Development of recombinant Lecithin Cholesterol Acyltransferase as a therapeutic

在过去的一年里，我们完成了重组LCAT第一阶段临床试验(RLCAT)的样本分析，该试验于2012年秋季完成。这项研究是在美国国立卫生研究院进行的，是对18名受试者进行的单次剂量递增研究。没有明显的不良反应，重组LCAT在提高高密度脂蛋白水平的能力方面表现出了预期的效果。我们还被批准用于治疗家族性LCAT缺乏症(FLD)和rLCAT患者的单一患者IND。RLCAT治疗将胆固醇酯水平恢复到接近正常水平和高于阈值，这是防止LPX形成所必需的，LPX是一种积聚在FLD中并导致肾脏疾病的异常脂蛋白颗粒。RLCAT治疗还纠正了这种疾病中的其他脂蛋白异常，如低密度脂蛋白，这可能导致心血管疾病。

In the past year, we completed the analysis of samples from a Phase I clinical trial of recombinant LCAT (rLCAT), which we completed in the fall of 2012. The study was done at the NIH and was a single dose escalation study in 18 subjects. There was no significant adverse events and the the recombinant LCAT behaved like expected based on animal studies in regard to its ability to increase HDL levels. We were also granted approval for a single patient IND for the treatment of a patient with Familial LCAT Deficiency (FLD) with rLCAT. The rLCAT treatment restored cholesteryl ester levels to near normal levels and above threshold necessary to prevent LpX formation, the abnormal lipoprotein particle that accumulates in FLD and causes renal disease. The rLCAT treatment also corrected the other lipoprotein abnormalities in this disorder, such as low HDL, which may contribute to cardiovascular disease.