Epygenix, Therapeutics, Inc. is an advanced clinical-stage, precision-medicine biopharmaceutical company in the US focused on developing drugs to treat rare and intractable forms of genetic epilepsy in childhood, such as Dravet Syndrome(DS) and Lennox Gastaut Syndrome(LGS).

Polaryx, located in New Jersey, is a life science-based clinical biopharmaceutical company primarily focused on developing treatments for rare diseases, particularly in the field of brain neurodegenerative disorders. Specifically, they are developing a new drug to treat lysosomal storage disorders (LSD), a rare genetic disease. They are researching three candidate compounds (PLX-100, PLX-200, PLX-300).

PLX-200 and PLX-100 target a rare genetic disease called neuronal ceroid lipofuscinosis (NCL), also known as Batten disease, which leads to mental impairment, motor function decline, vision loss, seizures, and early death. These drugs represent the first non-invasive oral treatment for patients with NCL.

PLX-300 is focused on lysosomal storage disorders such as Batten disease, Krabbe disease, Tay Sachs/Sandhoff disease, Niemann-Pick Type A/B, and others.

Estimating the number of patients in the United States and major European countries, there are approximately 1,200 CLN3 patients (market value of $419 million), about 2,800 overall CLN patients (market value of $1 billion), and around 24,000 LSD patients (market value exceeding $2 billion). Due to the lack of available treatments for these diseases, there is a significant demand for therapy development, driving Polaryx's research and development efforts.