Vincere gets $5M MJFF grant to develop Parkinson’s treatment
USP30 inhibitors target mitochondrial root of Parkinson's disease
- Vincere Biosciences received a $5M grant for Parkinson's disease treatment development.
- The therapy targets mitochondrial dysfunction, a key factor in Parkinson's progression.
- USP30 inhibitors aim to restore mitophagy, with clinical trials planned for 2026.
Vincere Biosciences won a $5 million grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to advance development of USP30 small molecule inhibitors, its candidate therapy aimed at slowing or halting Parkinson’s disease progression.
The grant, provided through MJFF’s Therapeutics Pipeline Program, will support preclinical studies on USP30 inhibitors, as well as the development of biomarkers to evaluate the inhibitors’ target engagement and guide their translation into clinical studies.
The company said the studies will support a U.S. Food and Drug Administration (FDA) application to initiate a Phase 1 clinical trial in 2026.
“It’s been exciting to see growing enthusiasm for USP30 since our AI platform prioritized this target in 2018,” Andy Lee, co-founder and chief business officer of Vincere, said in a company press release. “The new support from MJFF positions the company well for ongoing partnering discussions with larger organizations who may accelerate clinical development of this promising approach.”
Parkinson’s disease is caused by the progressive loss of dopaminergic neurons, nerve cells that produce dopamine, a chemical signaling molecule involved in motor control. Problems with mitophagy, the process by which cells clear damaged mitochondria (the cell’s powerhouses) and mitochondrial dysfunction are thought to contribute to neuronal loss.
Targeting mitochondria
Vincere aims to restore mitochondrial quality control and prevent the progression of nerve damage by using an oral, brain-penetrant small molecule to selectively block USP30, an enzyme in the mitochondrial membrane that slows mitophagy.
“Mitochondria sit at the crossroads of Parkinson’s and aging (the biggest risk factor for Parkinson’s),” said Spring Behrouz, PhD, co-founder and CEO of Vincere. “Fix the mitochondria and you strike at the root of the disease. MJFF’s support helps us move that science from the lab to the clinic.”
MJFF funded a previous Vincere study to determine whether USP30 inhibitors could improve mitophagy in rodent brains. The study evaluated whether the inhibitor interacted with USP30 in the brain and reduced neurodegeneration, building on initial data obtained from treating cells derived from people with Parkinson’s.
“Our collaboration with Vincere Biosciences over the years has supported the advancement of research targeting mitochondrial dysfunction, a key driver of Parkinson’s pathology,” said Jessica Tome Garcia, PhD, MJFF’s lead scientific program manager. “This next phase of work builds on that foundation and represents important progress toward disease-modifying therapies that could meaningfully improve patients’ lives.”
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