AU2020379046B2 - CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy - Google Patents

CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy Download PDF

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AU2020379046B2
AU2020379046B2 AU2020379046A AU2020379046A AU2020379046B2 AU 2020379046 B2 AU2020379046 B2 AU 2020379046B2 AU 2020379046 A AU2020379046 A AU 2020379046A AU 2020379046 A AU2020379046 A AU 2020379046A AU 2020379046 B2 AU2020379046 B2 AU 2020379046B2
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nucleic acid
protein
sequence
acid construct
coding sequence
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AU2020379046A1 (en
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Guochun Gong
Charleen Hunt
Yang Liu
Carmelo Romano
Brian Zambrowicz
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Regeneron Pharmaceuticals Inc
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/14—Hydrolases (3)
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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AU2020379046A 2019-11-08 2020-11-07 CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy Active AU2020379046B2 (en)

Applications Claiming Priority (3)

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US201962932608P 2019-11-08 2019-11-08
US62/932,608 2019-11-08
PCT/US2020/059568 WO2021092513A1 (en) 2019-11-08 2020-11-07 Crispr and aav strategies for x-linked juvenile retinoschisis therapy

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AU2020379046A1 AU2020379046A1 (en) 2022-04-21
AU2020379046B2 true AU2020379046B2 (en) 2025-03-13

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EP (1) EP4054651A1 (enExample)
JP (1) JP7756639B2 (enExample)
KR (2) KR20260018182A (enExample)
CN (1) CN114746125B (enExample)
AU (1) AU2020379046B2 (enExample)
BR (1) BR112022008861A2 (enExample)
CA (1) CA3156277A1 (enExample)
IL (1) IL292605B2 (enExample)
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CN113025618B (zh) * 2019-12-24 2024-02-06 朗信启昇(苏州)生物制药有限公司 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用
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WO2023081739A1 (en) * 2021-11-03 2023-05-11 Teamedon International, Inc. Methods of treating human x-linked retinoschisis using gene therapy
EP4722357A1 (en) 2023-07-07 2026-04-08 Shandong Shunfeng Biotechnology Co., Ltd. Engineered cas protein and use thereof
CN116790559B (zh) * 2023-07-07 2023-11-28 山东舜丰生物科技有限公司 一种融合HNH结构域的V型Cas酶及其应用

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