IL309626A - A combination of antisense oligomers - Google Patents
A combination of antisense oligomersInfo
- Publication number
- IL309626A IL309626A IL309626A IL30962623A IL309626A IL 309626 A IL309626 A IL 309626A IL 309626 A IL309626 A IL 309626A IL 30962623 A IL30962623 A IL 30962623A IL 309626 A IL309626 A IL 309626A
- Authority
- IL
- Israel
- Prior art keywords
- base sequence
- seq
- oligomer comprises
- unit
- antisense
- Prior art date
Links
Classifications
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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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
-
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
-
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
-
- 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
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/50—Physical structure
- C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
-
- 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
- C12N2320/31—Combination therapy
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (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)
- External Artificial Organs (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021104145 | 2021-06-23 | ||
| PCT/JP2022/025120 WO2022270585A1 (ja) | 2021-06-23 | 2022-06-23 | アンチセンスオリゴマーの組み合わせ |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL309626A true IL309626A (en) | 2024-02-01 |
Family
ID=84544436
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL309626A IL309626A (en) | 2021-06-23 | 2022-06-23 | A combination of antisense oligomers |
Country Status (13)
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12370264B1 (en) | 2018-08-02 | 2025-07-29 | Dyne Therapeutics, Inc. | Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject |
| WO2020028832A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| EP3830259A4 (en) | 2018-08-02 | 2022-05-04 | Dyne Therapeutics, Inc. | MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF 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 |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US20230111212A1 (en) * | 2020-01-10 | 2023-04-13 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2021154477A1 (en) | 2020-01-31 | 2021-08-05 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (tfr) antibody and uses thereof |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of myotonic dystrophy |
| WO2025076006A2 (en) * | 2023-10-02 | 2025-04-10 | Avidity Biosciences, Inc. | Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 45 skipping |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE312834T1 (de) | 1989-12-20 | 2005-12-15 | Avi Biopharma Inc | Ungeladene, auf morpholin basierende polymere mit chiralen, phosphor enthaltenden brücken zwischen den untereinheiten |
| JP2924179B2 (ja) | 1993-02-19 | 1999-07-26 | 日本新薬株式会社 | グリセロール誘導体，デバイス及び医薬組成物 |
| JP2005535318A (ja) * | 2002-08-12 | 2005-11-24 | ユニヴェルシテ ドゥ シェルブルック | プレメッセンジャーｒｎａのスプライス部位選択の再プログラミング方法 |
| EP2530155B1 (en) | 2002-11-25 | 2016-03-16 | Masafumi Matsuo | ENA nucleic acid drugs modifying splicing in mRNA precursor |
| AU2003225410A1 (en) * | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| ES2627917T3 (es) | 2004-06-28 | 2017-08-01 | The University Of Western Australia | Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos |
| LT2735568T (lt) | 2006-05-10 | 2017-11-27 | Sarepta Therapeutics, Inc. | Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų |
| EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| CA2691673A1 (en) | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
| MX2010004955A (es) | 2007-11-15 | 2010-06-30 | Avi Biopharma Inc | Metodo de sintesis de oligomeros de morfolina. |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| EP3133160B1 (en) | 2008-10-24 | 2018-12-12 | Sarepta Therapeutics, Inc. | Exon skipping compositions for dmd |
| JP5858285B2 (ja) | 2009-07-29 | 2016-02-10 | 東亞合成株式会社 | キャリアペプチドフラグメント及びその利用 |
| HUE040445T2 (hu) | 2009-11-12 | 2019-03-28 | Univ Western Australia | Antiszensz molekulák és eljárások betegségek kezelésére |
| AU2011367230B2 (en) | 2011-05-05 | 2017-08-10 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| CN108588073A (zh) | 2011-12-28 | 2018-09-28 | 日本新药株式会社 | 反义核酸 |
| CN104603271B (zh) | 2012-07-03 | 2019-07-19 | 马林生物科技有限公司 | 用于治疗肌肉萎缩症患者的寡核苷酸 |
| KR102644053B1 (ko) | 2015-05-19 | 2024-03-06 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 펩티드 올리고뉴클레오티드 콘주게이트 |
| HUE050061T2 (hu) * | 2015-09-15 | 2020-12-28 | Nippon Shinyaku Co Ltd | Antiszensz nukleinsav |
| KR20190024977A (ko) * | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
| 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 |
| JP2021521140A (ja) | 2018-04-12 | 2021-08-26 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド組成物及びその使用方法 |
| WO2020028832A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2020219820A1 (en) * | 2019-04-25 | 2020-10-29 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| CN115427448A (zh) | 2020-01-10 | 2022-12-02 | 达因疗法公司 | 肌肉靶向复合物及其用途 |
| US20230111212A1 (en) | 2020-01-10 | 2023-04-13 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| AU2021313058A1 (en) | 2020-07-23 | 2023-03-30 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof |
| MX2023000984A (es) | 2020-07-23 | 2023-05-03 | Dyne Therapeutics Inc | Complejos dirigidos al musculo y usos de los mismos para el tratamiento de distrofinopatias. |
| JP7041853B1 (ja) | 2020-09-14 | 2022-03-25 | 東亞合成株式会社 | キャリアペプチドフラグメントおよびその利用 |
-
2022
- 2022-06-23 JP JP2023530119A patent/JPWO2022270585A1/ja active Pending
- 2022-06-23 AU AU2022298028A patent/AU2022298028A1/en active Pending
- 2022-06-23 EP EP22828501.1A patent/EP4361269A4/en active Pending
- 2022-06-23 KR KR1020247000786A patent/KR20240024176A/ko active Pending
- 2022-06-23 TW TW111123470A patent/TW202307208A/zh unknown
- 2022-06-23 IL IL309626A patent/IL309626A/en unknown
- 2022-06-23 US US18/570,778 patent/US20240301416A1/en active Pending
- 2022-06-23 CN CN202280044513.5A patent/CN117940564A/zh active Pending
- 2022-06-23 BR BR112023027298A patent/BR112023027298A2/pt unknown
- 2022-06-23 CA CA3224782A patent/CA3224782A1/en active Pending
- 2022-06-23 WO PCT/JP2022/025120 patent/WO2022270585A1/ja not_active Ceased
- 2022-06-23 MX MX2023014417A patent/MX2023014417A/es unknown
-
2023
- 2023-12-04 CO CONC2023/0016805A patent/CO2023016805A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| JPWO2022270585A1 (cg-RX-API-DMAC7.html) | 2022-12-29 |
| WO2022270585A1 (ja) | 2022-12-29 |
| EP4361269A4 (en) | 2026-01-14 |
| CO2023016805A2 (es) | 2023-12-29 |
| CN117940564A (zh) | 2024-04-26 |
| MX2023014417A (es) | 2023-12-15 |
| BR112023027298A2 (pt) | 2024-03-12 |
| TW202307208A (zh) | 2023-02-16 |
| AU2022298028A1 (en) | 2023-12-21 |
| CA3224782A1 (en) | 2022-12-29 |
| KR20240024176A (ko) | 2024-02-23 |
| US20240301416A1 (en) | 2024-09-12 |
| EP4361269A1 (en) | 2024-05-01 |
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