Gain Therapeutics
Unveiling New Treatments With its Drug Discovery Platform

Today, the biotech industry embraces innovative approaches and technologies to introduce ground-breaking treatments to combat severe and rare diseases. The industry is specifically benefiting from advanced computing technologies that allow businesses to expand the scope and scale of their research and discover new treatments. A pioneer that is making a huge difference in this segment is Gain Therapeutics. The company offers a robust platform that identifies novel allosteric binding sites in a fraction of time and targets never described before. Gain Therapeutics is the first company to successfully combine advancements in computational biology, structural biology, and supercomputers to develop drugs. Using these advancements, the company unlocks new treatments for difficult-to-treat disorders characterized by protein misfolding, including lysosomal storage disorders. Gain Therapeutics started predicting novel binding sites on protein structures a decade ago when no one else was even remotely planning to do it. “We are one of the most experienced companies to integrate structural biology and supercomputing to develop novel drugs for patients suffering from diseases with inadequate or no treatments,” remarks Eric Richman, CEO at Gain Therapeutics.

Gain Therapeutics started its journey when a drug discovery technology was developed for predicting druggable protein sites by a professor at the University of Barcelona. The technology was licensed to a clinical-stage biotech company, Minoryx Therapeutics, further developing the platform. It, however, required additional funding and more recognition. Hence, Minoryx was spun off into an independent firm, which marked the beginning of Gain Therapeutics and its supercomputing-driven drug discovery platform.

Gain Therapeutics’ technology analyzes molecules to uncover structures that fit the allosteric binding sites. “We named our company Gain Therapeutics because we were able to identify binding sites, develop small molecule compounds that bind to them, restore protein function, and gain enzyme function, which is one of the most significant aspects of our strategy,” mentions Eric.

Gain Therapeutics is a trailblazer in identifying protein structures and developing innovative medicines for diseases with inadequate treatments.

The company’s drug discovery platform has produced an internal pipeline of preclinical products. Gain Therapeutics prioritizes lysosomal storage disorders and central nervous system disorders in its product pipeline because of the ability for the small molecules to cross the blood brain barrier and enter the central nervous system. Furthermore, the organization also focuses on Gaucher disease, which is linked to Parkinson’s disease by virtue of a genetic mutation. This is one of the first approaches to addressing a specific group of Parkinson’s patients using innovative technology. After decades of trying various treatments for Parkinson’s disease, it wasn’t possible until the company combined two areas, massive supercomputers and big data, with the advancements in structural biology. It allowed the company to identify new ways of treating diseases, which is its lead program.

While large pharmaceutical corporations take years to explore new targets and techniques to cure diseases, Gain Therapeutics identifies allosteric binding sites in three to four weeks, bringing the drug discovery process down from years to a few weeks. This results not only in reduced costs but also a faster time-to-market. Considering these factors, it certainly is a technology that can be transformational to the pharmaceutical industry and bring forth new treatments for incurable diseases.

Since its inception, Gain Therapeutics has been working consistently towards its objectives to get the key programs into clinical studies in 2022 and establish high-value partnerships. Today, the organization’s major focus is on treating central nervous system disorders linked to lysosomal storage disorders. In the future, the company intends to grow its internal pipeline while focusing on identifying new targets outside the central nervous system because these are the regions with significant unmet medical needs. “I envision us as a fully integrated pharmaceutical firm with exceptional drug development and research skills as well as a reliable partner for pharmaceutical companies in the future,” concludes Eric.