BR112022019889A2 - Inibição direcionada com o uso de oligonucleotídeos modificados geneticamente - Google Patents
Inibição direcionada com o uso de oligonucleotídeos modificados geneticamenteInfo
- Publication number
- BR112022019889A2 BR112022019889A2 BR112022019889A BR112022019889A BR112022019889A2 BR 112022019889 A2 BR112022019889 A2 BR 112022019889A2 BR 112022019889 A BR112022019889 A BR 112022019889A BR 112022019889 A BR112022019889 A BR 112022019889A BR 112022019889 A2 BR112022019889 A2 BR 112022019889A2
- Authority
- BR
- Brazil
- Prior art keywords
- genetically modified
- modified oligonucleotides
- targeted inhibition
- activity
- disclosed
- Prior art date
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- 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
- A61K47/54—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 an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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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
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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
- G16B15/00—ICT specially adapted for analysing two-dimensional or three-dimensional molecular structures, e.g. structural or functional relations or structure alignment
- G16B15/30—Drug targeting using structural data; Docking or binding prediction
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- G—PHYSICS
- G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
- G16H—HEALTHCARE INFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR THE HANDLING OR PROCESSING OF MEDICAL OR HEALTHCARE DATA
- G16H50/00—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics
- G16H50/20—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/316—Phosphonothioates
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- 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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- C12N2310/32—Chemical structure of the sugar
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/35—Nature of the modification
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- C12N2310/3521—Methyl
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A90/00—Technologies having an indirect contribution to adaptation to climate change
- Y02A90/10—Information and communication technologies [ICT] supporting adaptation to climate change, e.g. for weather forecasting or climate simulation
Abstract
INIBIÇÃO DIRECIONADA COM O USO DE OLIGONUCLEOTÍDEOS MODIFICADOS GENETICAMENTE. São divulgados aqui oligonucleotídeos projetados para inibição seletiva da expressão e atividade de polipeptídeos. Também são divulgados neste documento métodos para inibir seletivamente a expressão e a atividade do polipeptídeo contatando um oligonucleotídeo manipulado com um polinucleotídeo que codifica o polipeptídeo.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063004045P | 2020-04-02 | 2020-04-02 | |
PCT/US2021/025639 WO2021203043A2 (en) | 2020-04-02 | 2021-04-02 | Targeted inhibition using engineered oligonucleotides |
Publications (1)
Publication Number | Publication Date |
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BR112022019889A2 true BR112022019889A2 (pt) | 2023-03-07 |
Family
ID=77927597
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022019889A BR112022019889A2 (pt) | 2020-04-02 | 2021-04-02 | Inibição direcionada com o uso de oligonucleotídeos modificados geneticamente |
Country Status (12)
Country | Link |
---|---|
US (4) | US20230132602A1 (pt) |
EP (1) | EP4127175A2 (pt) |
JP (1) | JP2023521042A (pt) |
KR (1) | KR20230023612A (pt) |
CN (1) | CN116096890A (pt) |
AU (1) | AU2021246530A1 (pt) |
BR (1) | BR112022019889A2 (pt) |
CA (1) | CA3174286A1 (pt) |
CO (1) | CO2022015141A2 (pt) |
IL (1) | IL296957A (pt) |
MX (1) | MX2022012265A (pt) |
WO (1) | WO2021203043A2 (pt) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023288289A2 (en) | 2021-07-14 | 2023-01-19 | Mirecule, Inc. | Oligonucleotides and compositions thereof for neuromuscular disorders |
WO2023122347A2 (en) | 2021-12-23 | 2023-06-29 | Mirecule, Inc. | Compositions for delivery of polynucleotides |
KR20230102595A (ko) * | 2021-12-30 | 2023-07-07 | 주식회사 이노제닉스 | 포스파티딜이노시톨 3-키나아제 촉매 소단위 알파 유전자의 3'비번역 부위에 특이적으로 결합하는 핵산 절편 및 그 응용 |
CN117402251B (zh) * | 2023-12-15 | 2024-02-23 | 中国医学科学院基础医学研究所 | 一种抗小g蛋白rbj的抗体及其应用 |
Family Cites Families (71)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
US4415732A (en) | 1981-03-27 | 1983-11-15 | University Patents, Inc. | Phosphoramidite compounds and processes |
US4373071A (en) | 1981-04-30 | 1983-02-08 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
US4401796A (en) | 1981-04-30 | 1983-08-30 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
US5153319A (en) | 1986-03-31 | 1992-10-06 | University Patents, Inc. | Process for preparing polynucleotides |
US5047524A (en) | 1988-12-21 | 1991-09-10 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
US5262530A (en) | 1988-12-21 | 1993-11-16 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
US5700642A (en) | 1995-05-22 | 1997-12-23 | Sri International | Oligonucleotide sizing using immobilized cleavable primers |
PT1504126E (pt) | 2002-05-03 | 2014-06-02 | Univ Duke | Um método para regular a expressão génica |
US8106180B2 (en) * | 2003-08-07 | 2012-01-31 | Whitehead Institute For Biomedical Research | Methods and products for expression of micro RNAs |
WO2005017145A1 (ja) | 2003-08-13 | 2005-02-24 | Japan Biological Informatics Consortium | 機能性rnaが制御する被制御遺伝子の同定・予測方法及びその利用方法 |
US7795419B2 (en) * | 2004-05-26 | 2010-09-14 | Rosetta Genomics Ltd. | Viral and viral associated miRNAs and uses thereof |
EP1959012A3 (en) * | 2004-12-29 | 2009-12-30 | Exiqon A/S | Novel oligonucleotide compositions and probe sequences useful for detection and analysis of microRNAs and their target mRNAs |
JP2006248978A (ja) | 2005-03-10 | 2006-09-21 | Mebiopharm Co Ltd | 新規なリポソーム製剤 |
WO2006133022A2 (en) | 2005-06-03 | 2006-12-14 | The Johns Hopkins University | Compositions and methods for decreasing microrna expression for the treatment of neoplasia |
ITMI20051248A1 (it) * | 2005-07-01 | 2007-01-02 | Vimar Spa | Modem per bus per impianti elettrici civili ed industriali |
CN103028120B (zh) | 2005-09-12 | 2015-08-12 | 俄亥俄州立大学研究基金会 | 用于诊断或治疗bcl2相关癌症的组合物和方法 |
US8603991B2 (en) | 2005-11-18 | 2013-12-10 | Gradalis, Inc. | Individualized cancer therapy |
WO2007070483A2 (en) | 2005-12-12 | 2007-06-21 | The University Of North Carolina At Chapel Hill | Micrornas that regulate muscle cell proliferation and differentiation |
US8148069B2 (en) | 2006-01-05 | 2012-04-03 | The Ohio State University | MicroRNA-based methods and compositions for the diagnosis, prognosis and treatment of solid cancers |
WO2007143752A2 (en) | 2006-06-09 | 2007-12-13 | The Regents Of The University Of California | Targets in breast cancer for prognosis or therapy |
US20100292299A1 (en) | 2006-06-20 | 2010-11-18 | Mendell Joshua T | Nucleotide Motifs Providing Localization Elements and Methods of Use |
US8114982B2 (en) | 2006-12-06 | 2012-02-14 | Albert Einstein College Of Medicine Of Yeshiva University | Multi-microRNA methods and compositions |
JP2010516249A (ja) | 2007-01-17 | 2010-05-20 | ザ・ジョンズ・ホプキンス・ユニバーシティ | 腫瘍治療のためのミクロrnaに関する組成物及び方法 |
WO2009012263A2 (en) * | 2007-07-18 | 2009-01-22 | The Trustees Of Columbia University In The City Of New York | Tissue-specific micrornas and compositions and uses thereof |
CA2694928A1 (en) | 2007-07-31 | 2009-02-05 | Board Of Regents, The University Of Texas System | Micro-rnas that control myosin expression and myofiber identity |
WO2009075817A1 (en) | 2007-12-06 | 2009-06-18 | Minerva Biotechnologies Corporation | Method for treating cancer using interference rna |
JP2010094122A (ja) | 2008-06-12 | 2010-04-30 | Keio Gijuku | バイオマーカーとしてのマイクロrnaを用いた頭頸部腫瘍の診断・治療選択 |
US8729041B2 (en) | 2008-12-03 | 2014-05-20 | The Johns Hopkins University | Compositions and methods for treating hepatic neoplasia |
CA2765129A1 (en) | 2009-06-08 | 2010-12-16 | Miragen Therapeutics | Chemical modification motifs for mirna inhibitors and mimetics |
WO2011034811A1 (en) | 2009-09-17 | 2011-03-24 | Sigma-Aldrich Co. | Short rna mimetics |
WO2011069100A2 (en) * | 2009-12-04 | 2011-06-09 | Duke University | Microrna and use thereof in identification of b cell malignancies |
US8768630B2 (en) * | 2010-02-19 | 2014-07-01 | The Regents Of The University Of Michigan | miRNA target prediction |
JP5935030B2 (ja) | 2010-05-14 | 2016-06-15 | ダナ−ファーバー キャンサー インスティテュート, インコーポレイテッド | 白血病を治療するための組成物および方法 |
US20120225034A1 (en) | 2010-09-02 | 2012-09-06 | Universite De Mons | Agents useful in treating facioscapulohumeral muscular dystrophy |
JP2014506791A (ja) | 2011-02-03 | 2014-03-20 | マーナ セラピューティクス インコーポレイテッド | miR−34の合成模倣体 |
US9222085B2 (en) | 2011-02-03 | 2015-12-29 | Mirna Therapeutics, Inc. | Synthetic mimics of MIR-124 |
EP3290055A3 (en) | 2011-07-25 | 2018-05-30 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of dux4 |
WO2013019623A2 (en) | 2011-07-29 | 2013-02-07 | Fred Hutchinson Cancer Research Center | Methods and compositions for modulating the innate immune response and/or myogenesis in a mammalian subject |
AU2013201303C1 (en) | 2011-10-06 | 2016-06-23 | MiRagen Therapeutics, Inc. | Control of whole body energy homeostasis by microRNA regulation |
TWI516267B (zh) | 2011-11-07 | 2016-01-11 | 臺北榮民總醫院 | 抑制癌症或腫瘤細胞之類癌幹細胞特性及抗化學及放射治療特性的醫藥組合物 |
WO2013120038A2 (en) | 2012-02-10 | 2013-08-15 | Regents Of The University Of Minnesota | Morpholino targeting dux4 for treating fshd |
WO2014179765A2 (en) * | 2013-05-02 | 2014-11-06 | Thomas Jefferson University | Novel human mirnas for use in diagnosis, prognosis and therapy of human conditions and diseases |
US9518300B2 (en) | 2014-04-14 | 2016-12-13 | Dana-Farber Cancer Institute, Inc. | Composition and method for treating a hematological malignancy |
KR102390629B1 (ko) | 2014-04-25 | 2022-04-26 | 칠드런'즈 메디컬 센터 코포레이션 | 헤모글로빈병증을 치료하기 위한 조성물 및 방법 |
WO2016115490A1 (en) | 2015-01-16 | 2016-07-21 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of dux4 |
WO2016170348A2 (en) | 2015-04-22 | 2016-10-27 | Mina Therapeutics Limited | Sarna compositions and methods of use |
WO2017007886A2 (en) | 2015-07-08 | 2017-01-12 | Idera Pharmaceuticals, Inc. | Compositions for inhibiting dux4 gene expression and uses thereof |
WO2017156015A2 (en) * | 2016-03-07 | 2017-09-14 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Micrornas and methods of their use |
EP3684378A4 (en) | 2017-09-19 | 2021-06-16 | Children's National Medical Center | GAPMERS AND THEIR USES IN THE TREATMENT OF MUSCLE DYSTROPHY |
AU2018345772A1 (en) | 2017-10-02 | 2020-04-23 | Research Institute At Nationwide Children's Hospital | MiRNA detargeting system for tissue specific interference |
US10676744B2 (en) * | 2017-10-13 | 2020-06-09 | University Of South Carolina | Modulation of adipose tissue macrophage phenotype by use of MicroRNA-30 |
US11268305B2 (en) | 2018-05-17 | 2022-03-08 | Kiekert Ag | Latching system for a motor vehicle |
EA202190416A1 (ru) | 2018-08-02 | 2021-06-23 | Дайн Терапьютикс, Инк. | Мышечно-специфические комлексы и их применение для лечения плече-лопаточно-лицевой мышечной дистрофии |
KR20210142699A (ko) | 2019-03-29 | 2021-11-25 | 미쓰비시 타나베 파마 코퍼레이션 | Dux4의 발현을 조절하기 위한 화합물, 방법 및 의약조성물 |
AU2020313255A1 (en) | 2019-07-12 | 2022-01-06 | Daiichi Sankyo Company, Limited | Antisense oligonucleotide capable of altering splicing of DUX4 pre-mRNA |
JP2023512442A (ja) | 2020-01-10 | 2023-03-27 | ダイン セラピューティクス,インコーポレーテッド | 顔面肩甲上腕型筋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 |
AU2021237465A1 (en) | 2020-03-19 | 2022-10-13 | Avidity Biosciences, Inc. | Compositions and methods of treating Facioscapulohumeral muscular dystrophy |
MX2022012965A (es) | 2020-04-17 | 2023-01-18 | Nevada Res & Innovation Corporation | Inhibición por crispr para la distrofia muscular facioscapulohumeral. |
GB202009618D0 (en) | 2020-06-24 | 2020-08-05 | Ucl Business Ltd | Therapeutics for the treatment of FSHD |
BR112023001003A2 (pt) | 2020-07-23 | 2023-03-28 | Dyne Therapeutics Inc | Complexos de direcionamento muscular e usos dos mesmos para tratar distrofia muscular facioescapuloumeral |
AU2021313058A1 (en) | 2020-07-23 | 2023-03-30 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof |
WO2022043566A1 (en) | 2020-08-31 | 2022-03-03 | Merck Patent Gmbh | Method and device for manufacturing of solid oral dosage forms |
US20230348906A1 (en) | 2020-09-01 | 2023-11-02 | Ultragenyx Pharmaceutical Inc. | Dux4 inhibitors and methods of use thereof |
CN116323633A (zh) | 2020-09-11 | 2023-06-23 | 箭头药业股份有限公司 | 骨骼肌递送平台及使用方法 |
UY39417A (es) | 2020-09-11 | 2022-03-31 | Arrowhead Pharmaceuticals Inc | Agentes de arni para inhibir la expresión de dux4, composiciones de dichos agentes, y métodos de uso |
JP2022055361A (ja) | 2020-09-28 | 2022-04-07 | 田辺三菱製薬株式会社 | Dux4の発現を調節するための医薬組成物 |
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- 2021-04-02 KR KR1020227038205A patent/KR20230023612A/ko unknown
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- 2021-04-02 EP EP21780542.3A patent/EP4127175A2/en active Pending
- 2021-04-02 US US17/915,215 patent/US20230132602A1/en active Pending
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WO2021203043A3 (en) | 2021-11-11 |
CN116096890A (zh) | 2023-05-09 |
MX2022012265A (es) | 2023-01-11 |
WO2021203043A2 (en) | 2021-10-07 |
CO2022015141A2 (es) | 2022-12-30 |
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AU2021246530A1 (en) | 2022-11-03 |
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US11795459B2 (en) | 2023-10-24 |
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