PE20212248A1 - Molecula oligomerica de acido nucleico y uso de esta - Google Patents

Molecula oligomerica de acido nucleico y uso de esta

Info

Publication number
PE20212248A1
PE20212248A1 PE2021001108A PE2021001108A PE20212248A1 PE 20212248 A1 PE20212248 A1 PE 20212248A1 PE 2021001108 A PE2021001108 A PE 2021001108A PE 2021001108 A PE2021001108 A PE 2021001108A PE 20212248 A1 PE20212248 A1 PE 20212248A1
Authority
PE
Peru
Prior art keywords
nucleic acid
acid molecule
small
target gene
promoter
Prior art date
Application number
PE2021001108A
Other languages
English (en)
Inventor
Longcheng Li
Moorim Kang
Original Assignee
Ractigen Therapeutics
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 Ractigen Therapeutics filed Critical Ractigen Therapeutics
Publication of PE20212248A1 publication Critical patent/PE20212248A1/es

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • 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
    • A01K2217/072Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • 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
    • 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
    • A01K2217/077Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
    • 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
    • A01K2267/0306Animal model for genetic diseases
    • A01K2267/0318Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications

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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)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Epidemiology (AREA)
  • Environmental Sciences (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

La presente invencion se relaciona con una molecula de acido nucleico activador pequeno para tratar la atrofia muscular espinal y el uso de esta. La molecula de acido nucleico activador pequeno comprende una cadena de acido nucleico sentido y una cadena de acido nucleico antisentido, en donde la cadena de acido nucleico sentido y la cadena de acido nucleico antisentido son independientemente una cadena oligonucleotidica de 16 a 35 nucleotidos de longitud, en la que una cadena nucleotidica tiene al menos 75 % de homologia o complementariedad de bases con respecto a una diana seleccionada de una region promotora de un gen diana SMN2. La presente invencion tambien se relaciona con una composicion farmaceutica que comprende la molecula de acido nucleico activador pequeno descrita en la presente y, opcionalmente, un transportador farmaceuticamente aceptable, y un metodo para aumentar la expresion de un gen diana en celulas y metodos para tratar una enfermedad inducida por la expresion insuficiente de un gen diana con la molecula de acido nucleico activador pequeno o la composicion farmaceutica que comprende la molecula de acido nucleico activador pequeno descrita en la presente.
PE2021001108A 2018-12-29 2019-12-27 Molecula oligomerica de acido nucleico y uso de esta PE20212248A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN201811634268 2018-12-29
PCT/CN2019/129025 WO2020135677A1 (zh) 2018-12-29 2019-12-27 寡聚核酸分子及其应用

Publications (1)

Publication Number Publication Date
PE20212248A1 true PE20212248A1 (es) 2021-11-24

Family

ID=71125998

Family Applications (1)

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PE2021001108A PE20212248A1 (es) 2018-12-29 2019-12-27 Molecula oligomerica de acido nucleico y uso de esta

Country Status (13)

Country Link
US (1) US20220064642A1 (es)
EP (1) EP3904517A4 (es)
JP (1) JP2022515881A (es)
KR (1) KR20210110310A (es)
CN (1) CN112996913B (es)
AU (1) AU2019414608A1 (es)
BR (1) BR112021012422A2 (es)
CA (1) CA3120534A1 (es)
IL (1) IL284333A (es)
MX (1) MX2021007932A (es)
PE (1) PE20212248A1 (es)
SG (1) SG11202106327QA (es)
WO (1) WO2020135677A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230287416A1 (en) * 2020-07-31 2023-09-14 Ractigen Therapeutics Combinatory treatment of sma with sarna and mrna modulators
CN112779252B (zh) * 2020-12-31 2023-05-26 首都儿科研究所 靶向SMN2启动子区MeCP2结合的关键甲基化区域的反义寡核苷酸
CN117377764A (zh) * 2021-02-08 2024-01-09 中美瑞康核酸技术(南通)研究院有限公司 多价寡核苷酸试剂及其使用方法

Family Cites Families (14)

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WO2006005042A2 (en) * 2004-06-30 2006-01-12 Cemines, Inc. Methods and compositions for the diagnosis,prognosis,and treatment of cancer
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
CN1904900A (zh) * 2005-07-28 2007-01-31 中国科学院生物物理研究所 人类的内源性siRNA序列及其应用和筛选方法
US20080187512A1 (en) * 2007-02-07 2008-08-07 Academia Sinica Treatment for spinal muscular atrophy
JP2015518714A (ja) * 2012-05-16 2015-07-06 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
JP2016534035A (ja) * 2013-10-04 2016-11-04 ラナ セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症を治療するための組成物及び方法
CN104630219B (zh) * 2013-11-15 2019-05-07 中美瑞康核酸技术(南通)研究院有限公司 抑癌基因IRX1的saRNA分子及其组合物和其应用
CN106032532A (zh) * 2015-03-17 2016-10-19 中国医学科学院北京协和医院 一种小激活rna及其制备方法和应用
EP3286318A2 (en) * 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
WO2017075030A1 (en) * 2015-10-26 2017-05-04 Rana Therapeutics, Inc. Methods and compositions for increasing smn expression
WO2017087486A1 (en) * 2015-11-16 2017-05-26 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein
US9669109B1 (en) * 2015-11-24 2017-06-06 Universita Di Ferrara Modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy
WO2017218884A1 (en) * 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
WO2018017991A1 (en) * 2016-07-21 2018-01-25 The General Hospital Corporation Extracellular mrna markers of muscular dystrophies in human urine

Also Published As

Publication number Publication date
CN112996913B (zh) 2022-01-04
US20220064642A1 (en) 2022-03-03
EP3904517A1 (en) 2021-11-03
BR112021012422A2 (pt) 2021-10-26
JP2022515881A (ja) 2022-02-22
IL284333A (en) 2021-08-31
WO2020135677A1 (zh) 2020-07-02
SG11202106327QA (en) 2021-07-29
KR20210110310A (ko) 2021-09-07
EP3904517A4 (en) 2022-12-28
CA3120534A1 (en) 2020-07-02
AU2019414608A1 (en) 2021-07-22
MX2021007932A (es) 2021-08-16
CN112996913A (zh) 2021-06-18

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