Gain granted $2.8M to develop lead therapy for GBA1 Parkinson’s

Innosuisse award for GT-02287 to fund preclinical, clinical studies

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Gain Therapeutics has been awarded about $2.8 million to advance the development of GT-02287, its lead therapeutic candidate for GBA1-associated Parkinson’s disease.

Innosuisse, the Swiss Innovation Agency, granted the 2.5 million Swiss francs to the company under its Swiss Accelerator program, which provides funding for new and innovative products. The program aims to facilitate the growth of Swiss companies and will support both preclinical and clinical studies of the experimental therapy.

The funded project is titled “Harnessing Structurally Targeted Allosteric Regulators to treat Parkinson’s Disease.”

“We are grateful for the continued support from Innosuisse, which speaks to the wealth of promising data we have generated to date and the disease-modifying potential of our lead program for patients suffering from GBA1 Parkinson’s disease and other neurodegenerative disorders,” Matthias Alder, CEO of Gain, said in a company press release.

“This funding provides significant financial support for the further development of GT-02287 as we remain on track to submit the dossier for the start of the Phase 1 clinical study in mid-2023,” Alder added.

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GT-02287 Helps Prevent Toxic Alpha-synuclein in Patient-derived Cells

GT-02287 designed to ease effects of GBA1 gene mutations

Mutations in the GBA1 gene are found in about 5% of Parkinson’s patients, and are associated with earlier disease onset, fast progression, and more rapid cognitive decline.

Such mutations cause the formation of a misfolded and dysfunctional version of the beta-glucocerebrosidase (GCase) enzyme that’s important for the function of lysosomes, the cellular compartments that recycle cellular waste.

Consequently, lysosomes can’t effectively clear this waste, causing molecules to accumulate to toxic levels inside cells. That includes the alpha-synuclein protein that’s known to damage Parkinson’s patients’ nerve cells.

Even in patients without GBA1 mutations, low GCase activity is common, which is thought to contribute to alpha-synuclein buildup.

A type of compound called an allosteric modulator, GT-02287 is an oral small molecule designed to bind to GCase, allowing it to fold normally, thereby restoring its activity. In turn, the therapy is expected to lead to more normal lysosome function that will prevent alpha-synuclein buildup.

GT-02287 was developed with the company’s Site-Directed Enzyme Enhancement Therapy system, which uses the three-dimensional structure of a target enzyme to predict its interaction with therapeutic molecules.

Gain believes its therapeutic candidate may hold potential for slowing or stopping nerve cell damage in Parkinson’s patients.

Preclinical studies have demonstrated GT-02287’s ability to reduce alpha-synuclein buildup, lower inflammation, ease behavior problems, and boost fine motor skills in animal models.

Moreover, the investigational treatment exhibited a number of beneficial effects in human nerve cell models, including improved lysosomal function and nerve cell health, increased GCase levels, as well as lower alpha-synuclein accumulation.

The funding from Innosuisse will now support additional preclinical studies, in addition to the launch of clinical studies to evaluate the therapy’s in-human pharmacological properties.

Gain’s project was one of 53 of 752 evaluated projects selected for funding after a three-stage assessment process.  The company also was awarded about $1.7 million from Innosuisse in 2o21 to support the development of allosteric modulators for GBA1-related diseases.

“The Swiss Accelerator program under which we are supported was a highly competitive process, and we are pleased with the validation of our science and data that is reflected in the decision of Innosuisse to support the program,” said Manolo Bellotto, PhD, chief strategy officer and general manager at Gain.

“We look forward to continuing to build on this work and advance the development of a treatment for this devastating disease,” Bellotto said.