XellSmart Receives Fourth Consecutive US FDA & China NMPA IND Clearance for MSA-P iPSC-derived Cell Therapy

SUZHOU, China, March 2, 2026 /PRNewswire/ -- In February 2026, XellSmart Pharmaceutical Co., Ltd. (XellSmart) announced that it had secured its fourth US FDA and China NMPA clearance for Phase I/II registrational clinical trial entry with its independently developed, off-the-shelf, allogeneic, universal, iPSC-derived, subtype-specific neuron progenitor cell therapy targeting Multiple System Atrophy- Parkinsonian Type (MSA-P). This joins the previous three consecutive FDA-NMPA registrational trial clearances for its universal iPSC-derived cell therapies targeting Parkinson's Disease (PD), Spinal Cord Injury (SCI), and Amyotrophic Lateral Sclerosis (ALS), respectively. Building on its promising clinical data, the rapid pipeline progress fully attests to XellSmart's expertise in iPSC-for-CNS space, the potential clinical value and viability for its products, and its unwavering commitment in addressing the unmet clinical needs in the highly underserved neurodegenerative disease field. 

MSA, recognized by the World Health Organization (WHO) as a rare and fatal neurodegenerative disease with huge unmet needs, meets with this commitment. The Parkinsonian type of multiple system atrophy (MSA-P) mainly presents with progressive Parkinsonian syndrome, such as bradykinesia, rigidity, and gait disorders, accompanied by severe autonomic nerve dysfunction, including orthostatic hypotension and urinary incontinence. This disease is triggered by abnormal aggregation of α-synuclein in oligodendrocytes, causing extensive loss of neuronal function and rapid progress. The median survival for patients after diagnosis is merely 6 to10 years. 

Although MSA-P has a wider pathological scope than Parkinson's disease, the two diseases overlap to a certain extent in their pathogenesis, especially in the manifestation of Parkinsonian motor symptoms. The current standard clinical treatment for MSA-P includes dopaminergic drugs for Parkinson's-like motor symptoms, such as levodopa/carbidopa, combined with other dopaminergic therapies when necessary. However, the overall response is often not as good as that for Parkinson's disease and the efficacy is limited. Non-pharmacological treatments for MSA-P includes management of autonomic dysfunction and rehabilitation training, fall prevention and nutritional support to maintain patients' physical function and quality of life as much as possible. 

Currently, there are no approved therapies anywhere in the world that can effectively slow or reverse the disease progression. Yet, XellSmart's innovative approach attempts to address this challenging neurodegenerative disease. Through employing a potential mechanism of regenerating dopamine secretion and promoting circuit and brain environment repair, it explores this novel treatment approach for MSA-P, and the potential to deliver a new therapeutic option and hope to patients worldwide.

Standing at the Forefront of iPSC-derived Cell Therapies for CNS Diseases 

This pipeline extension into MSA-P naturally taps into XellSmart's expertise in CNS diseases, evidenced by its multiple cleared "China or global first" INDs. Overall, nine INDs from XellSmart have been cleared by US FDA and China NMPA for Phase I/II registrational clinical trials. These commenced trials employ clinical-grade, allogeneic, universal, off-the-shelf, iPSC-derived cell therapeutic products — the laser-sharp development focus of the company, all targeting major or critical CNS diseases, i.e., Parkinson's Diseases, Spinal Cord Injury, ALS and MSA-P. The key trials are:

• China Phase I/II & the US Phase I registrational clinical trials, targeting primary Parkinson's disease, the world's second most prevalent neurodegenerative disorder. The trial is led by a prestigious National Center for Neurological Disorders — Beijing Tiantan Hospital.
• China's first randomized, double-blind, controlled clinical trial of universal cell therapy, targeting early-onset Parkinson's disease. The trial is led by Huashan Hospital Fudan University, a leading National Center for Neurological Disorders.
• China and the US Phase I/II registrational clinical trials targeting MSA-P, projected to be kicked off in 2026.
• China and the US Phase I registrational clinical trials targeting Spinal Cord Injury, employing XellSmart's potentially First-in-Class product, led by the Third Affiliated Hospital of Sun Yat-sen University.
• China Phase I/II & the US Phase I registrational clinical trials, targeting Amyotrophic Lateral Sclerosis (ALS). The trial employs the first China-developed iPSC-derived cell therapy that had received Orphan Drug Designation from the US FDA in 2023. The trial is led by Peking University Third Hospital.

Key Clinical Results Emerged from PD Registrational Trials

This pipeline extension into MSA-P also builds on XellSmart's promising clinical data in PD trials. The trials carried out at the National Centers for Neurological Disorders have enrolled more than a dozen patients with moderate to severe PD. After receiving cell transplantation treatment, these patients showed:

• No unexpected safety signals observed to date.
• A positive trend for efficacy indicators such as 'ON/OFF state' and MDS-UPDRS motor scores. The average "ON state" has been extended by several hours, coupled with a significant relief in "OFF state", in contrast to pre-treatment difficulty in movement and significant limitations after medication failure. The time for daily free movement and maintaining a relatively high quality of life has significantly increased.
• A trend of long-term survival and maturation of the engrafted neurons, as brain PET examination suggests potential survival and function from newly generated dopaminergic cells in the putamen region.

The clinical results demonstrated XellSmart's clinical implementation capability and the significant clinical potential of using iPSC-derived cell therapies via stereotactic neurosurgery for safe and effective PD treatment.

A Strong Market Vote of Confidence Following Regulatory Votes of Confidence

Since its inception in 2021, XellSmart has embraced rapid clinical development and GMP-grade facility upgrade supported by five consecutive years of market-driven funding, collectively led by renowned venture capital firms including FreeS Fund, Qiming Venture Partners, LAV, Sequoia Capital China (Hongshan), 3SBIO and more.

The proceeds from this financing have been boosting its key pipeline products into pivotal validation clinical stages. Additionally, the funds will ensure future commercial roll-out of XellSmart's products through strengthening XellSmart's fully-fledged, large-scale, standardized production capacity, quality management and control systems for its off-the-shelf iPSC-derived cell products. A firm market vote of confidence, underpinned by successive regulatory votes of confidence by the NMPA and FDA.

About XellSmart

XellSmart is a clinical-stage, regenerative medicine company focusing on treating CNS diseases with clinical-grade, allogeneic, universal, off-the-shelf, iPSC-derived cell therapies. Founded by Dr. Xiang Li (Michael Lee) in 2021 in Suzhou and Shanghai, China, the company has built up a strong portfolio of global patents and secured US FDA and China NMPA clearance for nine INDs of allogeneic iPSC-derived cell therapies targeting major or critical CNS diseases: Parkinson's Diseases, Spinal Cord Injury, ALS and MSA-P, moving wholly-owned cell therapeutic products through clinical developments in China and the US, based on its own 5,000㎡ R&D center, B+A-grade GMP manufacturing facility, and quality control center in Shanghai and Suzhou, China. XellSmart, standing at the forefront of iPSC-derived cell therapies for underserved CNS diseases, is committed to transforming this disease treatment landscape and helping millions of suffering patients with its cutting-edge innovation.   

CONTACT:
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SOURCE XellSmart Biopharmaceutical (Suzhou/Shanghai) Co., Ltd.