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公开(公告)号：US11084864B2
公开(公告)日：2021-08-10
申请号：US16197194
申请日：2018-11-20
Applicant: Genzyme Corporation
Inventor： Peter Pechan , Jeffery Ardinger , Hillard Rubin , Samuel Wadsworth , Abraham Scaria
Abstract: The present provides fusion proteins comprising PDGF and VEGF binding portions, and recombinant viral particles encoding the fusion proteins. Compositions comprising the fusion proteins and viral particles as well as methods of using the same are also provided.
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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.
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公开(公告)号：US10982228B2
公开(公告)日：2021-04-20
申请号：US15308335
申请日：2015-05-02
Applicant: Genzyme Corporation
Inventor： Abraham Scaria , Jennifer Sullivan
IPC: C12N15/86 , A61K48/00 , C07K14/71 , C07K14/015 , 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.
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公开(公告)号：US10183983B2
公开(公告)日：2019-01-22
申请号：US15471725
申请日：2017-03-28
Applicant: Genzyme Corporation
Inventor： Peter Pechan , Jeffery Ardinger , Hillard Rubin , Samuel Wadsworth , Abraham Scaria
Abstract: The present provides fusion proteins comprising PDGF and VEGF binding portions, and recombinant viral particles encoding the fusion proteins. Compositions comprising the fusion proteins and viral particles as well as methods of using the same are also provided.
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5.发明申请公开(公告)号：US20170007719A1
公开(公告)日：2017-01-12
申请号：US15113720
申请日：2015-02-06
Applicant: GENZYME CORPORATION
Inventor： Abraham Scaria
CPC classification number: A61K48/005 , A61K9/0048 , C07K14/71 , C07K2319/02 , C07K2319/30 , C12N2750/14143 , C12N2750/14171
Abstract: Compositions and methods for treating macular degeneration are disclosed. The methods utilize gene delivery to human eyes of soluble Flt-1 receptors, as well fusion proteins including a soluble Flt-1 receptor.
Abstract translation: 公开了治疗黄斑变性的组合物和方法。 该方法利用可溶性Flt-1受体的人眼的基因递送，以及包括可溶性Flt-1受体的融合蛋白。
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Patent Agency Ranking
公开(公告)号：US11896651B2
公开(公告)日：2024-02-13
申请号：US15574420
申请日：2016-04-15
Applicant: Genzyme Corporation
Inventor： Guoxiang Ruan , Abraham Scaria
IPC: A61K38/46 , A61K31/7105 , A61K31/7088 , A61P27/02 , C12N9/22 , C12N15/11 , C12N15/90
CPC classification number: A61K38/465 , A61K31/7088 , A61K31/7105 , A61P27/02 , C12N9/22 , C12N15/11 , C12N15/907 , C12N2310/20 , C12N2750/14143 , C12N2800/80 , A61K31/7105 , A61K2300/00 , A61K31/7088 , A61K2300/00 , A61K38/465 , A61K2300/00
Abstract: Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non-naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self-limiting CRISPER-Cas system.
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公开(公告)号：US20180155417A1
公开(公告)日：2018-06-07
申请号：US15785775
申请日：2017-10-17
Applicant: Genzyme Corporation
Inventor： Abraham Scaria , Peter Pechan , Samuel Wadsworth
IPC: C07K16/22 , C12P21/00 , C07K14/475 , C07K14/52 , C07K14/705
CPC classification number: C07K16/22 , A61K38/00 , C07K14/475 , C07K14/52 , C07K14/705 , C07K2319/30 , C07K2319/32 , C07K2319/735 , C12P21/00
Abstract: Multimeric fusion proteins of an Ig-like domain of Flt-1 are rendered functional by inclusion of a linker moiety. Vectors encoding the fusion proteins and host cells expressing the fusion proteins can be used therapeutically to block neovascularization in individuals with pathological conditions related to neovascularization. Such conditions include age-related macular degeneration, cancer, psoriasis, proliferative diabetic retinopathy, asthma, uveitis, osteoarthritis, and rheumatoid arthritis. The same means of multimerization used for an Ig like domain of Flt-1, i.e., a linker and a multimerization domain, can be used for other polypeptides, including extracellular receptors, antibody variable regions, cytokines, chemokines, and growth factors.
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公开(公告)号：US09815892B2
公开(公告)日：2017-11-14
申请号：US14170257
申请日：2014-01-31
Applicant: Genzyme Corporation
Inventor： Abraham Scaria , Peter Pechan , Samuel Wadsworth
IPC: C07K16/22 , C07K14/475 , C07K14/52 , C07K14/705 , C12P21/00 , A61K38/00
CPC classification number: C07K16/22 , A61K38/00 , C07K14/475 , C07K14/52 , C07K14/705 , C07K2319/30 , C07K2319/32 , C07K2319/735 , C12P21/00
Abstract: Multimeric fusion proteins of an Ig-like domain of Flt-1 are rendered functional by inclusion of a linker moiety. Vectors encoding the fusion proteins and host cells expressing the fusion proteins can be used therapeutically to block neovascularization in individuals with pathological conditions related to neovascularization. Such conditions include age-related macular degeneration, cancer, psoriasis, proliferative diabetic retinopathy, asthma, uveitis, osteoarthritis, and rheumatoid arthritis. The same means of multimerization used for an Iglike domain of Flt-1, i.e., a linker and a multimerization domain, can be used for other polypeptides, including extracellular receptors, antibody variable regions, cytokines, chemokines, and growth factors.
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公开(公告)号：US11299715B2
公开(公告)日：2022-04-12
申请号：US14758785
申请日：2014-01-07
Applicant: Genzyme Corporation
Inventor： Peter Pechan , Jeffery Ardinger , Abraham Scaria , Samuel Wadsworth
IPC: C12N7/00
Abstract: The use of iNOS inhibitors, including aurintricarboxylic acid, dexamethasone and valproic acid, to increase the yield of a variety of viruses in culture, including recombinant herpesviruses is described.
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公开(公告)号：US10821193B2
公开(公告)日：2020-11-03
申请号：US15511595
申请日：2015-09-16
Applicant: Genzyme Corporation
Inventor： Peter Pechan , Abraham Scaria , Jeffery Ardinger
IPC: C12N15/864 , A61K48/00 , C07K14/47 , C12N15/113 , C12N7/00 , C12N15/86 , C12N15/11 , A61K9/00 , A61K38/17 , C12N15/12
Abstract: Provided herein are methods for treating myocilin (MYOC) glaucoma using adeno-associated viral (AAV) vectors. In some aspects, the AAV vectors encode R-spondin 1 (RSPO1), R-spondin 2 (RSPO2), R-spondin 3 (RSPO3) or R-spondin 4 (RSPO4) and/or RNAi that targets myocilin (MYOC). In one aspect, viral particles are administered to the eye of a human subject. Viral particles encoding RSPO1, RSPO2, RSPO3 and/or RSPO4 and/or MYOC RNAi are contemplated. In some aspects, variant AAV2 particles that transduce the trabecular meshwork are provided.
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