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公开(公告)号：US20170087212A1
公开(公告)日：2017-03-30
申请号：US15160949
申请日：2016-05-20
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：US20240350584A1
公开(公告)日：2024-10-24
申请号：US18616511
申请日：2024-03-26
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：US20240033323A1
公开(公告)日：2024-02-01
申请号：US18488346
申请日：2023-10-17
Applicant: Genzyme Corporation
Inventor： Marco A. PASSINI , Lamya Shihabuddin , Seng H. Cheng
CPC classification number: A61K38/1709 , A61K31/7088 , A61K48/005 , C12N15/86 , A61K48/00 , A61K48/0075 , C12N7/00 , A61K48/0008 , C07K14/4702 , C12N2750/14171 , C12N2750/14143 , C12N2750/14133 , C12N2750/14121
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：US20230084580A1
公开(公告)日：2023-03-16
申请号：US18046363
申请日：2022-10-13
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：US20210008227A1
公开(公告)日：2021-01-14
申请号：US16910834
申请日：2020-06-24
Applicant: Genzyme Corporation
Inventor： James DODGE , Lamya S. SHIHABUDDIN , Marco A. PASSINI , Seng H. CHENG , Catherine O'RIORDAN
IPC: A61K48/00 , C12N15/864 , C07K14/475
Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.
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Patent Agency Ranking
公开(公告)号：US20160074474A1
公开(公告)日：2016-03-17
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.
Abstract translation: 本发明提供使用包含表达SMN的转基因的自身互补重组腺相关病毒（rAAV）病毒颗粒治疗脊髓性肌肉萎缩的方法。 在一个方面，病毒颗粒在人类受试者中施用脊柱或大叶黄水母; 例如，儿科人类受试者。 考虑包含AAV9衣壳的病毒颗粒。
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公开(公告)号：US20230135379A1
公开(公告)日：2023-05-04
申请号：US18054500
申请日：2022-11-10
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：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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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：US20200306387A1
公开(公告)日：2020-10-01
申请号：US16808206
申请日：2020-03-03
Applicant: Genzyme Corporation
Inventor： Marco A. PASSINI , James DODGE
IPC: A61K48/00 , C12N15/86 , C12N15/864 , C07K14/47
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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