Core Viewpoint - The article highlights the successful administration of the first patient with hyperchylomicronemia receiving the targeted base editing drug CS-121, developed by Zhengxu Biotech, marking a significant advancement in gene therapy for hyperlipidemia [2][10]. Group 1: Clinical Research and Patient Outcomes - The first patient treated in the clinical research (IIT) for CS-121 injection has been discharged after showing a significant decrease in fasting triglyceride (TG) levels within three days post-administration [2][10]. - The patient had a history of fasting TG levels exceeding 12.5 mmol/L, which led to multiple episodes of acute pancreatitis [2][10]. Group 2: Disease Background - Hyperchylomicronemia is a metabolic disorder characterized by elevated chylomicron levels in the blood, often leading to severe hypertriglyceridemia (sHTG) and potential complications such as acute pancreatitis [5][6]. - The condition includes familial chylomicronemia syndrome (FCS) and multifactorial chylomicronemia syndrome (MCS), with FCS being a rare autosomal recessive disorder and MCS influenced by various factors [6]. Group 3: Treatment Challenges and Innovations - Current treatment strategies for hyperchylomicronemia focus on maintaining fasting TG levels below 5.7 mmol/L to mitigate the risk of acute pancreatitis, but existing medications have limited efficacy [7]. - The CS-121 injection utilizes a novel base editing technology (tBE) to precisely edit the APOC3 gene, aiming for a long-lasting reduction in TG levels with a single administration [9]. Group 4: Mechanism and Safety - The tBE technology allows for targeted editing without causing double-strand breaks in DNA, thus minimizing safety risks associated with traditional CRISPR methods [9]. - Preclinical studies have validated the safety and long-term efficacy of CS-121, showing no off-target effects in various tissues [9].