A phase I clinical trial titled "Evaluation of the Safety and Tolerability of GC304 Adeno-Associated Virus Injection in Patients with Primary Hypertriglyceridemia and a History of Acute Pancreatitis" has commenced recruitment. The clinical study has been approved by the National Medical Products Administration and the ethics committee of the research hospital. The study plans to recruit 7 participants nationwide and is now publicly recruiting.

This trial is suitable for patients with primary hypertriglyceridemia who have a history of acute pancreatitis. The trial has obtained tacit approval from the CDE, with the clinical trial approval number CXSL2200432, and has been approved by the ethics committee of Peking Union Medical College Hospital.

Research Institution: Peking Union Medical College Hospital
Principal Investigator: Zhang Shuyang
Executing Investigator: Tian Zhuang

1. At the time of signing the informed consent form, age between **18 and 60 years old**, gender is not limited; 

2. Clinically diagnosed with primary hypertriglyceridemia, must meet the criteria of acute pancreatitis attacks ≥2 times or ≥1 time severe acute pancreatitis within 5 years, and poorly controlled with conventional treatment or low-fat diet; 

3. Fasting (at least 10 hours) plasma TG levels (using the average value of the V1 and V2 visit test results during the screening period) ≥5.65mmol/L; 

4. Genetic testing shows a homozygous or heterozygous mutation in the GPIHBP1 gene, and/or homozygous or heterozygous mutation in the LPL gene;

 5. Participants have been taking a stable dose of fixed lipid-lowering drugs for ≥1 month before dosing; 

6. Participants fully understand and can comply with the requirements of the trial protocol and are willing to complete the trial as planned, including voluntarily adhering to the trial procedures, accepting low-fat diet requirements, and cooperating in providing biological samples; 

7. Able to understand the procedures and methods of this clinical trial, having given fully informed consent, the patient voluntarily participates and signs the informed consent form by themselves or their guardian.

Institution Name:Peking Union Medical College Hospital
Institution Address: No. 1 Shuai Fu Yuan, Dongcheng District, Beijing, Peking Union Medical College Hospital
Authorized Physician: Li Jingyi
Contact Phone: 13811849157

Recruitment Poster

About GC304 Adeno-Associated Virus Injection

GC304 adeno-associated virus injection is a recombinant AAV viral vector carrying the LPL gene expression cassette. LPL is a key enzyme in the human body for hydrolyzing plasma-rich lipoprotein triglycerides. GC304 injection, through the efficient expression of the artificially optimized LPLS447X gene, a natural beneficial mutant of LPL, in the human body, can degrade triglycerides in circulating blood over the long term and efficiently, providing a new type of gene drug for the prevention and/or treatment of severe hypertriglyceridemia.

About Genecradle Therapeutics

Beijing Genecradle Therapeutics Technology Co., Ltd. is a national high-tech enterprise with core business in the development of gene therapy drugs mediated by AAV vector delivery technology. The company is committed to promoting Chinese rare disease gene drugs from the foundation to clinical and market applications, benefiting patients and families. The company focuses on the development of gene therapy drugs for genetic neuromuscular diseases, inherited metabolic diseases, lysosomal diseases, and ophthalmic diseases, and through advancing the research and clinical application of rare disease gene drugs, it deepens the understanding of life and health, transitioning gene therapy technology and products from rare diseases to the treatment and rehabilitation of chronic and other major diseases.