A female patient with moderate-to-severe Parkinson's disease has shown remarkable clinical improvement following treatment with UX-DA001, an investigational iPSC-derived autologous cell therapy, according to data presented at the 2025 International Congress of Parkinson's Disease and Movement Disorders in Honolulu, Hawaii.
The first patient in the ongoing phase 1 open-label dose-escalation trial (NCT06778265) demonstrated significant motor function improvements at 6-month follow-up. Despite taking four daily anti-Parkinson's medications pre-operatively, the patient continued to experience substantial motor fluctuations and disabling nonmotor symptoms that severely impacted her quality of life.
Substantial Motor Function Gains
Treatment results showed the patient improved by 21 points in the OFF stage (47.7%) and by 9 points in the ON state (exceeding 45% improvement) as measured by Movement Disorders Society Unified PD Rating Scale (MDS-UPDRS) Part III scores. Daily OFF time was reduced by an average of 3.6 hours, while ON time without troublesome dyskinesia increased by 3.3 hours.
"The reduction of UPDRS part III score, indication an improvement in motor function of the first patient, is very encouraging and promising," said coauthor Zhou Liche, MD, physician in the Department of Neurology at Ruijin Hospital, who delivered the presentation selected as a "Late Breaking" oral platform presentation.
Objective Evidence of Graft Function
¹⁸F-FP-CIT positron emission tomography (PET) imaging provided objective evidence for treatment efficacy, showing consecutively increased PET uptake signals in the transplanted bilateral putamen. The specific binding ratios (SBR) of ¹⁸F-FP-CIT directly mirror nigrostriatal dopaminergic terminal integrity, and serial scans revealed increasing UX-DA001 graft-derived dopaminergic innervation in putamen.
"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," Zhou explained. "This approach strengthens the validation of clinical findings and accelerates go/no-go decisions for cell therapies in PD."
Safety Profile and Non-Motor Benefits
The patient tolerated the procedure well, with no serious adverse events or adverse events related to the transplanted cells reported. Surgery-related adverse events, including headache, leukocytosis, and dyskinesia, were mild and transient. Non-motor symptoms were also alleviated, with early positive improvements observed in both the NMSS scale and PDQ-39 quality of life scores, including improvements in sleep, anxiety, and urinary symptoms.
Advanced Technology Platform
UX-DA001 leverages proprietary technology platforms that enable higher target cell proportion and purity compared to other approaches. "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," Zhou noted.
The therapy is derived from the patient's own peripheral blood cells, which are reprogrammed into induced pluripotent stem cells and then differentiated into dopaminergic neurons in vitro before transplantation into the brain through minimally invasive surgery. This autologous approach significantly reduces the risk of immune rejection, eliminating the need for immunosuppressive drugs.
Preclinical Foundation and Trial Design
Preclinical studies demonstrated that grafted cells consistently yielded high numbers of in vivo midbrain dopaminergic neurons across batches in the 6-OHDA-lesioned SCID model at 6 months post transplantation. More than 50% of grafted cells expressed dopaminergic markers tyrosine hydroxylase and EN1. GLP safety assessments up to 6 months demonstrated no adverse effects, and tumorigenicity studies through 52 weeks revealed no tumor formation or abnormal cellular proliferation.
The phase 1 trial, led by Liu Jun, MD, professor and director of the Department of Neurology at Ruijin Hospital in China, will enroll patients with moderate-to-advanced PD aged 50 to 75 years. Participants receive bilateral putamen injections of UX-DA001 at either a low dose (0.9 × 10⁶ cells/hemisphere) or a high dose (1.8 × 10⁶ cells/hemisphere), with safety monitored by an independent committee.
Regulatory Milestones
UX-DA001 has received clinical trial approvals 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. This makes it the world's first autologous cell therapy product for Parkinson's disease to receive clinical trial approval in both countries, and the second globally to enter the clinical stage.
Shanghai Unixell Biotech, founded in 2021, operates a 4,000-square-meter R&D center and GMP facility. The company is actively seeking strategic partnerships with biopharmaceutical companies and investors to accelerate development and expand access to this innovative therapy for Parkinson's disease patients worldwide.
