#China #Shanghai #Parkinson's Disease #UniXell Biotech #UX-DA001

UniXell Biotechnology Marks First Patient Dosed with UX-DA001

In a remarkable advancement towards treating Parkinson's disease, Shanghai UniXell Biotechnology Co., Ltd. has announced that the first patient has been dosed with UX-DA001, a groundbreaking investigational autologous iPSC-based cell therapy. The milestone event took place during the clinical trial's Phase 1 study, emphasizing UniXell's commitment to innovative cell therapies designed to transform the landscape of treatment for neurodegenerative diseases.

The administration of UX-DA001 occurred on March 1, 2025, conducted by Dr. Li Dianyou at Ruijin Hospital in Shanghai. This pivotal moment not only marks a significant step for UniXell but also establishes it as the first registration-directed clinical trial for autologous iPSC-derived cell therapy involving Parkinson's disease in China.

Postoperative assessments conducted a month after the procedure have shown promising results, with reported enhancements in the patient's sleep quality and motor function. Notably, the initial recipient shared a heartfelt testimony, expressing, "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." The absence of severe adverse events during this period is a testament to the safety of this innovative therapy.

Dr. Liu Jun, the Principal Investigator of the trial and Director of the Neurology Department at Ruijin Hospital, further explained the potential benefits of cell therapy compared to conventional drug treatments. Current standard approaches predominantly rely on medications, which may lead to diminishing effectiveness over time and uncontrollable disease progression. In contrast, UX-DA001 offers an exciting alternative, with the potential for long-term benefits following a single treatment.

Exploring UX-DA001

So, what is UX-DA001? This investigational drug is derived from the patient's own peripheral blood cells, which are reprogrammed into induced pluripotent stem cells (iPSCs) before being differentiated into dopaminergic neurons in vitro. This meticulous process allows for the creation of effective neural progenitor cells, which are then transplanted into the patient’s brain through a minimally invasive procedure. The therapy minimizes the risk of immune rejection since it uses the patient’s cells, eliminating the need for immunosuppressive drugs—a notable advantage that also circumvents potential side effects such as increased susceptibility to infections, organ toxicity, and metabolic disorders.

The clinical trial, which is conducted under the supervision of experienced professionals at Ruijin Hospital, aims to assess the safety and tolerability of the UX-DA001 cell transplantation. Moreover, it evaluates the survival and integration of the transplanted cells, as well as their impact on motor functions for subjects diagnosed with Parkinson's disease.

UX-DA001 has received approvals from both China's National Medical Products Administration (NMPA) and the U.S. Food and Drug Administration (FDA), demonstrating the compound's significant promise not just in China, but potentially across the globe. Already, UniXell has an impressive roadmap, with another compound—UX-DA002, an allogenic cell therapy for Parkinson's—having its IND application accepted by the NMPA.

About UniXell Biotechnology

Founded in 2021, UniXell Biotechnology focuses on developing novel cell therapy drugs targeting neurological disorders such as Parkinson's disease and epilepsy. Their extensive 4,000 square-meter R&D center and GMP facilities support the creation of therapies utilizing advanced technologies. The company has drawn considerable interest from prominent investors like Hillhouse Capital and CDH Investments, highlighting confidence in its ability to innovate and provide effective treatments for neurological conditions.

As the clinical trials move forward, the scientific and medical communities remain keenly interested in the results, eagerly anticipating advancements in treatment options for patients suffering from Parkinson's disease.

For further details about the ongoing trial, prospective participants can visit clinicaltrials.gov (NCT#06778265).