BR112019024222A2 - elementos reguladores de alta atividade - Google Patents

elementos reguladores de alta atividade Download PDF

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Publication number
BR112019024222A2
BR112019024222A2 BR112019024222-8A BR112019024222A BR112019024222A2 BR 112019024222 A2 BR112019024222 A2 BR 112019024222A2 BR 112019024222 A BR112019024222 A BR 112019024222A BR 112019024222 A2 BR112019024222 A2 BR 112019024222A2
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BR
Brazil
Prior art keywords
transgene
expression
promoter
cases
expression cassette
Prior art date
Application number
BR112019024222-8A
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English (en)
Portuguese (pt)
Inventor
Kartik Ramamoorthi
Stephanie Tagliatela
Anne Tanenhaus
Andrew Young
Szu-Ying Chen
Chi Zhang
Stephanie Martin
David Oberkofler
Original Assignee
Encoded Therapeutics, Inc.
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Application filed by Encoded Therapeutics, Inc. filed Critical Encoded Therapeutics, Inc.
Publication of BR112019024222A2 publication Critical patent/BR112019024222A2/pt

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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/035Animal model for multifactorial diseases
    • A01K2267/0381Animal model for diseases of the hematopoietic system
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/001Vector systems having a special element relevant for transcription controllable enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
BR112019024222-8A 2017-05-19 2018-05-18 elementos reguladores de alta atividade BR112019024222A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762508968P 2017-05-19 2017-05-19
US62/508,968 2017-05-19
PCT/US2018/033515 WO2018213786A1 (en) 2017-05-19 2018-05-18 High activity regulatory elements

Publications (1)

Publication Number Publication Date
BR112019024222A2 true BR112019024222A2 (pt) 2020-08-18

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019024222-8A BR112019024222A2 (pt) 2017-05-19 2018-05-18 elementos reguladores de alta atividade

Country Status (11)

Country Link
US (2) US12104175B2 (https=)
EP (1) EP3624858A4 (https=)
JP (3) JP7315475B2 (https=)
KR (2) KR102636351B1 (https=)
CN (2) CN110944674B (https=)
AU (1) AU2018269050A1 (https=)
BR (1) BR112019024222A2 (https=)
CA (1) CA3063464A1 (https=)
MA (1) MA49153A (https=)
TW (1) TWI811219B (https=)
WO (1) WO2018213786A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
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GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
AU2018269050A1 (en) 2017-05-19 2020-01-16 Encoded Therapeutics, Inc. High activity regulatory elements
EP3717505A4 (en) 2017-12-01 2021-12-01 Encoded Therapeutics, Inc. GENENICALLY MODIFIED DNA-BINDING PROTEINS
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
MX2021008739A (es) * 2019-01-24 2021-10-13 Valorisation Hsj Lp Secuencias reguladoras de la transcripción específicas de células y usos de estas.
EP3952924A4 (en) * 2019-04-12 2023-05-24 Encoded Therapeutics, Inc. COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS
PL3976798T3 (pl) 2019-05-29 2026-04-07 Encoded Therapeutics, Inc. Kompozycje i sposoby selektywnej regulacji genów
JP7759311B2 (ja) 2019-07-19 2025-10-23 フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー リコンビナーゼ組成物及び使用方法
CN113088519B (zh) * 2019-12-23 2024-02-02 苏州华毅乐健生物科技有限公司 一种分离的核酸分子及其应用
JP2023514204A (ja) * 2020-02-14 2023-04-05 ウルトラジェニックス ファーマシューティカル インコーポレイテッド Cdkl5欠損障害を処置するための遺伝子治療
KR20230003569A (ko) * 2020-04-27 2023-01-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Cdkl5 결핍 장애 (cdd)의 치료에 유용한 조성물
US20250041450A1 (en) * 2021-08-05 2025-02-06 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
JP2025533994A (ja) * 2022-10-11 2025-10-09 メイラグティーエックス ユーケー アイアイ リミティド Atp7b遺伝子療法
CN117925624B (zh) * 2024-03-22 2024-05-31 上海凌医生物科技有限公司 一种金属响应调控元件
CN117947035A (zh) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 一种增强基因表达的启动子

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ES2284545T3 (es) * 1999-11-16 2007-11-16 Genzyme Corporation Vectores y transgenes con elementos reguladores para la administracion de genes del higado.
US6436703B1 (en) * 2000-03-31 2002-08-20 Hyseq, Inc. Nucleic acids and polypeptides
EP1783645A1 (en) * 2005-11-08 2007-05-09 Actigenics Methods for the identification of microRNA and their applications in research and human health
WO2008073303A2 (en) * 2006-12-07 2008-06-19 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
WO2013033627A2 (en) * 2011-09-01 2013-03-07 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
ES2786051T3 (es) 2012-10-25 2020-10-08 Tocagen Inc Vector retroviral con casete de mini-promotor
US11147887B2 (en) * 2014-12-17 2021-10-19 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson disease
TWI707951B (zh) * 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
AU2018269050A1 (en) 2017-05-19 2020-01-16 Encoded Therapeutics, Inc. High activity regulatory elements
EP3717505A4 (en) * 2017-12-01 2021-12-01 Encoded Therapeutics, Inc. GENENICALLY MODIFIED DNA-BINDING PROTEINS
SG11202109362XA (en) * 2019-02-28 2021-09-29 Encoded Therapeutics Inc Compositions and methods for treating laminopathies

Also Published As

Publication number Publication date
JP7315475B2 (ja) 2023-07-26
CA3063464A1 (en) 2018-11-22
MA49153A (fr) 2021-03-24
CN110944674A (zh) 2020-03-31
KR102636351B1 (ko) 2024-02-15
TWI811219B (zh) 2023-08-11
EP3624858A1 (en) 2020-03-25
US20200231943A1 (en) 2020-07-23
CN118956965A (zh) 2024-11-15
KR20200026814A (ko) 2020-03-11
WO2018213786A1 (en) 2018-11-22
EP3624858A4 (en) 2021-06-23
JP2020520643A (ja) 2020-07-16
JP2022173348A (ja) 2022-11-18
JP2024015408A (ja) 2024-02-01
US12104175B2 (en) 2024-10-01
TW201903152A (zh) 2019-01-16
KR20240024326A (ko) 2024-02-23
CN110944674B (zh) 2024-07-19
US20240376441A1 (en) 2024-11-14
AU2018269050A1 (en) 2020-01-16

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