SG11202103938UA - Bispecific antisense oligonucleotides for dystrophin exon skipping - Google Patents
Bispecific antisense oligonucleotides for dystrophin exon skippingInfo
- Publication number
- SG11202103938UA SG11202103938UA SG11202103938UA SG11202103938UA SG11202103938UA SG 11202103938U A SG11202103938U A SG 11202103938UA SG 11202103938U A SG11202103938U A SG 11202103938UA SG 11202103938U A SG11202103938U A SG 11202103938UA SG 11202103938U A SG11202103938U A SG 11202103938UA
- Authority
- SG
- Singapore
- Prior art keywords
- antisense oligonucleotides
- exon skipping
- dystrophin exon
- bispecific antisense
- bispecific
- Prior art date
Links
Classifications
-
- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- 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
- C12N2310/315—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
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- 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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18204170 | 2018-11-02 | ||
| PCT/EP2019/079714 WO2020089325A1 (en) | 2018-11-02 | 2019-10-30 | Bispecific antisense oligonucleotides for dystrophin exon skipping |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11202103938UA true SG11202103938UA (en) | 2021-05-28 |
Family
ID=64172289
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11202103938UA SG11202103938UA (en) | 2018-11-02 | 2019-10-30 | Bispecific antisense oligonucleotides for dystrophin exon skipping |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220025368A1 (en) |
| EP (1) | EP3874044A1 (en) |
| JP (1) | JP2022506219A (en) |
| KR (1) | KR20210091180A (en) |
| CN (1) | CN113286887A (en) |
| AU (1) | AU2019370937A1 (en) |
| BR (1) | BR112021008069A2 (en) |
| CA (1) | CA3118167A1 (en) |
| IL (1) | IL282818A (en) |
| MX (1) | MX2021004822A (en) |
| SG (1) | SG11202103938UA (en) |
| WO (1) | WO2020089325A1 (en) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20240004609A (en) | 2021-04-30 | 2024-01-11 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Treatment Methods for Muscular Dystrophy |
| CN114540461B (en) * | 2022-01-26 | 2024-09-06 | 深圳市儿童医院 | Kit for detecting Du's muscular dystrophy by PLGA microsphere-CRISPR (hybrid-enhanced fluorescence surface plasmon resonance) immunoprecipitation method |
| KR20240155352A (en) | 2022-03-17 | 2024-10-28 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Phosphorodiamidate morpholino oligomer conjugates |
| WO2023215781A1 (en) * | 2022-05-05 | 2023-11-09 | Biomarin Pharmaceutical Inc. | Method of treating duchenne muscular dystrophy |
Family Cites Families (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6683173B2 (en) | 1998-04-03 | 2004-01-27 | Epoch Biosciences, Inc. | Tm leveling methods |
| EP2305813A3 (en) * | 2002-11-14 | 2012-03-28 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| US7807816B2 (en) * | 2004-06-28 | 2010-10-05 | University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1857548A1 (en) * | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| DK3133160T3 (en) * | 2008-10-24 | 2019-04-01 | Sarepta Therapeutics Inc | EXON SKIP COMPOSITIONS FOR DMD |
| CN103003430A (en) * | 2009-11-12 | 2013-03-27 | 西澳大利亚大学 | Antisense molecules and methods for treating pathologies |
| WO2011097641A1 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
| JP2014513946A (en) * | 2011-04-22 | 2014-06-19 | プロセンサ テクノロジーズ ビー．ブイ． | Novel compounds for the treatment, delay and / or prevention of human genetic diseases such as myotonic dystrophy type 1 (DM1) |
| CA2848753C (en) | 2011-09-14 | 2022-07-26 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| EP3812370A1 (en) | 2012-07-13 | 2021-04-28 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| US8859754B2 (en) | 2012-07-31 | 2014-10-14 | Ased, Llc | Synthesis of deuterated ribo nucleosides, N-protected phosphoramidites, and oligonucleotides |
| JP2015529469A (en) | 2012-09-14 | 2015-10-08 | ラナ セラピューティクス インコーポレイテッド | Multimeric oligonucleotide compounds |
| WO2014093924A1 (en) | 2012-12-13 | 2014-06-19 | Moderna Therapeutics, Inc. | Modified nucleic acid molecules and uses thereof |
| JPWO2014112463A1 (en) | 2013-01-15 | 2017-01-19 | 国立大学法人大阪大学 | Nucleosides and nucleotides having a sulfonamide structure |
| EP2957567B1 (en) | 2013-02-18 | 2019-06-05 | Shionogi & Co., Ltd. | Nucleoside and nucleotide, having nitrogen-containing hetercycle structure |
| DK2970968T3 (en) | 2013-03-15 | 2018-03-05 | Miragen Therapeutics Inc | CONNECTED BICYCLIC Nucleosides |
| RU2730681C2 (en) * | 2014-03-12 | 2020-08-24 | Ниппон Синяку Ко., Лтд. | Antisense nucleic acids |
| WO2015142910A1 (en) | 2014-03-17 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom |
| BR112016029369B1 (en) * | 2014-06-17 | 2020-12-08 | Nippon Shinyaku Co., Ltd. | antisense oligomer, pharmaceutical composition use of an antisense oligomer or a pharmaceutically acceptable salt or hydrate thereof, and methods for making and screening an antisense oligomer |
| JP6562517B2 (en) | 2014-07-31 | 2019-08-21 | 国立大学法人大阪大学 | Bridged nucleosides and nucleotides |
| EP3207135A2 (en) | 2014-10-17 | 2017-08-23 | Celgene Alpine Investment Company II, LLC | Isotopologues of smad7 antisense oligonucleotides |
| CN108699555A (en) * | 2015-10-09 | 2018-10-23 | 萨勒普塔医疗公司 | Composition for treating Duchenne's dystrophy and associated disease and method |
| RU2769249C2 (en) * | 2016-07-05 | 2022-03-29 | Биомарин Текноложис Б.В. | Oligonucleotides switching or modulating pre-rna splicing and containing bicyclic framework fragments with improved characteristics for treating genetic diseases |
| EP3485015A4 (en) * | 2016-07-15 | 2020-07-29 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of dystrophin transcript |
-
2019
- 2019-10-30 SG SG11202103938UA patent/SG11202103938UA/en unknown
- 2019-10-30 AU AU2019370937A patent/AU2019370937A1/en active Pending
- 2019-10-30 CN CN201980078359.1A patent/CN113286887A/en active Pending
- 2019-10-30 WO PCT/EP2019/079714 patent/WO2020089325A1/en unknown
- 2019-10-30 CA CA3118167A patent/CA3118167A1/en active Pending
- 2019-10-30 JP JP2021523431A patent/JP2022506219A/en active Pending
- 2019-10-30 MX MX2021004822A patent/MX2021004822A/en unknown
- 2019-10-30 KR KR1020217015633A patent/KR20210091180A/en active Search and Examination
- 2019-10-30 BR BR112021008069A patent/BR112021008069A2/en unknown
- 2019-10-30 US US17/309,140 patent/US20220025368A1/en active Pending
- 2019-10-30 EP EP19791293.4A patent/EP3874044A1/en active Pending
-
2021
- 2021-04-29 IL IL282818A patent/IL282818A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20210091180A (en) | 2021-07-21 |
| MX2021004822A (en) | 2021-07-06 |
| JP2022506219A (en) | 2022-01-17 |
| AU2019370937A1 (en) | 2021-05-27 |
| CN113286887A (en) | 2021-08-20 |
| US20220025368A1 (en) | 2022-01-27 |
| BR112021008069A2 (en) | 2021-11-03 |
| CA3118167A1 (en) | 2020-05-07 |
| WO2020089325A1 (en) | 2020-05-07 |
| EP3874044A1 (en) | 2021-09-08 |
| IL282818A (en) | 2021-06-30 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL282818A (en) | Bispecific antisense oligonucleotides for dystrophin exon skipping | |
| IL284972A (en) | Antisense oligonucleotides for nucleic acid editing | |
| GB201904709D0 (en) | Chemically modified oligonucleotides | |
| HK1255100A1 (en) | Effective gene therapy tools for dystrophin exon 53 skipping | |
| LT3277814T (en) | Oligonucleotide compounds for targeting huntingtin mrna | |
| SG11202100682YA (en) | Method for treating epilepsy | |
| IL254173B (en) | Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy | |
| IL289968A (en) | Synthetic genetic elements for biomanufacture | |
| SG10201913477UA (en) | G-quadruplex-containing antisense oligonucleotides | |
| EP3497114A4 (en) | Androgen receptor antisense oligonucleotides | |
| GB2585889B (en) | Harnesses | |
| SG10201609048RA (en) | Antisense oligonucleotides | |
| IL309445A (en) | Pikfyve antisense oligonucleotides | |
| GB201909538D0 (en) | Deposition apparatus | |
| CA185591S (en) | Drop planter | |
| EP4079329A4 (en) | Antisense nucleic acid enabling exon skipping | |
| EP3638253A4 (en) | Methods for treating multiple sclerosis using antisense oligonucleotides | |
| SG10201607303YA (en) | Antisense oligonucleotides to induce exon skipping | |
| IL280299A (en) | Exon skipping oligomers for muscular dystrophy | |
| SG11202012829RA (en) | Acetyl-coa carboxylase2 antisense oligonucleotides | |
| GB201714403D0 (en) | Process for oligonucleotide synthesis | |
| GB201908441D0 (en) | Antisense oligonucleotides for immunotherapy | |
| GB201904647D0 (en) | Antisense oligonucleotides for immunotherapy | |
| GB201814450D0 (en) | Antisense oligonucleotides | |
| GB201815781D0 (en) | Antisense oligonucleotides for RNA editing |