UniXell Biotechnology announces the first patient dosed with UX-DA001, an investigational autologous iPSC based cell therapy for the treatment of Parkinson's disease, in its Phase 1 study

SHANGHAI, May 23, 2025 /PRNewswire/ -- Shanghai UniXell Biotechnology Co., Ltd. (UniXell Biotech), a biotech company dedicated to innovative cell therapies, announced the successful first administration of its proprietary Parkinson's disease cell drug UX-DA001 in China in March 2025. 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 surgical path planning and the subsequent stereotactic surgery for transplantation. The successful surgery marks the official initiation of China's first registration-directed clinical trial for an autologous iPSC-derived cell therapy for Parkinson's disease.

The one-month postoperative follow-up revealed significant improvements in the patient's sleep quality and motor function, with no severe adverse events reported. The first recipient of UniXell's autologous stem cell-derived drug, UX-DA001, shared, "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, noted "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 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.

The phase 1 study is conducted at Ruijin hospital in Shanghai, China, under the guidance of Dr. Liu Jun, MD, PhD, Principal Investigator (PI). The transplantation is performed by Dr. Li Dianyou, the director of the Functional Neurosurgery Department. This clinical study is designed to evaluate the safety and tolerability of UX-DA001 cell transplantation. It 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).

About UniXell Biotechnology Co., Ltd.

Shanghai UniXell Biotechnology Co., Ltd. was established in 2021, specializing in the research and development of cell therapy drugs for neurological diseases, including Parkinson's disease and epilepsy. In addition to its autologous UX-DA001, UniXell`s IND application for UX-DA002, an allogenic cell therapy for Parkinson`s disease, has been recently accepted by NMPA.

The company is equipped with a 4000 m² R&D center and GMP facilities. It has developed four major innovative technology platforms based on reprogramming technology, stem cell differentiation technology, SISBAR lineage tracing technology, and high-precision gene editing. These platforms facilitate the development and production of neural cell drugs with higher purity and more stable efficacy, ensuring efficient and safe clinical treatment outcomes. As of the present, UniXell has successfully obtained several rounds of funding from esteemed investors, such as Hillhouse Capital, CDH Investments, Fosun Group, Sherpa Healthcare Partners, TF Capital, and Tianshi Capital, among others. This underscores the robust market confidence in its pioneering therapeutic solutions and its potential for growth.

Contact:
UniXell Biotechnology Co., Ltd.
[email protected]

SOURCE UniXell Biotechnology