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Our Therapeutic Focus

What We Do

Our lead clinical-stage candidates are focused on treating orphan diseases in metabolic and neurological settings, followed by expansion into larger disease populations. Some examples of our lead programs include:

Orphan Disease – Myotonic Dystrophy
Myotonic Dystrophy (DM) is the most common genetic disease causing muscle degeneration (also known as muscular dystrophy). The genetic defect in DM is complex and leads to many down-stream consequences. The most frequent effects of DM are muscle weakness, myotonia (the inability to relax muscle) and muscle atrophy (muscle wasting that worsens over time). DM also affects other organs, including the brain, heart, lungs and gastrointestinal system.

There is a very high unmet medical need in DM and no currently approved therapies. Our candidates can deliver a specific amino acid profile that has the potential to restore function, ameliorate or improve symptoms and potentially benefit patients with DM.

Orphan Disease – Intractable (drug-resistant) Epilepsy Syndromes
Excessive and abnormal brain cell activity leads to seizures that can greatly disrupt a person’s life, causing physical injury and even death. About one-third of people with epilepsy are being treated with medicines that do not work well, or at all, to control their seizures.

We are developing proteins and peptides as amino acid delivery vehicles that have shown the potential to treat intractable seizures by promoting the normalization of excessive brain signaling. This leads to a significant reduction in the number and duration of seizures.

Subpopulation – Pediatric NAFLD/NASH
Nonalcoholic fatty liver disease (NAFLD) is a disease in which excessive fat accumulates in the liver of a patient with no history of alcohol abuse. This disease includes simple steatosis and nonalcoholic steatohepatitis (NASH). NAFLD/NASH is recognized as a hepatic manifestation of metabolic syndrome. In recent years, pediatric NAFLD has increased in line with the rise of pediatric obesity.

In NASH models our lead candidates demonstrate safety and improvement in multiple markers of the disease.

Large population – Sarcopenia
Sarcopenia is defined as aging and chronic disease-related skeletal muscle loss and dysfunction. Starting around age 25, skeletal muscle mass begins to decline. Progressive muscle loss in sarcopenia correlates with co-morbidities including physical disability, increased likelihood of falls, poor quality of life and death. There is currently no approved treatment for sarcopenia.

We are developing specific proteins that have been shown to combat muscle loss by maximizing skeletal muscle anabolism.

Other Disease Populations
We have pioneered computational, biophysical and systems medicine studies to establish the central role amino acid biology disruptions play in disease. More than 2,000 diseases have been linked to amino acid imbalances, providing a tremendous breadth of opportunity to expand our pipeline and transform lives.