XellSmart Biomedical Achieves Dual U.S.-China Regulatory Approvals for iPSC-Derived Cell Therapy in Parkinson’s Disease, Demonstrating >12-Month Clinical Improvement in Long-Term Patients

Recently, XsellSmart Biopharmaceuticals (Suzhou/Shanghai) Co., Ltd., a leading pharmaceutical company in the field of iPSC-derived cells, has achieved multiple breakthroughs in succession. XsellSmart’s independently developedMultiple novel universal cell therapy drugs have officially obtained seven formal registration clinical trial approvals from China’s National Medical Products Administration (NMPA) and the U.S. Food and Drug Administration (U.S. FDA), all focusing on: universal iPSC-derived cell therapies for the treatment of central nervous system (CNS) diseases.

XsellSmart has been approved and completedTwo nationally filed stem cell clinical research projects (both "first in China"),Possesses a novel drug with global orphan drug designation certified by the U.S. FDA; multiple "first-in-class (FIC)" novel drug products, being the first of their kind in China or globally, have entered registration clinical trial stages in both China and the United States, and allRegistered clinical trials led and jointly conducted by the neurology and neurosurgery departments of top-tier hospitals, represented by the National Center for Neurological Disorders., including for the treatment of:

The world's second most common neurodegenerative disease, primary Parkinson's disease (Phase I clinical trials registered in China and the United States);

Early-onset Parkinson's disease (China Registered Clinical Phase I/II);

Major Neurological Disorder: Spinal Cord Injury (China-U.S. Phase I Clinical Trial Registration);

Amyotrophic Lateral Sclerosis (ALS) (China registrational Phase I/II and US registrational Phase I clinical trials): The first independently developed iPSC-derived cell therapy in China to receive US FDA approval and be granted global Orphan Drug designation.

In 2024, XsellSmart collaborated with the team led by Dr. Liu Zhongmin, President of Shanghai East Hospital, and has completed including“"First case in China"Including multiple cases of iPSC-derived cell therapy for Parkinson’s disease patients, this clinical study is a national-level stem cell registered clinical research project officially approved by the two national bureaus, and has passed the National Health Commission’s clinical GCP compliance inspection and GMP production site inspection: XsellSmart’s clinical-grade iPSC-derived dopaminergic neural precursor cells were transplanted into the putamen region of the striatum in patients with moderate to severe Parkinson’s disease.The longest follow-up period exceeded 12 months. Long-term clinical follow-up results showed no cell therapy-related adverse events, and key efficacy endpoints, including on-off time and MDS-UPDRS scores, as well as multiple non-motor indicators, demonstrated significant improvement in many subjects.

In addition to clinical follow-up results demonstrating the favorable long-term safety and efficacy of cell therapy, all enrolled patients actively reported significant improvements in multiple aspects of their overall quality of life:

Representing the patient: Mr. Hu (pseudonym; male, with a 10-year history of Parkinson's disease), after long-term cell transplantation therapy, the "on" period has been significantly prolonged. Clinical observations and patient feedback indicate that facial expressions have normalized, speech is fluent, there is no obvious stiffness in the limbs, no significant bradykinesia, and no tremors. Initiating movement and walking are relatively easy, gait is acceptable, balance is good, and fine motor skills are well-preserved; activities such as writing, using chopsticks, and operating a mobile phone are performed normally. Activities of daily living, including washing up, bathing, dressing, and tying shoelaces, are carried out effectively. During the "on" period, the patient is able to work daily, commute to work by e-scooter, and perform routine tasks such as cash handling, medication dispensing, intramuscular injections, and intravenous infusions. The patient can walk for 2–3 hours and run intermittently for 4–5 kilometers.

Representing the patient: Ms. Li (pseudonym; female, with an 8-year history of Parkinson's disease), after long-term cell transplantation therapy, the "on" period has been significantly prolonged. Clinical observations and patient feedback indicate that during the medication "on" phase, there are virtually no obvious symptoms of rigidity or tremor. The patient experiences no difficulty with activities of daily living (ADLs) or fine motor tasks, enabling a normal lifestyle, regular exercise, and essentially normal work capacity. The patient exhibits a positive mood and mental state, with normal facial expressions and relatively fluent speech. Limb rigidity is markedly alleviated, with essentially no tremors in the extremities. Limb mobility is normal, without significant bradykinesia or difficulty initiating movement. Gait has improved, balance is normal, and fine motor skills are well-preserved; activities such as writing, using chopsticks, and operating a mobile phone are performed normally. Self-care activities, including washing, bathing, dressing, and tying shoelaces, are carried out effectively. Each morning, the patient can jog approximately 500 meters, followed by walking 3–4 kilometers.

XsellSmart has long been dedicated to developing clinical-grade, allogeneic, off-the-shelf iPSC-derived cell therapies for central nervous system (CNS) diseases that currently lack effective clinical solutions. XsellSmart has achieved pioneering breakthroughs in the treatment of major or critical neurological disorders, including Parkinson’s disease, spinal cord injury, and amyotrophic lateral sclerosis (ALS).

The breakthrough R&D and clinical progress achieved by XsellSmart signifies that China is on par with or leading the world, and in some cases pioneering globally, in the development of novel clinical-grade iPSC-derived subtype neuronal cell therapies for major or critical central nervous system (CNS) diseases, such as Parkinson’s disease, spinal cord injury, and amyotrophic lateral sclerosis (ALS).

We look forward to the multiple registrational clinical trials conducted by XsellSmart in strong collaboration with top-tier hospitals and leading clinical expert teams, aiming to achieve more innovative breakthroughs and contributions in the development of novel clinical-grade iPSC-derived subtype neuronal cell therapies for central nervous system (CNS) diseases, represented by Parkinson’s disease, spinal cord injury, and amyotrophic lateral sclerosis (ALS).With a groundbreaking innovative stem cell regenerative therapy drug of unparalleled national significance, may we soon bring relief to the vast number of neurological disease patients suffering in China and around the world!

(Clinical Trial Patient Recruitment Guidelines)

1. Voluntarily participate in this study and sign the informed consent form. Commit to complying with the study procedures and cooperating to complete the entire study process;

2. Patients with idiopathic Parkinson's disease aged 50 to 75 years, of either sex, with a disease history of more than 5 years;

3. Requirement for responsiveness to levodopa therapy.

1. Patients with Atypical Parkinsonism

2. Presence of peak-dose dyskinesia or biphasic dyskinesia and/or unpredictable off periods;

3. Severe psychiatric symptoms or dementia;

4. History of malignancy; history of seizures or prophylactic use of antiepileptic drugs; sleep apnea, chronic obstructive pulmonary disease, infectious diseases, coagulation abnormalities, and hepatic dysfunction;

5. Contraindications to MRI and PET scans;

6. Previously received cell therapy or surgical treatment for Parkinson's disease.

You will receive transportation allowances and related subsidies. For inquiries, please call 4001122987 or 17317916592, and specify “Inquiry regarding registration for the XsellSmart clinical trial on cell therapy for primary Parkinson’s disease.”

(Scan the QR code to register)

1. Voluntarily participate in this study and sign the informed consent form. Commit to complying with the study procedures and cooperating to complete the entire study process;

2. Patients with early-onset Parkinson's disease, aged 18–70 years, with medical records confirming disease onset between 18 and 50 years of age, regardless of gender;

3. Disease duration of more than 5 years;

4. Requirement for responsiveness to levodopa therapy;

5. Motor fluctuations remain inadequately controlled despite treatment with stable doses of Parkinson’s disease medications.

1. Patients with non-primary Parkinson’s disease (secondary causes include parkinsonism due to vascular, toxic, drug-induced, metabolic, or infectious etiologies; hereditary degenerative parkinsonian syndromes [such as Huntington’s disease and Wilson’s disease]; or Parkinson-plus syndromes such as progressive supranuclear palsy, multiple system atrophy, and corticobasal degeneration)

2. In the advanced stage of Parkinson’s disease, experiencing severe, disabling peak-dose dyskinesia or biphasic dyskinesia, and/or unpredictable or markedly fluctuating symptoms;

3. Severe psychiatric symptoms or dementia;

4. History of malignancy; history of epilepsy or prophylactic use of antiepileptic drugs; chronic obstructive pulmonary disease (COPD); severe cardiovascular and cerebrovascular diseases; infectious diseases; coagulation abnormalities or hepatic dysfunction, etc.;

5. Contraindications to MRI and PET scans;

6. Previously received cell therapy or surgical treatment for Parkinson's disease.

You will receive transportation allowances and related subsidies. For inquiries, please contact: 17317916592 (Huashan Hospital Recruitment Team) or 18896516857 (The Second Affiliated Hospital of Soochow University Recruitment Team). Please state: “Inquiry about enrollment in XsellSmart’s clinical trial of cell therapy for early-onset Parkinson’s disease.”

(Scan QR Code to Register)

1. Age 18–65 years (inclusive), regardless of gender;

2. Spinal cord injury from cervical level C4 to lumbar level L2 caused by trauma or injury-related factors (surgery);

3. ASIA Impairment Scale grades A, B, and C; magnetic resonance imaging (MRI) indicates spinal cord injury;

4. Patients with spinal cord injury whose primary spinal cord injury occurred within 14 to 60 days prior to screening (medical record documentation required);

5. Patients of childbearing potential must agree to use effective non-pharmacological contraceptive measures during the trial and for 6 months after the end of the trial;

6. Voluntary participation in the clinical study, with understanding of and compliance with the study procedures; the participant or their legal guardian must be able to understand and sign the informed consent form;

You will receive transportation subsidies and related allowances. Hotline: 16607590610/15768990134/17317916592 (Recruitment Team of The Third Affiliated Hospital of Sun Yat-sen University), 18811288338 (Recruitment Team of The First Affiliated Hospital of Dalian Medical University). Please state: “Inquiry for Registration in the Clinical Trial of XsellSmart’s Regenerative Neural Cell Therapy for Spinal Cord Injury.”

(Scan the QR code to register)

1. Aged 18–75 years (inclusive), regardless of gender;

2. Diagnosed with definite, probable, or laboratory-supported probable amyotrophic lateral sclerosis (ALS) based on the revised El Escorial criteria and clinical manifestations, with a disease duration of no more than 2 years from the onset of initial symptoms to screening;

3. Forced vital capacity (FVC%) ≥70% of the predicted value during the screening/baseline period;

1. The patient presents with symptoms and signs of neuromuscular weakness, and muscle weakness due to other causes cannot be excluded;

2. Patient body mass index (BMI) < 18.5 kg/m²²；

3. Primary lateral sclerosis in patients presenting exclusively with upper motor neuron symptoms;

4. Diseases involving neurological or muscular dysfunction, such as metabolic myopathies and myasthenia gravis;

5. Diagnosed with autoimmune diseases with uncontrolled conditions, severe arthritis, claudication, or other diseases that the investigator assesses as affecting the evaluation;

6. History of tracheostomy, or use of mechanical ventilatory support;

7. Poorly controlled hypertension, with systolic blood pressure >160 mmHg and/or diastolic blood pressure >100 mmHg;

You will receive transportation allowances and related subsidies. For inquiries, please call 17317916592 and specify: “Inquiry about Registration for XsellSmart’s Clinical Trial on Regenerative Neural Cell Therapy for ALS.”

(Scan the QR code to register)