CL2022002279A1 - Ácidos nucleicos antisentido que inducen la omisión del exón 51 - Google Patents
Ácidos nucleicos antisentido que inducen la omisión del exón 51Info
- Publication number
- CL2022002279A1 CL2022002279A1 CL2022002279A CL2022002279A CL2022002279A1 CL 2022002279 A1 CL2022002279 A1 CL 2022002279A1 CL 2022002279 A CL2022002279 A CL 2022002279A CL 2022002279 A CL2022002279 A CL 2022002279A CL 2022002279 A1 CL2022002279 A1 CL 2022002279A1
- Authority
- CL
- Chile
- Prior art keywords
- exon
- nucleic acids
- antisense nucleic
- inducing skipping
- skipping
- Prior art date
Links
Classifications
-
- 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
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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
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- 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
- C12N2310/32—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
- C12N2320/33—Alteration of splicing
Abstract
La presente memoria descriptiva proporciona un fármaco que provoca una omisión altamente eficaz del exón 51 en el gen de la distrofina humana. La presente memoria descriptiva proporciona un oligómero antisentido que tiene una actividad para inducir la omisión del exón 51 en el gen de la distrofina humana.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP2020033483 | 2020-02-28 |
Publications (1)
Publication Number | Publication Date |
---|---|
CL2022002279A1 true CL2022002279A1 (es) | 2023-02-10 |
Family
ID=77491251
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2022002279A CL2022002279A1 (es) | 2020-02-28 | 2022-08-22 | Ácidos nucleicos antisentido que inducen la omisión del exón 51 |
Country Status (17)
Country | Link |
---|---|
US (2) | US20230140736A1 (es) |
EP (1) | EP4112083A1 (es) |
JP (2) | JPWO2021172498A1 (es) |
KR (1) | KR20220145865A (es) |
CN (1) | CN115210376A (es) |
AU (1) | AU2021226089A1 (es) |
BR (1) | BR112022017066A2 (es) |
CA (1) | CA3173049A1 (es) |
CL (1) | CL2022002279A1 (es) |
CO (1) | CO2022013685A2 (es) |
EC (1) | ECSP22074446A (es) |
IL (1) | IL295967A (es) |
MX (1) | MX2022010545A (es) |
PE (1) | PE20230237A1 (es) |
TW (1) | TW202200162A (es) |
WO (1) | WO2021172498A1 (es) |
ZA (1) | ZA202209294B (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 |
---|---|---|---|---|
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 |
JP3398378B2 (ja) | 1989-12-20 | 2003-04-21 | アンチビラルス・インコーポレイテツド | リン含有キラルインターサブユニットリンケージを有する非電荷モルホリノ―基体ポリマー |
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 |
EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
CA2796924C (en) | 2002-11-25 | 2016-12-13 | Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy | Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors |
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 |
US7807816B2 (en) | 2004-06-28 | 2010-10-05 | University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
DK2024499T3 (da) | 2006-05-10 | 2018-01-29 | Sarepta Therapeutics Inc | Oligonukleotidanaloger med kationiske intersubunit-koblinger |
ES2694726T3 (es) | 2007-06-29 | 2018-12-26 | Sarepta Therapeutics, Inc. | Conjugados peptídicos específicos de tejido y métodos |
AU2008317566B2 (en) | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
CA2884340C (en) | 2007-11-15 | 2017-07-25 | Sarepta Therapeutics, Inc. | Method of synthesis of morpholino oligomers |
EP2350281B1 (en) * | 2008-10-24 | 2014-05-14 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
CA2741629C (en) | 2008-10-27 | 2022-07-05 | Academisch Ziekenhuis Leiden | Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
KR102183273B1 (ko) | 2011-05-05 | 2020-11-27 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 펩타이드 올리고뉴클레오타이드 접합체 |
BR112014018427B1 (pt) * | 2012-01-27 | 2021-11-03 | Biomarin Technologies B.V. | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker |
JP6519842B2 (ja) | 2013-10-04 | 2019-05-29 | 国立大学法人神戸大学 | 福山型筋ジストロフィー治療用アンチセンス核酸 |
CN110951732A (zh) | 2014-03-12 | 2020-04-03 | 日本新药株式会社 | 反义核酸 |
KR20240035901A (ko) | 2015-05-19 | 2024-03-18 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 펩티드 올리고뉴클레오티드 콘주게이트 |
CN108350005B (zh) | 2015-08-05 | 2024-02-06 | 卫材R&D管理有限公司 | 用于制备均一低聚物的手性试剂 |
TW201722439A (zh) | 2015-10-09 | 2017-07-01 | 波濤生命科學有限公司 | 寡核苷酸組合物及其方法 |
US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
CN110636866A (zh) | 2016-12-19 | 2019-12-31 | 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 |
SG10202100491QA (en) | 2016-12-19 | 2021-02-25 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
HUE059843T2 (hu) | 2016-12-19 | 2023-01-28 | Sarepta Therapeutics Inc | Exonátugró oligomerkonjugátumok izomdisztrófiára |
WO2019241385A2 (en) | 2018-06-13 | 2019-12-19 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystropy |
-
2021
- 2021-02-26 JP JP2022503739A patent/JPWO2021172498A1/ja active Pending
- 2021-02-26 IL IL295967A patent/IL295967A/en unknown
- 2021-02-26 PE PE2022001832A patent/PE20230237A1/es unknown
- 2021-02-26 KR KR1020227032708A patent/KR20220145865A/ko unknown
- 2021-02-26 WO PCT/JP2021/007286 patent/WO2021172498A1/ja active Application Filing
- 2021-02-26 MX MX2022010545A patent/MX2022010545A/es unknown
- 2021-02-26 US US17/802,720 patent/US20230140736A1/en active Pending
- 2021-02-26 AU AU2021226089A patent/AU2021226089A1/en active Pending
- 2021-02-26 TW TW110107021A patent/TW202200162A/zh unknown
- 2021-02-26 EP EP21760603.7A patent/EP4112083A1/en active Pending
- 2021-02-26 CA CA3173049A patent/CA3173049A1/en active Pending
- 2021-02-26 BR BR112022017066A patent/BR112022017066A2/pt unknown
- 2021-02-26 CN CN202180017313.6A patent/CN115210376A/zh active Pending
-
2022
- 2022-08-18 ZA ZA2022/09294A patent/ZA202209294B/en unknown
- 2022-08-22 CL CL2022002279A patent/CL2022002279A1/es unknown
- 2022-08-25 US US17/895,186 patent/US11781140B2/en active Active
- 2022-09-06 JP JP2022141656A patent/JP7292636B2/ja active Active
- 2022-09-23 EC ECSENADI202274446A patent/ECSP22074446A/es unknown
- 2022-09-23 CO CONC2022/0013685A patent/CO2022013685A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
US20230097387A1 (en) | 2023-03-30 |
CN115210376A (zh) | 2022-10-18 |
EP4112083A1 (en) | 2023-01-04 |
JP7292636B2 (ja) | 2023-06-19 |
IL295967A (en) | 2022-10-01 |
BR112022017066A2 (pt) | 2022-11-16 |
AU2021226089A1 (en) | 2022-09-15 |
PE20230237A1 (es) | 2023-02-07 |
MX2022010545A (es) | 2022-09-21 |
JP2022180420A (ja) | 2022-12-06 |
KR20220145865A (ko) | 2022-10-31 |
TW202200162A (zh) | 2022-01-01 |
US20230140736A1 (en) | 2023-05-04 |
CA3173049A1 (en) | 2021-09-02 |
JPWO2021172498A1 (es) | 2021-09-02 |
ECSP22074446A (es) | 2022-10-31 |
CO2022013685A2 (es) | 2022-10-11 |
ZA202209294B (en) | 2024-04-24 |
US11781140B2 (en) | 2023-10-10 |
WO2021172498A1 (ja) | 2021-09-02 |
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