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Gain Therapeutics, Inc. (NASDAQ: GANX) is a pioneering biotechnology company committed to the discovery and development of novel small molecule therapeutics. Specializing in addressing rare genetic diseases, Gain Therapeutics focuses on orphan diseases caused by inborn errors of metabolism. With a strong emphasis on lysosomal storage disorders, central nervous system disorders, metabolic disorders, and oncology, the company's innovative approach aims to provide solutions where there is a high unmet medical need.
At the forefront of Gain Therapeutics' pipeline is GT-02287, a promising clinical-stage product candidate targeting GBA1 Parkinson's disease. Currently in Phase 1 clinical trials, GT-02287 is designed to be a disease-modifying treatment. It functions as an allosteric protein modulator to restore the activity of the lysosomal enzyme glucocerebrosidase (GCase), tackling the underlying cause of disease rather than just alleviating symptoms.
The company's proprietary Magellan™ platform leverages AI-supported structural biology and advanced algorithms to discover novel allosteric binding sites on disease-implicated proteins. This innovative computational technology identifies potential small molecules that can bind to these sites, offering new avenues for therapeutic development. Magellan builds upon Gain’s original SEE-Tx® platform, enhancing it with AI and virtual screening capabilities to explore vast chemical spaces.
Recent achievements highlight Gain Therapeutics' impact on the biotech landscape. Data presented at the 20th WORLDSymposium™ showcased the progressive reversal of motor deficits in preclinical models, alongside a significant reduction of neurodegeneration biomarkers. This underscores the potential of GT-02287 as a best-in-class treatment for neurodegenerative diseases.
Gain Therapeutics collaborates with notable organizations including The Michael J. Fox Foundation for Parkinson’s Research and The Silverstein Foundation for Parkinson’s with GBA. These partnerships provide essential support and validation for the company's mission to transform therapeutic landscapes with innovative treatments.
For more information, visit GainTherapeutics.com and follow their updates on LinkedIn.
Gain Therapeutics (GANX) reported a net loss of $3.27 million for Q1 2022, improving from a loss of $2.54 million in Q1 2021. Revenue increased to $45,000, driven by collaboration agreements. R&D expenses rose to $1.56 million, while general and administrative costs surged to $1.78 million. The company holds $34.34 million in cash, providing operational runway into the latter half of 2023. Notable preclinical data was presented for its lead candidate GT-02287 targeting Parkinson’s and Gaucher's diseases, suggesting its potential therapeutic benefits.
Gain Therapeutics (Nasdaq: GANX) showcased promising data for its lead compound GT-02329 targeting Gaucher disease at the 1st IWGDD Symposium. The compound boosts GCase levels and activity, effectively reducing toxic substrate accumulation in both human and mouse models. Specifically, the data highlighted increased GCase activity and decreases in glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph). The company aims to further investigate GT-02329's potential as a therapy to alleviate neurological effects associated with Gaucher disease.
Gain Therapeutics, Inc. (Nasdaq: GANX) has announced promising pre-clinical results for its lead compound, GT-02287, targeting Parkinson’s Disease. Presented at the XXVII World Congress on Parkinson’s Disease, the compound successfully penetrates the brain, reduces alpha-synuclein levels, and improves dopamine production in neuronal models. Data showcases its ability to bind with the GCase protein, enhancing lysosomal function. The findings suggest potential disease-modifying effects, positioning GT-02287 as a candidate for clinical advancement this year.
Gain Therapeutics (Nasdaq: GANX) announced that CEO Eric Richman will participate in a fireside chat at the B. Riley Securities 2022 Virtual Neuro & Ophthalmology Conference on April 28, 2022, from 2:00 to 2:30 p.m. ET. Investors are invited to join via webcast. Gain Therapeutics specializes in drug discovery using its proprietary computational platform, SEE-Tx®, targeting allosteric regulators for various diseases. The company’s lead program focuses on Parkinson’s disease, supported by funding from notable organizations.
Gain Therapeutics (Nasdaq: GANX) presented promising pre-clinical data for its lead candidate GT-02287 at the 2022 Synuclein Meeting in Belgium. The compound enhances GCase levels, promotes lysosomal health, and depletes alpha-synuclein, indicating potential benefits for Parkinson's Disease patients. Key findings include increased GCase activity and neuron survival in relevant models. CEO Eric Richman emphasized the candidate's disease-modifying potential, while the data supports a therapeutic strategy targeting GCase for neurodegenerative diseases.
Gain Therapeutics (Nasdaq: GANX) announced promising pre-clinical data for its Gaucher Disease program, presented at the 2022 Glycolipid and Sphingolipid Biology GRC. The study highlighted that lead compounds significantly increase the levels and activity of the beta-glucocerebrosidase (GCase) protein, enhancing its co-localization with lysosomes and reducing toxic substrate accumulation in neurons. These orally bioavailable compounds may offer a potential treatment for patients with neuronopathic Gaucher disease, addressing a significant unmet medical need.
Gain Therapeutics, Inc. (Nasdaq: GANX) announced significant progress in its lead program for GBA1 Parkinson’s Disease, highlighted by compelling preclinical data and a strong cash position of $36.88 million as of December 31, 2021. The company plans to initiate a first-in-human clinical trial by the end of 2022, following their collaboration with Zentalis Pharmaceuticals to identify novel oncology molecules. Despite increased operating expenses leading to a net loss of $13.89 million, the increase in revenue to $165,000 indicates growing traction in their development efforts.
Gain Therapeutics (Nasdaq: GANX) presented new pre-clinical data at the International Conference on Alzheimer’s and Parkinson’s Diseases demonstrating the potential of their STAR small molecules in treating neurodegenerative diseases. The data shows that enhancing glucocerebrosidase (GCase) activity improves key pathological features linked to Parkinson’s and Alzheimer’s. Results from both Parkinson’s and Alzheimer’s models indicated reductions in neurotoxicity, inflammation, and toxic proteins, suggesting a promising therapeutic avenue for CNS diseases caused by protein misfolding.
Gain Therapeutics (NASDAQ: GANX) announced its participation in key investor and scientific conferences, emphasizing its commitment to neurodegenerative disease research. CEO Eric Richman will present at the Oppenheimer 32nd Annual Healthcare Conference on March 17, 2022, with a webcast available for investors. Additionally, Gain will showcase scientific data at the AD/PD™ 2022 Conference, running from March 15-20, 2022, and sponsor the Krabbe Translational Research Network Meeting from March 15-18, 2022. The company focuses on innovative treatments using its SEE-Tx™ platform.
Gain Therapeutics (NASDAQ: GANX) has unveiled promising pre-clinical data on its Gaucher Disease program at the 18th Annual WORLDSymposium. The study demonstrated that its STARs compounds significantly increase GCase protein levels, diminish inflammatory cytokines, and enhance lysosomal functions in GD-iPSC-derived dopaminergic neurons. These findings validate the company’s drug discovery platform, SEE-Tx®, and indicate potential for a first-in-class therapy. Gain aims to commence IND-enabling studies in Q4 2022, addressing unmet needs in neuronopathic Gaucher Disease treatment.
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