Dosing begins in clinical trial of Parkinson’s cell therapy RNDP-001
Treatment aims to replace lost neurons, addressing disease's root cause
- RNDP-001 is a cell therapy for Parkinson's disease, aiming to replace lost dopaminergic neurons.
- A Phase 1 clinical trial (REPLACE) has begun dosing patients to assess safety and efficacy.
- The therapy seeks to restore motor function and potentially halt disease progression.
Kenai Therapeutics said the first patient has been dosed in a Phase 1 clinical trial testing the safety and efficacy of RNDP-001, its experimental cell replacement therapy for Parkinson’s disease.
The REPLACE trial (NCT07106021) is recruiting up to 12 people with moderate to severe idiopathic (of unknown cause) Parkinson’s, ages 45 to 75, at three sites in the U.S. Initial safety, tolerability, and brain imaging data from all patients are expected in 2026.
“Initiating this first-in-human trial represents the culmination of decades of foundational research in regenerative neuroscience,” Howard Federoff, MD, PhD, chief medical officer and executive vice president of corporate medicine and science at Kenai, said in a company press release. “What sets RNDP-001 apart is not just the depth of research behind it, but also its potential to intervene at the level of disease by replacing the dopaminergic neurons that are central to Parkinson’s progression.”
The treatment has received U.S. Food and Drug Administration (FDA) fast-track designation, a status that aims to accelerate the development and review of therapies meant to address unmet medical needs in serious or life-threatening conditions. Benefits for drug developers include more frequent interactions with the FDA and eligibility for priority review.
Parkinson’s disease is caused by the gradual loss of dopaminergic neurons, the nerve cells responsible for producing dopamine, a chemical messenger involved in motor control. Many Parkinson’s treatments focus on enhancing dopamine signaling in the brain.
Replacing lost neurons
RNDP-001 is designed to replace lost dopaminergic neurons. It uses induced pluripotent stem cells created from mature cells from a donor’s skin or blood, and reprograms them into a stem-cell-like state to produce dopaminergic neuron progenitor cells. These cells are produced in advance and stored at low temperatures for use as needed.
The stored progenitor cells are surgically delivered directly into the patients’ brains, where they are expected to differentiate into dopaminergic neurons, increasing dopamine levels. This may ease Parkinson’s symptoms and potentially halt disease progression.
“While most Parkinson’s disease treatments focus on symptom management, RNDP-001 aims to replace lost neurons, repair damaged neural circuits and restore motor function for patients with moderate to moderate-severe forms of the disease,” said Nick Manusos, Kenai’s CEO. “By moving beyond symptom control to address the root cause of disease, our goal is to offer patients a future with improved motor function and better overall quality of life.”
Preclinical studies demonstrated that transplanting a large quantity of dopaminergic neuron progenitor cells into a rat model of Parkinson’s increased the growth of new nerves, resulting in improved motor function and longer survival.
REPLACE is an open-label trial that will test RNDP-001 at two dose levels. The first group of patients will receive a lower dose, while the second group will get a higher dose. A third group will be treated with the dose deemed the best.
The trial’s main goal is to evaluate the safety and tolerability of the treatment. Secondary goals include changes in dopaminergic signaling and in on time, or periods when symptoms are well controlled with the medication.
The company announced earlier this year that it would set up research and laboratory facilities at Lilly Gateway Labs in San Diego to help advance RNDP-001.
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