【First in Class】XellSmart iPSC-derived Drug granted ODD by FDA

2024-08-27

In November 2023, XellSmart Biomedical (Suzhou) Co., Ltd. (XellSmart Bio)'s clinical-grade iPSC-derived subtype-specific neural precursor cell product to treat ALS (amyotrophic lateral sclerosis) was granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA).

This is the worldwide first iPS-derived cell therapeutic medicine from China Granted by the FDA and granted orphan drug designation, and is also the worldwide first iPS-derived cell therapeutic medicine with global Orphan Drug Designation for the treatment of ALS to date.

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According to the US Orphan Drug Act, Drugs that are granted orphan drug designation in the United States can have a series of accelerated review and special support policies such as seven years of marketing exclusivity after approval, exemption of NDA/BLA filing fees, possible exemption of some clinical data reporting requirements, and tax deductions for clinical study expenses.

ALS is a type of motor neuronal and progressively neurodegenerative disease. Commonly known as ALS, motor neurons of ALS patients continue to deteriorate and die, resulting in combined damages to the upper and lower motor neural system, reflecting in the inability of ALS patients to walk, speak, swallow and breathing, and eventually leading to paralysis.

Clinical data show that the average survival period of an ALS patient is about 39 months, and there were almost no effective clinical drugs or clinical solutions to substantially alleviate ALS or save the lives of these patients.

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Dr. Li Xiang (Michael Lee), Founder and CEO of XellSmart:

There is clear unmet clinical need for ALS. The development of a safe, effective and affordable clinical-grade iPS-derived cell drug for ALS is one of my major focus besides PD for the past 14 years of my career in the iPSC field.

Starting from the birthday of XellSmart, our team has been tirelessly working on this project, driven by the conviction that our innovative FIC medicine will potentially bring new substantial solution to ALS patients around the world.

XellSmart has established a number of world-leading new technologies and platforms protected by global IP rights, which laid solid foundations for sustainable developments of innovative iPS-derived cell therapeutic pipelines from XellSmart. Being granted the orphan drug designation from FDA for XellSmart 's worldwide first clinical-grade iPS-derived neural precursor cell drug is a significant step of XellSmart to participate in global competition. It also marked the FDA’s recognition of a Chinese home-grown iPS-derived cell drug.

About XellSmart Bio

Dr. Xiang Li, founder of XellSmart, has focused on iPSC field for 15 years. Since Dr. Li returned to China and founded XellSmart, XellSmart has focused on development of clinical-grade iPS-derived cell drugs for the treatment of neurological diseases such as Parkinson’s disease and ALS, which have clear unmet clinical needs.

XellSmart has established a world-class full-time team with strong industrial backgrounds. The core members of the team hold degrees conferred by Peking University, Tsinghua University, Fudan University, Shanghai Jiao Tong University, Tongji University, Chinese Academy of Sciences, University of Wisconsin, Johns Hopkins University and other famous universities or companies in the cell therapy industry in China and abroad. The team possesses world and industry-leading stem-cell therapy technologies and translational expertise.

XellSmart has been operating ＞5000m²+ R&D center, B+A grade GMP base. XellSmart has completed core CMC for leading pipelines of iPS-derived cell drugs and is cooperating with multiple top hospitals for clinical developments and studies. Among them, the first pipeline of clinical-grade iPS-derived dopamine neural precursor cells for the treatment of PD is expected to enter Phase I clinical trial in China and US in 2024.

In 2023, XellSmart out stood from hundreds of pharmaceutical companies and was officially selected to enter the Roche Accelerator. With the supports from the Roche global advisory team and its scientific and clinical resources, XellSmart is accelerating the developments of its iPS-derived cell therapeutic pipeline for treating PD.

XellSmart has established a number of world-leading new technologies and platforms with protection of global IP rights, for sustainable developments of innovative iPSC-derived cell therapeutic pipelines:

In 2023, XellSmart's clinical-grade iPS-derived subtype neural precursor cells for the treatment of ALS was designated by the US Food and Drug Administration (FDA) as Orphan Drug.

It is the worldwide first iPS-derived cell therapeutic medicine from China approved by the FDA and granted orphan drug designation, and is also the worldwide first iPS-derived cell therapeutic medicine with global Orphan Drug Designation for the treatment of ALS to date.