UniXell Biotechnology begins dosing first patient with UX-DA001 in Parkinson's Phase 1 trial

Shanghai UniXell Biotechnology Co., Ltd. (UniXell Biotech), a company focused on cell therapy innovation, recently announced a breakthrough in Parkinson's disease treatment with its proprietary drug, UX-DA001. The first administration of this drug took place in China in March 2025, with a Phase I clinical trial designed to assess its safety, tolerability, and preliminary effectiveness.

On March 1, 2025, an important surgical procedure was conducted by Dr. Li Dianyou, head of the Functional Neurosurgery Department at Ruijin Hospital. The surgery marked the start of China's inaugural registration-directed clinical trial for autologous iPSC-derived cell therapy targeting Parkinson's disease. The early postoperative results have been promising, showing notable improvements in the patient’s sleep quality and motor functions without any severe side effects. The recipient of the treatment expressed optimism, noting significant enhancements in sleep and a hopeful outlook for continued progress.

Dr. Liu Jun, a key figure in the trial and director of the Neurology Department at Ruijin Hospital, highlighted the limitations of current Parkinson's therapies, which mostly involve medications to manage symptoms. Over time, these treatments can lose effectiveness and fail to halt disease progression. In contrast, cell therapy presents the possibility of a one-time intervention with lasting benefits.

The investigational drug UX-DA001 is unique in that it uses autologous iPSC-derived neural progenitor cells. These cells are derived from the patient’s blood cells, reprogrammed into induced pluripotent stem cells, and then transformed into dopaminergic neurons. This process aims to minimize the risk of immune rejection since the cells originate from the patient themselves, thereby eliminating the need for immunosuppressive drugs. This approach reduces the potential for side effects like increased infection risks, organ toxicity, and metabolic issues, ultimately providing a safer and more effective treatment option that improves the patient's quality of life.

UX-DA001 has received clinical approval for trial in both China and the United States, positioning it as a promising global treatment for Parkinson's disease. The Phase I trial is being conducted at Ruijin Hospital, guided by Dr. Liu Jun and performed by Dr. Li Dianyou. The study is vital in assessing the safety, tolerability, and effectiveness of UX-DA001 in improving motor functions in Parkinson's patients.

Shanghai UniXell Biotechnology was founded in 2021 and focuses on developing cell therapy drugs for neurological conditions such as Parkinson's disease and epilepsy. Apart from UX-DA001, the company has also progressed with UX-DA002, an allogeneic cell therapy, which has received regulatory interest.

The company operates a substantial research and development center and GMP-compliant facilities, enabling the advancement of its innovative technology platforms. These include reprogramming and stem cell differentiation technologies, lineage tracing, and high-precision gene editing. These platforms support the creation of neural cell drugs that offer high purity and stability, ensuring successful clinical outcomes. UniXell Biotech's progress is further reinforced by significant financial backing from prominent investors like Hillhouse Capital and others, reflecting strong market confidence in its pioneering treatments and potential growth.