XellSmart’s iPSC Therapy for Spinal Injury Approved for Phase I Trial by U.S. FDA

SUZHOU, China, May 26, 2025 /PRNewswire/ -- In May 2025, XellSmart Biopharmaceutical (Suzhou/Shanghai) Co., Ltd. officially announced that XellSmart-developed off-the-shelf allogeneic iPSC-derived subtype-specific neural regenerative cell therapy has received official approval from both China's National Medical Products Administration (NMPA) and the U.S. Food and Drug Administration (FDA) for a Phase I clinical trial, to treat spinal cord injury (SCI) - a severe and currently untreatable disease of the central nervous system, affecting more than 15 million patients globally.

As the first regenerative clinical trial globally employing a subtype-specific neural progenitor cell therapeutic product to treat spinal cord injury, the study will be led by the Third Affiliated Hospital of Sun Yat-sen University, a nationally recognized leader in spinal cord injury treatment.

Spinal cord injury (SCI) is a globally prevalent neurological disorder. It represents a critical unmet clinical need, marked by a high rate of disability, irreversibility, early onset, and a significant burden on patients, families, and society. SCI primarily affects young and middle-aged adults, typically resulting from traffic accidents, sports injuries, or other traumas. As a condition that deeply affects a patient's social function, it requires lifelong care and rehabilitation, leading to a significant economic burden and heavy societal costs. Beyond physical disability, SCI patients often face severe challenges in employment, social integration, and mental health. In high-income countries, lifetime healthcare and caregiving costs per patient may exceed USD 1 million, placing a heavy strain on both families and public health systems.

Worldwide, over 15 million people are estimated to suffer from spinal cord injury (SCI). This figure includes over 3 million patients in China and 300,000 patients in the US. Each year, China and the US report approximately 100,000 and 18,000 new cases of acute or subacute SCI — equivalent to nearly 10 and 2 new cases every hour, respectively.

SCI often leads to partial or complete paralysis, with the loss of motor and sensory function. Most patients experience permanent disability, severely compromising their quality of life. Due to the limited regenerative capacity of the central nervous system, nerve repair following SCI remains extremely challenging. Current treatments are largely limited to rehabilitation and supportive care, with no effective therapies available to promote neural regeneration during the injury phase.

Following over four years of rigorous development and preclinical studies, XellSmart has partnered with leading clinical experts in spinal cord injury to initiate the world's first registrational clinical trial of an off-the-shelf, allogeneic, iPSC-derived, subtype-specific, regenerative neural cell therapy for spinal cord injury. This pioneering trial represents a landmark achievement in the global effort to develop regenerative therapies for spinal cord injury. Committed to advancing breakthrough treatments, XellSmart aims to redefine possibilities for SCI recovery — bringing a new hope to patients in China and around the world, and delivering tangible relief to families and communities affected by this devastating condition.