UniXell Biotechnology doses first Parkinson’s patient with iPSCs, reports improvements

Shanghai UniXell Biotechnology Co., Ltd. (UniXell Biotech) announced that the first patient has received its investigational Parkinson’s disease cell therapy drug, UX-DA001, in China. The ongoing Phase I will assess the safety, tolerability, and preliminary efficacy of UX-DA001 for Parkinson’s disease.

On March 1, 2025, Dr. Li Dianyou, Director of the Functional Neurosurgery Department at Ruijin Hospital, performed the surgical planning and stereotactic surgery (a minimally invasive procedure using 3D coordinates to precisely target areas in the brain). This marks the start of China’s first registration-focused clinical trial involving autologous induced pluripotent stem cell (iPSC)-derived cell therapy (cells derived from the patient’s own body and reprogrammed to a pluripotent state) for Parkinson’s disease.

At the one-month postoperative evaluation, the patient reportedly showed notable improvements in sleep quality and motor function, and no serious adverse events were reported. The patient said, “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, stated, “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.'”

UX-DA001 is an investigational autologous neural progenitor cell drug derived from patient-specific induced pluripotent stem cells. The cells originate from the patient’s peripheral blood cells, which are reprogrammed into induced pluripotent stem cells and subsequently differentiated into dopaminergic neurons (nerve cells producing dopamine, a neurotransmitter critical for motor control) in vitro. These neurons are then transplanted into the patient’s brain through minimally invasive surgery.

Because UX-DA001 originates from the patient’s own cells, the company states that this significantly reduces the risk of immune rejection, eliminating the need for immunosuppressive medications and their associated risks, such as infections, tumors, organ toxicity, and metabolic disorders.

Clinical trial approvals (Investigational New Drug, IND) for UX-DA001 were granted by China’s National Medical Products Administration (NMPA) in December 2024 and the U.S. Food and Drug Administration (FDA) in February 2025. 

The Phase I trial is being conducted at Ruijin Hospital in Shanghai, China, led by Dr. Liu Jun, MD, PhD, as the Principal Investigator. Transplantations are performed by Dr. Li Dianyou, Director of the Functional Neurosurgery Department. The trial aims to evaluate safety and tolerability, as well as investigate whether the transplanted cells survive, integrate into the brain, and improve motor function in patients with Parkinson’s disease.

Further information about the trial is available on clinicaltrials.gov (NCT#06778265).