HRP20211407T1 - Genetska konstrukcija - Google Patents

Genetska konstrukcija Download PDF

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Publication number
HRP20211407T1
HRP20211407T1 HRP20211407TT HRP20211407T HRP20211407T1 HR P20211407 T1 HRP20211407 T1 HR P20211407T1 HR P20211407T T HRP20211407T T HR P20211407TT HR P20211407 T HRP20211407 T HR P20211407T HR P20211407 T1 HRP20211407 T1 HR P20211407T1
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Croatia
Prior art keywords
seq
sequence
fragment
variant
optionally
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HRP20211407TT
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English (en)
Croatian (hr)
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Peter Widdowson
Keith Martin
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Quethera Limited
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Publication of HRP20211407T1 publication Critical patent/HRP20211407T1/hr

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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
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/48—Nerve growth factor [NGF]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
    • 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
    • A61K48/0066—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • A—HUMAN NECESSITIES
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    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C12N15/1027—Mutagenizing nucleic acids by DNA shuffling, e.g. RSR, STEP, RPR
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86—Viral vectors
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    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • A—HUMAN NECESSITIES
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    • C12N2750/14011—Parvoviridae
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/10—Protein-tyrosine kinases (2.7.10)
    • C12Y207/10001—Receptor protein-tyrosine kinase (2.7.10.1)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Dermatology (AREA)
  • Heart & Thoracic Surgery (AREA)
HRP20211407TT 2015-10-26 2016-10-25 Genetska konstrukcija HRP20211407T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1518911.1A GB2547179A (en) 2015-10-26 2015-10-26 Genetic construct
EP16788748.8A EP3368678B1 (en) 2015-10-26 2016-10-25 Genetic construct
PCT/GB2016/053319 WO2017072498A1 (en) 2015-10-26 2016-10-25 Genetic construct

Publications (1)

Publication Number Publication Date
HRP20211407T1 true HRP20211407T1 (hr) 2021-12-10

Family

ID=55130230

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20211407TT HRP20211407T1 (hr) 2015-10-26 2016-10-25 Genetska konstrukcija

Country Status (21)

Country Link
US (2) US10342880B2 (OSRAM)
EP (2) EP3985119A1 (OSRAM)
JP (2) JP7073264B2 (OSRAM)
KR (1) KR102819739B1 (OSRAM)
CN (1) CN108350467B (OSRAM)
AU (1) AU2016344508B2 (OSRAM)
CY (1) CY1124588T1 (OSRAM)
DK (1) DK3368678T3 (OSRAM)
ES (1) ES2886009T3 (OSRAM)
GB (1) GB2547179A (OSRAM)
HR (1) HRP20211407T1 (OSRAM)
HU (1) HUE056911T2 (OSRAM)
LT (1) LT3368678T (OSRAM)
MX (1) MX2018005286A (OSRAM)
PL (1) PL3368678T3 (OSRAM)
PT (1) PT3368678T (OSRAM)
RS (1) RS62460B1 (OSRAM)
RU (1) RU2734678C2 (OSRAM)
SI (1) SI3368678T1 (OSRAM)
SM (1) SMT202100520T1 (OSRAM)
WO (1) WO2017072498A1 (OSRAM)

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GB201705484D0 (en) * 2017-04-05 2017-05-17 Quethera Ltd Genetic construct
CN111372612A (zh) * 2017-09-27 2020-07-03 西吉隆医疗股份有限公司 包含活性细胞的方法、组合物和可植入元件
IL321310A (en) 2017-10-20 2025-08-01 Res Inst Nationwide Childrens Hospital Methods and materials for NT-3 gene therapy
EP3720460A1 (en) * 2017-12-05 2020-10-14 University of Washington Compositions and methods for enhancing functional expression of therapeutic genes in photoreceptors
CN108250288A (zh) * 2018-01-17 2018-07-06 吉林省农业科学院 一种叶绿体表达的脑源性神经营养因子hBDNFb蛋白及其制备方法
CN109022487A (zh) * 2018-08-15 2018-12-18 上海市第人民医院 一种表达p65的基因载体及其用于治疗视网膜神经节细胞变性的基因治疗药物
WO2020077295A1 (en) * 2018-10-11 2020-04-16 Decibel Therapeutics, Inc. Aav1 vectors and uses thereof for treatment of otic indications
SG11202105267VA (en) * 2018-12-19 2021-07-29 Versameb Ag Rna encoding a protein
CN113557301B (zh) * 2019-03-04 2025-03-07 公益财团法人东京都医学综合研究所 编码Trk片段的核酸构建体及其利用
MA55223A (fr) * 2019-03-06 2022-01-12 Generation Bio Co Adn à extrémité fermée (cedna) et composés de modulation immunitaire
US20230123802A1 (en) * 2019-03-27 2023-04-20 Sigilon Therapeutics, Inc. Compositions, devices, and methods for factor vii therapy
US20230038502A1 (en) 2019-06-11 2023-02-09 Shire Human Genetic Therapies, Inc. Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
EP3999647A1 (en) * 2019-07-18 2022-05-25 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
RU2719013C1 (ru) * 2019-09-25 2020-04-16 Федеральное государственное бюджетное образовательное учреждение высшего образования "Московский государственный университет имени М.В. Ломоносова" (МГУ) Генно-инженерная конструкция для стимуляции посттравматической регенерации периферических нервов
WO2021067448A1 (en) 2019-09-30 2021-04-08 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of genetic hearing loss
EP4048800A1 (en) 2019-10-23 2022-08-31 Shire Human Genetic Therapies, Inc. Adeno-associated virus vectors based gene therapy for hereditary angioedema
US20220186252A1 (en) 2020-12-16 2022-06-16 Takeda Pharmaceutical Company Limited Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
MX2023009922A (es) * 2021-02-26 2023-10-25 Icahn School Med Mount Sinai Método para reducir la degeneración de células ganglionares retinianas.
CN116179495B (zh) * 2022-11-28 2025-06-06 上海恩凯细胞技术有限公司 转基因免疫细胞及其应用
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