbohydrate glycogen and convert it into the simple sugar glucose.

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What is Lafora Disease?

Lafora disease is an inherited, severe form of progressive myoclonus epilepsy. The condition most commonly begins with epileptic seizures in late childhood or adolescence. Other signs and symptoms include difficulty walking, muscle spasms (myoclonus) and dementia.

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What is Myotubular Myopathy?

X-linked myotubular myopathy is a condition that primarily affects muscles used for movement (skeletal muscles) and occurs almost exclusively in males. People with this condition have muscle weakness (myopathy) and decreased muscle tone (hypotonia) that are usually evident at birth.

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What is Myotonic Dystrophy?

The most common form of muscular dystrophy that begins in adulthood. Myotonic dystrophy is characterized by progressive muscle wasting and weakness. People with this disorder often have prolonged muscle contractions (myotonia) and are not able to relax certain muscles after use.

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Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease

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Valerion Reports Preclinical Potential for Pompe Treatment

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Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease

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Valerion Technology

Valerion is able to generate proprietary product candidates utilizing substrates (proteins, enzymes, oligonucleotides, etc.) not previously deliverable to target tissues (ex. heart & skeletal muscle fibers); thereby, providing a novel way to treat a number of diseases with limited or no current therapeutic options.

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