CN116234916A - Csrp3(富含半胱氨酸和甘氨酸的蛋白质3)基因疗法 - Google Patents

Csrp3(富含半胱氨酸和甘氨酸的蛋白质3)基因疗法 Download PDF

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CN116234916A
CN116234916A CN202180057650.8A CN202180057650A CN116234916A CN 116234916 A CN116234916 A CN 116234916A CN 202180057650 A CN202180057650 A CN 202180057650A CN 116234916 A CN116234916 A CN 116234916A
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polynucleotide
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C·D·赫尔佐格
C·B·萨克拉门多
R·普拉巴卡尔
D·里克斯
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Spaceship Seven LLC
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
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    • C07—ORGANIC CHEMISTRY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • 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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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Immunology (AREA)
CN202180057650.8A 2020-08-05 2021-08-03 Csrp3(富含半胱氨酸和甘氨酸的蛋白质3)基因疗法 Pending CN116234916A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063061727P 2020-08-05 2020-08-05
US63/061,727 2020-08-05
PCT/US2021/044412 WO2022031756A1 (en) 2020-08-05 2021-08-03 Csrp3 (cysteine and glycine rich protein 3) gene therapy

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Publication Number Publication Date
CN116234916A true CN116234916A (zh) 2023-06-06

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CN202180057650.8A Pending CN116234916A (zh) 2020-08-05 2021-08-03 Csrp3(富含半胱氨酸和甘氨酸的蛋白质3)基因疗法

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US (1) US20230257431A1 (https=)
EP (1) EP4192962A4 (https=)
JP (1) JP2023536618A (https=)
KR (1) KR20230042468A (https=)
CN (1) CN116234916A (https=)
AU (1) AU2021321410A1 (https=)
BR (1) BR112023001336A2 (https=)
CA (1) CA3184983A1 (https=)
IL (1) IL300187A (https=)
MX (1) MX2023000994A (https=)
WO (1) WO2022031756A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
MY209860A (en) 2020-02-13 2025-08-07 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
EP4479543A1 (en) * 2022-02-18 2024-12-25 Ginkgo Bioworks, Inc. Gene therapy for arrhythmogenic cardiomyopathy
CA3255650A1 (en) * 2022-03-18 2023-09-21 Aavantibio, Inc. METHODS AND COMPOSITIONS FOR TREATING RBM20-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10004857A1 (de) * 2000-02-03 2001-08-16 Schering Ag MLP-Gen, Nukleinsäuren, Polypeptide und deren Verwendung
WO2009012176A2 (en) 2007-07-14 2009-01-22 The University Of Iowa Research Foundation Methods and compositions for treating brain diseases
EP2792742A1 (en) * 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
IL248102B (en) 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
MX2019011046A (es) * 2017-03-17 2019-10-17 Res Inst Nationwide Childrens Hospital Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular.
SG11202002276VA (en) * 2017-09-20 2020-04-29 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
AU2019258830B2 (en) * 2018-04-27 2025-12-18 Universität Heidelberg Modified AAV capsid polypeptides for treatment of muscular diseases
US20210260215A1 (en) * 2018-06-08 2021-08-26 University Of Florida Research Foundation, Incorporated Aav cardiac gene therapy for cardiomyopathy
CN114450411A (zh) * 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒

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Publication number Publication date
US20230257431A1 (en) 2023-08-17
JP2023536618A (ja) 2023-08-28
IL300187A (en) 2023-03-01
MX2023000994A (es) 2023-03-01
EP4192962A1 (en) 2023-06-14
KR20230042468A (ko) 2023-03-28
WO2022031756A1 (en) 2022-02-10
EP4192962A4 (en) 2025-04-23
BR112023001336A2 (pt) 2023-02-14
CA3184983A1 (en) 2022-02-10
AU2021321410A1 (en) 2023-04-06

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