Kenai Therapeutics has dosed the first patient in its multi-centre Phase I REPLACE trial evaluating the allogeneic neuron replacement cell therapy, RNDP-001, for moderate to moderate-severe idiopathic Parkinson’s disease.
This follows clearance of Kenai’s investigational new drug application from the US Food and Drug Administration (FDA), allowing the clinical study of the off-the-shelf therapy intended to restore motor function by replacing lost dopamine-producing neurons.
The open-label study is focused on the safety and tolerability of RNDP-001 and also includes assessments of brain imaging evidence of cell survival, along with engraftment in adults with Parkinson’s.
The therapy is administered to targeted brain regions using a precision neurosurgical approach, aiming to replace cells that produce dopamine.
REPLACE plans to enrol up to 12 patients. Initial data on tolerability, safety, and brain imaging from the participants are anticipated in 2026.
Kenai Therapeutics CEO Nick Manusos said: “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.
“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. This milestone marks an important step forward for Kenai and for families impacted by Parkinson’s.”
The therapy has received fast track designation from the FDA, a status intended to expedite the development and review process.
It is based on an induced pluripotent stem cell platform, utilising donor-derived, cryopreserved cells to create a scalable, off-the-shelf product.
US-based company Kenai has an exclusive manufacturing partnership with FUJIFILM Cellular Dynamics to produce dopamine progenitor cells for clinical use.
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