SUZHOU, China, April 15, 2025 /PRNewswire/ — In January 2025 (U.S. time), the U.S. Food and Drug Administration (FDA) officially approved the Investigational New Drug (IND) application submitted by XellSmart Biopharmaceutical (Suzhou) Co., Ltd. for its universal allogeneic off-the-shelf iPS-derived dopaminergic neural progenitor cell therapy for the treatment of Parkinson’s disease (PD), the second most common neurodegenerative disease worldwide. Concurrently, the FDA also granted XellSmart a special exemption to support the clinical trial of XellSmart’s cell therapy for PD in the US.
The approval of XellSmart’s clinical trial, along with the special exemption granted, signifies the FDA’s recognition of XellSmart’s well-established clinical-grade iPSC-derived cell drug development, production, and quality control systems, as well as its completed preclinical safety and efficacy studies. This approval serves as a strong validation of the scientific rigor, professional expertise, and clinical value of XellSmart’s cell therapy for Parkinson’s disease.
Additionally, XellSmart’s allogeneic, off-the-shelf iPSC-derived dopaminergic neural progenitor cell injection (“XS-411 Injection”) has also been approved by China’s National Medical Products Administration (NMPA) to enter Phase I clinical trials. China has the largest population of Parkinson’s disease patients globally, with an estimated 5 million cases projected by 2030. The clinical trial approvals from both the U.S. FDA and China’s NMPA offer hope that XellSmart’s XS-411 could potentially benefit Parkinson’s disease patients not only in China and the U.S. but also worldwide.
Pioneering Achievements in XellSmart’s iPSC-Derived Cell Therapies to treat PD
In 2024, XellSmart successfully conducted a clinical study of GMP-grade iPSC-derived cell therapy for treating Parkinson’s disease patients in China. This study was officially approved by China’s National Health Commission, making XellSmart the first to complete an iPSC-derived cell therapy for Parkinson’s disease in China.
In the clinical study, GMP-grade XS411 cells were transplanted into the striatal putamen of multiple patients with moderate to moderate-to-severe Parkinson’s disease. To date, these patients have been followed for 6 to 12 months, with no adverse events related to XS411 reported. All treated patients showed significant improvements in key efficacy indicators, including increased ON time, reduced MDS-UPDRS scores, and enhancements in non-motor functions. These positive early-stage clinical results provide promising evidence supporting the continued development of XellSmart’s XS411 for Parkinson’s disease patients.
Pioneering Achievements in XellSmart’s FIC Cell Therapies to treat ALS
XellSmart has developed XS228, the world’s first-in-class, off-the-shelf, allogeneic, clinical-grade iPSC-derived cell therapy for ALS, which has been granted Orphan Drug Designation by the U.S. FDA. This designation provides market exclusivity, priority review, and special regulatory supports under the U.S. Orphan Drug Act.
In 2024, XellSmart received official approval from China’s National Health Commission to conduct a clinical study of XS228 for ALS in China, including the world’s first case of allogeneic, off-the-shelf iPSC-derived cell transplantation in an ALS patient. Clinical follow-up results showed that XS228 is safe and effectively slows disease progression compared to the current standard of care.
XellSmart’s off-the-shelf, allogeneic, clinical-grade iPSC-derived cell therapy (XS228) for ALS has been approved by the FDA to enter Phase I clinical trials, making it the first-in-class regenerative neural cell therapy for ALS.
These milestones position XellSmart as a global leader in advancing innovative iPSC-derived regenerative cell therapies for CNS diseases, including Parkinson’s disease and ALS.
Dr. Michael Lee (Xiang Li ), founder and CEO of XellSmart, stated:
“April 11th marks World Parkinson’s Disease Day. We named our clinical-grade iPS-derived dopaminergic neural progenitor cell therapy “XellSmart-411 Injection” (XS-411) as a constant reminder to stay true to our mission and remain dedicated to our original aspiration.
XellSmart is committed to developing universal, off-the-shelf, allogeneic, clinical-grade iPSC-derived regenerative cell therapies to address CNS diseases with significant unmet clinical needs.
The FDA’s unconditional approval of the INDs for XellSmart’s XS411 and XS228 products, targeting Parkinson’s disease and ALS, paves the way for these therapies to advance into clinical trials in the United States and lays the foundation for future commercialization in the global market.
XellSmart remains firmly committed to providing allogeneic, off-the-shelf, iPSC-derived regenerative cell therapies to patients with CNS diseases worldwide.”
Funding and BD cooperation contact: BD@xellsmart.com
About XellSmart
XellSmart is dedicated to developing universal, off-the-shelf, allogeneic, clinical-grade iPSC-derived cell therapies to address Parkinson’s disease and other CNS diseases that currently lack effective clinical solutions.
XellSmart has built a highly skilled, full-time team with extensive international expertise and established a portfolio of proprietary, cutting-edge technologies and platforms to drive sustained innovation in iPSC-derived cell therapies for CNS diseases.
XellSmart operates >5,000+ square meter facility comprising an advanced R&D center, GMP production facility, and quality control center. XellSmart has successfully achieved large-scale manufacturing of multiple GMP-grade iPSC-derived neural progenitor cell therapies.
XellSmart’s allogeneic iPSC-derived cell therapies, XS411 and XS228, targeting Parkinson’s disease and ALS, have been approved to enter Phase I clinical trials in both the United States and China.
To date, XellSmart has secured six rounds of financing from top-tier investors, including Frees Fund, Qiming Venture Partners, Lilly Asia Ventures, Sequoia China, and others, underscoring strong market confidence in its innovative therapies and growth potential.
Funding and BD cooperation contact: BD@xellsmart.com
