Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease
At the time of signing the informed consent, age ≥7, male or female
Clinical symptoms (at least one Fabry disease related symptom) and genetic diagnosis of Fabry disease,
Prior or no prior ERT treatment
Have renal or cardiac involvement (adults only)
Screening period laboratory test results: a) aspartate aminotransferase or alanine aminotransferase > 1.5× upper limit of normal (ULN);b) Total bilirubin > 1.5× upper limit of normal (ULN);c) Alkaline phosphatase > 2× upper limit of normal (ULN);d) Albumin < lower limit of normal (LLN)
There was a clinically significant increase in AFP during the screening period
Serum virology test: a) Hepatitis B: Hepatitis B virus surface antigen (HBsAg) positive, and hepatitis B virus-deoxyribonucleic acid (HBV-DNA) higher than the upper limit of normal detection;b) Hepatitis C: if the hepatitis C virus (HCV) antibody is positive, and the hepatitis C virus-ribonucleic acid (HCV-RNA) is higher than the upper limit of normal test value;c) Syphilis: positive for syphilis screening (Tp-Ab) and positive for syphile-specific antibodies;d) HIV: Known human immunodeficiency virus (HIV) positive history or HIV screening positive
AVT917 (>1:50), anti-AGA antibody positive(>1:2560)