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公开(公告)号：US11253485B2
公开(公告)日：2022-02-22
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：US10913956B2
公开(公告)日：2021-02-09
申请号：US15201268
申请日：2016-07-01
Applicant: Genzyme Corporation
Inventor： Marco A. Passini , Greg Stewart , James C. Dodge
Abstract: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
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公开(公告)号：US10369193B2
公开(公告)日：2019-08-06
申请号：US15160949
申请日：2016-05-20
Applicant: Genzyme Corporation
Inventor： Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng
Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.
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公开(公告)号：US11911440B2
公开(公告)日：2024-02-27
申请号：US18054500
申请日：2022-11-10
Applicant: Genzyme Corporation
Inventor： Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng
CPC classification number: A61K38/1709 , A61K31/7088 , A61K48/00 , A61K48/005 , A61K48/0008 , A61K48/0075 , C07K14/4702 , C12N7/00 , C12N15/86 , C12N2750/14121 , C12N2750/14133 , C12N2750/14143 , C12N2750/14171
Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.
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公开(公告)号：US10080783B2
公开(公告)日：2018-09-25
申请号：US14559851
申请日：2014-12-03
Applicant: Genzyme Corporation
Inventor： James Dodge , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng
CPC classification number: A61K38/465 , A61K9/0019 , A61K38/47 , C12Y301/04012 , C12Y301/06013 , C12Y302/0102 , C12Y302/01045 , C12Y302/01076 , C12Y304/14
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：US11975043B2
公开(公告)日：2024-05-07
申请号：US18046363
申请日：2022-10-13
Applicant: Genzyme Corporation
Inventor： Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng
CPC classification number: A61K38/1709 , A61K31/7088 , A61K48/00 , A61K48/0008 , A61K48/005 , A61K48/0075 , C07K14/4702 , C12N7/00 , C12N15/86 , C12N2750/14121 , C12N2750/14133 , C12N2750/14143 , C12N2750/14171
Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.
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公开(公告)号：US10821154B2
公开(公告)日：2020-11-03
申请号：US14888385
申请日：2013-05-01
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 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：US11957765B2
公开(公告)日：2024-04-16
申请号：US16808206
申请日：2020-03-03
Applicant: Genzyme Corporation
Inventor： Marco A. Passini , James Dodge
IPC: A61K48/00 , C07K14/47 , C12N15/86 , C12N15/864
CPC classification number: A61K48/0075 , A61K48/00 , C07K14/47 , C12N15/86 , C12N15/8645 , C12N2750/14041 , C12N2750/14143 , C12N2799/025
Abstract: The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
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公开(公告)号：US20240000886A1
公开(公告)日：2024-01-04
申请号：US18304519
申请日：2023-04-21
Applicant: Genzyme Corporation
Inventor： Marco A. Passini , Lamya S. Shihabuddin , Catherine R. O'Riordan , Seng H. Cheng
CPC classification number: A61K38/1709 , A61K48/0075 , A61K9/0019 , A61K9/0085 , A61K48/0058 , C12N2750/14171 , C12N2750/14141 , A61K48/00 , C12N2750/14143
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：US09415119B2
公开(公告)日：2016-08-16
申请号：US14876683
申请日：2015-10-06
Applicant: Genzyme Corporation
Inventor： Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng
IPC: A61K48/00 , C12N7/00 , A61K31/7088 , C12N15/86 , A61K38/17
CPC classification number: A61K38/1709 , A61K31/7088 , A61K48/00 , A61K48/0008 , A61K48/005 , A61K48/0075 , C07K14/4702 , C12N7/00 , C12N15/86 , C12N2750/14121 , C12N2750/14133 , C12N2750/14143 , C12N2750/14171
Abstract: Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.
Abstract translation: 公开了用于治疗影响运动功能的疾病的组合物和方法，例如受到疾病或脑和/或脊髓损伤的运动功能。
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