GANX Profile
Gain Therapeutics, Inc. is a biotechnology firm specializing in the development of innovative therapies targeting diseases associated with protein misfolding. The company concentrates on addressing rare genetic disorders and neurological conditions, leveraging its proprietary Site-Directed Enzyme Enhancement Therapy (SEE-Tx) platform to advance its research and therapeutic solutions.
Gain Therapeutics employs its SEE-Tx technology to identify allosteric sites on misfolded proteins. By discovering and targeting these specific sites, the company aims to develop small molecules that can bind to these locations, thereby restoring proper protein folding and mitigating disease symptoms. This approach is designed to address a range of genetic and neurological disorders with unmet medical needs.
The company's development pipeline includes several promising drug candidates targeting various conditions. These include therapies for Morquio B syndrome, GM1 gangliosidosis, neuronopathic Gaucher disease, GBA1 Parkinson's disease, Krabbe disease, and Mucopolysaccharidosis type 1. Gain Therapeutics' focus on these rare and challenging diseases underscores its commitment to advancing treatments for conditions with limited therapeutic options.
Established in 2017 and headquartered in Bethesda, Maryland, Gain Therapeutics is at the forefront of applying cutting-edge biotechnology to address complex medical issues. The company's ongoing research and development efforts are supported by a team of experts dedicated to improving patient outcomes through targeted and effective therapeutic interventions.
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