FI4122497T3 - Eksonin ohittavia oligomeerikonjugaatteja lihasdystrofiaan - Google Patents
Eksonin ohittavia oligomeerikonjugaatteja lihasdystrofiaan Download PDFInfo
- Publication number
- FI4122497T3 FI4122497T3 FIEP22180767.0T FI22180767T FI4122497T3 FI 4122497 T3 FI4122497 T3 FI 4122497T3 FI 22180767 T FI22180767 T FI 22180767T FI 4122497 T3 FI4122497 T3 FI 4122497T3
- Authority
- FI
- Finland
- Prior art keywords
- pharmaceutically acceptable
- acceptable salt
- oligomer conjugate
- antisense oligomer
- break
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title 1
- 230000000692 anti-sense effect Effects 0.000 claims 11
- 150000003839 salts Chemical class 0.000 claims 11
- 108010069091 Dystrophin Proteins 0.000 claims 7
- 102000001039 Dystrophin Human genes 0.000 claims 6
- 241000288906 Primates Species 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 6
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 206010064571 Gene mutation Diseases 0.000 claims 3
- 239000003937 drug carrier Substances 0.000 claims 3
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 2
- 102100025250 C-X-C motif chemokine 14 Human genes 0.000 claims 1
- 101000858068 Homo sapiens C-X-C motif chemokine 14 Proteins 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
Classifications
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- 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
- A61K47/54—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 an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- 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
- 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
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/3513—Protein; Peptide
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (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)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662436199P | 2016-12-19 | 2016-12-19 | |
| US201762443481P | 2017-01-06 | 2017-01-06 | |
| US201762479177P | 2017-03-30 | 2017-03-30 | |
| US201762562119P | 2017-09-22 | 2017-09-22 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| FI4122497T3 true FI4122497T3 (fi) | 2024-06-04 |
Family
ID=61006319
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| FIEP22180767.0T FI4122497T3 (fi) | 2016-12-19 | 2017-12-14 | Eksonin ohittavia oligomeerikonjugaatteja lihasdystrofiaan |
Country Status (31)
| Country | Link |
|---|---|
| US (2) | US11382981B2 (enExample) |
| EP (3) | EP4406601A3 (enExample) |
| JP (4) | JP7125940B2 (enExample) |
| KR (2) | KR20240006023A (enExample) |
| CN (2) | CN117298290A (enExample) |
| AU (1) | AU2017382741B2 (enExample) |
| BR (1) | BR112019012651A2 (enExample) |
| CA (1) | CA3046801A1 (enExample) |
| CO (1) | CO2019007398A2 (enExample) |
| DK (2) | DK4122497T3 (enExample) |
| ES (2) | ES2980686T3 (enExample) |
| FI (1) | FI4122497T3 (enExample) |
| HR (2) | HRP20240705T1 (enExample) |
| HU (2) | HUE066576T2 (enExample) |
| IL (2) | IL304284B2 (enExample) |
| LT (2) | LT3554554T (enExample) |
| MA (1) | MA65895B1 (enExample) |
| MD (2) | MD3554554T2 (enExample) |
| MX (1) | MX2019006989A (enExample) |
| MY (1) | MY205600A (enExample) |
| NZ (1) | NZ755422A (enExample) |
| PL (2) | PL4122497T3 (enExample) |
| PT (2) | PT3554554T (enExample) |
| RS (2) | RS63705B1 (enExample) |
| SA (1) | SA519402143B1 (enExample) |
| SG (1) | SG10202100491QA (enExample) |
| SI (2) | SI4122497T1 (enExample) |
| SM (2) | SMT202200440T1 (enExample) |
| TW (1) | TWI780095B (enExample) |
| WO (1) | WO2018118627A1 (enExample) |
| ZA (1) | ZA201903940B (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| SG10202101830WA (en) | 2016-05-24 | 2021-04-29 | Sarepta Therapeutics Inc | Processes for preparing oligomers |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| IL304284B2 (en) | 2016-12-19 | 2025-03-01 | Sarepta Therapeutics Inc | Exon-skipping oligomer fusions for muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| GB201821269D0 (en) | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| CA3134165A1 (en) * | 2019-03-28 | 2020-10-01 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy with casimersen |
| US20220193246A1 (en) * | 2019-04-18 | 2022-06-23 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| EP3987029A1 (en) * | 2019-06-19 | 2022-04-27 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
| MY209476A (en) | 2019-12-26 | 2025-07-11 | Nippon Shinyaku Co Ltd | Antisense nucleic acid that induces skipping of exon 50 |
| KR20220145865A (ko) | 2020-02-28 | 2022-10-31 | 니뽄 신야쿠 가부시키가이샤 | 엑손 51의 스키핑을 유도하는 안티센스 핵산 |
| CA3187301A1 (en) * | 2020-06-17 | 2021-12-23 | Stealth Biotherapeutics Inc. | Methods and compositions for the treatment of muscular dystrophy |
| CA3211038A1 (en) * | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| CA3216839A1 (en) * | 2021-04-30 | 2022-11-03 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| TW202307208A (zh) | 2021-06-23 | 2023-02-16 | 日商日本新藥股份有限公司 | 反義寡聚物之組合 |
| KR20240035503A (ko) | 2021-07-08 | 2024-03-15 | 니뽄 신야쿠 가부시키가이샤 | 석출 억제제 |
| JPWO2023282344A1 (enExample) | 2021-07-08 | 2023-01-12 | ||
| IL310003A (en) | 2021-07-08 | 2024-03-01 | Nippon Shinyaku Co Ltd | Nephrotoxicity reducing agent |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| TW202346589A (zh) | 2022-03-10 | 2023-12-01 | 日商日本新藥股份有限公司 | 抗病毒反義寡聚物 |
| JP7523012B1 (ja) * | 2023-05-17 | 2024-07-26 | 東亞合成株式会社 | ペプチドフラグメント及びその利用 |
| WO2025030155A1 (en) * | 2023-08-03 | 2025-02-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
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