世界上第一个——XellSmart的同种异体iPSC衍生的脊髓损伤再生细胞疗法正式获得美国FDA批准进行注册阶段：临床试验-动脉网

World's First -- XellSmart's Allogeneic iPSC-derived Regenerative Cell Therapy for Spinal Cord Injury Officially Approved by the U.S. FDA for a Registrational Phase：Clinical Trial

CISION 等信源发布 2025-05-26 09:00

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可切换为仅中文

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SUZHOU,

苏州，

China

中国

，

May 25, 2025

2025年5月25日

/PRNewswire/ --

In May 2025, XellSmart Biopharmaceutical (Suzhou/

2025年5月，XellSmart生物制药（苏州/

Shanghai

上海

) 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

）有限公司正式宣布，XellSmart开发的现成同种异体iPSC衍生的亚型特异性神经再生细胞疗法已获得双方的正式批准。

China's

中国的

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)

国家药品监督管理局（NMPA）和美国食品药品监督管理局（FDA）批准进行一期临床试验，以治疗脊髓损伤（SCI）。

—

a severe and currently untreatable disease of the central nervous system, affecting more than 15 million patients globally.

一种严重的、目前无法治疗的中枢神经系统疾病，影响着全球超过1500万名患者。

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

作为全球首个使用特定亚型神经祖细胞治疗产品治疗脊髓损伤的再生临床试验，该研究将由第三附属医院牵头进行。

Sun Yat

孙逸仙

-sen University, a nationally recognized leader in spinal cord injury treatment.

中山大学，全国公认的脊髓损伤治疗领域的领导者。

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.

脊髓损伤（SCI）是一种全球范围内普遍存在的神经系统疾病。它代表着一个关键的未满足临床需求，具有高致残率、不可逆性、早发性，并给患者、家庭和社会带来沉重负担。SCI 主要影响青壮年，通常由交通事故、运动损伤或其他创伤引起。

。

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.

作为一种严重影响患者社会功能的疾病，需要终身照料和康复，造成严重的经济负担和沉重的社会成本。

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

除了身体残疾之外，脊髓损伤患者通常在就业、社会融入和心理健康方面面临严峻挑战。在高收入国家，每位患者的终身医疗和护理费用可能

exceed

超过

USD 1 million

100万美元

, placing a heavy strain on both families and public health systems.

，给家庭和公共卫生系统都带来了沉重的压力。

Worldwide,

全球范围内，

over 15 million

超过1500万

people are estimated to suffer from spinal cord injury (SCI). This figure includes over 3 million patients in

据估计，有人患有脊髓损伤（SCI）。这个数字包括超过300万患者在内。

China

中国

and 300,000 patients in the US. Each year,

美国每年有 300,000 名患者，

China

中国

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.

美国报告的急性或亚急性脊髓损伤新发病例大约为100,000例和18,000例——分别相当于每小时近10例和2例新发病例。

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.

脊髓损伤往往会导致部分或完全瘫痪，伴随运动和感觉功能的丧失。大多数患者会经历永久性残疾，严重损害其生活质量。由于中枢神经系统的再生能力有限，脊髓损伤后的神经修复仍然极具挑战性。

。

Current treatments are largely limited to rehabilitation and supportive care, with no effective therapies available to promote neural regeneration during the injury phase.

当前的治疗主要局限于康复和支持性护理，在损伤阶段尚无有效的治疗方法来促进神经再生。

Following over four years of rigorous development and preclinical studies, XellSmart has partnered with leading clinical experts in spinal cord injury to initiate the

经过四年多的严格开发和临床前研究，XellSmart已与脊髓损伤领域的领先临床专家合作，启动了

world's first registrational clinical trial of an off-the-shelf, allogeneic, iPSC-derived, subtype-specific, regenerative neural cell therapy for spinal cord injury.

世界上首个现成的、同种异体的、iPSC衍生的、亚型特异性的脊髓损伤再生神经细胞治疗的注册临床试验。

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

这项开创性的试验代表了全球开发脊髓损伤再生疗法的一个里程碑成就。XellSmart 致力于推进突破性治疗，旨在重新定义脊髓损伤康复的可能性——为患者带来新的希望。

China

中国

and around the world, and delivering tangible relief to families and communities affected by this devastating condition.

并在全球范围内，为受这种破坏性状况影响的家庭和社区提供切实的救济。

For funding and BD cooperation, contact:

对于资金和商务合作，请联系：

BD@xellsmart.com

XellSmart has been dedicated to the development of clinical-grade, allogeneic, off-the-shelf iPSC-derived cell therapies for central nervous system (CNS) diseases that currently lack effective treatment options. Up until now, XellSmart has secured formal approvals from both

赛尔智能一直致力于开发临床级别的、同种异体的、现成的iPSC衍生细胞疗法，用于目前缺乏有效治疗选择的中枢神经系统（CNS）疾病。截至目前，赛尔智能已获得双方的正式批准。

China's

中国的

NMPA and the U.S. FDA for seven registered and registrational clinical trials spanning

中国国家药品监督管理局（NMPA）和美国食品药品监督管理局（FDA），涵盖七项已注册和注册中的临床试验

China

中国

and

和

the United States

美国

, for iPSC-derived cell therapies to treat CNS diseases

，用于治疗中枢神经系统疾病的iPSC衍生细胞疗法

—

including Parkinson's disease (Approved Phase I in both

包括帕金森病（均已批准进入第一阶段）

China

中国

and US), amyotrophic lateral sclerosis (ALS; Approved Phase I in both

和美国），肌萎缩侧索硬化症（ALS；在两地均获准进入第一阶段

China

中国

and US; Global orphan drug designation by FDA), and spinal cord injury (SCI; Approved Phase I in both

和美国；FDA授予的全球孤儿药资格），和脊髓损伤（SCI；均已获批进入第一阶段）

China

中国

and US), hence pioneering breakthrough advancements in tackling major and severe CNS diseases:

以及美国），因此在应对重大和严重的中枢神经系统疾病方面开创了突破性的进展：

In 2023,

2023年，

China's

中国的

first

第一

iPSC-derived cell therapy (XS228 injection), developed by XellSmart, received FDA approval and was granted global orphan drug designation (ODD).

XellSmart开发的iPSC衍生细胞疗法（XS228注射剂）获得FDA批准，并被授予全球孤儿药资格（ODD）。

In 2024,

2024年，

China's

中国的

first

第一

national-level registered clinical study of iPSC-derived cell therapy for neurological diseases was approved to treat Parkinson's disease patients—including

国家级注册临床研究批准使用诱导多能干细胞衍生的细胞疗法治疗神经系统疾病，包括帕金森病患者——

China's

中国的

first

第一

case. More than 12 months of follow-up demonstrated good safety and significant improvements in 'off' time, MDS-UPDRS scores, and multiple non-motor symptoms.

病例。超过12个月的随访显示，安全性良好，并且“关”期时间、MDS-UPDRS评分和多种非运动症状均有显著改善。

In 2024, the

2024年，

world's first

世界第一

national-level registered clinical study of iPSC-derived cells (XS228 injection) for amyotrophic lateral sclerosis (ALS) was approved. Multiple ALS patients including the world's first case were treated safely, with preliminary data showing effective slowing of disease progression.

国家层面注册的iPSC衍生细胞（XS228注射）治疗肌萎缩侧索硬化症（ALS）的临床研究获批开展。包括世界首例在内的多名ALS患者已安全接受治疗，初步数据显示疾病进展得到了有效延缓。

In 2025,

2025年，

China's

中国的

first

第一

allogeneic off-the-shelf iPSC-derived dopaminergic neural progenitor cell (XS411 injection) registrational Phase I clinical trial was launched, led by the National Neurological Disease Medical Center at Beijing Tiantan Hospital, targeting Parkinson's disease — the world's second most common neurodegenerative disorder..

同种异体现成的iPSC衍生多巴胺能神经祖细胞（XS411注射）注册性I期临床试验已启动，由北京天坛医院国家神经系统疾病医学中心主导，针对帕金森病——全球第二常见的神经退行性疾病。

In 2025,

2025年，

China's

中国的

first

第一

randomized, double-blind controlled registrational Phase I/II clinical trial was launched, led by Huashan Hospital affiliated with Fudan University (also a National Neurological Disease Medical Center), targeting early-onset Parkinson's disease (EOPD).

由复旦大学附属华山医院（同时也是国家神经疾病医学中心）牵头，针对早发型帕金森病（EOPD）的随机、双盲对照注册性I/II期临床试验已启动。

In 2025, the

2025年，

world's first

世界第一

registrational Phase I/II clinical trial of an allogeneic off-the-shelf iPSC-derived subtype-specific neural progenitor cell therapy (XS228 injection) was launched by Peking University Third Hospital, targeting amyotrophic lateral sclerosis (ALS), one of the world's four major incurable diseases.

北京大学第三医院开展了针对世界四大顽症之一的肌萎缩侧索硬化（ALS）的异体通用型iPSC来源的亚型特异性神经前体细胞疗法（XS228注射液）的注册性I/II期临床试验。

In 2025, the

2025年，

world's first

世界第一

registrational Phase I clinical trial of an allogeneic off-the-shelf iPSC-derived subtype-specific neural progenitor cell therapy was initiated by the Third Affiliated Hospital of

第三附属医院启动了同种异体现成的iPSC衍生的亚型特异性神经前体细胞疗法的注册性I期临床试验。

Sun Yat

孙逸仙

-sen University, targeting spinal cord injury (SCI), a major neurological disease.

中山大学，针对脊髓损伤（SCI）这一主要的神经系统疾病。

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.

XellSmart组建了一支完全专注、具有国际竞争力的“All-In”团队，致力于开发针对目前缺乏有效治疗选择的中枢神经系统（CNS）疾病的临床级同种异体即用型iPSC衍生细胞疗法。XellSmart构建了拥有自主知识产权、全球领先的工业技术与平台组合，为其核心业务持续推动内部创新提供强大动力。

XellSmart has independently built and operates a R&D center, a B+A-grade GMP manufacturing facility and a quality control center with a total area exceeding .

XellSmart独立建造并运营着一个研发中心、一个B+A级GMP制造工厂和一个总面积超过的质量控制中心。

5000m

5000米

。

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 national, registered or registrational clinical studies and global clinical trials..

多家由XellSmart开发的临床级iPSC衍生细胞治疗候选药物已完成核心CMC开发，并建立了完全集成的临床级制造工艺和质量控制系统。XellSmart已成功生产出多个GMP级iPSC衍生的特定亚型神经祖细胞治疗的临床批次，目前这些产品已被应用于多个国家注册或注册性质的临床研究及全球临床试验中。

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.

XellSmart已获得多轮融资，由知名市场化风险投资机构启明创投、礼来亚洲基金、红杉中国（洪杉）等共同领投。

For funding and BD cooperation, contact:

有关资金和商务合作，请联系：

BD@xellsmart.com

SOURCE XellSmart

来源 XellSmart

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