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    METHODS FOR DETECTING AAV
    2.
    发明申请
    METHODS FOR DETECTING AAV 有权

    公开(公告)号：US20250138024A1

    公开(公告)日：2025-05-01

    申请号：US19013863

    申请日：2025-01-08

    Applicant: Genzyme Corporation

    Inventor： Xiaoying JIN ,  Catherine O'RIORDAN ,  Lin LIU ,  Kate ZHANG

    IPC: G01N33/68 ,  C12N15/86 ,  G01N30/02 ,  G01N30/72

    Abstract: Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.

    VARIANT RNAi
    3.
    发明公开
    VARIANT RNAi 审中-公开

    公开(公告)号：US20240182896A1

    公开(公告)日：2024-06-06

    申请号：US18483474

    申请日：2023-10-09

    Applicant: Genzyme Corporation

    Inventor： Lisa M. STANEK ,  Adam PALERMO ,  Brenda RICHARDS ,  Sergio Pablo SARDI ,  Catherine O'RIORDAN ,  Antonius SONG

    IPC: C12N15/113

    CPC classification number: C12N15/113 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2310/533

    Abstract: Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

    VARIANT RNAi
    4.
    发明申请
    VARIANT RNAi 审中-公开

    公开(公告)号：US20180023082A1

    公开(公告)日：2018-01-25

    申请号：US15549895

    申请日：2016-02-09

    Applicant: Genzyme Corporation

    Inventor： Lisa M. STANEK ,  Adam PALERMO ,  Brenda RICHARDS ,  Sergio Pablo SARDI ,  Catherine O'RIORDAN ,  Antonius SONG

    IPC: C12N15/113

    Abstract: Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

    GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS
    5.
    发明申请
    GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS 审中-公开
    脊柱侧凸性脊柱炎及其他脊髓损伤的基因治疗

    公开(公告)号：US20160067312A1

    公开(公告)日：2016-03-10

    申请号：US14692469

    申请日：2015-04-21

    Applicant: GENZYME CORPORATION

    Inventor： James DODGE ,  Lamya SHIHABUDDIN ,  Catherine O'RIORDAN

    IPC: A61K38/30

    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 a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.

    Abstract translation: 本公开提供了用于治疗影响受试者的运动功能和控制的疾病或损伤的方法和组合物。 一方面，本发明通过将包含转基因的重组神经营养病毒载体给予脑，将转基因产物递送至受试者的脊髓。 病毒载体将转基因递送到易受病毒感染并表达编码的重组病毒基因产物的脑区域。 还提供了通过将含有转基因的重组神经营养病毒载体给予受试者的脑，将转基因产物递送至受试者的脊髓的组合物。

    METHODS FOR DETECTING AAV
    6.
    发明申请
    METHODS FOR DETECTING AAV 有权

    公开(公告)号：US20250147042A1

    公开(公告)日：2025-05-08

    申请号：US19013836

    申请日：2025-01-08

    Applicant: Genzyme Corporation

    Inventor： Xiaoying JIN ,  Catherine O'RIORDAN ,  Lin LIU ,  Kate ZHANG

    IPC: G01N33/68 ,  C12N15/86 ,  G01N30/02 ,  G01N30/72

    Abstract: Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.

    VARIANT RNAi
    8.
    发明申请
    VARIANT RNAi 有权

    公开(公告)号：US20210047641A1

    公开(公告)日：2021-02-18

    申请号：US16941303

    申请日：2020-07-28

    Applicant: Genzyme Corporation

    Inventor： Lisa M. STANEK ,  Adam PALERMO ,  Brenda RICHARDS ,  Sergio Pablo SARDI ,  Catherine O'RIORDAN ,  Antonius SONG

    IPC: C12N15/113

    Abstract: Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

    GENE THERAPY FOR SPINAL CORD DISORDERS
    9.
    发明申请
    GENE THERAPY FOR SPINAL CORD DISORDERS 有权

    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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