The study will evaluate safety and tolerance of intravenous delivery of GC304 gene therapy drug as a treatment of primary hypertriglyceridemic patients with previous onset of acute pancreatitis.
Diagnosed as primary hypertriglyceridemia poorly managed by regular treatment and dietary control, with episode of acute pancreatitis twice or once of severe acute pancreatitis within 5 years;
Fasting plasma triglycerides (TG) levels above 5.65 mmol/L (intake of dietary fat <30 g within 24 hours before blood taken);
Homozygous or heterozygous mutations in GPIHBP1 or LPL genes by genetic screening;
The patients within reproductive age take effective contraceptive measures voluntarily entering screening stage until 6 months after the trial;
Patient who is known to be allergic to any ingredient of a trial drug (including immunosuppressants) or has any disease prohibited from the treatment;
Patient who is having active bacteria, fungi, viruses or other infections;
Patient who is intolerant of immunosuppressive drugs or steroids;
unrelieved abdominal pain caused by acute onset of pancreatitis or by other causes；
Sequential
GC304
GeneticSelf-complementary adeno-associated virus serotype 5 (AAV5) carrying a codon-optimized LPL coding sequence(coLPL) driven by a liver-specific promoter (LP)
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])
Frequency of treatment-related adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
The incidence of dose limited tolerance (DLT)
Changes of plasma triglyceride levels from baseline
The proportion of patients who stop using lipid-lowering medications;
Copy numbers of viral vector DNA [Shedding of viral vectors];
Titers of antibody against viral vector
Changes of LPL activity in post-heparin plasma from baseline;
Frequency of onset of acute pancreatitis after administration of GC304;
Changes of plasma triglyceride levels from baseline；
The proportion of patients who stop taking hypolipidemic drugs;
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