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MA58655B1 - Acide nucléique optimisé par codons qui code la protéine smn1 - Google Patents

Acide nucléique optimisé par codons qui code la protéine smn1

Info

Publication number
MA58655B1
MA58655B1 MA58655A MA58655A MA58655B1 MA 58655 B1 MA58655 B1 MA 58655B1 MA 58655 A MA58655 A MA 58655A MA 58655 A MA58655 A MA 58655A MA 58655 B1 MA58655 B1 MA 58655B1
Authority
MA
Morocco
Prior art keywords
encodes
nucleic acid
codon
optimized nucleic
smn1
Prior art date
Application number
MA58655A
Other languages
English (en)
Other versions
MA58655A1 (fr
Inventor
Dmitriy Aleksandrovich Madera
Pavel Mikhailovich Gershovich
Anna Sergeevna Veselova
Tatiana Evgenievna Shugaeva
Maria Andreevna Lomunova
Margarita Aleksandrovna Shkliaeva
Dmitry Valentinovich Morozov
Original Assignee
Biocad Joint Stock Co
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 Biocad Joint Stock Co filed Critical Biocad Joint Stock Co
Publication of MA58655A1 publication Critical patent/MA58655A1/fr
Publication of MA58655B1 publication Critical patent/MA58655B1/fr

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C07K14/4707—Muscular dystrophy
    • 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
    • A61K48/0066—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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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

Landscapes

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

Abstract

La présente invention se rapporte au domaine de la génétique, de la thérapie génique et de la biologie moléculaire. L'invention concerne plus précisément un acide nucléique optimisé par codons séparé qui code la protéine smn1 (protéine de survivance des neurones moteurs), une cassette d'expression et un vecteur à base de celle-ci, ainsi qu'un virus recombinant à base de aav9 (virus adéno-associé de sérotype 9) afin d'augmenter l'expression du gène smn1 dans des cellules cibles, ainsi que l'utilisation de celui-ci.
MA58655A 2020-06-02 2021-06-02 Acide nucléique optimisé par codons qui code la protéine smn1 MA58655B1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
RU2020118148A RU2742837C1 (ru) 2020-06-02 2020-06-02 Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
PCT/RU2021/000238 WO2021246909A1 (fr) 2020-06-02 2021-06-02 Acide nucléique optimisé par codons qui code la protéine smn1

Publications (2)

Publication Number Publication Date
MA58655A1 MA58655A1 (fr) 2023-06-28
MA58655B1 true MA58655B1 (fr) 2023-12-29

Family

ID=74665909

Family Applications (1)

Application Number Title Priority Date Filing Date
MA58655A MA58655B1 (fr) 2020-06-02 2021-06-02 Acide nucléique optimisé par codons qui code la protéine smn1

Country Status (22)

Country Link
US (1) US20230212609A1 (fr)
EP (1) EP4159863A4 (fr)
JP (1) JP7765413B2 (fr)
KR (1) KR20230019162A (fr)
CN (1) CN116234914A (fr)
AR (1) AR122500A1 (fr)
AU (1) AU2021282898A1 (fr)
BR (1) BR112022024708A2 (fr)
CA (1) CA3181288A1 (fr)
CL (1) CL2022003428A1 (fr)
CO (1) CO2022017334A2 (fr)
CR (1) CR20220619A (fr)
EC (1) ECSP22092206A (fr)
IL (1) IL298772A (fr)
MA (1) MA58655B1 (fr)
MX (1) MX2022015231A (fr)
PE (1) PE20240079A1 (fr)
RU (1) RU2742837C1 (fr)
TW (1) TWI893130B (fr)
UY (1) UY39245A (fr)
WO (1) WO2021246909A1 (fr)
ZA (1) ZA202213092B (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA62178A1 (fr) * 2021-01-29 2023-12-29 Biocad Joint Stock Co Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
UY40304A (es) * 2022-06-10 2023-12-29 Biocad Joint Stock Co Ácido nucleico con actividad promotora y uso del mismo
WO2023246734A1 (fr) * 2022-06-21 2023-12-28 Skyline Therapeutics (Shanghai) Co., Ltd. Vaa recombinant pour la thérapie génique de la maladie sma

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4634665A (en) 1980-02-25 1987-01-06 The Trustees Of Columbia University In The City Of New York Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials
US4399216A (en) 1980-02-25 1983-08-16 The Trustees Of Columbia University Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials
US5179017A (en) 1980-02-25 1993-01-12 The Trustees Of Columbia University In The City Of New York Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials
DE3431140A1 (de) 1984-08-24 1986-03-06 Behringwerke Ag, 3550 Marburg Enhancer fuer eukaryotische expressionssysteme
GB8717430D0 (en) 1987-07-23 1987-08-26 Celltech Ltd Recombinant dna product
EP0708178A1 (fr) * 1994-10-19 1996-04-24 Institut National De La Sante Et De La Recherche Medicale (Inserm) Gène de la survie des neurones moteurs: gène de l'atrophie musculaire spinale
RU2312896C2 (ru) * 2001-09-20 2007-12-20 Глаксо Груп Лимитед Последовательность нуклеиновой кислоты, кодирующая белок gag вич-1, способ получения указанной последовательности, вектор, содержащий ее, белок, кодируемый ею, фармацевтическая композиция и их применение для профилактики и/или лечения вич-инфекции и спида
US20080276327A1 (en) 2004-05-21 2008-11-06 University Of Utah Research Foundation Methods and Compositions Related to Delivery of Chemical Compounds to Invertebrate Embryos
US10821154B2 (en) * 2013-05-01 2020-11-03 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
EP4036241A1 (fr) * 2013-10-22 2022-08-03 Translate Bio, Inc. Administration de l'arnm dans le snc et leurs utilisation
US9668980B2 (en) * 2014-07-02 2017-06-06 Rana Therapeutics, Inc. Encapsulation of messenger RNA
MX2017005834A (es) * 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
KR20180086266A (ko) * 2015-12-14 2018-07-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 척수성 근위축증의 치료에 유용한 아데노-관련 바이러스 벡터
EP3452101A2 (fr) 2016-05-04 2019-03-13 CureVac AG Arn codant pour une protéine thérapeutique
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
US20200370069A1 (en) * 2017-07-08 2020-11-26 Genethon Treatment of spinal muscular atrophy
CN111566220A (zh) * 2017-11-08 2020-08-21 阿维克斯公司 制备病毒载体的手段和方法及其用途
CN108795946B (zh) * 2018-06-28 2022-03-04 北京锦篮基因科技有限公司 携带设计smn1基因表达框的重组腺相关病毒及应用
TW202019398A (zh) 2018-06-28 2020-06-01 張翔毓 用於治療或預防構形疾病之方法及藥物篩選方法

Also Published As

Publication number Publication date
ECSP22092206A (es) 2023-01-31
UY39245A (es) 2021-12-31
WO2021246909A1 (fr) 2021-12-09
TWI893130B (zh) 2025-08-11
CA3181288A1 (fr) 2021-12-09
ZA202213092B (en) 2024-09-25
RU2742837C1 (ru) 2021-02-11
US20230212609A1 (en) 2023-07-06
JP2023529855A (ja) 2023-07-12
CL2022003428A1 (es) 2023-06-09
MX2022015231A (es) 2023-02-09
AR122500A1 (es) 2022-09-14
CN116234914A (zh) 2023-06-06
EP4159863A1 (fr) 2023-04-05
IL298772A (en) 2023-02-01
AU2021282898A1 (en) 2023-02-02
CR20220619A (es) 2023-06-14
CO2022017334A2 (es) 2023-04-17
TW202214862A (zh) 2022-04-16
PE20240079A1 (es) 2024-01-16
MA58655A1 (fr) 2023-06-28
KR20230019162A (ko) 2023-02-07
EP4159863A4 (fr) 2024-08-28
BR112022024708A2 (pt) 2023-04-11
JP7765413B2 (ja) 2025-11-06

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