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AR126727A1 - Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4 - Google Patents

Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4

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Publication number
AR126727A1
AR126727A1 ARP220102119A ARP220102119A AR126727A1 AR 126727 A1 AR126727 A1 AR 126727A1 AR P220102119 A ARP220102119 A AR P220102119A AR P220102119 A ARP220102119 A AR P220102119A AR 126727 A1 AR126727 A1 AR 126727A1
Authority
AR
Argentina
Prior art keywords
nucleic acid
sequence
recombinant nucleic
mitochondrial
protein
Prior art date
Application number
ARP220102119A
Other languages
English (en)
Inventor
Li Bin
Original Assignee
Wuhan Neurophth Biotechnology Ltd Company
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Wuhan Neurophth Biotechnology Ltd Company filed Critical Wuhan Neurophth Biotechnology Ltd Company
Publication of AR126727A1 publication Critical patent/AR126727A1/es

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
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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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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Se proporciona un ácido nucleico recombinante que comprende: una secuencia mitocondrial diana; y una secuencia de codificación de una proteína mitocondrial, en la cual la secuencia de codificación de una proteína mitocondrial codifica un polipéptido que comprende una proteína mitocondrial; una secuencia del ácido nucleico del extremo 3’ UTR, y una secuencia señal de poliA. También se proporciona una composición farmacéutica que comprende el ácido nucleico recombinante y un método para el tratamiento de la neuropatía óptica hereditaria de Leber (NOHL) utilizando la composición farmacéutica. Reivindicación 1: Un ácido nucleico recombinante que comprende (secuencialmente desde el extremo 5’ hasta el extremo 3’) una secuencia mitocondrial diana y una secuencia de codificación de una proteína mitocondrial, en el cual, opcionalmente, la secuencia de codificación de una proteína mitocondrial codifica una proteína ND4; y, opcionalmente, la proteína ND4 comprende una secuencia de aminoácidos que tiene al menos un 90%, al menos un 95%, al menos un 96%, al menos un 97%, al menos un 98%, al menos un 99% o un 100% de identidad con la SEC ID Nº 160. Reivindicación 32: Un vector viral que comprende el ácido nucleico recombinante según cualquiera de las reivindicaciones 1 a 31. Reivindicación 35: Una composición farmacéutica que comprende un virus adenoasociado (AAV), en el cual el AAV comprende el ácido nucleico recombinante según cualquiera de las reivindicaciones 1 a 31.
ARP220102119A 2021-08-06 2022-08-05 Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4 AR126727A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CN202110899561 2021-08-06

Publications (1)

Publication Number Publication Date
AR126727A1 true AR126727A1 (es) 2023-11-08

Family

ID=85155306

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220102119A AR126727A1 (es) 2021-08-06 2022-08-05 Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4

Country Status (11)

Country Link
US (1) US20240376164A1 (es)
EP (1) EP4382601A4 (es)
JP (1) JP2024529033A (es)
KR (1) KR20240042489A (es)
CN (1) CN116024266A (es)
AR (1) AR126727A1 (es)
AU (1) AU2022322957A1 (es)
BR (1) BR112024002305A2 (es)
CA (1) CA3228344A1 (es)
MX (1) MX2024001597A (es)
WO (1) WO2023011632A1 (es)

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3625214A (en) 1970-05-18 1971-12-07 Alza Corp Drug-delivery device
US4906474A (en) 1983-03-22 1990-03-06 Massachusetts Institute Of Technology Bioerodible polyanhydrides for controlled drug delivery
US4789734A (en) 1985-08-06 1988-12-06 La Jolla Cancer Research Foundation Vitronectin specific cell receptor derived from mammalian mesenchymal tissue
JP2876058B2 (ja) 1986-08-18 1999-03-31 エミスフィア・テクノロジーズ・インコーポレイテッド 薬物送達システム
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US5811128A (en) 1986-10-24 1998-09-22 Southern Research Institute Method for oral or rectal delivery of microencapsulated vaccines and compositions therefor
US4897268A (en) 1987-08-03 1990-01-30 Southern Research Institute Drug delivery system and method of making the same
AU679041B2 (en) 1993-01-11 1997-06-19 Dana-Farber Cancer Institute Inducing cytotoxic T lymphocyte responses
US6207392B1 (en) 1997-11-25 2001-03-27 The Regents Of The University Of California Semiconductor nanocrystal probes for biological applications and process for making and using such probes
CN102634527B (zh) * 2012-04-11 2013-11-06 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
WO2020001657A1 (en) * 2018-06-29 2020-01-02 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CN111068071A (zh) * 2018-10-22 2020-04-28 武汉纽福斯生物科技有限公司 基因治疗Leber遗传学视神经病变
CN110876269B (zh) * 2018-06-29 2021-07-30 武汉纽福斯生物科技有限公司 治疗遗传性视神经病变的组合物和方法
WO2020038352A1 (en) * 2018-08-20 2020-02-27 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
EP3840785A4 (en) * 2018-08-20 2022-07-13 Wuhan Neurophth Biotechnology Limited Company COMPOSITIONS AND METHODS FOR THE TREATMENT OF LEBERIAN OPTIC ATROPHY
WO2020146379A1 (en) * 2019-01-07 2020-07-16 California Institute Of Technology Gas vesicle expression systems, gas vesicle constructs and related genetic circuits, vectors, mammalian cells, hosts, compositions, methods and systems
CN113025633B (zh) * 2019-12-09 2024-08-27 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
CN116096904A (zh) * 2019-12-31 2023-05-09 天鹅生物疗法有限公司 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途

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Publication number Publication date
CA3228344A1 (en) 2023-02-09
JP2024529033A (ja) 2024-08-01
KR20240042489A (ko) 2024-04-02
BR112024002305A2 (pt) 2024-04-30
WO2023011632A1 (zh) 2023-02-09
AU2022322957A1 (en) 2024-03-21
CN116024266A (zh) 2023-04-28
EP4382601A4 (en) 2025-07-30
US20240376164A1 (en) 2024-11-14
EP4382601A1 (en) 2024-06-12
MX2024001597A (es) 2024-02-15

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