UniXell Biotech Announces Successful First-in-Human Dosing of Autologous iPSC-Derived Cell Therapy (UX-DA001) for Parkinson’s Disease

SHANGHAI, May 23, 2025 – UniXell Biotechnology Co., Ltd. (UniXell Biotech), a biotech company dedicated to innovative cell therapies, today announced a significant milestone with the successful first administration of its proprietary Parkinson’s disease cell drug, UX-DA001, in China in March 2025. This achievement marks the official initiation of China’s first registration-directed clinical trial for an autologous iPSC-derived cell therapy for Parkinson’s disease.

The Phase I clinical trial aims to evaluate the safety, tolerability, and preliminary efficacy of UX-DA001 in patients with Parkinson’s disease. On March 1, 2025, Dr. Li Dianyou, Director of the Functional Neurosurgery Department at Ruijin Hospital, performed the intricate surgical path planning and subsequent stereotactic transplantation.

Early results from the one-month postoperative follow-up have been encouraging, revealing significant improvements in the patient’s sleep quality and motor function, with no severe adverse events reported. The first recipient of UX-DA001 expressed profound hope, stating, “After receiving the stem cell treatment, I experienced a remarkable improvement in my sleep quality. I fall asleep quickly and notice gradual progress every day. Although recovery takes time, the ‘day by day improvement’ fills me with hope for the future.”

Dr. Liu Jun, Principal Investigator of the trial and Director of the Neurology Department at Ruijin Hospital, emphasized the transformative potential of this approach: “Current standard treatment for Parkinson’s disease primarily relies on medications to alleviate symptoms. However, after prolonged oral drug treatment, patients often experience fluctuations in efficacy and disease progression continues. In contrast, cell therapy offers the prospect of ‘one-time treatment with long-term benefits.'”

About UX-DA001 and the Phase 1 Trial: UX-DA001 is an investigational autologous iPSC-derived neural progenitor cell drug. It is meticulously derived from the patient’s own peripheral blood cells, which are first reprogrammed into induced pluripotent stem cells (iPSCs) and then precisely differentiated into dopaminergic neurons in vitro. These cells are subsequently transplanted into the patient’s brain through a minimally invasive surgical procedure.

A key advantage of UX-DA001 is its autologous nature, utilizing the patient’s own cells, which significantly reduces the risk of immune rejection. This eliminates the need for immunosuppressive drugs, thereby avoiding potential side effects such as increased infection and tumor risks, organ toxicity, and metabolic disorders, promising a safer and more effective treatment that significantly improves the quality of life for patients.

UX-DA001 has received clinical trial approvals (Investigational New Drug, IND) from both the National Medical Products Administration (NMPA) of China (December 2024) and the U.S. Food and Drug Administration (FDA) (February 2025), positioning UX-DA001 with potential global implications for Parkinson’s disease patients.

The Phase 1 study is being conducted at Ruijin Hospital in Shanghai, China, under the guidance of Dr. Liu Jun, MD, PhD, Principal Investigator. Dr. Li Dianyou, Director of the Functional Neurosurgery Department, is performing the transplantation surgeries. This clinical study is designed to evaluate the safety and tolerability of UX-DA001 cell transplantation, and also investigates whether the transplanted cells survive, integrate, and improve motor function in subjects with Parkinson’s disease.

More information about this trial is available at clinicaltrials.gov (NCT#06778265).