XellSmart Gains FDA Approval for INDs in CNS Cell Therapies Targeting Parkinson's, Spinal Cord Injury, and ALS

XellSmart Biomedical Co., Ltd., based in Suzhou and Shanghai, China, is at the forefront of biotechnology advancements, focusing on pioneering iPSC-derived cell therapies. The company has achieved recognition with seven Investigational New Drug (IND) approvals from both China's National Medical Products Administration (NMPA) and the U.S. Food and Drug Administration (FDA). These approvals are centered on clinical-grade, off-the-shelf, allogeneic iPSC-derived cell therapy candidates targeting central nervous system (CNS) diseases, which currently have significant unmet medical needs.

Among the conditions being targeted are Parkinson's Disease (PD), Spinal Cord Injury (SCI), and Amyotrophic Lateral Sclerosis (ALS). Parkinson's Disease, the second most prevalent neurodegenerative disease worldwide, affects over 10 million people, and XellSmart is conducting Phase I clinical trials both in China and the United States. For Spinal Cord Injury, another major CNS condition impacting over 10 million individuals globally, XellSmart has initiated first-in-class Phase I clinical trials in both countries. ALS, a rare and severe neurodegenerative disease, is also in the company's focus, with Phase I/II clinical trials underway in China and Phase I trials in the United States. Notably, the FDA has granted Orphan Drug designation to XellSmart's treatment for ALS.

XellSmart has made significant strides in the treatment of Parkinson's Disease through iPSC-derived dopaminergic neural progenitor cell therapy, marking a milestone with the first case treated in China. This study, approved by China's National Health Commission (NHC), involved the transplantation of clinical-grade iPSC-derived cells into patients' putamen of the striatum. Following over a year of monitoring, no adverse events related to the cell therapy were reported. Several patients exhibited notable improvements in key effectiveness measures such as "on/off" time duration and MDS-UPDRS scores, along with enhancements in non-motor symptoms.

The year 2023 marked a significant achievement for XellSmart with the FDA's Orphan Drug Designation for its iPSC-derived cell therapy (XS228 injection) targeting ALS. In 2024, XellSmart established itself further by initiating China’s first national-level registered clinical study for an iPSC-derived cell therapy for Parkinson's Disease, subsequently treating the first case in the country. The same year saw the initiation of the world’s first national-level clinical study for a subtype-specific iPSC-derived neural progenitor cell therapy for ALS, yielding promising early results.

In 2025, XellSmart launched China's first registrational Phase I clinical trial for an off-the-shelf allogeneic iPSC-derived dopaminergic neural progenitor cell therapy (XS411 injection) for Parkinson's Disease. This was spearheaded by Beijing Tiantan Hospital, a National Neurological Disease Medical Center. Additionally, the company initiated China’s first randomized, double-blind, controlled Phase I/II clinical trial of the XS411 injection to treat early-onset Parkinson's disease (EOPD), led by Huashan Hospital of Fudan University.

XellSmart also broke new ground with the world’s first Phase I/II registrational clinical trial of an off-the-shelf, allogeneic iPSC-derived subtype-specific neural progenitor cell therapy targeting ALS, launched by Peking University Third Hospital. In another pioneering move, The Third Affiliated Hospital of Sun Yat-sen University commenced the world's first registrational Phase I clinical trial for an off-the-shelf iPSC-derived cell therapy targeting Spinal Cord Injury.

The company has built a comprehensive team dedicated to the development of clinical-grade, allogeneic, off-the-shelf iPSC-derived therapies for CNS diseases that currently lack effective treatment options. XellSmart's continued innovations are backed by a robust R&D center, GMP manufacturing facilities, and a quality control center. The company has also secured substantial funding from esteemed venture capital firms such as Qiming Venture Partners, Eli Lilly Asia Ventures, and Sequoia Capital China, facilitating its ongoing research and development efforts in this cutting-edge field.