Grant awarded to consortium led by Gain Therapeutics to advance the development of proprietary allosteric small molecule regulators against Alpha-1 Antitrypsin (AAT) Deficiency

Bethesda, Md. — Gain Therapeutics, Inc. (Nasdaq: GANX), a biotechnology company transforming drug discovery with its proprietary computational discovery platform identifying novel allosteric binding sites and creating small molecule treatments, announced that Eurostars and Innosuisse have awarded a grant in the aggregate amount of €1.2 million to a consortium led by Gain Therapeutics which includes the Institute for Research in Biomedicine, Newcells Biotech and the University of Helsinki. This grant supports a research project to develop novel small molecule allosteric regulators against Alpha-1 Antitrypsin (AAT) Deficiency, a rare genetic condition that can result in serious lung and liver diseases.

“We are pleased that Eurostars and Innosuisse recognize that Gain Therapeutics’ allosteric small molecule regulators provide a new approach to address AAT deficiency-related metabolic diseases. We look forward to advancing our AAT program with this consortium, which combines our unique targeting approach with novel in vitro and in vivo models. This grant validates the capability of our computational discovery platform SEE-Tx to identify previously unknown allosteric binding sites on protein targets in CNS, oncology and now metabolic disease,” said Dr. Manolo Bellotto, Chief Strategic Officer and General Manager at Gain Therapeutics.

Prof. Dr. Maurizio Molinari, group leader of the Protein Folding and Quality Control research team from the IRB Bellinzona and Adjunct Professor at the École Polytechnique Fédérale de Lausanne (EPFL) added, “The support by Eurostars and Innosuisse is a rewarding recognition for our long-standing activity in the field of rare diseases. The transnational collaboration with Gain Therapeutics, Newcells Biotech and the University of Helsinki will hopefully offer the opportunity to translate into the clinic, the research activity performed at the IRB and aims at understanding how perturbations in protein folding may cause severe diseases.”

Dr. Mike Nicholds, CEO of Newcells Biotech added, “Newcells is excited to contribute to this project and collaborate to advance an innovative therapy by applying our novel in-vitro stem cell tissue models of the liver and lung to enable efficacy and safety studies.”

Dr. Satu Kuure, Principal Investigator and Head of the GM-unit at University of Helsinki commented: “We are excited to participate in the Eurostars-3 project, where our task is to generate unique in-vivo models of A1AT deficiency manifesting both liver and lung symptoms. These models encompass the localized organ specific and systemic effects of A1AT deficiency. Consequently, these models will be used as a disease model where lead small molecules therapeutic power can be tested in a physiological setting.”

About Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin deficiency is a genetic disorder that affects the production of a protein called alpha-1 antitrypsin (AAT), which is mainly produced in the liver and released into the bloodstream. AAT plays an important role in protecting the lungs from damage caused by inflammation. In people with AAT deficiency, mutations of the SERPINA1 gene cause misfolding of AAT, which then cannot be effectively released into the bloodstream. As a result, there is a buildup of the misfolded protein in the liver cells, which can cause liver damage and may lead to liver disease. Additionally, insufficient amounts of AAT in the bloodstream can lead to lung damage and the development of chronic obstructive pulmonary disease (COPD). AAT deficiency occurs in approximately 100,000 people in the United States and may qualify as an orphan disease. Gain Therapeutics has applied its drug discovery platform SEE-Tx® to identify small molecules binding to a novel allosteric site on AAT to restore proper folding of the protein and halt the disease cascade leading to AAT deficiency and its associated diseases.

About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is transforming drug discovery with its proprietary computational discovery platform identifying novel allosteric binding sites and creating small molecule treatments to address unmet medical needs. The ability to identify never-seen-before allosteric targets on proteins involved in diseases across the full spectrum of therapeutic areas provides opportunities for a range of drug-protein interactions, including protein stabilization, protein destabilization, targeted protein degradation, allosteric inhibition, and allosteric activation. Gain’s pipeline spans neurodegenerative diseases, lysosomal storage disorders (LSDs), metabolic disorders, as well as other diseases that can be targeted through protein degradation, such as oncology. Gain’s lead program in Parkinson’s disease has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as funding support from Eurostars-3 which is part of the European Partnership on Innovative SMEs: the partnership is co-funded by the European Union through Horizon Europe and Innosuisse – Swiss Innovation Agency and SERI – The State Secretariat for Education, Research and Innovation. For more information, please visit

About Institute for Research in Biomedicine (IRB)
The Institute for Research in Biomedicine was founded in 2000 with the clear and ambitious goal of advancing the study of human immunology, with particular emphasis on the mechanisms of host defense. The activities of the 13 research groups now extend beyond immunology to include the fields of DNA repair, rare diseases, structural and cell biology. Located in Bellinzona, capital of the Italian-speaking Canton of Ticino, the IRB is an affiliated institute of the USI Faculty of Biomedical Sciences. For more information, visit:

About Newcells Biotech
Newcells Biotech Ltd applies its expertise and proprietary technologies in stem and primary cell biology to develop and commercialize micro-physiological systems (MPS) that closely mimic human and other species in vivo biology. Newcells unique models of the kidney and retina comprise a comprehensive suite of in-vitro MPS based assays that provide predictive insights on efficacy, safety and pharmacokinetics. The recently launched lung tissue fibrosis assay is part of a range of pulmonary models for disease modelling created by the development team that has also developed an advanced in vitro liver model.

About University of Helsinki
The University of Helsinki is one of the world’s leading multidisciplinary research universities, renowned for its high-quality teaching, research and innovation. It is proud to be the only Finnish university constantly ranked among the top one hundred best universities in the world. The University of Helsinki has a long standing commitment for sustainability, responsibility and reflected in the motto: With the Power of Knowledge. Established in 1640, the University of Helsinki is the oldest university in Finland.

About Eurostars
Eurostars is a part of the Horizon Europe program that supports SMEs and project partners (universities, research organizations and other types of organizations) by funding international collaborative R&D and innovation projects. Eurostars is run by EUREKA, an intergovernmental network, which involves 37 countries.