MX2022007937A - Acido nucleico antisentido que induce la omision del exon 50. - Google Patents
Acido nucleico antisentido que induce la omision del exon 50.Info
- Publication number
- MX2022007937A MX2022007937A MX2022007937A MX2022007937A MX2022007937A MX 2022007937 A MX2022007937 A MX 2022007937A MX 2022007937 A MX2022007937 A MX 2022007937A MX 2022007937 A MX2022007937 A MX 2022007937A MX 2022007937 A MX2022007937 A MX 2022007937A
- Authority
- MX
- Mexico
- Prior art keywords
- exon
- nucleic acid
- antisense nucleic
- induces skipping
- skipping
- 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
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- 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
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- 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
-
- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/4965—Non-condensed pyrazines
-
- 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
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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
-
- 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
- C12N2310/314—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
- C12N2310/321—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
- 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)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
Abstract
La presente especificación proporciona un fármaco que provoca el salto altamente eficiente de exón 50 en el gen de distrofina humana. La presente especificación proporciona un oligómero antisentido que induce el salto de exón 50 en el gen de distrofina humana.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP2019236704 | 2019-12-26 | ||
PCT/JP2020/048803 WO2021132591A1 (ja) | 2019-12-26 | 2020-12-25 | エクソン50のスキッピングを誘導するアンチセンス核酸 |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2022007937A true MX2022007937A (es) | 2022-07-27 |
Family
ID=76574765
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022007937A MX2022007937A (es) | 2019-12-26 | 2020-12-25 | Acido nucleico antisentido que induce la omision del exon 50. |
Country Status (14)
Country | Link |
---|---|
US (2) | US20230073008A1 (es) |
EP (1) | EP4083208A4 (es) |
JP (2) | JPWO2021132591A1 (es) |
KR (1) | KR20220122673A (es) |
CN (1) | CN114901823A (es) |
AU (1) | AU2020411964A1 (es) |
BR (1) | BR112022012622A2 (es) |
CA (1) | CA3165961A1 (es) |
CO (1) | CO2022008664A2 (es) |
IL (1) | IL294271A (es) |
MX (1) | MX2022007937A (es) |
TW (1) | TW202138560A (es) |
WO (1) | WO2021132591A1 (es) |
ZA (1) | ZA202206667B (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
Family Cites Families (28)
Publication number | Priority date | Publication date | Assignee | Title |
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US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
EP0962463B1 (en) | 1989-12-20 | 2002-07-10 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorus-containing chiral intersubunit linkages |
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 |
IL115849A0 (en) | 1994-11-03 | 1996-01-31 | Merz & Co Gmbh & Co | Tangential filtration preparation of liposomal drugs and liposome product thereof |
EP2530156B1 (en) | 2002-11-25 | 2015-11-18 | Masafumi Matsuo | ENA nucleic acid drugs modifying splicing in mRNA precursor |
DK1766010T3 (da) | 2004-06-28 | 2011-06-06 | Univ Western Australia | Antisense-oligonukleotider til induktion af exon-skipping og fremgangsmåder til anvendelse deraf |
SI2735568T1 (en) | 2006-05-10 | 2018-01-31 | Sarepta Therapeutics, Inc. | Analogues of the oligonucleotide, with cationic links between subunits |
AU2008271050B2 (en) | 2007-06-29 | 2014-11-06 | Sarepta Therapeutics, Inc. | Tissue specific peptide conjugates and methods |
CN101861318A (zh) | 2007-11-15 | 2010-10-13 | Avi生物制药公司 | 合成吗啉代低聚物的方法 |
TR201902952T4 (tr) * | 2008-10-24 | 2019-03-21 | Sarepta Therapeutics Inc | Dmd için ekson atlama bileşimleri. |
ES2532634T5 (es) | 2008-10-27 | 2018-04-30 | Biomarin Technologies B.V. | Procedimientos y medios para el salto eficiente del exón 45 en el pre-ARNm de la distrofia muscular de Duchenne |
CN101388842B (zh) | 2008-10-30 | 2012-04-04 | 华为技术有限公司 | 一种存储方法和装置 |
KR102366851B1 (ko) | 2009-11-12 | 2022-02-23 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
CN103619356B (zh) | 2011-05-05 | 2017-09-12 | 萨勒普塔医疗公司 | 肽寡核苷酸缀合物 |
JP2013100190A (ja) | 2011-11-07 | 2013-05-23 | Fumihiro Mitamura | コンクリート組成物の製造方法 |
IN2014DN06220A (es) | 2011-12-28 | 2015-10-23 | Nippon Shinyaku Co Ltd | |
US20140329762A1 (en) * | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
KR20240035901A (ko) | 2015-05-19 | 2024-03-18 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 펩티드 올리고뉴클레오티드 콘주게이트 |
UA123995C2 (uk) | 2015-08-05 | 2021-07-07 | ЕЙСАЙ Ар ЕНД Ді МЕНЕДЖМЕНТ КО., ЛТД. | Хіральні реагенти для одержання гомогенних олігомерів |
WO2017062862A2 (en) | 2015-10-09 | 2017-04-13 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
EP3359668A4 (en) * | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF |
WO2018014042A1 (en) * | 2016-07-15 | 2018-01-18 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of dystrophin transcript |
IL267246B2 (en) | 2016-12-19 | 2023-03-01 | Sarepta Therapeutics Inc | Exon-skipping oligomer conjugates for muscular dystrophy |
NZ755416A (en) | 2016-12-19 | 2023-05-26 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
KR102639633B1 (ko) | 2016-12-19 | 2024-02-26 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
EP3894558A1 (en) | 2018-12-13 | 2021-10-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
-
2020
- 2020-12-25 CN CN202080090310.0A patent/CN114901823A/zh active Pending
- 2020-12-25 EP EP20906005.2A patent/EP4083208A4/en active Pending
- 2020-12-25 BR BR112022012622A patent/BR112022012622A2/pt unknown
- 2020-12-25 JP JP2021567687A patent/JPWO2021132591A1/ja active Pending
- 2020-12-25 TW TW109146242A patent/TW202138560A/zh unknown
- 2020-12-25 WO PCT/JP2020/048803 patent/WO2021132591A1/ja active Application Filing
- 2020-12-25 MX MX2022007937A patent/MX2022007937A/es unknown
- 2020-12-25 IL IL294271A patent/IL294271A/en unknown
- 2020-12-25 US US17/788,826 patent/US20230073008A1/en active Pending
- 2020-12-25 KR KR1020227024957A patent/KR20220122673A/ko active Search and Examination
- 2020-12-25 CA CA3165961A patent/CA3165961A1/en active Pending
- 2020-12-25 AU AU2020411964A patent/AU2020411964A1/en active Pending
-
2022
- 2022-06-15 ZA ZA2022/06667A patent/ZA202206667B/en unknown
- 2022-06-16 JP JP2022097020A patent/JP7201192B2/ja active Active
- 2022-06-22 CO CONC2022/0008664A patent/CO2022008664A2/es unknown
- 2022-06-23 US US17/847,333 patent/US11655472B2/en active Active
Also Published As
Publication number | Publication date |
---|---|
EP4083208A4 (en) | 2024-01-03 |
KR20220122673A (ko) | 2022-09-02 |
IL294271A (en) | 2022-08-01 |
EP4083208A1 (en) | 2022-11-02 |
BR112022012622A2 (pt) | 2022-09-06 |
JP7201192B2 (ja) | 2023-01-10 |
US11655472B2 (en) | 2023-05-23 |
CA3165961A1 (en) | 2021-07-01 |
TW202138560A (zh) | 2021-10-16 |
CN114901823A (zh) | 2022-08-12 |
ZA202206667B (en) | 2023-12-20 |
US20230073008A1 (en) | 2023-03-09 |
AU2020411964A1 (en) | 2022-06-16 |
CO2022008664A2 (es) | 2022-07-19 |
US20220333112A1 (en) | 2022-10-20 |
WO2021132591A1 (ja) | 2021-07-01 |
JPWO2021132591A1 (es) | 2021-07-01 |
JP2022133312A (ja) | 2022-09-13 |
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