Unixell Biotech reported a case study of UX-DA001, an iPSC-derived autologous cell therapy for Parkinson Diseases at MDS Congress 2025

SHANGHAI, Oct. 16, 2025 /PRNewswire/ -- Unixell Biotech, a clinical-stage bitotech company focused on developing innovative cell therapies for Parkinson's disease (PD) and other neurological disorders, announced positive 6-month follow-up data from the first patient in its phase 1 clinical trial for UX-DA001, an iPSC-derived autologous cell therapy for PD. These data were presented on October 8th at the International Congress of Parkinson's Disease and Movement Disorders (MDS), Honolulu, USA. UX-DA001 is the world's first autologous cell therapy product for Parkinson's Disease which has both received clinical trial approval in China and the United States.

The study is led by Principal Investigator Dr. Liu Jun, Professor and Director of the Department of Neurology at Ruijin Hospital Affiliated with School of Medicine, Shanghai Jiao Tong University. The transplantation surgery was performed by Dr. Li Dianyou, Director of the Functional Neurosurgery Department at Ruijin Hospital. Dr. Zhou Liche, a member of  the study team, delivered the presentation, which has been selected as a "Late Breaking" oral platform presentation.

The Phase I clinical trial of UX-DA001 officially commenced  in early 2025. The first patient enrolled in the trial is a female one with moderate-to-severe PD who, despite taking four anti-Parkinson's medications daily pre-operatively, still experienced significant symptom fluctuations, along with debilitating non-motor symptoms such as sleep disorders and autonomic dysfunction, severely impacting her quality of life.

The 6-month post-treatment data demonstrated a favorable safety profile and significant therapeutic effects for UX-DA001:

• The patient tolerated the procedure well, with no serious adverse events or adverse events related to the transplanted cells.
• Significant improvement in motor function: The MDS-UPDRS-III score improved by 21 points during the "OFF-period" and 9 points during the "ON-period", representing an improvement ratio exceeding 45% in both states.
• Daily "OFF" time reduced by an average of 3.6 hours, while "ON" time without troublesome dyskinesia increased by 3.3 hours.
• Non-motor symptoms were alleviated, with early positive improvements were observed in both the NMSS scale and PDQ-39 quality of life scores.
• ¹⁸ F-FP-CIT PET imaging showed consecutively increased PET uptake signals in the transplanted bilateral putamen, providing objective evidence for dopamine transporter expression, cell survival and functional integration.

Dr. Zhou Liche commented, "The reduction of Unified Parkinson's Disease Rating Scale (UPDRS) part III score, indication an improvement in motor function of the first patient, is very encouraging and promising. Furthermore, in addition to the UPDRS scores, which remains the most frequently employed primary endpoint, neuroimaging biomarkers are increasingly recognized as critical outcome measures in PD clinical trials owing to their intrinsic objectivity. In this study, we used ¹⁸F-FP-CIT PET to measure the density of presynaptic dopamine transporter. The specific binding ratios (SBR) of ¹⁸F-FP-CIT directly mirrors nigrostriatal dopaminergic terminal integrity and serial scans revealed an increasing UX-DA001 graft-derived dopaminergic innervation in putmen. By integrating these imaging readouts with UPDRDS-based clinical scores, the trial achieves a dual-layer assessment—subjective functional status is supported by objective biological evidence. This approach strengthens the validation of clinical findings and accelerates go/no-go decisions for cell therapies in PD."

"The key advantage of UX-DA001 lies in its technology platforms enabling higher target cell proportion and purity. This allows for achieving comparable efficacy with a lower total number of cells injected, consequently shortening the transplantation procedure time and enhancing its reliability and safety." Dr. Zhou remarked, in response to a question about the study's distinctive features compared to other autologous stem cell research.

The Phase I clinical trial for UX-DA001 is still ongoing. Encouraged by the positive results from the first patient, the UniXell Biotech and the clinical team are confident with the promising prospects of the trial. Recognizing that strategic partnerships can accelerate development and expand access to innovative therapies, we welcome discussions with biopharmaceutical companies and investors, both domestically and internationally, who share our vision of providing a new and improved treatment option for Parkinson's disease patients worldwide.

[About the International Parkinson and Movement Disorder Society (MDS)]

The International Parkinson and Movement Disorder Society(MDS) is the most globally authoritative premier professional organization in the field of Parkinson's disease and movement disorders. It brings together top-tier international clinical experts, scientists, and healthcare professionals, dedicated to advancing research, clinical care, education, and awareness. The Society was founded in 1985 by Professors Stanley Fahn and C. David Marsden and formally renamed the International Parkinson and Movement Disorder Society in 2013.

About UX-DA001 and Phase 1 Trial

UX-DA001 is an investigational autologous iPSC-derived neural progenitor cell drug. It is derived from the patient's own peripheral blood cells, which are firstly reprogrammed into induced pluripotent stem cells and then differentiated into dopaminergic neurons in vitro. It is then transplanted into the patient's brain through a minimally invasive surgery. UX-DA001 leverages the patient's own cells, significantly reducing the risk of immune rejection. Patients do not require immunosuppressive drugs, thereby avoiding potential side effects such as increased infection and tumor risks, organ toxicity, and metabolic disorders, thus achieving a safer and more effective treatment that significantly improves the quality of life for patients. The UX-DA001 has received clinical trial approvals (Investigational New Drug, IND) from both the National Medical Products Administration (NMPA) of China and the U.S. Food and Drug Administration (FDA) in December 2024 and February 2025, respectively, offering UX-DA001 a potential to have implications on patients with Parkinson's disease in not only China but also globally.

[About UX-DA001]

UX-DA001 is human midbrain dopaminergic neural precursor cells, developed using Unixell's proprietary technology platforms: a high-throughput stem cell differentiation lineage tracing platform, a highly efficient neural differentiation platform, and an iPSC reprogramming platform. Leveraging these advanced technologies, the product can generate highly pure, stable, and potent human midbrain dopaminergic neural precursor cells, aiming to effectively repair damaged neural circuits and restore dopamine levels in the brain.

[About Unixell Biotech]

Shanghai Unixell Biotech, founded in 2021, focuses on developing cell therapies for neurological diseases such as Parkinson's disease and epilepsy. The company operates a 4,000-square-meter R&D center and GMP facility. It has received numerous honors and awards. In December 2024, Unixell's UX-DA001 successfully obtained clinical trial approvals from both the National Medical Products Administration (NMPA) of China and the U.S. FDA, making it the first iPSC-derived autologous cell therapy for Parkinson's disease in China, and the second globally, to enter the clinical stage.

SOURCE UniXell Biotechnology