JP2023536618A - Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療 - Google Patents

Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療 Download PDF

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JP2023536618A
JP2023536618A JP2023507388A JP2023507388A JP2023536618A JP 2023536618 A JP2023536618 A JP 2023536618A JP 2023507388 A JP2023507388 A JP 2023507388A JP 2023507388 A JP2023507388 A JP 2023507388A JP 2023536618 A JP2023536618 A JP 2023536618A
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polynucleotide
vector
mlp
promoter
seq
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JP2023536618A5 (enExample
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クリストファー ディーン ハーツォグ
チェスター ビッテンコート サクラメント
ラジ プラバカール
デイビッド リックス
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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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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
    • 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
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Cardiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2023507388A 2020-08-05 2021-08-03 Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療 Pending JP2023536618A (ja)

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

Publications (2)

Publication Number Publication Date
JP2023536618A true JP2023536618A (ja) 2023-08-28
JP2023536618A5 JP2023536618A5 (enExample) 2024-08-08

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JP2023507388A Pending JP2023536618A (ja) 2020-08-05 2021-08-03 Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療

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

Families Citing this family (10)

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Publication number Priority date Publication date Assignee Title
KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12539316B2 (en) 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
IL280465B2 (en) 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
FI4103214T3 (fi) 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
WO2023159190A1 (en) * 2022-02-18 2023-08-24 Ginkgo Bioworks, Inc. Gene therapy for arrhythmogenic cardiomyopathy
IL315695A (en) * 2022-03-18 2024-11-01 Univ Florida Methods and compositions for treating RBM20-associated cardiomyopathy with a viral vector
JP2025512574A (ja) * 2022-04-22 2025-04-17 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ベクターを使用するＢ細胞リンパ腫２関連Ａｎｔｈａｎｏｇｅｎｅ ３ （ＢＡＧ３）遺伝子治療

Citations (3)

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JP2003523743A (ja) * 2000-02-03 2003-08-12 シエーリング アクチエンゲゼルシャフト Ｍｌｐ遺伝子、核酸、ポリペプチド、及びその使用
JP2016516423A (ja) * 2013-04-17 2016-06-09 ウニヴェルジテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 心筋症を処置するための遺伝子治療ベクター
JP2020513811A (ja) * 2017-03-17 2020-05-21 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達

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LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
DK3684423T5 (da) * 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
CA3097375A1 (en) * 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
CN112272705A (zh) * 2018-06-08 2021-01-26 佛罗里达大学研究基金会有限公司 用于心肌病的aav心脏基因治疗
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JP2016516423A (ja) * 2013-04-17 2016-06-09 ウニヴェルジテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 心筋症を処置するための遺伝子治療ベクター
JP2020513811A (ja) * 2017-03-17 2020-05-21 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達

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BR112023001336A2 (pt) 2023-02-14
WO2022031756A1 (en) 2022-02-10
AU2021321410A1 (en) 2023-04-06
IL300187A (en) 2023-03-01
CA3184983A1 (en) 2022-02-10
EP4192962A4 (en) 2025-04-23
KR20230042468A (ko) 2023-03-28
CN116234916A (zh) 2023-06-06
MX2023000994A (es) 2023-03-01
EP4192962A1 (en) 2023-06-14
US20230257431A1 (en) 2023-08-17

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