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Gain Therapeutics Priced, Nasdaq: GANX

Preclinical biotech developing small molecule therapies for lysosomal storage disorders.

Industry: Health Care

First Day Return: +1.8%

Industry: Health Care

We are a development stage biotechnology company developing novel therapeutics to treat diseases caused by protein misfolding, with an initial focus on lysosomal storage disorders (“LSDs”), including rare genetic diseases and neurological disorders. We use our exclusively in-licensed proprietary platform, Site-Directed Enzyme Enhancement Therapy (“SEE-Tx”), to discover novel allosteric sites on misfolded proteins and identify proprietary small molecules that bind these sites and restore protein folding, potentially treating the underlying disease. These small molecule binding sites, away from the protein’s active areas, are called allosteric sites. We believe targeting the allosteric binding site instead of the active binding site can provide a number of advantages: superior regulation of misfolded proteins implicated in disease, enhanced specificity by being non-competitive with the natural substrate and the potential for molecules with favorable drug-like properties. While the SEE-Tx platform is novel and has not been used to develop approved drugs yet, these advantages have the potential to ultimately enhance both tolerability and response. We expect to obtain additional preclinical data and commence IND-enabling studies for lead compound candidates beginning 2021, at which point we will begin the process to prepare and submit IND submissions to the FDA.
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Gain Therapeutics (GANX) Performance