Gain Therapeutics Announces Grant Award by Eurostars with Innosuisse for Alpha-1 Antitrypsin Deficiency Research Program
Gain Therapeutics (Nasdaq: GANX) has received a grant totaling €1.2 million from Eurostars and Innosuisse to develop small molecule regulators for Alpha-1 Antitrypsin (AAT) Deficiency. This rare genetic disorder can result in serious lung and liver diseases. The grant will fund collaborative research involving the Institute for Research in Biomedicine, Newcells Biotech, and the University of Helsinki. Gain’s proprietary platform, SEE-Tx, aims to identify allosteric sites and enhance therapeutic strategies for metabolic diseases. This recognition marks a significant validation of Gain’s innovative approach in addressing rare diseases.
- Awarded a grant of €1.2 million, highlighting financial support for AAT deficiency research.
- Involvement of reputable institutions, indicating strong collaborative effort and expertise.
- Validation of Gain's computational platform SEE-Tx for identifying novel therapeutic targets.
- None.
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., March 21, 2023 (GLOBE NEWSWIRE) -- 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, today announced that Eurostars and Innosuisse have awarded a grant in the aggregate amount of
“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 https://www.gaintherapeutics.com.
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: www.irb.usi.ch
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.
Cautionary Note Regarding Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases, you can identify these statements by forward-looking words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "predict," "goal, " "intend," "seek, " "potential" or "continue," the negative of these terms and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. All statements, other than historical facts, included in this press release are forward-looking statements. These statements include, but are not limited to, statements regarding: the development of the Company’s current or future product candidates; expectations regarding timing for reporting data from ongoing preclinical studies or the initiation of future clinical trials, including the timing for completion of IND-enabling toxicology studies and submission of the dossier requirement for commencement of a Phase 1 clinical program for GT-02287 for GBA1 Parkinson’s disease and the potential therapeutic and clinical benefits of the Company’s product candidates; the selection and development, and timing thereof, of future programs, or any potential business development opportunities for product candidates; the Company’s financial position and ability to execute on the next phase of its strategy; and the Company’s anticipated cash runway guidance, including the ability for the Company’s current and projected cash to allow the Company to meet value inflection points. Each of these forward-looking statements involves risks and uncertainties that could cause the Company’s preclinical and future clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. Many factors may cause differences between current expectations and actual results, including the impacts of the COVID-19 pandemic and other global and macroeconomic conditions on the Company’s business; clinical trials and financial position; unexpected safety or efficacy data observed during preclinical studies or clinical trials, clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; the uncertainties and timing of the regulatory approval process; and unexpected litigation or other disputes. Other factors that may cause the Company’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are identified in the sections titled “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in the Company’s Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission from time to time. New risks and uncertainties arise over time, and it is not possible for us to predict all such factors or how they may affect us. You should not place undue reliance on forward-looking statements. All information in this press release is as of the date of the release, and we are under no duty to update this information after the date of this release, except as required by law. You should not rely on it as representing our views as of any date subsequent to the date of this press release.
Investor & Media Contact:
Argot Partners
Noor Pahlavi
(212) 600-1902
Gain@argotpartners.com
FAQ
What is the purpose of the grant awarded to Gain Therapeutics?
How much funding did Gain Therapeutics receive for their project?
Which organizations are involved in the consortium led by Gain Therapeutics?
What condition is Gain Therapeutics targeting with their research?
What is the significance of the SEE-Tx platform for Gain Therapeutics?
