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GANX-Investor-Factsheet-Q1-2023

Discovering treatments for patients that currently have no available disease-modifying options is the priority of Gain Therapeutics. Specifically, there is a large unmet need for patients suffering from protein misfolding disorders that alter enzyme function.<br><br>https://perspectives.gaintherapeutics.com/combining-physics-biochemistry-and-supercomputing-how-gain-therapeutics-is-revolutionizing-drug-discovery/

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GANX-Investor-Factsheet-Q1-2023

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  1. FACTSHEET | Q1 2023 Leading the Discovery of New Therapies with Allosteric Modulators We are a biotechnology company utilizing our proprietary computational drug discovery platform SEE-Tx® to target unknown allosteric binding sites with small molecules for the treatment of devastating diseases with high unmet medical need. SEE-Tx® Drug Discovery Platform Using AI-supported structural biology and our supercomputer-powered physics-based methods, SEE-Tx® radically transforms and accelerates drug discovery. EQUITY OVERVIEW GANX (NASDAQ-GM) Ticker (Exchange) Stock Price (USD) $3.82 Market Cap ~$45M Cash ~$25.7M 1 2 Outstanding Shares 11.88M Fast – SEE-Tx® enables the identification of novel protein binding sites and hit compounds for experimental testing in 4-6 weeks. 52-Week Range $2.33 - $6.70 Avg. Daily Volume ~34,000 Versatile – SEE-Tx® can be applied in any therapeutic area and generates small molecule hits that can elicit either a gain of function or a loss of function of the target protein through the full range of possible compound-protein interactions, including stabilization, activation, inhibition, degradation or destabilization. BTIG –Thomas Schrader H.C. Wainwright - Raghuram Selvaraju Oppenheimer –Hartaj Singh Chardan–KeayNakae Analyst Coverage Powerful – SEE-Tx® expands the target universe to the 90% of protein targets that have so far been undruggable and generates a more than 100-fold greater output of chemically diverse hit compounds compared to traditional high-throughput screening. Our Lead Program and Pipeline Our lead program is a brain penetrant small molecule that restores the enzymatic function of misfolded GCase and enables the protein to eliminate the build-up of toxic substrates, which if left unchecked, will cause neuronal cell death. Due to this mechanism of action, the lead candidate has the potential to have a disease-modifying effect in GBA Parkinson’s disease, a genetically defined subset of Parkinson’s that accounts for ~15% of PD patients, and in neuronopathic Gaucher disease, a rare childhood disease with no available treatments. The lead candidate was shown to have a positive impact preclinically in disease-relevant animal models, including restoration of locomotion deficits, and is expected to enter the clinic in 2023. All figures as of 01/20/2023 unless otherwise noted (Source: NASDAQ) 1 Cash, cash equivalents and marketable securities as of 9/30/2022 2Form 10-Q Filed on November 10, 2022 INVESTMENT HIGHLIGHTS SEE-Tx® platform accelerates drug discovery from 2+ years to 2-3 months and enabling targeting of all human proteins Lead program with disease-modifying potential in GBA-PD and GD entering the clinic in 2023 Highly experienced management team with extensive biotech and pharmaceutical experience UPCOMING MILESTONES THERAPEUTIC AREA INDICATION GENE DISCOVERY RESEARCH PRECLINICAL PHASE 1 Neurodegenerative Diseases Parkinson’s Disease GBA1 GBA1 Program (PD and GD) Presentation of data at key data conferences (1H 2023) Completion of GLP Tox. Studies (1H 2023) HREC Submission (Mid 2023) Phase 1 Study Initiation (2H 2023) - Lysosomal Storage Disorders Gaucher Disease GBA1 GM1 Gangliosidosis GLB1 - - - Mucopolysaccharidosis Type 1 IDUA Krabbe Disease GALC Pipeline Metachromatic Leukodystrophy ARSA - Identify Lead Series for GALC program in Krabbe disease (1H 2023) Identify Lead Series for 2 solid tumor programs (2H 2023) Metabolic Diseases Liver/Lung UNDISCLOSED - Oncology Solid Tumors UNDISCLOSED CONTACT Gain Therapeutics, Inc. 4800 Montgomery Lane, Ste. 220 Bethesda, MD 20814 P: +1 301-500-1556 www.gaintherapeutics.com Investor Contact Argot Partners - Noor Pahlavi P: +1 301-641-2525 E: gain@argotpartners.com Solid Tumors UNDISCLOSED Our Strategy for Value Creation We create intrinsic value in Gain by progressing our differentiated pipeline programs and by applying SEE-Tx® to generate new programs in oncology. addition, we are focused on generating non-dilutive cash in-flow through partnering of pipeline programs, research collaborations applying SEE-Tx® to targets of interest to our collaboration partners. In © 2023 - GAIN THERAPEUTICS Non-Confidential Factsheet

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