First patient in US trial gets new off-the-shelf cell therapy for Parkinson’s
Single dose of NouvNeu001 delivered to both sides of brain region
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- NouvNeu001, an off-the-shelf cell therapy for Parkinson's, has been administered to the first patient in U.S. clinical trial.
- The one-time treatment aims to replace lost dopamine-producing neurons, targeting the disease's underlying cause.
- In a trial in China, the therapy was shown to be safe and to improve patients' motor function.
The first patient has been dosed in a U.S. clinical trial testing Iregene Therapeutics’ off-the-shelf, or ready-to-use, cell therapy NouvNeu001 for Parkinson’s disease.
The therapy was delivered to both sides of the putamen — a brain region deeply affected in Parkinson’s and central to movement control — in a patient at Weill Cornell Medical Center in New York, according to a company press release from Iregene announcing the milestone.
The U.S. Phase 2 trial follows previously reported data from a Phase 1/2 trial (NCT06167681) in China, where NouvNeu001 was found to be generally safe and well tolerated. Participants in that early-stage study showed improvements in motor function as measured by standard Parkinson’s disease rating scales, and some reduced their dopaminergic medication use.
Building on these positive data, the U.S. Food and Drug Administration cleared NouvNeu001 for direct Phase 2 entry. That followed the U.S. regulatory agency’s granting of fast track status and regenerative medicine advanced therapy (RMAT) designation to the Parkinson’s cell therapy. Both programs are designed to accelerate the development and review of promising therapies for serious conditions and allow for closer interaction with the agency during clinical development.
Alongside the U.S. Parkinson’s milestone, the company also announced enrollment of the first patient in China in a clinical trial evaluating NouvNeu004, a related cell therapy candidate for multiple system atrophy (MSA). MSA is a rare and rapidly progressive neurological disorder with symptoms that can overlap with Parkinson’s.
“Dosing the first U.S. patient for Parkinson’s disease and enrolling the first MSA patient in China are pivotal milestones in our mission to deliver transformative, disease-modifying therapies to patients worldwide,” said Meng Cai, PhD, Iregene’s chief medical officer.
Cell therapy aims to replace lost neurons in Parkinson’s
Parkinson’s is caused by the progressive loss of dopaminergic neurons, which are the nerve cells that produce dopamine, a chemical messenger essential for controlling movement. While available treatments can ease symptoms, they do not stop or reverse the underlying neurodegeneration.
This new treatment is designed to target the causes of Parkinson’s.
NouvNeu001 is an off-the-shelf cell therapy derived from induced pluripotent stem cells (iPSCs). Using what the company describes as its “AI + Chemical Induction” platform, the cells are directed to become dopaminergic progenitor cells — immature cells intended to mature into functional dopamine-producing neurons after transplantation. iPSCs are stem cells that can give rise to different cell types, including nerve cells, depending on the particular chemical cues they are given.
NouvNeu001 is being developed as a potential one-time treatment to replace lost neurons and modify disease progression through what the company describes as a “single dose, sustained benefit” strategy.
Parallel Phase 2 trials of NouvNeu001 are ongoing in both the U.S. and China, as researchers continue evaluating whether the therapy can safely restore dopamine-producing neurons and potentially alter the course of Parkinson’s disease.
“These parallel advances reflect the productivity of our ‘AI + Chemical Induction’ platform, yielding two pioneering ‘off-the-shelf’ cell therapies: NouvNeu001 for Parkinson’s disease and NouvNeu004 for Multiple System Atrophy. Together, they demonstrate our commitment to a robust global development pathway and our continued contribution to the advancement of cell therapy for neurodegenerative diseases,” Cai said.
