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METHODS AND COMPOSITIONS FOR DELIVERY TO THE CNS ARILSULFATASE A
专利权人:
ИНК. (US);ШИР ХЬЮМАН ДЖЕНЕТИК ТЕРАПИС
发明人:
САЛАМАТ-МИЛЛЕР Назила (US),ТЭЙЛОР Кэтрин (US),КАМПОЛИЕТО Пол (US),ШАХРОКХ Захра (US),ПАН Цзинь (US),ЧАРНАС Лоуренс (US),РАЙТ Тереса Леах (US),КАЛИАС Периклс (US)
申请号:
RU2012154574/15
公开号:
RU2012154574A
申请日:
2011.06.25
申请国别(地区):
RU
年份:
2014
代理人:
摘要:
1. A stable composition for intrathecal administration, containing aryl sulfatase A protein (ASA), salt and polysorbate surfactant. 2. A stable composition according to claim 1, characterized in that the ASA protein is present in a concentration range of approximately 0-100 mg / ml. A stable composition according to claim 1, characterized in that the ASA protein is present in a concentration selected from 10 mg / ml, 30 mg / ml, 50 mg / ml or 100 mg / ml. The stable composition according to claim 1, characterized in that the ASA protein contains the amino acid sequence of SEQ ID NO: 1.5. A stable composition according to any one of claims 1 to 4, characterized in that the ASA protein is obtained from a human cell line. A stable composition according to any one of claims 1 to 4, characterized in that the ASA protein is obtained from CHO cells. A stable composition according to any one of claims 1 to 4, characterized in that the salt is NaCl. A stable composition according to claim 7, characterized in that NaCl is present in a concentration range of approximately 0-300 mM. A stable composition according to claim 7, characterized in that NaCl is present in a concentration range of approximately 137-154 mmol. A stable composition according to claim 9, characterized in that NaCl is present at a concentration of approximately 154 mM. A stable composition according to any one of claims 1 to 4, characterized in that the polysorbate surfactant is selected from the group consisting of polysorbate 20, polysorbate 40, polysorbate 60, polysorbate 80, and combinations thereof. The stable composition according to claim 11, characterized in that the surfactant polysorbate is polysorbate 20.13. A stable composition according to claim 12, characterized in that polysorbate 20 is present in a concentration range of about 0-0.2%. Stable composition according to claim 13, characterized in that polysorbate 20 is present1. Стабильный состав для интратекального введения, содержащий белок ар
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