KR102810425B1 - 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 - Google Patents
근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 Download PDFInfo
- Publication number
- KR102810425B1 KR102810425B1 KR1020237022506A KR20237022506A KR102810425B1 KR 102810425 B1 KR102810425 B1 KR 102810425B1 KR 1020237022506 A KR1020237022506 A KR 1020237022506A KR 20237022506 A KR20237022506 A KR 20237022506A KR 102810425 B1 KR102810425 B1 KR 102810425B1
- Authority
- KR
- South Korea
- Prior art keywords
- antisense oligomer
- exon
- administered
- dystrophin
- present disclosure
- Prior art date
- Legal status (The legal status 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 status listed.)
- Active
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)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (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)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (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 (10)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662436182P | 2016-12-19 | 2016-12-19 | |
| US62/436,182 | 2016-12-19 | ||
| US201762443476P | 2017-01-06 | 2017-01-06 | |
| US62/443,476 | 2017-01-06 | ||
| US201762479173P | 2017-03-30 | 2017-03-30 | |
| US62/479,173 | 2017-03-30 | ||
| US201762562080P | 2017-09-22 | 2017-09-22 | |
| US62/562,080 | 2017-09-22 | ||
| PCT/US2017/066222 WO2018118599A1 (en) | 2016-12-19 | 2017-12-13 | Exon skipping oligomer conjugates for muscular dystrophy |
| KR1020197020418A KR102552428B1 (ko) | 2016-12-19 | 2017-12-13 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020197020418A Division KR102552428B1 (ko) | 2016-12-19 | 2017-12-13 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| KR20230110811A KR20230110811A (ko) | 2023-07-25 |
| KR102810425B1 true KR102810425B1 (ko) | 2025-05-21 |
Family
ID=61006315
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020237022506A Active KR102810425B1 (ko) | 2016-12-19 | 2017-12-13 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| KR1020197020418A Active KR102552428B1 (ko) | 2016-12-19 | 2017-12-13 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020197020418A Active KR102552428B1 (ko) | 2016-12-19 | 2017-12-13 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
Country Status (27)
| Country | Link |
|---|---|
| US (4) | US10888578B2 (enExample) |
| EP (1) | EP3554553B1 (enExample) |
| JP (3) | JP7118063B2 (enExample) |
| KR (2) | KR102810425B1 (enExample) |
| CN (1) | CN110290812B (enExample) |
| AU (1) | AU2017382723B2 (enExample) |
| CA (1) | CA3046793A1 (enExample) |
| CO (1) | CO2019007397A2 (enExample) |
| DK (1) | DK3554553T3 (enExample) |
| ES (1) | ES2927079T3 (enExample) |
| HR (1) | HRP20220943T1 (enExample) |
| HU (1) | HUE059843T2 (enExample) |
| IL (2) | IL267244B (enExample) |
| LT (1) | LT3554553T (enExample) |
| MD (1) | MD3554553T2 (enExample) |
| MX (1) | MX2019006882A (enExample) |
| MY (1) | MY195801A (enExample) |
| NZ (1) | NZ755416A (enExample) |
| PL (1) | PL3554553T3 (enExample) |
| PT (1) | PT3554553T (enExample) |
| RS (1) | RS63610B1 (enExample) |
| SA (1) | SA519402154B1 (enExample) |
| SG (1) | SG10202012839TA (enExample) |
| SM (1) | SMT202200366T1 (enExample) |
| TW (1) | TWI760402B (enExample) |
| WO (1) | WO2018118599A1 (enExample) |
| ZA (1) | ZA201904092B (enExample) |
Families Citing this family (22)
| 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 |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| BR112018074346B1 (pt) | 2016-05-24 | 2023-01-03 | Sarepta Therapeutics, Inc. | Composto oligomérico e processo para preparar um composto oligomérico |
| HUE066393T2 (hu) | 2016-05-24 | 2024-07-28 | Sarepta Therapeutics Inc | Eljárás foszfordiamidát morfolino oligomerek elõállítására |
| CN110337308B (zh) | 2016-12-19 | 2023-09-01 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 |
| SMT202200403T1 (it) * | 2016-12-19 | 2022-11-18 | Sarepta Therapeutics Inc | Coniugati di oligomeri per salto di esone per distrofia muscolare |
| MX2019006882A (es) * | 2016-12-19 | 2019-08-16 | Sarepta Therapeutics Inc | Conjugados de oligomeros de omision de exon para distrofia muscular. |
| MX2019008199A (es) | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| 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 |
| EP3955966A1 (en) * | 2019-04-18 | 2022-02-23 | 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 |
| EP4112083A4 (en) | 2020-02-28 | 2024-06-26 | Nippon Shinyaku Co., Ltd. | 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 |
| EP4330395A1 (en) | 2021-04-30 | 2024-03-06 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| MX2023014417A (es) | 2021-06-23 | 2023-12-15 | Nippon Shinyaku Co Ltd | Combinacion de oligomeros antisentido. |
| AU2022306542A1 (en) | 2021-07-08 | 2024-01-18 | Nippon Shinyaku Co., Ltd. | Precipitation suppressing 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 |
| JPWO2023282345A1 (enExample) | 2021-07-08 | 2023-01-12 | ||
| US20250188469A1 (en) | 2022-03-10 | 2025-06-12 | Nippon Shinyaku Co., Ltd. | Antiviral antisense oligomer |
| CA3252177A1 (en) | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics, Inc. | MORPHOLINO PHOSPHORODIAMIDATE OLIGOMER CONJUGATE |
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