SG11202012044QA - Compositions and methods for treating leber's hereditary optic neuropathy - Google Patents

Compositions and methods for treating leber's hereditary optic neuropathy

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Publication number
SG11202012044QA
SG11202012044QA SG11202012044QA SG11202012044QA SG11202012044QA SG 11202012044Q A SG11202012044Q A SG 11202012044QA SG 11202012044Q A SG11202012044Q A SG 11202012044QA SG 11202012044Q A SG11202012044Q A SG 11202012044QA SG 11202012044Q A SG11202012044Q A SG 11202012044QA
Authority
SG
Singapore
Prior art keywords
compositions
methods
optic neuropathy
hereditary optic
treating leber
Prior art date
Application number
SG11202012044QA
Other languages
English (en)
Inventor
Bin Li
Original Assignee
Wuhan Neurophth Biological Tech Limited 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
Priority claimed from CN201810703168.7A external-priority patent/CN110724695A/zh
Priority claimed from CN201810702492.7A external-priority patent/CN110656117A/zh
Priority claimed from PCT/CN2018/095023 external-priority patent/WO2020010491A1/zh
Priority claimed from CN201810948193.1A external-priority patent/CN110846392A/zh
Priority claimed from PCT/CN2018/103937 external-priority patent/WO2020000641A1/zh
Priority claimed from CN201811221305.XA external-priority patent/CN111073899B/zh
Priority claimed from CN201811230856.2A external-priority patent/CN111068071A/zh
Application filed by Wuhan Neurophth Biological Tech Limited Company filed Critical Wuhan Neurophth Biological Tech Limited Company
Publication of SG11202012044QA publication Critical patent/SG11202012044QA/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
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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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
    • 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
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
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    • 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
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    • 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
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
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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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
    • C12N9/0036—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on NADH or NADPH (1.6)
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    • C12Y106/00—Oxidoreductases acting on NADH or NADPH (1.6)
    • C12Y106/99—Oxidoreductases acting on NADH or NADPH (1.6) with other acceptors (1.6.99)
    • C12Y106/99003—NADH dehydrogenase (1.6.99.3)
    • 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
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    • A01K2227/10—Mammal
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C07K2319/00—Fusion polypeptide
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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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    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Genetics & Genomics (AREA)
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  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Inorganic Chemistry (AREA)
  • Immunology (AREA)
  • Dispersion Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SG11202012044QA 2018-06-29 2019-07-01 Compositions and methods for treating leber's hereditary optic neuropathy SG11202012044QA (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
CN201810703168.7A CN110724695A (zh) 2018-06-29 2018-06-29 一种编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
CN201810702492.7A CN110656117A (zh) 2018-06-29 2018-06-29 一种编码人nadh脱氢酶亚单位6蛋白的核酸及其应用
PCT/CN2018/095023 WO2020010491A1 (zh) 2018-07-09 2018-07-09 编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
CN201810948193.1A CN110846392A (zh) 2018-08-20 2018-08-20 一种重组腺相关病毒或含其的试剂盒及其应用
PCT/CN2018/103937 WO2020000641A1 (zh) 2018-06-29 2018-09-04 编码人nadh脱氢酶亚单位蛋白的核酸及其应用
CN201811221305.XA CN111073899B (zh) 2018-10-19 2018-10-19 一种编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
CN201811230856.2A CN111068071A (zh) 2018-10-22 2018-10-22 基因治疗Leber遗传学视神经病变
PCT/CN2018/113799 WO2020082417A1 (zh) 2018-10-22 2018-11-02 基因治疗Leber遗传性视神经病变
PCT/CN2018/118662 WO2020077756A1 (zh) 2018-10-19 2018-11-30 Nd4蛋白的编码序列及其应用
PCT/CN2019/070461 WO2020037938A1 (zh) 2018-08-20 2019-01-04 一种重组腺相关病毒或含其的试剂盒及其应用
PCT/CN2019/094136 WO2020001657A1 (en) 2018-06-29 2019-07-01 Compositions and methods for treating leber's hereditary optic neuropathy

Publications (1)

Publication Number Publication Date
SG11202012044QA true SG11202012044QA (en) 2021-01-28

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SG11202012044QA SG11202012044QA (en) 2018-06-29 2019-07-01 Compositions and methods for treating leber's hereditary optic neuropathy

Country Status (11)

Country Link
US (3) US11034954B2 (https=)
EP (1) EP3814492A4 (https=)
JP (2) JP2021529001A (https=)
KR (2) KR102627561B1 (https=)
CN (3) CN113528510A (https=)
AU (3) AU2019296451B2 (https=)
BR (1) BR112020026361A2 (https=)
CA (1) CA3103740A1 (https=)
MX (1) MX2020013772A (https=)
SG (1) SG11202012044QA (https=)
WO (1) WO2020001657A1 (https=)

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JP7067744B2 (ja) * 2016-06-30 2022-05-16 株式会社ヘリオス 移植用媒体
CN113528510A (zh) 2018-06-29 2021-10-22 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
SG11202101032VA (en) 2018-08-20 2021-02-25 Wuhan Neurophth Biotechnology Ltd Company Compositions and methods for treating leber's hereditary optic neuropathy
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
CN112159841B (zh) * 2020-10-10 2022-03-01 苏州大学 基于cox10基因插入缺失多态性位点的心源性猝死易感性检测试剂盒
AR126727A1 (es) * 2021-08-06 2023-11-08 Wuhan Neurophth Biotechnology Ltd Company Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4
CN115710585B (zh) * 2021-08-23 2025-03-28 纽福斯(苏州)生物科技有限公司 编码nd4的核酸及其用途

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