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发明公开
CN110753556A 治疗同型半胱氨酸尿症的酶替代疗法优化 无效 - 驳回

治疗同型半胱氨酸尿症的酶替代疗法优化
摘要：
本发明提供了一种PEG化人截短的胱硫醚合酶蛋白的方法，该蛋白在氨基酸位置15处具有半胱氨酸至丝氨酸的突变(htCBS C15S)。用5kDa、10kDa或20kDa NHS酯PEG分子之一将htCBS C15SPEG化。在该方法中使用了对PEG化过程的过程监控，以降低未PEG化的htCBS C15S和PEG化不足的htCBS C15S的水平。PEG化htCBS C15S的给药在同型半胱氨酸尿症的整个治疗过程中均具有功效。
摘要（英）：

The present invention provides a method of PEGylating a human truncated cystathionine -synthase protein containing a mutation of a cysteine to a serine at amino acid position 15 (htCBS C15S). The htCBS C15S was PEGylated with one of 5 kDa, 10 kDa, or 20 kDa NHS ester PEG molecules. In-process monitoring of the PEGylation process was used in the method to reduce levels of unPEGylated htCBS C15S andhtCBS C15S with insufficient PEGylation. Administration of the PEGylated htCBS C15S had efficacy throughout the course of treatment for homocystinuria.

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