XellSmart Gets FDA OK for 3 iPSC Cell Therapies for Key CNS Diseases

XellSmart Biomedical Co., Ltd. (Suzhou/Shanghai, China) is a leading biotechnology company dedicated to the development of innovative iPSC-derived cell therapies.

To date, the company has received seven Investigational New Drug (IND) approvals for registered clinical trials from both the National Medical Products Administration (NMPA) of China and the U.S. Food and Drug Administration (FDA).

All approvals focus on clinical-grade, off-the-shelf and allogeneic iPSC-derived cell therapy candidates targeting central nervous system (CNS) diseases with significant unmet medical needs, including:

• Parkinson's Disease (PD), the world's second most prevalent neurodegenerative disease affecting over 10 million patients globally (Phase I clinical trials in both China and the U.S.);
• Spinal Cord Injury (SCI), a major CNS disease impacting more than 10 million patients globally (First-in-Class; Phase I clinical trials in China and the U.S.); and
• Amyotrophic lateral sclerosis (ALS), a devastating rare neurodegenerative disease (First-in-Class; Phase I/II clinical trial in China and Phase I in the U.S.; designated as an Orphan Drug by the U.S. FDA).

Parkinson's Disease Milestone: First Case Treated in China

XellSmart has completed multiple cases of iPSC-derived dopaminergic neural progenitor cell therapy for patients with Parkinson's disease, including "the first case" in China. This clinical study was approved by the National Health Commission (NHC) of China.

In the clinical study, clinical-grade iPSC-derived dopaminergic neural progenitor cells were transplanted into the putamen of the striatum in patients with moderate to advanced Parkinson's disease.

With follow-up periods exceeding 12 months, no cell therapy-related adverse events have been observed. Multiple patients showed significant improvements in key efficacy indicators, including "on/off" time duration and MDS-UPDRS scores, as well as notable enhancements in non-motor symptoms.

Commitment to CNS Diseases with No Effective Treatments

XellSmart is committed to developing clinical-grade, off-the-shelf allogeneic iPSC-derived cell therapies targeting central nervous system (CNS) diseases with no effective treatment options.

XellSmart is at the forefront of advancing innovative iPSC-based therapies to address major and intractable CNS disorders:

Timeline of Key Milestones (2023–2025)

2023

• China's first iPSC-derived cell therapy (XS228 injection), developed by XellSmart, was granted by FDA as Orphan Drug Designation (ODD) for the treatment of amyotrophic lateral sclerosis (ALS).

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.
• 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 therapy with good safety profiles and preliminary data showing slowed disease progression.

2025

• China's first registrational Phase I clinical trial of an off-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.
• 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.
• 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 spinal cord injury (SCI) a major neurological disorder with no effective treatment.
• 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, a devastating rare neurodegenerative disease.

XellSmart’s Infrastructure: Facilities and Manufacturing

XellSmart has established a fully dedicated, internationally competitive "All-In" 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
• A quality control center
• Combined footprint: Over 5,000 square meters

Clinical Manufacturing and CMC Readiness

Multiple clinical-grade iPSC-derived cell therapy candidates developed by XellSmart have completed core CMC development, with 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.

Financial Support and Strategic Backing

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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