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MX2019006882A - Conjugados de oligomeros de omision de exon para distrofia muscular. - Google Patents

Conjugados de oligomeros de omision de exon para distrofia muscular.

Info

Publication number
MX2019006882A
MX2019006882A MX2019006882A MX2019006882A MX2019006882A MX 2019006882 A MX2019006882 A MX 2019006882A MX 2019006882 A MX2019006882 A MX 2019006882A MX 2019006882 A MX2019006882 A MX 2019006882A MX 2019006882 A MX2019006882 A MX 2019006882A
Authority
MX
Mexico
Prior art keywords
oligomer conjugates
muscular dystrophy
exon skipping
skipping oligomer
conjugates
Prior art date
Application number
MX2019006882A
Other languages
English (en)
Inventor
J Hanson Gunnar
A Passini Marco
Original Assignee
Sarepta Therapeutics Inc
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 Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of MX2019006882A publication Critical patent/MX2019006882A/es

Links

Classifications

    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • 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
    • 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
    • C12N15/11—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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente invencion se refiere a conjugados de oligomeros antisentido complementarios a un sitio de objetivo seleccionado en el gen de distrofina humana para inducir la omision del exon 51.
MX2019006882A 2016-12-19 2017-12-13 Conjugados de oligomeros de omision de exon para distrofia muscular. MX2019006882A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662436182P 2016-12-19 2016-12-19
US201762443476P 2017-01-06 2017-01-06
US201762479173P 2017-03-30 2017-03-30
US201762562080P 2017-09-22 2017-09-22
PCT/US2017/066222 WO2018118599A1 (en) 2016-12-19 2017-12-13 Exon skipping oligomer conjugates for muscular dystrophy

Publications (1)

Publication Number Publication Date
MX2019006882A true MX2019006882A (es) 2019-08-16

Family

ID=61006315

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019006882A MX2019006882A (es) 2016-12-19 2017-12-13 Conjugados de oligomeros de omision de exon para distrofia muscular.

Country Status (28)

Country Link
US (4) US10888578B2 (es)
EP (1) EP3554553B1 (es)
JP (3) JP7118063B2 (es)
KR (2) KR102810425B1 (es)
CN (1) CN110290812B (es)
AU (1) AU2017382723B2 (es)
BR (1) BR112019012647A2 (es)
CA (1) CA3046793A1 (es)
CO (1) CO2019007397A2 (es)
DK (1) DK3554553T3 (es)
ES (1) ES2927079T3 (es)
HR (1) HRP20220943T1 (es)
HU (1) HUE059843T2 (es)
IL (2) IL267244B (es)
LT (1) LT3554553T (es)
MD (1) MD3554553T2 (es)
MX (1) MX2019006882A (es)
MY (1) MY195801A (es)
NZ (1) NZ755416A (es)
PL (1) PL3554553T3 (es)
PT (1) PT3554553T (es)
RS (1) RS63610B1 (es)
SA (1) SA519402154B1 (es)
SG (1) SG10202012839TA (es)
SM (1) SMT202200366T1 (es)
TW (1) TWI760402B (es)
WO (1) WO2018118599A1 (es)
ZA (1) ZA201904092B (es)

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TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45154B1 (fr) 2016-05-24 2024-11-29 Sarepta Therapeutics, Inc. Procédés de préparation d'oligomères
CN109311920B (zh) 2016-05-24 2021-11-09 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
PT3554553T (pt) * 2016-12-19 2022-08-04 Sarepta Therapeutics Inc Conjugados oligoméricos de salto de exão para a distrofia muscular
BR112019012651A2 (pt) 2016-12-19 2020-01-28 Sarepta Therapeutics Inc conjugados de oligômero de salto de éxon para distrofia muscular
LT3554552T (lt) * 2016-12-19 2022-10-10 Sarepta Therapeutics, Inc. Egzoną praleidžiantys oligomerų konjugatai nuo raumenų distrofijos
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
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WO2020214763A1 (en) * 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
EP3987029A1 (en) * 2019-06-19 2022-04-27 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
CN118109468A (zh) 2019-12-26 2024-05-31 日本新药株式会社 诱导外显子50的跳读的反义核酸
AU2021226089A1 (en) 2020-02-28 2022-09-15 National Center Of Neurology And Psychiatry Antisense nucleic acid inducing skipping of exon 51
AU2021427595A1 (en) * 2021-02-12 2023-09-28 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
JP2024519451A (ja) 2021-04-30 2024-05-14 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーのための治療法
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