MX2020003227A - Conjugados de oligomero con omision de exon para la distrofia muscular. - Google Patents
Conjugados de oligomero con omision de exon para la distrofia muscular.Info
- Publication number
- MX2020003227A MX2020003227A MX2020003227A MX2020003227A MX2020003227A MX 2020003227 A MX2020003227 A MX 2020003227A MX 2020003227 A MX2020003227 A MX 2020003227A MX 2020003227 A MX2020003227 A MX 2020003227A MX 2020003227 A MX2020003227 A MX 2020003227A
- Authority
- MX
- Mexico
- Prior art keywords
- oligomer conjugates
- muscular dystrophy
- exon skipping
- skipping oligomer
- conjugates
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 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
- A61K47/62—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
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- 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
- 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
-
- 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
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- 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
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- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
-
- 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
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- 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
-
- 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
- C12N2320/33—Alteration of splicing
-
- 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)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762562146P | 2017-09-22 | 2017-09-22 | |
| PCT/US2017/066509 WO2018118662A1 (en) | 2016-12-19 | 2017-12-14 | Exon skipping oligomer conjugates for muscular dystrophy |
| PCT/US2018/035660 WO2019059973A1 (en) | 2017-09-22 | 2018-06-01 | OLIGOMERIC CONJUGATES FOR THE EXON JUMP FOR MUSCLE DYSTROPHY |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2020003227A true MX2020003227A (es) | 2020-09-28 |
Family
ID=65810491
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020003227A MX2020003227A (es) | 2017-09-22 | 2018-06-01 | Conjugados de oligomero con omision de exon para la distrofia muscular. |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20200377886A1 (enExample) |
| JP (1) | JP2020537501A (enExample) |
| KR (1) | KR20200057029A (enExample) |
| AU (1) | AU2018336569A1 (enExample) |
| CA (1) | CA3075964A1 (enExample) |
| EA (2) | EA201991450A1 (enExample) |
| IL (1) | IL273289A (enExample) |
| MX (1) | MX2020003227A (enExample) |
| WO (1) | WO2019059973A1 (enExample) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017205496A1 (en) | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
| ES2928486T3 (es) | 2016-12-19 | 2022-11-18 | Sarepta Therapeutics Inc | Conjugados de oligómeros con omisión de exón para la distrofia muscular |
| HRP20221316T1 (hr) | 2016-12-19 | 2022-12-23 | Sarepta Therapeutics, Inc. | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
| WO2020028864A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| JP7611825B2 (ja) | 2018-08-02 | 2025-01-10 | ダイン セラピューティクス，インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体およびそれらの使用 |
| 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 |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
| JP7687682B2 (ja) * | 2019-04-08 | 2025-06-03 | 国立大学法人東京科学大学 | 筋疾患治療用医薬組成物 |
| WO2020214763A1 (en) * | 2019-04-18 | 2020-10-22 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| WO2020257489A1 (en) * | 2019-06-19 | 2020-12-24 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
| WO2021077000A1 (en) | 2019-10-16 | 2021-04-22 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
| US12378267B2 (en) | 2021-06-17 | 2025-08-05 | Entrada Therapeutics, Inc. | Synthesis of FMOC-protected morpholino monomers and oligomers |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| CA3226366A1 (en) | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations 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 |
| KR20240168981A (ko) * | 2022-03-02 | 2024-12-02 | 웨이브 라이프 사이언시스 리미티드 | 엑손 스키핑을 위한 올리고뉴클레오티드 조성물 및 이의 방법 |
| US20250289851A1 (en) | 2022-04-22 | 2025-09-18 | Entrada Therapeutics, Inc. | Cyclic peptides for delivering therapeutics |
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-
2017
- 2017-12-14 EA EA201991450A patent/EA201991450A1/ru unknown
-
2018
- 2018-06-01 JP JP2020515158A patent/JP2020537501A/ja active Pending
- 2018-06-01 US US16/647,728 patent/US20200377886A1/en not_active Abandoned
- 2018-06-01 CA CA3075964A patent/CA3075964A1/en active Pending
- 2018-06-01 WO PCT/US2018/035660 patent/WO2019059973A1/en not_active Ceased
- 2018-06-01 KR KR1020207010926A patent/KR20200057029A/ko not_active Ceased
- 2018-06-01 EA EA202090744A patent/EA202090744A1/ru unknown
- 2018-06-01 AU AU2018336569A patent/AU2018336569A1/en not_active Abandoned
- 2018-06-01 MX MX2020003227A patent/MX2020003227A/es unknown
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2020
- 2020-03-15 IL IL273289A patent/IL273289A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2018336569A1 (en) | 2020-05-07 |
| EA202090744A1 (ru) | 2020-07-13 |
| IL273289A (en) | 2020-04-30 |
| WO2019059973A1 (en) | 2019-03-28 |
| JP2020537501A (ja) | 2020-12-24 |
| KR20200057029A (ko) | 2020-05-25 |
| US20200377886A1 (en) | 2020-12-03 |
| CA3075964A1 (en) | 2019-03-28 |
| EA201991450A1 (ru) | 2019-12-30 |
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