Gain Therapeutics presents preclinical data from Gaucher Disease Program

Gain Therapeutics presented new pre-clinical data from its Gaucher disease program in a poster presentation at the 19th Annual WORLDSymposium held February 22-26, 2023, in Orlando, Florida. The data generated in an animal model of neuronopathic Gaucher disease show that GT-02329 restores beta-glucocerebrosidase activity, depletes accumulation of toxic lipid substrates, reduces neuroinflammation and improves neuromuscular function. The effect of Gain’s small molecule structurally targeted allosteric regulators of GCase that act on lysosomal function was shown in studies conducted in the conduritol beta epoxide mouse model. CBE is a covalent inhibitor that causes a reduction in GCase activity by reacting with the catalytic site of GCase and inactivating the GCase enzyme, which results in neurological features that occur in nGD patients, including the accumulation of the toxic lipid substrate glucosylsphingosine. The data presented in the poster titled "GT-02329, a structurally targeted allosteric regulator of GCase, restores GCase activity, reduces microgliosis and improves fine locomotor skills in the CBE model of neuronopathic Gaucher’s disease" demonstrated that: GT-02329 is orally bioavailable and brain penetrant, restored GCase activity, reduced accumulation of GCase substrates glucosylceramide and glucosylsphingosine, reduced a marker of inflammation, and improved neuromuscular function in a dose-dependent manner in CBE-injured mice treated with the compound.