GT-02329 / Gain Therap - LARVOL DELTA

Allosteric Modulation of GCase Enhances Lysosomal Activity and Reduces ER Stress in GCase-Related Disorders. (PubMed, Int J Mol Sci) - "Using Site-directed Enzyme Enhancement Therapy (SEE-Tx®), two structurally targeted allosteric regulators (STARs), GT-02287 and GT-02329, were developed and tested in GD patient-derived fibroblasts with relevant GCase variants. This enhanced lysosomal function and reduced cellular stress. These findings validate the STARs' mechanism of action and highlight their therapeutic potential for GCase-related disorders, including GD, PD, and Dementia with Lewy Bodies."

Journal • Alzheimer's Disease • CNS Disorders • Dementia • Gaucher Disease • Genetic Disorders • Lewy Body Disease • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Parkinson's Disease • Rare Diseases • GBA • GBA1

Gain Therapeutics, Inc. Reports Full Year 2021 Financial Results and Business Update (Yahoo News) - "Gain Therapeutics, Inc...today announced its financial results for the full year ended December 31, 2021 and highlighted recent corporate accomplishments....Upcoming 2022 Milestones; Complete the preclinical development and submit the regulatory dossier to the Human Research Ethics Committee in Australia to initiate a first-in-human (FIH) Phase 1 clinical trial in its GBA1 Parkinson’s disease program."

Preclinical • CNS Disorders • Parkinson's Disease

Gain Therapeutics Presents Positive Data on its Structurally Targeted Allosteric Regulators in both Parkinson’s and Alzheimer’s Disease at the AD/PD 2022 Conference (GlobeNewswire) - "Gain Therapeutics, Inc...today presented new pre-clinical data from its Parkinson’s and Alzheimer’s Disease programs. These results were highlighted in a poster presentation at the International Conference on Alzheimer’s (AD) and Parkinson’s Diseases (PD) being held March 15 - 20, 2022 virtually and in Barcelona, Spain. The data shows that improvement of the lysosomal function through the enhancement of glucocerebrosidase (GCase) levels and activity can improve key pathological features in α-synucleinopathies and AD."

Preclinical • CNS Disorders • Parkinson's Disease

Gain Therapeutics, Inc. Announces Breakthrough Pre-Clinical Data of Novel Small Molecules for the Treatment of Parkinson’s Disease and Gaucher Disease (GlobeNewswire) - “Gain Therapeutics, Inc…announced data from the Company’s study conducted at the University of Maryland School of Medicine (UMSOM). The study, evaluating two lead Structurally Targeted Allosteric Regulators (STARs) compounds – GT-02287 and GT-02329 – for the treatment of Gaucher and GBA1 Parkinson’s Disease, demonstrated highly statistically significant effects on all tested phenotypes representing a key breakthrough in the potential to treat neurodegenerative disorders characterized by misfolded proteins including Parkinson’s Disease, Alzheimer’s, Gaucher’s Disease and Lewy Body Dementia. Study results were presented at The Michael J. Fox Foundation for Parkinson’s Research’s Innovating from Drug Discover to the Clinic: Novel Approaches to PD Therapeutic Development webinar.”

Preclinical • CNS Disorders • Gaucher Disease • Parkinson's Disease • Type 1 Gaucher Disease

Gain Therapeutics, Inc. Announces Positive Topline Data in Patient-derived iPSC Study Evaluating STAR Compounds as a Treatment for Gaucher and Parkinson’s Disease (GlobeNewswire) - "GT-02287and GT-02329 compounds display positive effects on GCase activity and related parameters; Study results demonstrate an increase in GCase protein levels with transport to the lysosomes and decrease of glucosylceramide and α-synuclein-p129 levels; Comprehensive study results to be presented at Michael J. Fox Foundation’s upcoming Innovating from Drug Discovery to the Clinic: Novel Approaches to PD Therapeutic Development webinar; IND-initiating study planned for Q4’21."

Preclinical • CNS Disorders • Parkinson's Disease

Gain Therapeutics, Inc. Reports Second Quarter 2021 Financial Results and Business Update (GlobeNewswire) - "Upcoming 2021 & 2022 Milestones: New Data Release – Top line data from collaboration with University of Maryland School of Medicine on GBA-associated Parkinson’s Disease in IPSC model. Industry Conference - Full data from collaboration with University of Maryland School of Medicine on GBA-associated Parkinson’s Disease. Initiate IND-enabling studies 2021 - Gaucher’s / Parkinson’s Disease."

Preclinical • CNS Disorders • Gaucher Disease • Parkinson's Disease

Gain Therapeutics and University of Maryland School of Medicine Announce Research Collaboration (GlobeNewswire) - "Gain Therapeutics, Inc....today announced a research collaboration with the University of Maryland School of Medicine (UMSOM), to investigate Gain’s structurally targeted allosteric regulators (STARs) in cellular models of neuronopathic Gaucher disease (nGD) and Parkinson’s disease (PD)....The research program will aim to further elucidate the mechanism of action of Gain’s STAR candidates by studying their effect on GCase, including GCase’s enzyme activity and transport to the lysosome....Gain and UMSOM intend to report initial data from the collaboration in the first half of 2021."

Licensing / partnership • Preclinical • CNS Disorders • Parkinson's Disease

Gain Therapeutics, Inc. Announces Close of $10 Million Series B Financing (GlobeNewswire) - "Gain Therapeutics, Inc....today announced that it has raised $10 million in a Series B financing with institutional and accredited investors. Proceeds from the financing will be used to complete IND-enabling studies for the company’s two lead development programs and fund the advancement of additional pipeline candidates. Concurrent with the close of the Series B financing, Gain, completed a share exchange with Gain Therapeutics, SA...."

Financing • CNS Disorders • Parkinson's Disease