The U.S. Food and Drug Administration (FDA) has officially approved XellSmart Biopharmaceutical's Investigational New Drug (IND) applications for its innovative cell therapies targeting two major neurodegenerative diseases. The approval, granted in January 2025, covers XellSmart's universal allogeneic off-the-shelf iPSC-derived dopaminergic neural progenitor cell therapy for Parkinson's disease and its first-in-class iPSC-derived cell therapy for amyotrophic lateral sclerosis (ALS).
The FDA also granted XellSmart a special exemption to support the clinical trial of its Parkinson's disease therapy in the United States, signifying recognition of the company's clinical-grade iPSC-derived cell drug development, production, and quality control systems, as well as its completed preclinical safety and efficacy studies.
Dual Regulatory Approvals Expand Global Reach
In a significant regulatory achievement, XellSmart's allogeneic, off-the-shelf iPSC-derived dopaminergic neural progenitor cell injection ("XS-411 Injection") has also received approval from China's National Medical Products Administration (NMPA) to enter Phase I clinical trials. This dual approval is particularly important as China has the largest population of Parkinson's disease patients globally, with projections indicating approximately 5 million cases by 2030.
Dr. Michael Lee (Xiang Li), founder and CEO of XellSmart, emphasized the significance of these approvals: "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."
Promising Clinical Evidence for Parkinson's Disease Therapy
XellSmart has already demonstrated promising results in early clinical studies. In 2024, the company conducted a clinical study of GMP-grade iPSC-derived cell therapy for treating Parkinson's disease patients in China, becoming the first to complete such a study in the country.
In this clinical investigation, GMP-grade XS411 cells were transplanted into the striatal putamen of multiple patients with moderate to moderate-to-severe Parkinson's disease. Follow-up data spanning 6 to 12 months revealed no adverse events related to XS411, while all treated patients showed significant improvements in key efficacy indicators, including:
- Increased ON time (periods when medication effectively controls symptoms)
- Reduced MDS-UPDRS scores (a standard measure of Parkinson's disease severity)
- Enhancements in non-motor functions
These positive early-stage clinical results provide compelling evidence supporting the continued development of XellSmart's XS411 for Parkinson's disease patients.
Breakthrough Therapy for ALS
XellSmart's innovation extends beyond Parkinson's disease to ALS, a devastating neurodegenerative condition with limited treatment options. The company has developed XS228, described as the world's first-in-class, off-the-shelf, allogeneic, clinical-grade iPSC-derived cell therapy for ALS. This pioneering therapy has been granted Orphan Drug Designation by the U.S. FDA, providing market exclusivity, priority review, and special regulatory support under the U.S. Orphan Drug Act.
In 2024, XellSmart received approval from China's National Health Commission to conduct a clinical study of XS228 for ALS, which included the world's first case of allogeneic, off-the-shelf iPSC-derived cell transplantation in an ALS patient. Clinical follow-up results demonstrated that XS228 is safe and effectively slows disease progression compared to the current standard of care.
The FDA's approval for XS228 to enter Phase I clinical trials positions it as the first-in-class regenerative neural cell therapy for ALS, addressing a significant unmet medical need.
Technology Platform and Manufacturing Capabilities
XellSmart's technological platform represents a significant advancement in regenerative medicine. The company has established a comprehensive infrastructure to support its innovative therapies, including:
- A 5,000+ square meter facility comprising an advanced R&D center
- GMP production facilities meeting international standards
- A dedicated quality control center
- Large-scale manufacturing capabilities for GMP-grade iPSC-derived neural progenitor cell therapies
This infrastructure has enabled XellSmart to develop universal, off-the-shelf, allogeneic, clinical-grade iPSC-derived cell therapies that could potentially transform treatment paradigms for CNS diseases.
Financial Backing and Future Prospects
XellSmart's innovative approach has attracted substantial financial support from leading investment firms. To date, the company has secured six rounds of financing from top-tier investors, including Frees Fund, Qiming Venture Partners, Lilly Asia Ventures, and Sequoia China. This strong financial backing underscores market confidence in XellSmart's innovative therapies and growth potential.
The company's focus on allogeneic, off-the-shelf therapies addresses key challenges in cell therapy, including manufacturing scalability and accessibility. By developing universal therapies that don't require patient-specific manufacturing, XellSmart aims to make these advanced treatments available to a broader patient population.
As XellSmart advances its therapies through clinical trials in both the United States and China, the company is positioned to potentially deliver transformative treatments for patients with Parkinson's disease, ALS, and potentially other CNS disorders with significant unmet clinical needs.
