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公开(公告)号：US20190083582A1
公开(公告)日：2019-03-21
申请号：US16111009
申请日：2018-08-23
Applicant: Genzyme Corporation
Inventor： James DODGE , Marco A. PASSINI , Lamya SHIHABUDDIN , Seng H. CHENG
Abstract: Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
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公开(公告)号：US20170014520A1
公开(公告)日：2017-01-19
申请号：US15280864
申请日：2016-09-29
Applicant: Genzyme Corporation
Inventor： Yunxiang ZHU , Seng H. CHENG , Canwen JIANG , Luis Z. AVILA
Abstract: The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate.
Abstract translation: 本发明提供了合成包含氨基氧基的寡糖的方法。 本发明还提供了包含氨基氧基的寡糖，将包含氨基氧基的寡糖与糖蛋白偶联的方法和寡糖 - 蛋白质缀合物。 还提供了通过施用寡糖 - 蛋白质缀合物来治疗哺乳动物中的溶酶体储存障碍的方法。
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公开(公告)号：US20220204963A1
公开(公告)日：2022-06-30
申请号：US17472367
申请日：2021-09-10
Applicant: Genzyme Corporation
Inventor： Yunxiang ZHU , Seng H. CHENG , Canwen JIANG , Luis Z. AVILA
Abstract: The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate.
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公开(公告)号：US20210228692A1
公开(公告)日：2021-07-29
申请号：US17137060
申请日：2020-12-29
Applicant: Genzyme Corporation
Inventor： James DODGE , Marco A. PASSINI , Lamya SHIHABUDDIN , Seng H. CHENG
Abstract: Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
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Patent Agency Ranking
公开(公告)号：US20210121523A1
公开(公告)日：2021-04-29
申请号：US17033443
申请日：2020-09-25
Applicant: Genzyme Corporation
Inventor： Marco A. PASSINI , Lamya S. SHIHABUDDIN , Catherine R. O'RIORDAN , Seng H. CHENG
Abstract: The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN. In one aspect, the viral particles are administered in the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.
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公开(公告)号：US20200010825A1
公开(公告)日：2020-01-09
申请号：US16268989
申请日：2019-02-06
Applicant: Genzyme Corporation
Inventor： Yunxiang ZHU , Seng H. CHENG , Canwen JIANG , Luis Z. AVILA
Abstract: The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate.
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公开(公告)号：US20190328682A1
公开(公告)日：2019-10-31
申请号：US16281961
申请日：2019-02-21
Applicant: Genzyme Corporation
Inventor： James C. DODGE , Marco A. PASSINI , Lamya S. SHIHABUDDIN , Seng H. CHENG
Abstract: Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.
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公开(公告)号：US20180065957A1
公开(公告)日：2018-03-08
申请号：US15490385
申请日：2017-04-18
Applicant: Genzyme Corporation
Inventor： Elyse BOURQUE , Mario A. CABRERA-SALAZAR , Cassandra CELATKA , Seng H. CHENG , Bradford Hirth , Andrew GOOD , Katherine JANCSICS , John MARSHALL , Markus Metz , Ronald K. SCHEULE , Renato SKERLJ , Yibin XIANG , Zhong ZHAO , John LEONARD , Thomas NATOLI , Elina MAKINO , Herve HUSSON , Oxana BESKROVNAYA
IPC: C07D453/02 , C07D471/08 , A61K45/06 , A61K31/551 , A61K31/55 , A61K31/496 , A61K31/497 , A61K31/506 , A61K31/5377 , A61K31/439
CPC classification number: C07D453/02 , A61K31/439 , A61K31/496 , A61K31/497 , A61K31/506 , A61K31/5377 , A61K31/55 , A61K31/551 , A61K45/06 , C07D471/08
Abstract: The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.
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公开(公告)号：US20180036295A1
公开(公告)日：2018-02-08
申请号：US15556444
申请日：2016-03-09
Applicant: Genzyme Corporation
Inventor： Seng H. CHENG , Lamya SHIHABUDDIN , Sergio Pablo SARDI
IPC: A61K31/439 , A61K9/00 , A61K45/06
CPC classification number: A61K31/439 , A61K9/0053 , A61K31/00 , A61K45/06 , A61K2300/00
Abstract: This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. The disclosure also relates to a method of reducing, reversing or preventing the accumulation of protein aggregates in tissue of a subject diagnosed as having a proteinopathy, or being at risk of developing a proteinopathy, the method comprising administering to the subject an effective amount of a quinuclidine compound. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods. The proteinopathy may be a synucleinopathy or a tauopathy, such as Parkinson's disease, Alzheimer's disease or dementia with Lewy bodies.
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