LT3554553T - Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos - Google Patents

Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos

Info

Publication number
LT3554553T
LT3554553T LTEPPCT/US2017/066222T LT17066222T LT3554553T LT 3554553 T LT3554553 T LT 3554553T LT 17066222 T LT17066222 T LT 17066222T LT 3554553 T LT3554553 T LT 3554553T
Authority
LT
Lithuania
Prior art keywords
muscular dystrophy
exon skipping
oligomer conjugates
skipping oligomer
conjugates
Prior art date
Application number
LTEPPCT/US2017/066222T
Other languages
English (en)
Inventor
Marco A. Passini
Gunnar J. Hanson
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 LT3554553T publication Critical patent/LT3554553T/lt

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)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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)
LTEPPCT/US2017/066222T 2016-12-19 2017-12-13 Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos LT3554553T (lt)

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
LT3554553T true LT3554553T (lt) 2022-08-25

Family

ID=61006315

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEPPCT/US2017/066222T LT3554553T (lt) 2016-12-19 2017-12-13 Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos

Country Status (28)

Country Link
US (4) US10888578B2 (lt)
EP (1) EP3554553B1 (lt)
JP (3) JP7118063B2 (lt)
KR (2) KR102810425B1 (lt)
CN (1) CN110290812B (lt)
AU (1) AU2017382723B2 (lt)
CA (1) CA3046793A1 (lt)
CO (1) CO2019007397A2 (lt)
CY (1) CY1125549T1 (lt)
DK (1) DK3554553T3 (lt)
ES (1) ES2927079T3 (lt)
HR (1) HRP20220943T1 (lt)
HU (1) HUE059843T2 (lt)
IL (2) IL267244B (lt)
LT (1) LT3554553T (lt)
MD (1) MD3554553T2 (lt)
MX (1) MX2019006882A (lt)
MY (1) MY195801A (lt)
NZ (1) NZ755416A (lt)
PL (1) PL3554553T3 (lt)
PT (1) PT3554553T (lt)
RS (1) RS63610B1 (lt)
SA (1) SA519402154B1 (lt)
SG (1) SG10202012839TA (lt)
SM (1) SMT202200366T1 (lt)
TW (1) TWI760402B (lt)
WO (1) WO2018118599A1 (lt)
ZA (1) ZA201904092B (lt)

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KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
MX384727B (es) 2016-05-24 2025-03-14 Sarepta Therapeutics Inc Procesos para preparar oligómeros
TWI737736B (zh) 2016-05-24 2021-09-01 美商薩羅塔治療公司 製備磷醯二胺嗎啉代寡聚物之方法
NZ755416A (en) * 2016-12-19 2023-05-26 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
PT4122497T (pt) 2016-12-19 2024-06-12 Sarepta Therapeutics Inc Conjugados oligoméricos de salto de exão para a distrofia muscular
ES3051834T3 (en) * 2016-12-19 2025-12-30 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
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
IL294271A (en) 2019-12-26 2022-08-01 Nippon Shinyaku Co Ltd Antisense nucleic acid that induces skipping of exon 50
PE20230237A1 (es) 2020-02-28 2023-02-07 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido que inducen la omision del exon 51
US20240327831A1 (en) * 2020-12-23 2024-10-03 Sarepta Therapeutics, Inc. Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy
CA3211038A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
JPWO2022270585A1 (lt) 2021-06-23 2022-12-29
TW202317147A (zh) 2021-07-08 2023-05-01 日商日本新藥股份有限公司 析出抑制劑
US20240316093A1 (en) 2021-07-08 2024-09-26 Nippon Shinyaku Co., Ltd. Nephrotoxicity reducing agent
TW202308664A (zh) 2021-07-08 2023-03-01 日商日本新藥股份有限公司 腎毒性減輕劑
JPWO2023171804A1 (lt) 2022-03-10 2023-09-14
EP4493693A1 (en) 2022-03-17 2025-01-22 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates

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