Shenzhen-based Shenzhen CellX Receives FDA Orphan Drug Designation for First-in-Class iPSC-Derived Cell Therapy for ALS

November 2023,XsellSmart Biopharmaceuticals (Suzhou) Co., Ltd.(hereinafter referred to as: XsellSmart)Global First-in-Class Clinical-Grade iPSC-Derived Subtype Neural Progenitor Cell Product for the Treatment of Amyotrophic Lateral Sclerosis (ALS) Granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA)! It is the first independently developed and domestically produced iPSC-derived cell therapy from China to receive FDA recognition and Orphan Drug Designation, as well as the world’s first iPSC-derived cell therapy with Orphan Drug Designation for the treatment of ALS to date.

Under the U.S. Orphan Drug Act, drugs granted orphan drug designation in the United States are eligible for a series of accelerated review and special support policies, including seven years of marketing exclusivity following new drug approval, waivers of NDA/BLA application fees, potential exemptions from certain clinical data submission requirements, and tax credits for clinical research expenses.

Amyotrophic lateral sclerosis (ALS) is a motor neuron disease and a progressive neurodegenerative disorder; patients with ALS are commonly referred to as "ALS patients." In individuals with ALS, motor neurons undergo continuous degeneration and death, resulting in combined impairment of both upper and lower motor neurons. This ultimately leads to paralysis, progressing from the inability to walk to the loss of speech, swallowing, and breathing functions. Clinical data indicate that the median survival period for ALS patients is approximately 39 months. Currently, there are no effective clinical drugs or therapeutic solutions capable of substantially alleviating or halting the progression of ALS.

Dr. Li Xiang, Founder of XsellSmartHe stated, “Amyotrophic lateral sclerosis (ALS), often referred to as an ‘incurable cancer,’ presents a clearly defined and urgent unmet clinical need. Developing safe, effective, and controllable clinical-grade iPSC-derived cell therapies for ALS has been the focus of my attention, reflection, research, and implementation efforts in the iPSC field over the past decade and more. It is also the endeavor that the XsellSmart team has been diligently and quietly pursuing since the company’s inception. We look forward to XsellSmart’s novel drug ultimately bringing new hope and a ray of light to ALS patients worldwide.”

Since its inception, XsellSmart has established a portfolio of proprietary, internationally leading new technologies and platforms, providing critical support for the continuous development of its self-developed innovative pipeline. The company’s globally first-in-class clinical-grade iPSC-derived subtype neural progenitor cell therapy for amyotrophic lateral sclerosis (ALS) has received Orphan Drug Designation from the U.S. FDA. This milestone represents a significant step in XsellSmart’s internationalization strategy and underscores the U.S. FDA’s recognition of China’s domestically developed, original iPSC-derived cell therapies.

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Dr. Li Xiang, founder of XsellSmart, has dedicated fifteen years to the scientific R&D and industrialization of innovative iPS cell-derived therapeutics. Since Dr. Li returned to China to establish XsellSmart on a full-time basis, the company has focused on developing clinical-grade iPS cell-derived therapies for neurological disorders, such as Parkinson’s disease, that currently lack effective clinical solutions.

XsellSmart has assembled a full-time team with international competitiveness and industry background. The core members of XsellSmart come from renowned domestic and overseas universities such as Peking University, Tsinghua University, Fudan University, Shanghai Jiao Tong University, Tongji University, the Chinese Academy of Sciences, the University of Wisconsin–Madison, and Johns Hopkins University, as well as from leading companies in the cell therapy industry. The team possesses the most advanced stem cell therapy technologies and translational experience at the forefront of both international and industrial sectors.

XsellSmart operates an R&D center, a Grade B+A GMP facility, and a quality control center spanning over 5,000 square meters. The company has completed core CMC development for its primary iPS cell-derived therapeutic pipelines, established end-to-end clinical-grade manufacturing processes and quality control systems, and successfully conducted formal trial production of clinical-grade iPS cell-derived subtype-specific neural precursor cell products. XsellSmart collaborates with multiple research institutions and hospitals, including Shanghai East Hospital and Peking University Third Hospital, on related technological and clinical research. Notably, its lead pipeline—a clinical-grade iPS cell-derived dopaminergic neural precursor cell therapy for Parkinson’s disease—is expected to enter registrational clinical trials in 2024.

Since Dr. Li Xiang founded XsellSmart on a full-time basis, the company has completed multiple rounds of financing totaling hundreds of millions of yuan, led by prominent venture capital firms including FreeS Fund, Qiming Venture Partners, Lilly Asia Ventures, and Sequoia China. From 2022 to 2023, amidst the overall downturn in the pharmaceutical sector and the lingering effects of the COVID-19 pandemic, Dr. Li Xiang led XsellSmart to secure over RMB 200 million in Series A financing and additional Series A+ funding. This round was co-led by Qiming Venture Partners, Lilly Asia Ventures, Jinyuan Zhanhong, and Sino-Singapore Capital, with continued follow-on investments from Sequoia China and FreeS Fund, and joint investments from Yuanhe Holdings, Peking University Technology Transfer Fund, Ali Health, and Huatai Zijin.

In 2023, XsellSmart stood out among hundreds of pharmaceutical companies and was officially selected for the Roche Accelerator (Roche China Accelerator), becoming one of the fifteen partner companies in China. With the support of Roche’s global team of professional advisors and complementary scientific resources, XsellSmart is accelerating the research, development, and industrialization of its novel iPSC-derived cell therapy pipeline for Parkinson’s disease.

XsellSmart has established a portfolio of proprietary, internationally leading new technologies and platforms, providing critical support for the continuous development of its self-developed innovative pipeline. In 2023, XsellSmart’s clinical-grade iPS cell-derived subtype neural progenitor cell therapy for amyotrophic lateral sclerosis (ALS) was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA), conferring benefits such as seven years of market exclusivity upon approval. This marks the first domestically developed and manufactured iPS cell-derived therapeutic from China to receive FDA recognition and Orphan Drug Designation.