Gain Therapeutics presents preclinical data from GBA1 Program

Gain Therapeutics announced the presentation of new pre-clinical data supporting the potential of its structurally targeted allosteric regulators of glucocerebrosidase to prevent neurodegeneration in Alzheimer’s disease. The data will be shown in a poster presentation at the 2023 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders being held March 28 – April 1, 2023 in Gothenburg, Sweden. The data generated in two cell-based assays of Alzheimer’s disease showed that Gain’s orally bioavailable, brain-penetrant structurally targeted allosteric regulators of GCase show promising activity against Amyloid Beta 1-42 and oligomeric Tau toxicity, which are thought to underlie neurodegeneration and cognitive impairment in Alzheimer’s disease. The neuroprotective properties of STAR compounds were evaluated in cellular models of AD with primary rat cortical and hippocampal neurons challenged with Abeta-1-42 and human Tau oligomers, respectively. The data presented in the poster titled "Small-Molecule Structurally Targeted Allosteric Regulators of Glucocerebrosidase Show Neuroprotective Properties in Cell-Based Models of Alzheimer’s Disease," demonstrate in vitro evidence that a STAR compound improves neuronal survival and neurite network and reduces hyperphosphorylated tau in primary rat cortical neurons after Abeta-1-42 injury. Additionally, STAR compounds were shown to reduce hTauO-induced neurotoxicity in primary rat hippocampal neurons. The data therefore demonstrate that structurally targeted allosteric regulators are a potential disease-modifying, novel pharmacological option for the treatment of AD and other tauopathies that warrants further investigation.