Novel small molecule in development for Parkinson’s-related GBA1 mutations

Vanqua Bio has announced its lead candidate for Parkinson’s disease, a small molecule allosteric activator of glucocerebrosidase, will enter clinical development in the first quarter of 2024.

According to a company release, Vanqua plans to advance VQ-101 for those with PD and mutations in GBA1, the gene that encodes glucocerebrosidase (GCase), which are the most common genetic risk factor for the condition. Mutations in the GBA1 gene result in a decrease in the activity of GCase, which in turn disrupts the function of lysosomes, the recycling centers of the cells, that enable toxic forms of proteins to accumulate and harm neurons.

In preclinical studies of patient-derived neuronal cells, VQ-101 activated wild-type and mutant forms of GCase, reduced lipid substrates and blocked accumulation of pathogenic forms of alpha synuclein, according to the release.

Vanqua additionally announced the appointment of three executives to help with stewardship of the novel therapeutic: Jesse Cedarbaum, MD, as chief medical officer, Omer Siddiqui as chief development officer and Rand Sutherland, MD, MPH, as a member of the board of directors.

“The advancement of VQ-101 and expansion of the leadership team significantly furthers Vanqua’s mission of helping to usher in a new era of hope for patients living with neurodegenerative disorders,” Vanqua Bio CEO Jim Sullivan, PhD, said in the release.