HRP20221155T1 - Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju - Google Patents
Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju Download PDFInfo
- Publication number
- HRP20221155T1 HRP20221155T1 HRP20221155TT HRP20221155T HRP20221155T1 HR P20221155 T1 HRP20221155 T1 HR P20221155T1 HR P20221155T T HRP20221155T T HR P20221155TT HR P20221155 T HRP20221155 T HR P20221155T HR P20221155 T1 HRP20221155 T1 HR P20221155T1
- Authority
- HR
- Croatia
- Prior art keywords
- antisense oligomer
- pharmaceutically acceptable
- oligomer conjugate
- acceptable salt
- conjugate
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title 1
- 230000000692 anti-sense effect Effects 0.000 claims 15
- 150000003839 salts Chemical class 0.000 claims 10
- 102000001039 Dystrophin Human genes 0.000 claims 7
- 108010069091 Dystrophin Proteins 0.000 claims 7
- 239000000825 pharmaceutical preparation Substances 0.000 claims 7
- 206010064571 Gene mutation Diseases 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 2
- 230000008094 contradictory effect Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000002560 therapeutic procedure Methods 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
- A61K47/68—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
- A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
- A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
-
- 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
- A61K47/54—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
- 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
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Genetics & Genomics (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- Immunology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (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 (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662436223P | 2016-12-19 | 2016-12-19 | |
| US201762443484P | 2017-01-06 | 2017-01-06 | |
| US201762479178P | 2017-03-30 | 2017-03-30 | |
| US201762562146P | 2017-09-22 | 2017-09-22 | |
| EP17826645.8A EP3554552B1 (en) | 2016-12-19 | 2017-12-14 | Exon skipping oligomer conjugates for muscular dystrophy |
| PCT/US2017/066509 WO2018118662A1 (en) | 2016-12-19 | 2017-12-14 | Exon skipping oligomer conjugates for muscular dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20221155T1 true HRP20221155T1 (hr) | 2022-12-09 |
Family
ID=60953960
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20221155TT HRP20221155T1 (hr) | 2016-12-19 | 2017-12-14 | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
Country Status (27)
| Country | Link |
|---|---|
| US (3) | US11000600B2 (enExample) |
| EP (2) | EP3554552B1 (enExample) |
| JP (3) | JP7160807B2 (enExample) |
| KR (2) | KR20240006057A (enExample) |
| CN (1) | CN110636866B (enExample) |
| AU (1) | AU2017382773B2 (enExample) |
| BR (1) | BR112019012664A2 (enExample) |
| CA (1) | CA3047010A1 (enExample) |
| CO (1) | CO2019007399A2 (enExample) |
| DK (1) | DK3554552T3 (enExample) |
| ES (1) | ES2928486T3 (enExample) |
| HR (1) | HRP20221155T1 (enExample) |
| HU (1) | HUE059905T2 (enExample) |
| IL (2) | IL297528A (enExample) |
| LT (1) | LT3554552T (enExample) |
| MD (1) | MD3554552T2 (enExample) |
| MX (1) | MX2019006879A (enExample) |
| MY (1) | MY205772A (enExample) |
| NZ (1) | NZ755438A (enExample) |
| PL (1) | PL3554552T3 (enExample) |
| PT (1) | PT3554552T (enExample) |
| RS (1) | RS63621B1 (enExample) |
| SA (1) | SA519402226B1 (enExample) |
| SG (1) | SG10202100557UA (enExample) |
| SM (1) | SMT202200403T1 (enExample) |
| TW (2) | TW202332470A (enExample) |
| WO (1) | WO2018118662A1 (enExample) |
Families Citing this family (21)
| 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 |
| MA45183A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| WO2017205496A1 (en) | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MD3554554T2 (ro) | 2016-12-19 | 2022-12-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
| BR112019012664A2 (pt) | 2016-12-19 | 2020-01-21 | Sarepta Therapeutics Inc | conjugados de oligômero de salto de éxon para distrofia muscular |
| EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| GB201821269D0 (en) | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| EP3987029A1 (en) * | 2019-06-19 | 2022-04-27 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
| US20230174979A1 (en) * | 2019-10-09 | 2023-06-08 | Children’S Medical Center Corporation | Methods and compositions for treating ataxia telangiectasia |
| KR20220122673A (ko) | 2019-12-26 | 2022-09-02 | 니뽄 신야쿠 가부시키가이샤 | 엑손 50의 스키핑을 유도하는 안티센스 핵산 |
| JPWO2021172498A1 (enExample) | 2020-02-28 | 2021-09-02 | ||
| CA3211038A1 (en) * | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| KR20240004609A (ko) | 2021-04-30 | 2024-01-11 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근이영양증에 대한 치료 방법 |
| MX2023014417A (es) | 2021-06-23 | 2023-12-15 | Nippon Shinyaku Co Ltd | Combinacion de oligomeros antisentido. |
| KR20240035502A (ko) | 2021-07-08 | 2024-03-15 | 니뽄 신야쿠 가부시키가이샤 | 신독성 경감제 |
| EP4368176A4 (en) | 2021-07-08 | 2025-06-25 | Nippon Shinyaku Co., Ltd. | Nephrotoxicity reducing agent |
| JPWO2023282346A1 (enExample) | 2021-07-08 | 2023-01-12 | ||
| CN120129745A (zh) | 2022-03-10 | 2025-06-10 | 日本新药株式会社 | 抗病毒反义低聚物 |
| EP4493693A1 (en) | 2022-03-17 | 2025-01-22 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
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