FDA Approves XellSmart's iPSC Cell Therapy for Spinal Cord Injury Trial

In May 2025, XellSmart Biopharmaceutical (Suzhou/Shanghai) Co., Ltd. announced a significant milestone in the field of neuroregenerative medicine. The company received approval from China's National Medical Products Administration and the U.S. Food and Drug Administration to commence Phase I clinical trials for their innovative therapy aimed at treating spinal cord injury (SCI), a condition affecting over 15 million people worldwide. This groundbreaking trial is the first of its kind to utilize subtype-specific neural progenitor cell therapy derived from induced pluripotent stem cells (iPSCs) to potentially regenerate neural connections in the damaged spinal cord.

Spinal cord injury represents a major challenge in the medical field due to its irreversible nature and the profound impact it has on individuals and society. SCI is particularly prevalent among young and middle-aged adults, often resulting from accidents or trauma. The condition leads to varying degrees of paralysis, severely impairing motor and sensory functions, and necessitates lifelong care and rehabilitation. This places a significant financial burden on families and healthcare systems, with costs in high-income countries exceeding USD 1 million per patient. Furthermore, patients with SCI face difficulties integrating into society, finding employment, and maintaining mental health.

The clinical trial will be conducted at the Third Affiliated Hospital of Sun Yat-sen University, recognized for its leadership in spinal cord injury treatment. The trial seeks to evaluate the safety and efficacy of XellSmart's iPSC-derived neural regenerative cell therapy, a promising approach that may redefine treatment possibilities for SCI. Current therapies primarily focus on rehabilitation and supportive care, as the central nervous system's limited ability to regenerate has hindered progress. XellSmart's therapy aims to overcome these limitations by promoting neural regeneration during the injury phase, potentially offering a transformative solution for SCI patients.

Over the past four years, XellSmart has collaborated with leading experts to develop this pioneering therapy. The company is committed to advancing breakthrough treatments that bring hope to SCI patients in China and beyond, relieving the burden on families and communities affected by this debilitating condition. XellSmart's efforts extend beyond spinal cord injury, as they have secured approvals for several clinical trials targeting central nervous system diseases, including Parkinson's disease and amyotrophic lateral sclerosis (ALS). These trials mark significant advancements in tackling severe neurological disorders.

In 2023, XellSmart's XS228 injection, a cell therapy for CNS diseases, received FDA approval and global orphan drug designation. The following year, they launched China's first registered clinical study of iPSC-derived therapy for Parkinson's disease, demonstrating safety and notable improvements in patients' symptoms. The company also initiated a clinical study for ALS, showing promising results in slowing disease progression.

XellSmart's commitment to innovation is reflected in their robust portfolio of proprietary technologies and platforms, alongside their integrated R&D and manufacturing capabilities. With a dedicated team and competitive infrastructure, they have successfully produced multiple GMP-grade iPSC-derived neural progenitor therapies for clinical use. The company continues to attract significant investment from renowned venture capital firms, supporting their vision of transforming treatment paradigms for CNS diseases that currently lack effective solutions.

With the initiation of this clinical trial for spinal cord injury, XellSmart is poised to make a substantial impact in the field of regenerative medicine, paving the way for new therapeutic options and improving the lives of millions affected by neurological conditions.