FDA Approves XellSmart's iPSC-derived Cell Therapies for Parkinson's and ALS Phase I Trials

In April 2025, XellSmart Biopharmaceutical (Suzhou) Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) approved its Investigational New Drug (IND) application for an innovative cell therapy to treat Parkinson's disease, a major neurodegenerative disorder. This therapy involves the use of universal allogeneic off-the-shelf induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cells. This approval, alongside a special exemption for clinical trials in the U.S., highlights the FDA's recognition of XellSmart's advanced development, production, and quality control systems for clinical-grade iPSC-derived cell drugs. It also underlines the significance of XellSmart's completed preclinical safety and efficacy studies.

In concurrent developments, China's National Medical Products Administration (NMPA) also approved XellSmart's iPSC-derived dopaminergic neural progenitor cell injection, known as XS-411, for Phase I clinical trials. With China housing the world's largest population of Parkinson's disease patients, projected to reach 5 million by 2030, these trial approvals offer optimism for patients not only in China and the U.S. but potentially around the globe.

XellSmart's pioneering efforts in iPSC-derived cell therapies have been evident since 2024 when they successfully conducted a clinical study in China. Approved by China's National Health Commission, this study marked the first completion of an iPSC-derived cell therapy for Parkinson's disease in the country. The study involved the transplantation of GMP-grade XS411 cells into the striatal putamen of patients with moderate to severe Parkinson's disease. Follow-ups ranging from 6 to 12 months showed no adverse events related to XS411, with all patients experiencing significant improvements in key efficacy indicators such as increased ON time, reduced MDS-UPDRS scores, and enhancements in non-motor functions. These encouraging early results support further development of XellSmart's XS411 for Parkinson's patients.

In addition to Parkinson's disease, XellSmart is advancing its iPSC-derived therapies for Amyotrophic Lateral Sclerosis (ALS) with a first-in-class cell therapy product named XS228. This off-the-shelf, allogeneic clinical-grade iPSC-derived cell therapy has been granted Orphan Drug Designation by the FDA. This status ensures market exclusivity, priority review, and regulatory support under the U.S. Orphan Drug Act. In 2024, XellSmart gained approval from China's National Health Commission to conduct clinical studies for XS228 in ALS patients. Notably, this included the world's first case of allogeneic, off-the-shelf iPSC-derived cell transplantation in an ALS patient. Clinical follow-ups revealed that XS228 is safe and slows disease progression more effectively than current standard treatments.

The FDA's approval for XS228 to enter Phase I clinical trials marks it as the first-in-class regenerative neural cell therapy for ALS. These achievements position XellSmart as a global leader in the field of innovative iPSC-derived regenerative cell therapies for central nervous system (CNS) diseases, including Parkinson's disease and ALS.

The founder and CEO of XellSmart, Dr. Michael Lee, emphasized the company's commitment to developing universal, off-the-shelf, allogeneic iPSC-derived regenerative cell therapies to address unmet clinical needs in CNS diseases. The unconditional approval of the INDs for XS411 and XS228 by the FDA facilitates the progression of these therapies into clinical trials in the United States, setting the stage for future global market commercialization. XellSmart remains dedicated to providing these advanced therapies to patients worldwide suffering from CNS diseases.