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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 (NASDAQ: GANX) reported its Q3 2021 financial results and announced key corporate developments. The company has $40.98 million in cash, providing a strong financial runway into the second half of 2023. Significant progress was made in patient-derived iPSC studies, showing STAR compounds positively affecting Gaucher and Parkinson's disease markers. Matthias Alder was appointed COO, and a five-member Scientific Advisory Board was established to advance the company’s clinical programs. Q3 revenues increased to $27 thousand, while net loss rose to $4.66 million, reflecting higher R&D and administrative expenses.
Gain Therapeutics (Nasdaq: GANX) presented promising preclinical data on GT-02287, a compound targeting glucocerebrosidase (GCase) for Parkinson’s Disease, at the Society for Neuroscience Annual Meeting. The findings revealed that GT-02287 significantly reduced alpha-synuclein pathology and neuroinflammation while improving behavioral deficits in an animal model. With a favorable toxicity profile, this orally bioavailable compound shows potential in addressing unmet needs for patients suffering from Parkinson’s, particularly those with GBA1 mutations.
Gain Therapeutics (Nasdaq: GANX) announced breakthrough results from a study on its STAR compounds, GT-02287 and GT-02329, for treating Gaucher and GBA1 Parkinson's Disease. Conducted at the University of Maryland, the study demonstrated significant increases in GCase protein levels in iPSC-derived dopaminergic neurons and reductions in phosphorylated α-synuclein. These findings reveal a potential new approach for neurodegenerative disorders, showing a 129% increase in GCase enzyme levels. The company plans to initiate IND-enabling studies soon, promising hope for patients with alpha synucleinopathies.
Gain Therapeutics (Nasdaq: GANX) announced participation at the 12th Annual Jefferies London Healthcare Conference on November 16-19, 2021. CEO Eric Richman will present on November 17 from 9:20 to 9:55 a.m. GMT. Investors can engage in one-on-one meetings either in-person or virtually. The presentation will focus on Gain's innovative drug development strategies targeting neurodegenerative diseases and lysosomal storage disorders through its proprietary SEE-Tx™ platform. For more details, visit Gain’s official website.
Gain Therapeutics, Inc. (Nasdaq: GANX) announced that its management team will present at key medical conferences, including The Michael J. Fox Foundation’s Parkinson’s Disease Therapeutics Webinars on November 10, 2021, and The Society of Neuroscience Annual Meeting on November 9, 2021. The webinars aim to showcase advancements in Parkinson’s research, featuring topics like drug discovery and brain-penetrant allosteric regulators. These sessions will provide insights into ongoing research supported by the foundation. For registration and further information, visit the respective conference websites.
Gain Therapeutics has established a Scientific Advisory Board (SAB) to enhance its research efforts in neurodegenerative diseases and lysosomal storage disorders. Dr. Luca Benatti has been appointed as the chair, joined by esteemed members including Dr. Samuel Broder and Dr. Joanne Taylor. These experts bring decades of significant industry experience to shape the company's clinical strategies as it progresses its lead program in Parkinson’s Disease toward trials. Gain aims to redefine drug discovery through its innovative SEE-Tx™ platform.
Gain Therapeutics, Inc. (Nasdaq: GANX) recently appointed Matthias Alder as Chief Operating Officer to enhance its operational capabilities and prepare for clinical trials of its lead program targeting Gaucher and Parkinson's Diseases, expected in 2022. Alder brings over 25 years of industry experience, previously serving as Chief Business Officer at Autolus Therapeutics. CEO Eric Richman expressed confidence in Alder's ability to scale Gain's platform and expand partnerships. The company focuses on allosteric binding sites using its innovative SEE-Tx™ discovery platform to address neurodegenerative diseases.
Gain Therapeutics, Inc. (Nasdaq: GANX) will participate in the Cantor Virtual Global Healthcare Conference from September 27th-30th, showcasing its innovative approach to targeting allosteric binding sites for neurodegenerative diseases. CEO Eric Richman will present on September 30, from 8:00 to 8:30 a.m. ET, with a webcast available for interested investors. Gain Therapeutics aims to redefine drug discovery using its SEE-Tx™ platform, unlocking new treatment options for challenging disorders.
Gain Therapeutics (Nasdaq: GANX) announced that CEO Eric Richman will present at the Oppenheimer Fall Healthcare Life Sciences & MedTech Summit from September 20-23, 2021. The presentation is scheduled for September 22 from 1:15 to 1:55 p.m. ET. Investors can participate via webcast and one-on-one meetings. Gain Therapeutics focuses on optimizing allosteric binding sites in treatments for neurodegenerative diseases.
Gain Therapeutics, focused on neurodegenerative diseases, announced a presentation by Dr. Manolo Bellotto on their allosteric regulators for GM1 Gangliosidosis and Morquio B Disease. The presentation will take place on September 14, 2021, at 2:00 PM PT/5:00 PM ET during the GM1 Virtual Community Conference. These diseases are linked to mutations in the GLB1 gene, leading to toxic substrate accumulation. Gain's STARs aim to potentially treat neurological symptoms, marking a significant advancement in the field.
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