Gain Therapeutics, Inc. (GANX) is a biotechnology company specializing in the development of novel small molecule therapeutics to treat diseases caused by protein misfolding. The company leverages its proprietary Magellan™ drug discovery platform to identify allosteric binding sites on proteins, enabling the modulation of protein function to address underlying disease mechanisms. GANX focuses on therapeutic areas such as central nervous system disorders, lysosomal storage disorders, metabolic disorders, and oncology.
- Research and Development - Develops small molecule therapeutics targeting diseases caused by protein misfolding, utilizing the Magellan™ drug discovery platform to identify and modulate allosteric binding sites.
- Pipeline Programs - Includes GT-02287, a clinical-stage product candidate for Parkinson’s disease, aimed at restoring glucocerebrosidase function, reducing toxic lipid substrates, and improving mitochondrial health.
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| Name | Position | External Roles | Short Bio | |
|---|---|---|---|---|
Gene Mack ExecutiveBoard | Chief Executive Officer | Gene Mack has been serving as CEO of GANX since January 2025, after previously serving as CFO from April 8, 2024 and Interim CEO from June 25, 2024 to January 6, 2025. | View Report → | |
Gianluca Fuggetta Executive | Senior Vice President Finance and Principal Financial Officer | Gianluca Fuggetta is the Senior Vice President Finance and Principal Financial Officer at GANX since January 6, 2025. He has progressed through various finance roles at GANX and previously built his career as an Assurance Manager at PwC. | ||
Claude Nicaise Board | Member of the Board of Directors | Board Member at Sarepta Therapeutics, Inc. since 2015; Board Member at Chemomab Therapeutics since 2021; Board Member at Cassava Biosciences since January 2024 | Claude Nicaise, M.D. has over 35 years of experience in clinical and regulatory affairs and has been serving as an independent board member at GANX since January 2021. He has held leadership roles at major companies including Alexion Pharmaceuticals and Bristol-Myers Squibb. | |
Dov Goldstein Board | Board Member | Chief Financial Officer of BioAge Labs | Dov Goldstein, M.D. serves as a Board Member and Audit Committee Chair at GANX since January 2021, leveraging his extensive background in science and financial management. He has over 20 years of experience and currently serves as the Chief Financial Officer of BioAge Labs, among other executive roles in the industry. | |
Eric I. Richman Board | Board Member | Advisor at Broad Oak Capital; Director at Neubase Therapeutics, Inc.; Director at NovelStem International Corp; Director at LabConnect, Inc.; Co-founder and Chairman at InFuse Holdings | Eric I. Richman has served as a Board Member at GANX since July 2020 after previously serving as CEO from June 2020 to September 2022, bringing over 30 years of experience in biotechnology and pharmaceuticals. | |
Gwen Melincoff Board | Member of the Board of Directors | Managing Director at Gemini Advisors LLC; Advisor at Verge Genomics; Board Member at Protalix Biotherapeutics, Inc.; Board Member at Collegium Pharmaceutical, Inc. | Gwen Melincoff has over 25 years of experience in business development and venture capital in the biotechnology and pharmaceutical industries, and she has served as a board member at GANX since January 2021. She also contributes her expertise externally as Managing Director at Gemini Advisors LLC, Advisor at Verge Genomics, and as a board member at Protalix Biotherapeutics, Inc. and Collegium Pharmaceutical, Inc.. | |
Hans Peter Hasler Board | Member of the Board of Directors at GANX | Chairman of the Board at HBM Healthcare Investments AG; Director of the Board at Minerva Neuroscience Inc.; Nonexecutive Chairman of the Board at Shield Therapeutics PLC; Chief Advisor at SBTech Advisory | Hans Peter Hasler has been serving as a member of the Board of Directors at GANX since January 2021. He is an experienced leader in the biotechnology and pharmaceutical industries, having held positions such as Chairman of the Board at HBM Healthcare Investments AG and served as CEO at Vicarius Pharma AG until February 2020. | |
Jeffrey Riley Board | Board Member | CEO and Director of Caravan Biologix, Inc. since September 2021; Executive Chairman of Blink TBI since August 2020 | Jeffrey Riley has served as a Board Member at GANX since May 2019, also taking on key governance roles as Chair of the Compensation Committee and Audit Committee Member. With over 25 years in the biotechnology and pharmaceutical industries, he strengthens strategic oversight at GANX while contributing to multiple organizations. | |
Khalid Islam Board | Founder and Chairman of the Board of Directors | Chairman of the Board, Fennec Pharmaceuticals Inc.; Chairman of the Board, Minoryx Therapeutics S.L. | Khalid Islam, Ph.D., is the co-founder and Chairman of the Board of Gain Therapeutics, Inc. since 2017. He brings over 35 years of biotechnology experience and has held leadership roles in several pharmaceutical companies. | |
Matthias Alder | Matthias Alder served as COO of GANX starting in October 2021 and was promoted to CEO and Board member on September 20, 2022, until his departure on June 25, 2024. He has over 25 years of experience in the pharmaceutical and biotechnology industries. |
Research analysts covering Gain Therapeutics.
Competitors mentioned in the company's latest 10K filing.
| Company | Description |
|---|---|
Vanqua Bio | This company is targeting GBA-PD using small molecules, hypothesized to increase GCase levels, differing from the approach of acting as non-competitive pharmacological chaperones focused on stabilizing and restoring lysosomal function to misfolded GCase. |
BIAL | This company is targeting GBA-PD using small molecules, hypothesized to increase GCase levels, differing from the approach of acting as non-competitive pharmacological chaperones focused on stabilizing and restoring lysosomal function to misfolded GCase. |
Caraway Therapeutics | This company is targeting GBA-PD using small molecules, hypothesized to increase GCase levels, differing from the approach of acting as non-competitive pharmacological chaperones focused on stabilizing and restoring lysosomal function to misfolded GCase. |
Prevail Therapeutics | This company is evaluating a potential gene therapy candidate in a Phase 1/2 clinical trial targeting GBA-PD through gene therapies. |
This company is targeting GBA-PD through gene therapies. |
| Customer | Relationship | Segment | Details |
|---|---|---|---|
Zentalis Pharmaceuticals | Collaboration Agreement | All | Generated $0.055 million of revenue in 2023 (approximately 100% of total revenue). Collaboration ended 12/31/2023. |
Recent press releases and 8-K filings for GANX.
- Gain Therapeutics, Inc. hosted a virtual Key Opinion Leader (KOL) event on October 14, 2025, to discuss emerging data from its Parkinson's Disease candidate, GT-02287.
- Initial data from the Phase 1b trial of GT-02287, which enrolled 21 participants, showed a favorable safety profile with 85% of treatment-emergent adverse events (TEAEs) being mild and no serious adverse events (SAEs) as of September 3, 2025.
- The trial's initial MDS-UPDRS data indicated mean improvement by Day 90, suggesting a slower, non-acute therapeutic effect for GT-02287.
- The company anticipates an IND filing by the end of 2025 and plans to initiate Phase 2a in 1H26, with final Phase 1b extension results expected in 2H26.
- GAIN Therapeutics (GANX) held a KOL event on October 14, 2025, to discuss its lead program, GT-2287, for Parkinson's Disease, particularly in patients with a GBA1 genetic mutation.
- The ongoing Phase 1b study of GT-2287 in Parkinson's patients has completed enrollment with 21 participants as of early September. The 90-day dosing period will conclude by the end of 2025, with full safety, tolerability, PK, and UPDRS data expected.
- Preliminary data from the Phase 1b study indicates GT-2287 is overall safe and well-tolerated, with 85% of adverse events being mild. Early observations show a mean improvement in MDS UPDRS scores by day 90.
- Biomarker data, including alpha-synuclein, is anticipated later in Q4 2025. A Phase 2 placebo-controlled study is planned to begin in Q2 2026, with the Investigational New Drug (IND) application submission expected by the end of 2025.
- Gain Therapeutics (GANX) will host a virtual Key Opinion Leader (KOL) event on October 14th, 2025, at 4:00 p.m. EST, to discuss emerging data from its lead drug candidate, GT-02287, for Parkinson's disease.
- The event will feature KOLs Karl Kieburtz, M.D., M.P.H., and Kenneth Marek, M.D., who will contextualize data from the Phase 1b clinical study of GT-02287.
- Initial data from the Phase 1b study in Parkinson's disease patients indicates GT-02287 was generally well tolerated, with no treatment-emergent serious adverse events, and showed trending improvements in MDS-UPDRS scores.
- GT-02287 is an orally administered, brain-penetrant small molecule that aims to restore glucocerebrosidase (GCase) function, with preclinical data suggesting it may have the potential to slow or stop the progression of Parkinson's disease.
- Gain Therapeutics presented interim data from its Phase 1b clinical study of GT-02287 for Parkinson's disease, reporting improvement in MDS-UPDRS scores in the first 9 participants after 90 days of dosing.
- GT-02287 was generally well-tolerated with no treatment-emergent serious adverse events, and plasma exposures were within the projected therapeutic range.
- The data monitoring committee recommended continuation of the study, and Australian health authorities approved a Phase 1b study extension for up to 12 months.
- The company will host a webinar on October 14 at 4 p.m. ET to discuss these results further.