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Gain Therapeutics, Inc., a biotechnology company, engages in developing various therapies to treat diseases caused by protein misfolding. It focuses on rare genetic diseases and neurological disorders. The company uses its Site-Directed Enzyme Enhancement Therapy platform to discover allosteric sites on misfolded proteins and identify proprietary small molecules that bind these sites, restore protein folding, and treat disease. It is developing structurally targeted allosteric regulator candidates to treat various diseases, including Morquio B, GM1 gangliosidosis (GM1), neuronopathic Gaucher disease, GBA1 Parkinson's, Krabbe, and Mucopolysaccharidosis type 1 diseases. The company was founded in 2017 and is based in Bethesda, Maryland.

Gain Therapeutics, Inc.

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BETHESDA, MD., DEC. 18, 2025 (GLOBE NEWSWIRE) -- GAIN THERAPEUTICS, INC. (NASDAQ: GANX) (GAIN, OR THE COMPANY), A CLINICAL-STAGE BIOTECHNOLOGY COMPANY LEADING THE DISCOVERY AND DEVELOPMENT OF THE NEXT GENERATION OF ALLOSTERIC SMALL MOLECULE THERAPIES, TODAY ANNOUNCED THAT IT WILL HOST A VIRTUAL KEY OPINION LEADER (KOL) EVENT,UNDERSTANDING GCASE SUBSTRATES IN PARKINSON'S DISEASE: PERSPECTIVES ON BIOMARKERS AND DISEASE MODIFICATION, CONTEXTUALIZING EMERGING BIOMARKER DATA FROM THE PHASE 1B CLINICAL STUDY OF GT-02287.

GAIN THERAPEUTICS TO HOST VIRTUAL KOL EVENT ON GT-02287 FOR PARKINSON'S DISEASE

RESULTS SHOWED CNS TARGET ENGAGEMENT WITH REDUCTION IN GCASE SUBSTRATE GLUCOSYLSPHINGOSINE (GLUSPH) IN CEREBROSPINAL FLUID (CSF), A PRESPECIFIED ENDPOINT THE REDUCTION IN GLUSPH IN CSF, A FIRST-EVER OBSERVATION FOLLOWING THE ADMINISTRATION OF A GCASE MODULATOR TO PD PATIENTS, SUGGESTS INCREASED GCASE ACTIVITY IN THE BRAIN, WHICH IS EXPECTED TO IMPACT THE PROGRESSION OF PARKINSON'S DISEASE (PD) KOL EVENT PLANNED FOR EARLY JANUARY TO DISCUSS THE RESULTS; REGISTRATION INFORMATION HEREIN BETHESDA, MD., DEC. 18, 2025 (GLOBE NEWSWIRE) -- GAIN THERAPEUTICS, INC. (NASDAQ: GANX) (GAIN, OR THE COMPANY), A CLINICAL-STAGE BIOTECHNOLOGY COMPANY LEADING THE DISCOVERY AND DEVELOPMENT OF THE NEXT GENERATION OF ALLOSTERIC SMALL MOLECULE THERAPIES, TODAY PROVIDED EVIDENCE, FOR THE FIRST TIME IN PARKINSON'S DISEASE (PD), OF A REDUCTION IN GLUCOCEREBROSIDASE (GCASE) SUBSTRATE IN CEREBROSPINAL FLUID (CSF).

GAIN THERAPEUTICS ANNOUNCES POSITIVE RESULTS IN KEY EXPLORATORY ENDPOINT FROM ITS PHASE 1B CLINICAL STUDY OF GT-02287 IN PEOPLE WITH PARKINSON'S DISEASE

GT-02287 REDUCES MITOCHONDRIAL STRESS AND ENHANCES NEURONAL SURVIVAL IN MPP+ TREATED RAT DOPAMINERGIC NEURONS GT-02287 DECREASES STAINING FOR MIRO1, SUGGESTING IMPROVED MITOCHONDRIAL HEALTH IN -SYNUCLEIN-INDUCED MOUSE PD MODEL GT-02287 IMPROVES MITOCHONDRIAL FUNCTION BY FACILITATING GCASE TRAFFICKING TO THE MITOCHONDRIA IN PATIENT-DERIVED FIBROBLASTS HARBORING GBA1 MUTATION BETHESDA, MD., NOV. 20, 2025 (GLOBE NEWSWIRE) -- GAIN THERAPEUTICS, INC. (NASDAQ: GANX) (GAIN, OR THE COMPANY), A CLINICAL-STAGE BIOTECHNOLOGY COMPANY LEADING THE DISCOVERY AND DEVELOPMENT OF THE NEXT GENERATION OF ALLOSTERIC SMALL MOLECULE THERAPIES, TODAY ANNOUNCED A POSTER PRESENTATION WAS MADE AT THE SOCIETY FOR NEUROSCIENCE ANNUAL MEETING, NEUROSCIENCE 2025, HELD NOVEMBER 15TH-19TH IN SAN DIEGO, CA.

GAIN THERAPEUTICS PRESENTS PRECLINICAL GT-02287 DATA AT NEUROSCIENCE 2025

PRESENTED INITIAL DATA FROM PHASE 1B STUDY SUGGESTING GT-02287 HAS A DISEASE-SLOWING EFFECT CONSISTENT WITH THE PRECLINICAL MODELS IN VIVO AND THE PROPOSED MECHANISM OF ACTION COMPLETED ENROLLMENT OF 21 PARTICIPANTS IN PHASE 1B STUDY EVALUATING GT-02287 IN PARKINSON'S DISEASE WITH OR WITHOUT GBA1 MUTATIONS DURING 3Q 2025 RECEIVED APPROVAL FROM THE AUSTRALIAN HEALTH AUTHORITIES TO EXTEND THE DURATION OF THE PHASE 1B STUDY, ALLOWING PARTICIPANTS TO CONTINUE TREATMENT WITH GT-02287 FOR A TOTAL OF 12 MONTHS; WITH A MAJORITY OF SUBJECTS ELECTING TO PARTICIPATE IN THE STUDY EXTENSION ANALYSIS OF FUNCTIONAL CHANGES AND BIOMARKER ACTIVITY AT 90 DAYS EXPECTED TO BE AVAILABLE DURING 4Q 2025 BETHESDA, MD., NOV. 12, 2025 (GLOBE NEWSWIRE) -- GAIN THERAPEUTICS, INC. (NASDAQ: GANX) (GAIN, OR THE COMPANY), A CLINICAL-STAGE BIOTECHNOLOGY COMPANY LEADING THE DISCOVERY AND DEVELOPMENT OF THE NEXT GENERATION OF ALLOSTERIC SMALL MOLECULE THERAPIES, TODAY REPORTED FINANCIAL RESULTS FOR THE QUARTER ENDED SEPTEMBER 30, 2025, AND PROVIDED A CORPORATE UPDATE.

GAIN THERAPEUTICS REPORTS FINANCIAL RESULTS FOR THIRD QUARTER 2025 AND PROVIDES CORPORATE UPDATE

BETHESDA, MD., OCT. 30, 2025 (GLOBE NEWSWIRE) -- GAIN THERAPEUTICS, INC. (NASDAQ: GANX) (GAIN, OR THE COMPANY), A CLINICAL-STAGE BIOTECHNOLOGY COMPANY LEADING THE DISCOVERY AND DEVELOPMENT OF THE NEXT GENERATION OF ALLOSTERIC SMALL MOLECULE THERAPIES, TODAY ANNOUNCED A POSTER PRESENTATION AT THE SOCIETY FOR NEUROSCIENCE ANNUAL MEETING, NEUROSCIENCE 2025, BEING HELD NOVEMBER 15TH19TH IN SAN DIEGO, CA.

GAIN THERAPEUTICS TO PRESENT AT NEUROSCIENCE 2025

BETHESDA, MD., OCT. 16, 2025 (GLOBE NEWSWIRE) -- GAIN THERAPEUTICS, INC. (NASDAQ: GANX) (GAIN, OR THE COMPANY), A CLINICAL-STAGE BIOTECHNOLOGY COMPANY LEADING THE DISCOVERY AND DEVELOPMENT OF THE NEXT GENERATION OF ALLOSTERIC SMALL MOLECULE THERAPIES, TODAY ANNOUNCED IT WILL PARTICIPATE IN THE UPCOMING 2025 MAXIM GROWTH SUMMIT, TAKING PLACE FROM OCTOBER 22ND TO 23RD AT THE HARD ROCK HOTEL NYC.

GAIN THERAPEUTICS TO ATTEND THE 2025 MAXIM GROWTH SUMMIT

BETHESDA, MD., OCT. 09, 2025 (GLOBE NEWSWIRE) -- GAIN THERAPEUTICS, INC. (NASDAQ: GANX) (GAIN, OR THE COMPANY), A CLINICAL-STAGE BIOTECHNOLOGY COMPANY LEADING THE DISCOVERY AND DEVELOPMENT OF THE NEXT GENERATION OF ALLOSTERIC, SMALL-MOLECULE THERAPIES, TODAY ANNOUNCED THAT IT WILL HOST A VIRTUAL KEY OPINION LEADER (KOL) EVENT, BIOMARKERS, CLINICAL ENDPOINTS, AND THE PATH TODISEASE MODIFICATION : CONTEXTUALIZING THE EMERGING DATA FROM GT-02287 FEATURING KEY OPINION LEADERS (KOLS), KARL KIEBURTZ, M.D.

IMPROVEMENT OBSERVED IN MDS-UPDRS SCORES IN FIRST 9 PARTICIPANTS ENROLLED; 90-DAY FOLLOW-UP CONTINUING IN REMAINING PARTICIPANTS

GAIN THERAPEUTICS PRESENTS INITIAL DATA FROM PHASE 1B CLINICAL STUDY OF GT-02287 IN PARKINSON'S PATIENTS AT INTERNATIONAL CONGRESS OF PARKINSON'S DISEASE AND MOVEMENT DISORDERS

BETHESDA, MD., SEPT. 23, 2025 (GLOBE NEWSWIRE) -- GAIN THERAPEUTICS, INC. (NASDAQ: GANX) (GAIN, OR THE COMPANY), A CLINICAL-STAGE BIOTECHNOLOGY COMPANY LEADING THE DISCOVERY AND DEVELOPMENT OF THE NEXT GENERATION OF ALLOSTERIC, SMALL-MOLECULE THERAPIES, TODAY ANNOUNCED THAT DR. RINALDO MONTALVO, DIRECTOR OF ARTIFICIAL INTELLIGENCE AT GAIN THERAPEUTICS, WILL DELIVER A TALK AT THE WORLD BI SPONSORED DRUG DISCOVERY INNOVATION PROGRAMME (DDIP) 2025, BEING HELD SEPTEMBER 25TH -26TH IN BARCELONA, SPAIN.

GAIN THERAPEUTICS TO PARTICIPATE AT DRUG DISCOVERY INNOVATION PROGRAMME 2025

EXTENSION OF THE PHASE 1B STUDY WILL ALLOW PARTICIPANTS TO CONTINUE DOSING WITH GT-02287 FOR AN ADDITIONAL NINE MONTHS AFTER COMPLETING THE FIRST 90-DAY DOSING SCHEDULE

Gain Therapeutics, Inc.

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