CA3103740A1 - Compositions and methods for treating leber's hereditary optic neuropathy - Google Patents

Compositions and methods for treating leber's hereditary optic neuropathy Download PDF

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Publication number
CA3103740A1
CA3103740A1 CA3103740A CA3103740A CA3103740A1 CA 3103740 A1 CA3103740 A1 CA 3103740A1 CA 3103740 A CA3103740 A CA 3103740A CA 3103740 A CA3103740 A CA 3103740A CA 3103740 A1 CA3103740 A1 CA 3103740A1
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Prior art keywords
sequence
nucleic acid
recombinant nucleic
seq
set forth
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CA3103740A
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English (en)
French (fr)
Inventor
Bin Li
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Wuhan Neurophth Biotechnology Ltd Co
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Wuhan Neurophth Biotechnology Ltd Co
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Priority claimed from CN201810702492.7A external-priority patent/CN110656117A/zh
Priority claimed from CN201810703168.7A external-priority patent/CN110724695A/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 Biotechnology Ltd Co filed Critical Wuhan Neurophth Biotechnology Ltd Co
Priority claimed from PCT/CN2019/094136 external-priority patent/WO2020001657A1/en
Publication of CA3103740A1 publication Critical patent/CA3103740A1/en
Pending legal-status Critical Current

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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • 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
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    • 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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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • 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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    • A61K47/26—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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/107—Rabbit
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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CA3103740A 2018-06-29 2019-07-01 Compositions and methods for treating leber's hereditary optic neuropathy Pending CA3103740A1 (en)

Applications Claiming Priority (21)

Application Number Priority Date Filing Date Title
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
CN201810703168.7 2018-06-29
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
CN201810702492.7 2018-06-29
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
CNPCT/CN2018/095023 2018-07-09
CN201810948193.1 2018-08-20
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
CNPCT/CN2018/103937 2018-09-04
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
CN201811221305.X 2018-10-19
CN201811230856.2A CN111068071A (zh) 2018-10-22 2018-10-22 基因治疗Leber遗传学视神经病变
CN201811230856.2 2018-10-22
PCT/CN2018/113799 WO2020082417A1 (zh) 2018-10-22 2018-11-02 基因治疗Leber遗传性视神经病变
CNPCT/CN2018/113799 2018-11-02
CNPCT/CN2018/118662 2018-11-30
PCT/CN2018/118662 WO2020077756A1 (zh) 2018-10-19 2018-11-30 Nd4蛋白的编码序列及其应用
CNPCT/CN2019/070461 2019-01-04
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
CA3103740A1 true CA3103740A1 (en) 2020-01-02

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ID=72041345

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Application Number Title Priority Date Filing Date
CA3103740A Pending CA3103740A1 (en) 2018-06-29 2019-07-01 Compositions and methods for treating leber's hereditary optic neuropathy

Country Status (9)

Country Link
US (3) US11034954B2 (https=)
EP (1) EP3814492A4 (https=)
JP (2) JP2021529001A (https=)
KR (2) KR20240014102A (https=)
AU (3) AU2019296451B2 (https=)
BR (1) BR112020026361A2 (https=)
CA (1) CA3103740A1 (https=)
MX (1) MX2020013772A (https=)
SG (1) SG11202012044QA (https=)

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KR20240014102A (ko) 2018-06-29 2024-01-31 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
CA3109432A1 (en) 2018-08-20 2020-02-27 Wuhan Neurophth Biotechnology Limited 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

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