PE20230237A1 - Acidos nucleicos antisentido que inducen la omision del exon 51 - Google Patents
Acidos nucleicos antisentido que inducen la omision del exon 51Info
- Publication number
- PE20230237A1 PE20230237A1 PE2022001832A PE2022001832A PE20230237A1 PE 20230237 A1 PE20230237 A1 PE 20230237A1 PE 2022001832 A PE2022001832 A PE 2022001832A PE 2022001832 A PE2022001832 A PE 2022001832A PE 20230237 A1 PE20230237 A1 PE 20230237A1
- Authority
- PE
- Peru
- Prior art keywords
- exon
- skip
- nucleic acids
- antisense nucleic
- acids induce
- Prior art date
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Classifications
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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
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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
-
- 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
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- 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
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- 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
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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
- C12N2310/10—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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente memoria descriptiva proporciona un farmaco que provoca una omision altamente eficaz del exon 51 en el gen de la distrofina humana. La presente memoria descriptiva proporciona un oligomero antisentido que tiene una actividad para inducir la omision del exon 51 en el gen de la distrofina humana.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP2020033483 | 2020-02-28 | ||
PCT/JP2021/007286 WO2021172498A1 (ja) | 2020-02-28 | 2021-02-26 | エクソン51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 |
---|---|
PE20230237A1 true PE20230237A1 (es) | 2023-02-07 |
Family
ID=77491251
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2022001832A PE20230237A1 (es) | 2020-02-28 | 2021-02-26 | Acidos nucleicos antisentido que inducen la omision del exon 51 |
Country Status (17)
Country | Link |
---|---|
US (2) | US20230140736A1 (es) |
EP (1) | EP4112083A4 (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 (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN118488959A (zh) | 2021-12-27 | 2024-08-13 | 日本新药株式会社 | 低聚核酸化合物的制造方法 |
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 |
AU655164B2 (en) | 1989-12-20 | 1994-12-08 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorous-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 |
EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
ES2554660T3 (es) | 2002-11-25 | 2015-12-22 | Masafumi Matsuo | Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm |
WO2004083432A1 (en) | 2003-03-21 | 2004-09-30 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
EP3808845A1 (en) | 2004-06-28 | 2021-04-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
AU2007297861A1 (en) | 2006-05-10 | 2008-03-27 | Avi Biopharma, Inc. | Oligonucleotide analogs having cationic intersubunit linkages |
CA2691673A1 (en) | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
CA2704049A1 (en) | 2007-10-26 | 2009-04-30 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
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 |
US8871918B2 (en) * | 2008-10-24 | 2014-10-28 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for DMD |
ES2562658T3 (es) | 2008-10-27 | 2016-03-07 | Biomarin Technologies B.V. | Procedimientos y medios para el salto eficiente del exón 45 en el pre-ARNm de la distrofia muscular de Duchenne |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
IL273838B (en) | 2011-05-05 | 2022-09-01 | Sarepta Therapeutics Inc | Peptide and oligonucleotide conjugates, preparations containing them and their uses |
CN104203289B (zh) * | 2012-01-27 | 2020-11-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
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 |
SI3118311T1 (sl) | 2014-03-12 | 2019-05-31 | Nippon Shinyaku Co., Ltd. | Protismiselna nukleinska kislina |
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 |
IL295755A (en) | 2015-10-09 | 2022-10-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods thereof |
US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
EP3554553B1 (en) | 2016-12-19 | 2022-07-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
ES2980686T3 (es) | 2016-12-19 | 2024-10-02 | Sarepta Therapeutics Inc | Conjugados de oligómero de omisión de exones para distrofia muscular |
KR20240006057A (ko) | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
US20220251551A1 (en) | 2018-06-13 | 2022-08-11 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
-
2021
- 2021-02-26 AU AU2021226089A patent/AU2021226089A1/en active Pending
- 2021-02-26 PE PE2022001832A patent/PE20230237A1/es unknown
- 2021-02-26 MX MX2022010545A patent/MX2022010545A/es unknown
- 2021-02-26 EP EP21760603.7A patent/EP4112083A4/en active Pending
- 2021-02-26 KR KR1020227032708A patent/KR20220145865A/ko unknown
- 2021-02-26 CN CN202180017313.6A patent/CN115210376A/zh active Pending
- 2021-02-26 IL IL295967A patent/IL295967A/en unknown
- 2021-02-26 BR BR112022017066A patent/BR112022017066A2/pt unknown
- 2021-02-26 US US17/802,720 patent/US20230140736A1/en active Pending
- 2021-02-26 JP JP2022503739A patent/JPWO2021172498A1/ja active Pending
- 2021-02-26 TW TW110107021A patent/TW202200162A/zh unknown
- 2021-02-26 CA CA3173049A patent/CA3173049A1/en active Pending
- 2021-02-26 WO PCT/JP2021/007286 patent/WO2021172498A1/ja active Application Filing
-
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 CO CONC2022/0013685A patent/CO2022013685A2/es unknown
- 2022-09-23 EC ECSENADI202274446A patent/ECSP22074446A/es unknown
Also Published As
Publication number | Publication date |
---|---|
MX2022010545A (es) | 2022-09-21 |
JP2022180420A (ja) | 2022-12-06 |
IL295967A (en) | 2022-10-01 |
AU2021226089A1 (en) | 2022-09-15 |
WO2021172498A1 (ja) | 2021-09-02 |
TW202200162A (zh) | 2022-01-01 |
US20230140736A1 (en) | 2023-05-04 |
US20230097387A1 (en) | 2023-03-30 |
EP4112083A1 (en) | 2023-01-04 |
CN115210376A (zh) | 2022-10-18 |
CO2022013685A2 (es) | 2022-10-11 |
BR112022017066A2 (pt) | 2022-11-16 |
ZA202209294B (en) | 2024-04-24 |
CL2022002279A1 (es) | 2023-02-10 |
ECSP22074446A (es) | 2022-10-31 |
CA3173049A1 (en) | 2021-09-02 |
EP4112083A4 (en) | 2024-06-26 |
US11781140B2 (en) | 2023-10-10 |
JP7292636B2 (ja) | 2023-06-19 |
JPWO2021172498A1 (es) | 2021-09-02 |
KR20220145865A (ko) | 2022-10-31 |
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