BR112022020412A2 - Sequências antissenso para tratar esclerose lateral amiotrófica - Google Patents

Sequências antissenso para tratar esclerose lateral amiotrófica

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Publication number
BR112022020412A2
BR112022020412A2 BR112022020412A BR112022020412A BR112022020412A2 BR 112022020412 A2 BR112022020412 A2 BR 112022020412A2 BR 112022020412 A BR112022020412 A BR 112022020412A BR 112022020412 A BR112022020412 A BR 112022020412A BR 112022020412 A2 BR112022020412 A2 BR 112022020412A2
Authority
BR
Brazil
Prior art keywords
lateral sclerosis
amyotrophic lateral
sense sequences
treat amyotrophic
sequences
Prior art date
Application number
BR112022020412A
Other languages
English (en)
Inventor
Biferi Maria-Grazia
Cappella Marisa
Barkats Martine
Original Assignee
Association Inst De Myologie
Inst Nat Sante Rech Med
Univ Sorbonne
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 Association Inst De Myologie, Inst Nat Sante Rech Med, Univ Sorbonne filed Critical Association Inst De Myologie
Publication of BR112022020412A2 publication Critical patent/BR112022020412A2/pt

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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
    • C12N15/113—Non-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
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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    • C12N2320/00—Applications; Uses
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    • C12N2320/33—Alteration of splicing
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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
    • 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
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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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

SEQUÊNCIAS ANTISSENSO PARA TRATAR ESCLEROSE LATERAL AMIOTRÓFICA. A presente invenção se refere a sequências antissenso, construtos de ácido nucleico e vetores que compreendem as ditas sequências antissenso, e seu uso para tratar uma doença associada à expansão de repetição de hexanucleotídeo C9orf72 como esclerose lateral amiotrófica ou demência frontotemporal.
BR112022020412A 2020-04-09 2021-04-09 Sequências antissenso para tratar esclerose lateral amiotrófica BR112022020412A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP20169064 2020-04-09
PCT/EP2021/059313 WO2021205005A2 (en) 2020-04-09 2021-04-09 Antisense sequences for treating amyotrophic lateral sclerosis

Publications (1)

Publication Number Publication Date
BR112022020412A2 true BR112022020412A2 (pt) 2023-05-02

Family

ID=70285522

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022020412A BR112022020412A2 (pt) 2020-04-09 2021-04-09 Sequências antissenso para tratar esclerose lateral amiotrófica

Country Status (11)

Country Link
US (1) US20230139408A1 (pt)
EP (1) EP4133073A2 (pt)
JP (1) JP2023520730A (pt)
KR (1) KR20230009383A (pt)
CN (1) CN115516093A (pt)
AU (1) AU2021253183A1 (pt)
BR (1) BR112022020412A2 (pt)
CA (1) CA3171436A1 (pt)
IL (1) IL296702A (pt)
MX (1) MX2022012637A (pt)
WO (1) WO2021205005A2 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023183304A2 (en) * 2022-03-21 2023-09-28 Capsigen Inc. Transcription-dependent directed evolution of aav capsids having enhanced tropism

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
JP5894543B2 (ja) 2010-03-17 2016-03-30 アソシアシオン インスティテュト ドゥ ミョロジー 神経筋疾患の治療のための修飾型Ｕ７ｓｎＲＮＡ
ES2535654T3 (es) 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
CN104704123A (zh) 2012-06-21 2015-06-10 肌肉学研究协会 基因治疗载体的广泛的基因递送
US10577604B2 (en) * 2012-10-15 2020-03-03 Ionis Pharmaceuticals, Inc. Methods for monitoring C9ORF72 expression
BR112016007751A2 (pt) * 2013-10-11 2017-09-12 Ionis Pharmaceuticals Inc composições para modulação de expressão de c9orf72
WO2015057727A1 (en) * 2013-10-14 2015-04-23 Isis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016024205A1 (en) * 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
US10793855B2 (en) * 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
US10407678B2 (en) * 2015-04-16 2019-09-10 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2018064600A1 (en) * 2016-09-30 2018-04-05 Regeneron Pharmaceuticals, Inc. Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus
WO2019032612A1 (en) * 2017-08-08 2019-02-14 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED METHODS
WO2019094694A1 (en) * 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders

Also Published As

Publication number Publication date
EP4133073A2 (en) 2023-02-15
WO2021205005A2 (en) 2021-10-14
CA3171436A1 (en) 2021-10-14
MX2022012637A (es) 2022-11-07
WO2021205005A3 (en) 2021-11-18
IL296702A (en) 2022-11-01
KR20230009383A (ko) 2023-01-17
US20230139408A1 (en) 2023-05-04
JP2023520730A (ja) 2023-05-18
CN115516093A (zh) 2022-12-23
AU2021253183A1 (en) 2022-11-03

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