Gain Therapeutics Inc (NASDAQ: GANX) released preclinical data from a project. The company discussed the results of the Parkinson’s Disease project at the poster presentation in Belgium. Its presentation indicated that GT-02287 led to increased production and action of GCase or beta-glucocerebrosidase protein
Other effects of GT-02287 include neuronal survival, improved neural connection, correcting the mutated phenotypes of Parkinson’s disease such as alpha-synuclein, and improving the health of lysosomes.
According to Gain Therapeutics’ CEO, Eric Richman, treatment options for Parkinson’s disease are limited. For this reason, the company was glad to present data that could give people with the disease hope.
How the therapy works
The General Manager and President of Gain, Manolo Bellotto, adds that the company discovered GT-02287 via its SEE-Tx platform. The compound can bind allosterically to GCase and stabilise it. As a result, GCase is not metabolised and instead goes to lysosomes to perform its function.
Bellotto adds that these new findings support previous data on the drug’s effect on Parkinson’s Disease. It shows the drug’s impact on neural health, lysosomes health, substrate detention, enzyme levels, and disease pathology.
The company generated this data from two separate in vitro experiments covering WT and N370S, and L444P. N370S and L444P are the most significant mutations to the GBA1 seen in Parkinson’s Disease.
The effects the compound had on GCase
The company noticed an increased activity and production of GCase in N370S, L444P, and WT dopaminergic neurons. Researchers also discovered that phosphorylated alpha-synuclein decreased in N370S, L444, and WT dopaminergic neurons.
The GBA1 gene encodes Case, a lysosomal enzyme. Mutation in this gene is one of the most significant Parkinson’s Disease risk factors. Moreover, the levels of GCase also cause the disease’s pathology.
Gain Therapeutics is a pharmaceutical company that uses its SE-Tx platforms to discover allosteric regulators. The company can discover allosteric targets on proteins of diseases affecting multiple systems.
Some of the diseases the company can target include metabolic, lysosomal storage and neurodegenerative diseases. The company’s most recent top project is on GT-02287, which could potentially treat Parkinson’s Disease.
