Biopharmaceutical company developing novel therapies using proprietary drug discovery technologies.
Gain Therapeutics, Inc., a biotechnology firm, specializes in the development of therapies aimed at treating diseases attributed to protein misfolding. With a primary focus on rare genetic disorders and neurological conditions, the company leverages its innovative Site-Directed Enzyme Enhancement Therapy platform. This proprietary technology enables Gain Therapeutics to pinpoint allosteric sites on misfolded proteins and identify unique small molecules capable of binding to these sites. By restoring proper protein folding, these molecules have the potential to effectively treat a range of diseases.
Gain Therapeutics is actively advancing its pipeline of structurally targeted allosteric regulator candidates designed to address various medical conditions. These include Morquio B syndrome, GM1 gangliosidosis, neuronopathic Gaucher disease, GBA1-linked Parkinson's disease, Krabbe disease, and Mucopolysaccharidosis type 1. By harnessing molecular insights and therapeutic approaches, the company aims to introduce transformative treatments that address critical unmet medical needs within these challenging disease areas.
Established in 2017 and headquartered in Bethesda, Maryland, Gain Therapeutics continues to drive innovation in biotechnology with a dedicated focus on advancing novel therapies. The company's commitment to leveraging cutting-edge science and its strategic location within the biotechnology hub of Bethesda underscores its mission to deliver meaningful advancements in medical treatment modalities.