CL2023000662A1 - Agentes de rnai para inhibir la expresión de dux4, composiciones de los mismos y métodos de uso. - Google Patents
Agentes de rnai para inhibir la expresión de dux4, composiciones de los mismos y métodos de uso.Info
- Publication number
- CL2023000662A1 CL2023000662A1 CL2023000662A CL2023000662A CL2023000662A1 CL 2023000662 A1 CL2023000662 A1 CL 2023000662A1 CL 2023000662 A CL2023000662 A CL 2023000662A CL 2023000662 A CL2023000662 A CL 2023000662A CL 2023000662 A1 CL2023000662 A1 CL 2023000662A1
- Authority
- CL
- Chile
- Prior art keywords
- compositions
- methods
- rnai agents
- dux4 expression
- inhibiting dux4
- Prior art date
Links
- 108091030071 RNAI Proteins 0.000 title 1
- 230000009368 gene silencing by RNA Effects 0.000 title 1
- 230000002401 inhibitory effect Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
- A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
-
- 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
- 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.
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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
- C12N2310/312—Phosphonates
-
- 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
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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
- 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
-
- 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
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- 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
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063077272P | 2020-09-11 | 2020-09-11 | |
| US202163214742P | 2021-06-24 | 2021-06-24 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2023000662A1 true CL2023000662A1 (es) | 2024-01-05 |
Family
ID=78078423
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2023000662A CL2023000662A1 (es) | 2020-09-11 | 2023-03-08 | Agentes de rnai para inhibir la expresión de dux4, composiciones de los mismos y métodos de uso. |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20230416737A1 (es) |
| EP (1) | EP4211243A2 (es) |
| JP (1) | JP2023541404A (es) |
| KR (1) | KR20230064620A (es) |
| AU (1) | AU2021342155A1 (es) |
| CA (1) | CA3189065A1 (es) |
| CL (1) | CL2023000662A1 (es) |
| IL (1) | IL301187A (es) |
| MX (1) | MX2023002853A (es) |
| TW (1) | TW202227627A (es) |
| UY (1) | UY39417A (es) |
| WO (1) | WO2022056266A2 (es) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112020006169A2 (pt) * | 2017-10-05 | 2020-10-20 | Fulcrum Therapeutics, Inc. | inibidores de quinase p38 que reduzem dux4 e expressão de gene a jusante para o tratamento de fshd |
| 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 |
| CN112912499A (zh) | 2018-08-02 | 2021-06-04 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF DYSTROPHINOPATHIES |
| JP2023521042A (ja) | 2020-04-02 | 2023-05-23 | ミレキュール インコーポレイテッド | 操作されたオリゴヌクレオチドを使用する標的化された阻害 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11931421B2 (en) | 2022-04-15 | 2024-03-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
| WO2024006999A2 (en) | 2022-06-30 | 2024-01-04 | Alnylam Pharmaceuticals, Inc. | Cyclic-disulfide modified phosphate based oligonucleotide prodrugs |
| WO2024011135A2 (en) * | 2022-07-06 | 2024-01-11 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
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| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| US5744300A (en) | 1993-03-24 | 1998-04-28 | Geron Corporation | Methods and reagents for the identification and regulation of senescence-related genes |
| US5998203A (en) | 1996-04-16 | 1999-12-07 | Ribozyme Pharmaceuticals, Inc. | Enzymatic nucleic acids containing 5'-and/or 3'-cap structures |
| US5994076A (en) | 1997-05-21 | 1999-11-30 | Clontech Laboratories, Inc. | Methods of assaying differential expression |
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| JP2002537828A (ja) | 1999-03-10 | 2002-11-12 | フォゲン リミティド | 細胞への物質のデリバリー |
| WO2003035884A2 (de) | 2001-10-18 | 2003-05-01 | Heart Biosystems Gmbh | Transiente immortalisierung von zellen durch onkogenproteine oder telomerproteine |
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| US7250496B2 (en) | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| EP2305812A3 (en) * | 2002-11-14 | 2012-06-06 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
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| BR112020006169A2 (pt) | 2017-10-05 | 2020-10-20 | Fulcrum Therapeutics, Inc. | inibidores de quinase p38 que reduzem dux4 e expressão de gene a jusante para o tratamento de fshd |
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| CN111526880A (zh) * | 2017-11-01 | 2020-08-11 | 箭头药业股份有限公司 | 整联蛋白配体及其用途 |
| CA3083526A1 (en) * | 2017-12-06 | 2019-06-13 | Andrew John Geall | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
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| CN112912499A (zh) | 2018-08-02 | 2021-06-04 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 |
| JP2021533199A (ja) * | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | 筋強直症ジストロフィーを処置するための筋標的化複合体およびそれらの使用 |
| CA3124963A1 (en) | 2018-12-31 | 2020-07-09 | Research Institute At Nationwide Children's Hospital | Dux4 rna silencing using rna targeting crispr-cas13b |
| MX2022011499A (es) | 2020-03-19 | 2022-10-07 | Avidity Biosciences Inc | Composiciones y metodos para tratar la distrofia muscular facioescapulohumeral. |
| US20230323456A1 (en) | 2020-08-31 | 2023-10-12 | Modalis Therapeutics Corporation | Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene |
| TW202227626A (zh) | 2020-09-01 | 2022-07-16 | 美商奧崔基尼克斯製藥公司 | Ｄｕｘ４抑制劑及其使用方法 |
-
2021
- 2021-09-10 CA CA3189065A patent/CA3189065A1/en active Pending
- 2021-09-10 IL IL301187A patent/IL301187A/en unknown
- 2021-09-10 AU AU2021342155A patent/AU2021342155A1/en active Pending
- 2021-09-10 JP JP2023516187A patent/JP2023541404A/ja active Pending
- 2021-09-10 TW TW110133835A patent/TW202227627A/zh unknown
- 2021-09-10 UY UY0001039417A patent/UY39417A/es unknown
- 2021-09-10 KR KR1020237011805A patent/KR20230064620A/ko unknown
- 2021-09-10 WO PCT/US2021/049871 patent/WO2022056266A2/en active Application Filing
- 2021-09-10 EP EP21786710.0A patent/EP4211243A2/en active Pending
- 2021-09-10 MX MX2023002853A patent/MX2023002853A/es unknown
-
2023
- 2023-03-08 CL CL2023000662A patent/CL2023000662A1/es unknown
- 2023-03-09 US US18/181,311 patent/US20230416737A1/en active Pending
- 2023-03-09 US US18/181,199 patent/US11845937B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| US20230416737A1 (en) | 2023-12-28 |
| US11845937B2 (en) | 2023-12-19 |
| IL301187A (en) | 2023-05-01 |
| EP4211243A2 (en) | 2023-07-19 |
| KR20230064620A (ko) | 2023-05-10 |
| AU2021342155A1 (en) | 2023-04-13 |
| WO2022056266A3 (en) | 2022-04-28 |
| TW202227627A (zh) | 2022-07-16 |
| UY39417A (es) | 2022-03-31 |
| WO2022056266A8 (en) | 2023-04-13 |
| US20230265430A1 (en) | 2023-08-24 |
| CA3189065A1 (en) | 2022-03-17 |
| JP2023541404A (ja) | 2023-10-02 |
| MX2023002853A (es) | 2023-03-31 |
| WO2022056266A2 (en) | 2022-03-17 |
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