XellSmart Completed World's First Patient Dosing in its Registrational Clinical trial for iPSC-derived Subtype-specific Neural Cell Therapy for Spinal Cord Injury

SUZHOU, China, July 28, 2025 /PRNewswire/ -- XellSmart Biopharmaceutical (Suzhou/Shanghai) Co., Ltd. achieved a significant milestone in its "world's first" China NMPA & US FDA-approved registrational clinical trial. This Phase I China registrational clinical trial for XS228 Cell Injection – the world's first "off-the-shelf", allogeneic, iPSC-derived, spinal neural progenitor cell therapy targeting major neurological disorders, specifically spinal cord injury (SCI) – has commenced. The clinical trial initiation meeting was held at The Third Affiliated Hospital of Sun Yat-sen University (SYSU-3) in Guangzhou. Following this, the world's first patient dosing with the therapy was successfully administered.

Following the world's first transplantation of clinical-grade, iPSC-derived neural progenitor cells in the registrational clinical trial, the initial subject exhibited no surgical or perioperative complications or other adverse safety events. All monitored parameters remained within normal ranges. The subject successfully completed the observation period uneventfully and has now entered the formal follow-up period.

XellSmart has achieved a significant milestone in global central nervous system (CNS) injury repair — having completed the world's first patient dosing in its China registrational clinical trial. This landmark event serves as a benchmark for evaluating the safety of transplanted, iPSC-derived, spinal-cord neural progenitor cells and analyzing their potential therapeutic effects on patients with spinal cord injury (SCI).

XellSmart is a leading biotechnology company dedicated to developing innovative iPSC-derived cell therapies. To date, the company has secured seven Investigational New Drug (IND) approvals from both China's National Medical Products Administration (NMPA) and the U.S. Food and Drug Administration (FDA) for registrational clinical trials. All approved programs focus on clinical-grade, off-the-shelf, allogeneic, iPSC-derived cell therapies targeting central nervous system (CNS) diseases with significant unmet medical needs, including:

• Parkinson's Disease (PD): The world's second most common neurodegenerative disorder, affecting over 10 million patients globally (Phase I clinical trials ongoing in China and approved in the US);
• Spinal Cord Injury (SCI): A major CNS disorder impacting more than 10 million patients worldwide with no effective treatment (a first-in-class therapy; a Phase I clinical trial ongoing in China and approved in the US);
• Amyotrophic Lateral Sclerosis (ALS): A devastating rare neurodegenerative disease (a first-in-class therapy; a Phase I/II clinical trial ongoing in China and approved in the US; granted Orphan Drug Designation by the US FDA).

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XellSmart, standing at the forefront of advancing innovative iPSC-based therapies, is committed to developing clinical-grade, off-the-shelf, allogeneic, iPSC-derived cell therapies targeting major and intractable central nervous system (CNS) diseases with no effective treatment options. The following is an overview of its clinical milestones:

In 2023, China's first iPSC-derived cell therapy (XS228 injection), developed by XellSmart, received Orphan Drug Designation (ODD) from the US FDA for the treatment of ALS.

In 2024, China's first national-level registered clinical study of an iPSC-derived cell therapy for Parkinson's disease—also developed by XellSmart—was approved and initiated, including the first case treated in China.

In 2024, the world's first national-level registered clinical study of a subtype-specific, iPSC-derived, neural progenitor cell therapy (XS228 injection) for ALS was approved. Multiple patients, including the world's first ALS case treated with this approach, received the therapy with preliminary data showing good safety profiles and slowed disease progression.

In 2025, China's first registrational Phase I clinical trial of anoff-the-shelf, allogeneic, iPSC-derived, dopaminergic neural progenitor cell therapy (XS411 injection) for Parkinson's disease was launched, led by the National Neurological Disease Medical Center at Beijing Tiantan Hospital.

In 2025, China's first randomized, double-blind, controlled Phase I/II clinical trial of XS411 injection was initiated to treat early-onset Parkinson's disease (EOPD), led by Huashan Hospital of Fudan University, a National Neurological Disease Medical Center.

In 2025, the world's first registrational Phase I clinical trial of an off-the-shelf, allogeneic, iPSC-derived, subtype-specific, neural progenitor cell therapy was launched by The Third Affiliated Hospital of Sun Yat-sen University, targeting SCI.

In 2025, the world's first Phase I/II registrational clinical trial of XS228 injection — an off-the-shelf, allogeneic, iPSC-derived, subtype-specific, neural progenitor cell therapy — was initiated by Peking University Third Hospital, targeting ALS.

XellSmart has established a fully dedicated and internationally competitive team focusing on development of clinical-grade, allogeneic, off-the-shelf, iPSC-derived cell therapies for central nervous system (CNS) diseases that currently lack effective treatment options. XellSmart has built a portfolio of proprietary, globally leading industrial technologies and platforms, fueling sustained in-house innovation centered on its core business. To develop iPSC-derived cell therapy candidates, XellSmart has established and operates an R&D center, a B+ and A-grade GMP manufacturing facility, and a quality control center spanning over 5,000 square meters.

With completed core CMC development, fully integrated clinical-grade manufacturing processes and quality control systems in place, XellSmart has successfully produced clinical batches of multiple GMP-grade, iPSC-derived, subtype-specific, neural progenitor cell therapies, currently deployed in multiple clinical trials.

XellSmart has secured multiple rounds of financing, collectively led by renowned market-driven venture capital firms including Qiming Venture Partners, Eli Lilly Asia Ventures, Sequoia Capital China (Hongshan), and others.

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