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    AAV vectors for retinal and CNS gene therapy
    1.
    发明授权
    AAV vectors for retinal and CNS gene therapy 有权

    公开(公告)号：US12241078B2

    公开(公告)日：2025-03-04

    申请号：US17194009

    申请日：2021-03-05

    Applicant: Genzyme Corporation

    Inventor： Abraham Scaria ,  Jennifer Sullivan ,  Lisa M. Stanek ,  Lamya S. Shihabuddin

    IPC: C12N15/86 ,  A61K48/00 ,  C07K14/015 ,  C07K14/71 ,  C12N7/00 ,  C12N15/113

    Abstract: Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of rAAV particles. Further provided herein are improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of disorders of the CNS. The invention provides methods for delivering the rAAV to the CNS, methods for treating disorders of the CNS with improved compositions of rAAV particles, and kits for delivering the rAAV to the CNS and/or treating a CNS disorder.

    Variant RNAi
    2.
    发明授权
    Variant RNAi 有权

    公开(公告)号：US10760079B2

    公开(公告)日：2020-09-01

    申请号：US16566565

    申请日：2019-09-10

    Applicant: Genzyme Corporation

    Inventor： Lisa M. Stanek ,  Adam Palermo ,  Brenda Richards ,  Sergio Pablo Sardi ,  Catherine O'Riordan ,  Antonius Song

    IPC: C07H21/04 ,  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.

    Variant RNAi
    3.
    发明授权
    Variant RNAi 有权

    公开(公告)号：US10450563B2

    公开(公告)日：2019-10-22

    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: C07H21/04 ,  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.

    Variant RNAi
    5.
    发明授权
    Variant RNAi 有权

    公开(公告)号：US11781137B2

    公开(公告)日：2023-10-10

    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: C07H21/04 ,  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.

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