SNUG01 Clinical Study Officially Launched in the Third Hospital of Beijing Medical University

On September 22, 2023, a clinical research (IIT) project of SNUG01, a gene therapy drug for amyotrophic lateral sclerosis (ALS, also known as ALS), was successfully launched in the Third Hospital of Peking University (the Third Hospital of Beijing Medical University). Professor Fan Dongsheng's team from the Third Hospital of Beijing Medical University, Jia Yichang's laboratory team from Tsinghua University, founder of Shenji Changhua and clinical team attended the kick-off meeting.

kick-off meeting site

project is "a single-arm, open exploratory clinical study to evaluate SNUG01 single intrathecal injection in patients with amyotrophic lateral sclerosis". It is an IIT study sponsored by Professor Fan Dongsheng, a famous expert on ALS, as the main researcher and funded by Shenji Changhua. SNUG01 is a gene therapy drug with adeno-associated virus (AAV) as the carrier and the exclusive therapeutic target SG001 of Shenji Changhua, in order to achieve the therapeutic effect of "one-time administration, long-term effective.This project will rely onone of the largest treatment and research centers of progressive freezing disease in ChinaDepartment of Neurology, the Third Hospital of North Medical College, make full use of itson clinical resources.

Professor Fan Dongsheng

Professor Fan Dongsheng said at the kick-off meeting that there are few basic research clinical translational medicine projects for ALS in China. SNUG01 clinical research has solid preclinical data, and the first sympathetic drug administration has preliminarily verified its safety and efficacy. Its therapeutic target SG001 has significant neuron protection and has great clinical therapeutic potential. He is full of expectations for the SNUG01 clinical research launched in the Third Hospital of Beijing Medical University. He believes that the launch of SNUG01 clinical research will be a milestone event. The Third Hospital of Beijing Medical University will join hands with Shenji Changhua to actively promote this clinical research.

Professor Jia Yichang

Shenji Changhua Science and professor of medicine at Tsinghua University, said that SNUG01 uses a new therapeutic target for ALS, which has been rigorously scientifically verified. The successful start of this IIT clinical study is inseparable from the hard work of the front-line researchers in Jia Yichang Laboratory of Tsinghua University, the cooperation of Shenji Changhua clinical and non-clinical teams, and the strong support of Professor Fan Dongsheng's team from the Third Hospital of Beijing Medical University.

scene photo

This launch marks the first gene therapy drug of Shenji Changhua into a new stage of clinical research, the next step Shenji Changhua will continue to promote clinical research work with a scientific and pragmatic attitude, to help the research center clinical research high-quality development. The clinical application of SNUG01 is expected to open a new field of ALS treatment, bring new hope for patients and their families, and is of great significance to explore new treatment methods for ALS.

patient recruitment

dear patient friend:

is conducting "a single-arm, open exploratory clinical study to evaluate the single intrathecal injection of SNUG01 in patients with amyotrophic lateral sclerosis", which has been approved by the medical ethics committee of the hospital.
Based on the principle of voluntary participation, amyotrophic lateral sclerosis patients are recruited to participate in this study. If you wish and meet the following conditions, you can contact our department.
basic selection conditions:
1.18~65 years old (including 18 years old and 65 years old), men and women are not limited;
2. Meet the diagnostic criteria for amyotrophic lateral sclerosis (ALS) (clinically possible and auxiliary examination supports ALS or probable ALS or clinically confirmed ALS);
3. The course of ALS must be less than 24 months (counting from the first occurrence of any symptom of ALS to the date of signing the consent form);
4. Body mass index (BMI)≥ 19 kg/m2subjects;
5. Pre-screening ALSFRS-R Pre-study Slope & Delta;FS, measured as & Delta;FS =(48-ALSFRS-R at screening visit) \Duration (months) from onset to screening visit ≥ 0.5 min/month, ALSFRS-R total score ≥ 26 min;
6. Not currently receiving riluzole or have received a stable dose of riluzole for at least 4 weeks prior to Screening Visit. Subjects receiving riluzole are expected to maintain the same dose throughout the study;
7. Not currently receiving Edaravone or receiving approved standard regimen of Edaravone. Subjects who are being treated with edaravone must have completed at least one cycle of treatment prior to the screening visit and expect to continue treatment with edaravone throughout the study;
8. No previous tracheotomy and use of noninvasive ventilation for less than 16 hours per day prior to screening;
9. Negative for human immunodeficiency virus (HIV), syphilis, hepatitis B (negative for hepatitis B surface antigen HBsAg and anti-hepatitis B core antibody anti-HBcAb) or hepatitis C;
10. Has not had any previous gene therapy, hematopoietic stem cell transplantation or solid organ transplantation;
11. Able to understand and voluntarily sign the informed consent form.
If you meet the above main conditions and are willing to participate in this study, please contact us at 15710013912 (9-18 on weekdays). You will be further examined by the study doctor to assess compliance with all inclusion and exclusion criteria of the study protocol.