MX2021011565A - Composiciones y métodos que comprenden un arn guía de ttr y un polinucleótido que codifica un agente de fijacion a adn guiado por arn. - Google Patents
Composiciones y métodos que comprenden un arn guía de ttr y un polinucleótido que codifica un agente de fijacion a adn guiado por arn.Info
- Publication number
- MX2021011565A MX2021011565A MX2021011565A MX2021011565A MX2021011565A MX 2021011565 A MX2021011565 A MX 2021011565A MX 2021011565 A MX2021011565 A MX 2021011565A MX 2021011565 A MX2021011565 A MX 2021011565A MX 2021011565 A MX2021011565 A MX 2021011565A
- Authority
- MX
- Mexico
- Prior art keywords
- rna
- compositions
- binding agent
- dna binding
- methods including
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous 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
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
-
- 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
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- 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
- C12N2310/322—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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- 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
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
-
- 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
-
- 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
- C12N2320/31—Combination therapy
-
- 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
-
- 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
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962825637P | 2019-03-28 | 2019-03-28 | |
| PCT/US2020/025513 WO2020198697A1 (en) | 2019-03-28 | 2020-03-27 | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021011565A true MX2021011565A (es) | 2021-11-12 |
Family
ID=70296147
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021011565A MX2021011565A (es) | 2019-03-28 | 2020-03-27 | Composiciones y métodos que comprenden un arn guía de ttr y un polinucleótido que codifica un agente de fijacion a adn guiado por arn. |
Country Status (15)
| Country | Link |
|---|---|
| US (3) | US20230044994A1 (enExample) |
| EP (1) | EP3946598A1 (enExample) |
| JP (2) | JP7631215B2 (enExample) |
| KR (1) | KR20220004648A (enExample) |
| CN (1) | CN113874076A (enExample) |
| AU (1) | AU2020244887A1 (enExample) |
| BR (1) | BR112021019196A2 (enExample) |
| CA (1) | CA3134271A1 (enExample) |
| CO (1) | CO2021014559A2 (enExample) |
| EA (1) | EA202192636A1 (enExample) |
| IL (1) | IL286524A (enExample) |
| MX (1) | MX2021011565A (enExample) |
| PH (1) | PH12021552301A1 (enExample) |
| SG (1) | SG11202110434PA (enExample) |
| WO (1) | WO2020198697A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2923757T3 (es) * | 2011-12-16 | 2022-09-30 | Modernatx Inc | Composiciones de ARNm modificado |
| US12678515B2 (en) | 2015-12-30 | 2026-07-14 | Acuitas Therapeutics, Inc. | Lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| CA3116576A1 (en) | 2018-10-18 | 2020-04-23 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| CN117098541A (zh) | 2020-11-25 | 2023-11-21 | 阿卡格拉医药公司 | 用于递送核酸的脂质纳米粒及相关使用方法 |
| CA3220738A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| US20220396782A1 (en) * | 2021-06-04 | 2022-12-15 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof |
| WO2022271780A1 (en) * | 2021-06-22 | 2022-12-29 | Intellia Therapeutics, Inc. | Methods for in vivo editing of a liver gene |
| CN118974256A (zh) | 2022-03-29 | 2024-11-15 | 锐正基因(苏州)有限公司 | 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法 |
| WO2023230587A2 (en) | 2022-05-25 | 2023-11-30 | Akagera Medicines, Inc. | Lipid nanoparticles for delivery of nucleic acids and methods of use thereof |
| WO2024003805A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Methods and compositions for ttr gene editing and therapy using crispr system |
| EP4590820A2 (en) | 2022-09-22 | 2025-07-30 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease |
| EP4627083A1 (en) * | 2022-11-30 | 2025-10-08 | Arbor Biotechnologies, Inc. | Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2026003582A2 (en) * | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
| WO2026061971A1 (en) | 2024-09-17 | 2026-03-26 | Lonza Ltd | Nucleic acid untranslated regions |
Family Cites Families (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5585481A (en) | 1987-09-21 | 1996-12-17 | Gen-Probe Incorporated | Linking reagents for nucleotide probes |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| ATE515510T1 (de) | 1991-12-24 | 2011-07-15 | Isis Pharmaceuticals Inc | Durch dna-abschnitte unterbrochene modifizierte oligonukleotide |
| EP0760008A1 (en) | 1994-05-19 | 1997-03-05 | Dako A/S | Pna probes for detection of neisseria gonorrhoeae and chlamydia trachomatis |
| US6974667B2 (en) | 2000-06-14 | 2005-12-13 | Gene Logic, Inc. | Gene expression profiles in liver cancer |
| US7244764B2 (en) | 2003-06-23 | 2007-07-17 | Neurochem (International) Limited | Methods and compositions for treating amyloid-related diseases |
| US20050244869A1 (en) * | 2004-04-05 | 2005-11-03 | Brown-Driver Vickie L | Modulation of transthyretin expression |
| WO2006007712A1 (en) | 2004-07-19 | 2006-01-26 | Protiva Biotherapeutics, Inc. | Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents |
| EP2323667A4 (en) | 2008-08-07 | 2012-07-25 | Isis Pharmaceuticals Inc | MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES |
| EP2937418B1 (en) | 2008-10-20 | 2017-10-18 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of transthyretin |
| CA2754043A1 (en) | 2009-03-12 | 2010-09-16 | Alnylam Pharmaceuticals, Inc. | Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes |
| JP5723378B2 (ja) | 2009-11-03 | 2015-05-27 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | トランスサイレチン(ttr)を阻害する脂質製剤化組成物及び方法 |
| ES2625689T3 (es) | 2010-04-29 | 2017-07-20 | Ionis Pharmaceuticals, Inc. | Modulación de la expresión de transtiretina |
| UA118649C2 (uk) | 2011-11-18 | 2019-02-25 | Елнілем Фармасьютікалз, Інк. | ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR) |
| EP4397759A3 (en) | 2012-10-23 | 2024-10-09 | Toolgen Incorporated | Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| PT2931898E (pt) * | 2012-12-12 | 2016-06-16 | Harvard College | Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais |
| SG10201707569YA (en) | 2012-12-12 | 2017-10-30 | Broad Inst Inc | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications |
| IL308158A (en) | 2012-12-17 | 2023-12-01 | Harvard College | Rna-guided human genome engineering |
| ES2887254T3 (es) | 2013-03-08 | 2021-12-22 | Novartis Ag | Lípidos y composiciones lipídicas para el suministro de agentes activos |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| PL2992098T3 (pl) | 2013-05-01 | 2019-09-30 | Ionis Pharmaceuticals, Inc. | Kompozycje i sposoby modulowania ekspresji hbv i ttr |
| SG11201510327TA (en) | 2013-06-17 | 2016-01-28 | Broad Inst Inc | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| WO2015089419A2 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| KR20160097338A (ko) | 2013-12-12 | 2016-08-17 | 더 브로드 인스티튜트, 인코퍼레이티드 | 뉴클레오티드 반복 장애에서의 crispr-cas 시스템의 조성물 및 방법 및 용도 |
| US10059655B2 (en) | 2013-12-19 | 2018-08-28 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| WO2015183026A1 (ko) | 2014-05-28 | 2015-12-03 | 주식회사 툴젠 | 불활성화된 표적 특이적 뉴클레아제를 이용한 표적 dna의 분리 방법 |
| DK3161128T3 (en) | 2014-06-26 | 2018-11-05 | Regeneron Pharma | METHODS AND COMPOSITIONS FOR TARGETED GENTICAL MODIFICATIONS AND PROCEDURES FOR USE THEREOF |
| CN106794141B (zh) | 2014-07-16 | 2021-05-28 | 诺华股份有限公司 | 将核酸包封在脂质纳米粒主体中的方法 |
| CN105821072A (zh) | 2015-01-23 | 2016-08-03 | 深圳华大基因研究院 | 用于DNA组装的CRISPR-Cas9系统及DNA组装方法 |
| AU2016226077B2 (en) | 2015-03-03 | 2021-12-23 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| PL3298134T3 (pl) | 2015-05-16 | 2023-09-18 | Genzyme Corporation | Edycja genów mutacji głęboko intronowych |
| US20180208945A1 (en) * | 2015-07-28 | 2018-07-26 | Danisco Us Inc. | Genome editing systems and methods of use |
| DK4276189T3 (da) | 2015-09-21 | 2026-05-26 | Trilink Biotechnologies Llc | Initierende oligonukleotidprimere med kappe til syntesering af 5'-ende RNA'er med kappe |
| US10369232B2 (en) | 2015-09-21 | 2019-08-06 | Arcturus Therapeutics, Inc. | Allele selective gene editing and uses thereof |
| US11866727B2 (en) | 2015-11-06 | 2024-01-09 | Crispr Therapeutics Ag | Materials and methods for treatment of glycogen storage disease type 1A |
| WO2017093804A2 (en) | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| US11083799B2 (en) | 2016-03-16 | 2021-08-10 | Crispr Therapeutics Ag | Materials and methods for treatment of hereditary haemochromatosis |
| WO2017165704A1 (en) | 2016-03-23 | 2017-09-28 | University Of Miami | Treatment of cancer by activating endogenous cryptic amyloidogenic aggregating peptides |
| ES3030658T3 (en) | 2016-03-30 | 2025-07-01 | Intellia Therapeutics Inc | Lipid nanoparticle formulations for crispr/cas components |
| JP2019515914A (ja) | 2016-04-22 | 2019-06-13 | インテリア セラピューティクス,インコーポレイテッド | 転写因子4内のトリヌクレオチドリピートと関連する疾患の治療のための組成物および方法 |
| WO2018007871A1 (en) | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| WO2018058064A1 (en) | 2016-09-23 | 2018-03-29 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing |
| WO2018067447A1 (en) | 2016-10-03 | 2018-04-12 | Itellia Therapeutics, Inc. | Improved methods for identifying double strand break sites |
| EP3551757A1 (en) | 2016-12-08 | 2019-10-16 | Intellia Therapeutics, Inc. | Modified guide rnas |
| WO2018183808A1 (en) | 2017-03-31 | 2018-10-04 | Agenovir Corporation | Antiviral therapeutic |
| TWI904069B (zh) * | 2017-09-29 | 2025-11-11 | 美商英特利亞醫療公司 | 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法 |
| EP3688162B1 (en) * | 2017-09-29 | 2024-03-06 | Intellia Therapeutics, Inc. | Formulations |
| IL311278B2 (en) * | 2017-09-29 | 2026-05-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing |
| JP7832761B2 (ja) * | 2018-06-08 | 2026-03-18 | インテリア セラピューティクス,インコーポレイテッド | 遺伝子編集のための修飾されたガイドrna |
| BR112021005718A2 (pt) * | 2018-09-28 | 2021-06-22 | Intellia Therapeutics, Inc. | composições e métodos para edição de genes de lactato desidrogenase (ldha) |
-
2020
- 2020-03-27 KR KR1020217035004A patent/KR20220004648A/ko active Pending
- 2020-03-27 EA EA202192636A patent/EA202192636A1/ru unknown
- 2020-03-27 WO PCT/US2020/025513 patent/WO2020198697A1/en not_active Ceased
- 2020-03-27 PH PH1/2021/552301A patent/PH12021552301A1/en unknown
- 2020-03-27 BR BR112021019196A patent/BR112021019196A2/pt unknown
- 2020-03-27 MX MX2021011565A patent/MX2021011565A/es unknown
- 2020-03-27 AU AU2020244887A patent/AU2020244887A1/en active Pending
- 2020-03-27 JP JP2021557397A patent/JP7631215B2/ja active Active
- 2020-03-27 CA CA3134271A patent/CA3134271A1/en active Pending
- 2020-03-27 SG SG11202110434PA patent/SG11202110434PA/en unknown
- 2020-03-27 CN CN202080038208.6A patent/CN113874076A/zh active Pending
- 2020-03-27 EP EP20720292.0A patent/EP3946598A1/en active Pending
-
2021
- 2021-09-19 IL IL286524A patent/IL286524A/en unknown
- 2021-09-27 US US17/486,735 patent/US20230044994A1/en not_active Abandoned
- 2021-10-28 CO CONC2021/0014559A patent/CO2021014559A2/es unknown
-
2023
- 2023-07-31 US US18/362,867 patent/US12686876B2/en active Active
-
2025
- 2025-02-04 JP JP2025016673A patent/JP2025072483A/ja active Pending
-
2026
- 2026-03-26 US US19/630,254 patent/US20260201423A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020198697A1 (en) | 2020-10-01 |
| US12686876B2 (en) | 2026-07-21 |
| SG11202110434PA (en) | 2021-10-28 |
| JP2022525428A (ja) | 2022-05-13 |
| KR20220004648A (ko) | 2022-01-11 |
| CO2021014559A2 (es) | 2021-11-19 |
| IL286524A (en) | 2021-10-31 |
| JP7631215B2 (ja) | 2025-02-18 |
| EP3946598A1 (en) | 2022-02-09 |
| BR112021019196A2 (pt) | 2022-01-18 |
| US20230044994A1 (en) | 2023-02-09 |
| AU2020244887A1 (en) | 2021-11-11 |
| EA202192636A1 (ru) | 2022-03-17 |
| US20260201423A1 (en) | 2026-07-16 |
| PH12021552301A1 (en) | 2022-07-04 |
| JP2025072483A (ja) | 2025-05-09 |
| CA3134271A1 (en) | 2020-10-01 |
| CN113874076A (zh) | 2021-12-31 |
| US20240124897A1 (en) | 2024-04-18 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CO2021014559A2 (es) | Composiciones y métodos que comprenden un arn guía de ttr y un polinucleótido que codifica un agente de fijación a adn guiado por arn | |
| MX2025014378A (es) | Composiciones utiles para la edicion del gen ttr y el tratamiento de la amiloidosis attr | |
| PH12021550686A1 (en) | Compositions and methods for lactate dehydrogenase (ldha) gene editing | |
| CO2022012196A2 (es) | Composiciones y métodos para la edición de genes de calicreína (klkb1) | |
| CO2021014562A2 (es) | Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr que comprenden un corticosteroide o el uso de este | |
| MX2021001070A (es) | Composiciones y métodos para editar el gen hidroxiácido oxidasa 1 (hao1) para tratar la hiperoxaluria primaria tipo 1 (ph1). | |
| CO2021006301A2 (es) | Composiciones y métodos de inmunoterapia | |
| BR112021016613A2 (pt) | Agente capaz de inibir a sinalização mediada por interleucina 11 (il-11), uso deste na fabricação de um medicamento e métodos | |
| EA201890957A1 (ru) | Комбинированная терапия для лечения злокачественных опухолей | |
| ECSP20021323A (es) | Proteínas de unión condicionalmente activadas restringidas | |
| MX2025012087A (es) | Piridazinonas y metodos de uso de estas | |
| MX2023009712A (es) | Composiciones oligonucleotidicas y sus metodos. | |
| CO2021012310A2 (es) | Composiciones y métodos para tratar laminopatías | |
| BR112019007210A2 (pt) | métodos e composições para o tratamento da doença de fabry | |
| WO2016106406A3 (en) | Rna agents for gst-pi gene modulation | |
| BR112019008374A2 (pt) | inibidor seletivo de egfr mutante por inserção do éxon 20 | |
| MX2015013177A (es) | Combinacion de tratamientod de cancer usando microarns e inhibidores de egfr-tki. | |
| MX2019008921A (es) | Adenovirus recombinante de transgenes múltiples. | |
| MX2018016057A (es) | Composiciones y metodos para tratar enfermedades cardiovasculares. | |
| MX2019006552A (es) | Terapia génica para mucopolisacaridosis de tipo i. | |
| ZA202204072B (en) | Co-delivery of tgf-β sirna and pdl1 sirna to treat cancer | |
| MX2017000582A (es) | Marcadores geneticos para predecir la reactividad a terapia con agente que eleva la lipoproteina de alta densidad (hdl) o que imita la lipoproteina de alta densidad (hdl). | |
| CO2022006367A2 (es) | Tratamiento génico para la enfermedad de alzheimer | |
| EA202192806A1 (ru) | Системы редактирования генов для модифицирования гена scn9a или scn10a и способы их применения | |
| BR112017010428A2 (pt) | composição para tratar dor aguda, pós-operatória ou crônica em um indivíduo, método para tratar um indivíduo tendo dor aguda, pós-operatória ou crônica e kit para produzir a composição |