PEGylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria

The present disclosure provides a formulation of a drug comprising a PEGylated CBS protein having the amino acid sequence of SEQ ID NO: 1. Doses and dosing regimens for treating homocystinuria in a subject in need thereof are provided. In addition, doses and dosing regimens for reducing homocysteine...

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Bibliographic Details
Main Authors KRAUS JAN P, KOSVIK, OLE, MAJTAN TOMAS, SZELOS-MORA, MIREILLE, WANNER RUDOLF, GRAVEN FRANK, BUBLIL EREZ
Format Patent
LanguageChinese
English
Published 22.07.2022
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Summary:The present disclosure provides a formulation of a drug comprising a PEGylated CBS protein having the amino acid sequence of SEQ ID NO: 1. Doses and dosing regimens for treating homocystinuria in a subject in need thereof are provided. In addition, doses and dosing regimens for reducing homocysteine (Hcy) levels or increasing cysteine (Cys) and/or cystathionine (Cth) levels in a subject in need thereof are also provided. 本公开提供一种药品的制剂，所述药品包含具有SEQ ID NO:1的氨基酸序列的PEG化CBS蛋白质。提供用于治疗有需要的受试者的高胱氨酸尿症的剂量和给药方案。另外，还提供用于在有需要的受试者中降低高半胱氨酸(Hcy)水平或增加半胱氨酸(Cys)和/或胱硫醚(Cth)水平的剂量和给药方案。
Bibliography:Application Number: CN202080060487