RS65493B1 - Formulacije - Google Patents
FormulacijeInfo
- Publication number
- RS65493B1 RS65493B1 RS20240516A RSP20240516A RS65493B1 RS 65493 B1 RS65493 B1 RS 65493B1 RS 20240516 A RS20240516 A RS 20240516A RS P20240516 A RSP20240516 A RS P20240516A RS 65493 B1 RS65493 B1 RS 65493B1
- Authority
- RS
- Serbia
- Prior art keywords
- formulations
- Prior art date
Links
- 238000009472 formulation Methods 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
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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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
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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
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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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- 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
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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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
- 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 RNAses, DNAses
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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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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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
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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
- C12N2310/321—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
- C12N2310/34—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
- C12N2310/352—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
- C12N2320/30—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
- 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)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Dispersion Chemistry (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Dermatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762566240P | 2017-09-29 | 2017-09-29 | |
| EP18800355.2A EP3688162B1 (en) | 2017-09-29 | 2018-09-28 | Formulations |
| PCT/US2018/053559 WO2019067992A1 (en) | 2017-09-29 | 2018-09-28 | Formulations |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RS65493B1 true RS65493B1 (sr) | 2024-05-31 |
Family
ID=64267891
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RS20240516A RS65493B1 (sr) | 2017-09-29 | 2018-09-28 | Formulacije |
Country Status (22)
| Country | Link |
|---|---|
| US (1) | US20230140670A1 (sr) |
| EP (2) | EP4385514A2 (sr) |
| JP (2) | JP7284179B2 (sr) |
| KR (1) | KR20200079497A (sr) |
| CN (1) | CN111406108A (sr) |
| AR (1) | AR113031A1 (sr) |
| AU (1) | AU2018338915A1 (sr) |
| BR (1) | BR112020006300A2 (sr) |
| CA (1) | CA3077413A1 (sr) |
| DK (1) | DK3688162T3 (sr) |
| EA (1) | EA202090868A1 (sr) |
| FI (1) | FI3688162T3 (sr) |
| IL (1) | IL273541A (sr) |
| LT (1) | LT3688162T (sr) |
| MX (1) | MX2020007148A (sr) |
| PL (1) | PL3688162T3 (sr) |
| PT (1) | PT3688162T (sr) |
| RS (1) | RS65493B1 (sr) |
| SG (1) | SG11202002653UA (sr) |
| SI (1) | SI3688162T1 (sr) |
| TW (1) | TWI833708B (sr) |
| WO (1) | WO2019067992A1 (sr) |
Families Citing this family (63)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3134544A1 (en) | 2019-03-28 | 2020-10-01 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating attr amyloidosis comprising a corticosteroid or use thereof |
| MX2021011565A (es) | 2019-03-28 | 2021-11-12 | Intellia Therapeutics Inc | Composiciones y métodos que comprenden un arn guía de ttr y un polinucleótido que codifica un agente de fijacion a adn guiado por arn. |
| CN114423869A (zh) | 2019-07-19 | 2022-04-29 | 旗舰先锋创新Vi有限责任公司 | 重组酶组合物和使用方法 |
| EP4073249A1 (en) * | 2019-12-11 | 2022-10-19 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
| AU2021263745A1 (en) | 2020-04-28 | 2022-12-08 | Intellia Therapeutics, Inc. | Methods of in vitro cell delivery |
| AU2021275213A1 (en) | 2020-05-20 | 2023-02-02 | Flagship Pioneering Innovations Vi, Llc | Immunogenic compositions and uses thereof |
| JP2023526422A (ja) | 2020-05-20 | 2023-06-21 | フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー | コロナウイルス抗原組成物及びそれらの使用 |
| JP2023527413A (ja) | 2020-05-29 | 2023-06-28 | フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー | Ｔｒｅｍ組成物及びそれに関連する方法 |
| EP4158032A2 (en) | 2020-05-29 | 2023-04-05 | Flagship Pioneering Innovations VI, LLC | Trem compositions and methods relating thereto |
| MX2023002439A (es) | 2020-09-03 | 2023-05-09 | Flagship Pioneering Innovations Vi Llc | Composiciones immunogénicas y usos de las mismas. |
| JP2023549011A (ja) | 2020-09-15 | 2023-11-22 | ヴァーヴ・セラピューティクス，インコーポレーテッド | 遺伝子編集のための脂質製剤 |
| 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 |
| IL303506A (en) | 2020-12-11 | 2023-08-01 | Intellia Therapeutics Inc | Polynucleotides, compounds and methods for genome editing involving deamination |
| TW202235617A (zh) | 2020-12-11 | 2022-09-16 | 美商英特利亞醫療公司 | 用於減少細胞中ii類mhc之組合物及方法 |
| MX2023007630A (es) | 2020-12-23 | 2023-08-25 | Flagship Pioneering Innovations Vi Llc | Composiciones de trem modificadas y usos de las mismas. |
| CR20230320A (es) | 2020-12-23 | 2023-10-23 | Intellia Therapeutics Inc | Composiciones y métodos para reducir los hla-a en una célula |
| MX2023007465A (es) | 2020-12-23 | 2023-08-18 | Intellia Therapeutics Inc | Composiciones y metodos para modificar geneticamente ciita en una celula. |
| AU2021411521A1 (en) | 2020-12-30 | 2023-08-03 | Intellia Therapeutics, Inc. | Engineered t cells |
| WO2022212784A1 (en) | 2021-03-31 | 2022-10-06 | Flagship Pioneering Innovations V, Inc. | Thanotransmission polypeptides and their use in treating cancer |
| AU2022258733A1 (en) | 2021-04-17 | 2023-11-30 | Intellia Therapeutics, Inc. | Inhibitors of dna-dependent protein kinase and compositions and uses thereof |
| TW202317764A (zh) | 2021-06-10 | 2023-05-01 | 美商英特利亞醫療公司 | 用於基因編輯之包含內部連接子之經修飾引導rna |
| EP4359531A1 (en) | 2021-06-22 | 2024-05-01 | Intellia Therapeutics, Inc. | Methods for in vivo editing of a liver gene |
| EP4377457A1 (en) | 2021-07-26 | 2024-06-05 | Flagship Pioneering Innovations VI, LLC | Trem compositions and uses thereof |
| IL311225A (en) | 2021-09-08 | 2024-05-01 | Flagship Pioneering Innovations Vi Llc | Methods and compositions for genome modulation |
| TW202330916A (zh) | 2021-09-17 | 2023-08-01 | 美商旗艦先鋒創新有限責任公司 | 用於產生環狀多核糖核苷酸之組成物和方法 |
| WO2023069397A1 (en) | 2021-10-18 | 2023-04-27 | Flagship Pioneering Innovations Vi, Llc | Compositions and methods for purifying polyribonucleotides |
| CN118251491A (zh) | 2021-10-28 | 2024-06-25 | 瑞泽恩制药公司 | 用于敲除C5的CRISPR/Cas相关方法及组合物 |
| CA3237303A1 (en) | 2021-11-03 | 2023-05-11 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing |
| CA3239266A1 (en) | 2021-11-24 | 2023-06-01 | Flagship Pioneering Innovations Vi, Llc | Coronavirus immunogen compositions and their uses |
| CA3238735A1 (en) | 2021-11-24 | 2023-06-01 | Jennifer A. Nelson | Immunogenic compositions and their uses |
| WO2023096963A1 (en) | 2021-11-24 | 2023-06-01 | Flagship Pioneering Innovations Vi, Llc | Varicella-zoster virus immunogen compositions and their uses |
| WO2023102550A2 (en) | 2021-12-03 | 2023-06-08 | The Broad Institute, Inc. | Compositions and methods for efficient in vivo delivery |
| CA3241061A1 (en) | 2021-12-22 | 2023-06-29 | Alexandra Sophie DE BOER | Compositions and methods for purifying polyribonucleotides |
| WO2023122789A1 (en) | 2021-12-23 | 2023-06-29 | Flagship Pioneering Innovations Vi, Llc | Circular polyribonucleotides encoding antifusogenic polypeptides |
| WO2023150620A1 (en) | 2022-02-02 | 2023-08-10 | Regeneron Pharmaceuticals, Inc. | Crispr-mediated transgene insertion in neonatal cells |
| WO2023178425A1 (en) | 2022-03-23 | 2023-09-28 | Nanovation Therapeutics Inc. | High sterol-containing lipid nanoparticles |
| WO2023185697A2 (en) | 2022-03-29 | 2023-10-05 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of transthyretin amyloidosis |
| WO2023184038A1 (en) | 2022-04-01 | 2023-10-05 | Nanovation Therapeutics Inc. | Mrna delivery method and composition thereof |
| WO2023196634A2 (en) | 2022-04-08 | 2023-10-12 | Flagship Pioneering Innovations Vii, Llc | Vaccines and related methods |
| WO2023205148A1 (en) | 2022-04-19 | 2023-10-26 | Intellia Therapeutics, Inc. | Chimeric antigen receptor compositions and uses |
| WO2023212677A2 (en) | 2022-04-29 | 2023-11-02 | Regeneron Pharmaceuticals, Inc. | Identification of tissue-specific extragenic safe harbors for gene therapy approaches |
| WO2023220603A1 (en) | 2022-05-09 | 2023-11-16 | Regeneron Pharmaceuticals, Inc. | Vectors and methods for in vivo antibody production |
| WO2023220083A1 (en) | 2022-05-09 | 2023-11-16 | Flagship Pioneering Innovations Vi, Llc | Trem compositions and methods of use for treating proliferative disorders |
| TW202409283A (zh) | 2022-05-13 | 2024-03-01 | 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 | 雙股ｄｎａ組合物及相關方法 |
| WO2023235725A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
| WO2023235726A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr interference therapeutics for c9orf72 repeat expansion disease |
| TW202408595A (zh) | 2022-06-16 | 2024-03-01 | 美商英特利亞醫療公司 | 用於對細胞進行遺傳修飾之方法及組合物 |
| WO2023250112A1 (en) | 2022-06-22 | 2023-12-28 | Flagship Pioneering Innovations Vi, Llc | Compositions of modified trems and uses thereof |
| WO2024006955A1 (en) | 2022-06-29 | 2024-01-04 | Intellia Therapeutics, Inc. | Engineered t cells |
| WO2024026474A1 (en) | 2022-07-29 | 2024-02-01 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle |
| WO2024030856A2 (en) | 2022-08-01 | 2024-02-08 | Flagship Pioneering Innovations Vii, Llc | Immunomodulatory proteins and related methods |
| WO2024035952A1 (en) | 2022-08-12 | 2024-02-15 | Remix Therapeutics Inc. | Methods and compositions for modulating splicing at alternative splice sites |
| WO2024061296A2 (en) | 2022-09-22 | 2024-03-28 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease |
| WO2024077191A1 (en) | 2022-10-05 | 2024-04-11 | Flagship Pioneering Innovations V, Inc. | Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer |
| WO2024097664A1 (en) | 2022-10-31 | 2024-05-10 | Flagship Pioneering Innovations Vi, Llc | Compositions and methods for purifying polyribonucleotides |
| US20240182561A1 (en) | 2022-11-04 | 2024-06-06 | Regeneron Pharmaceuticals, Inc. | Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle |
| WO2024102799A1 (en) | 2022-11-08 | 2024-05-16 | Flagship Pioneering Innovations Vi, Llc | Compositions and methods for producing circular polyribonucleotides |
| WO2024102434A1 (en) | 2022-11-10 | 2024-05-16 | Senda Biosciences, Inc. | Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs |
| US20240173426A1 (en) | 2022-11-14 | 2024-05-30 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes |
| WO2024129988A1 (en) | 2022-12-14 | 2024-06-20 | Flagship Pioneering Innovations Vii, Llc | Compositions and methods for delivery of therapeutic agents to bone |
| CN118236344A (zh) * | 2022-12-15 | 2024-06-25 | 深圳瑞吉生物科技有限公司 | 一种核酸脂质纳米载体及其制备方法与应用 |
| WO2024138189A2 (en) | 2022-12-22 | 2024-06-27 | Intellia Therapeutics, Inc. | Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies |
| WO2024138115A1 (en) | 2022-12-23 | 2024-06-27 | Intellia Theraperutics, Inc. | Systems and methods for genomic editing |
Family Cites Families (21)
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| AU2010334911A1 (en) | 2009-12-23 | 2012-07-12 | Novartis Ag | Lipids, lipid compositions, and methods of using them |
| US8691750B2 (en) * | 2011-05-17 | 2014-04-08 | Axolabs Gmbh | Lipids and compositions for intracellular delivery of biologically active compounds |
| US8993233B2 (en) | 2012-12-12 | 2015-03-31 | The Broad Institute Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| EP3553174A1 (en) | 2012-12-17 | 2019-10-16 | President and Fellows of Harvard College | Rna-guided human genome engineering |
| EA030650B1 (ru) | 2013-03-08 | 2018-09-28 | Новартис Аг | Липиды и липидные композиции для доставки активных агентов |
| EP2971013B1 (en) * | 2013-03-15 | 2020-08-19 | The University Of British Columbia | Lipid nanoparticles for transfection and related methods |
| EP3971287A1 (en) * | 2013-07-11 | 2022-03-23 | ModernaTX, Inc. | Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use |
| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin 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 |
| US10059655B2 (en) * | 2013-12-19 | 2018-08-28 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| JP6778175B2 (ja) | 2014-07-16 | 2020-10-28 | ノバルティス アーゲー | 脂質ナノ粒子ホスト中に核酸を封入する方法 |
| CA2978314A1 (en) | 2015-03-03 | 2016-09-09 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| TW201642837A (zh) * | 2015-03-24 | 2016-12-16 | Kyowa Hakko Kirin Co Ltd | 含有核酸之脂質奈米粒子 |
| WO2016197133A1 (en) * | 2015-06-04 | 2016-12-08 | Protiva Biotherapeutics, Inc. | Delivering crispr therapeutics with lipid nanoparticles |
| US20190054112A1 (en) * | 2015-09-18 | 2019-02-21 | Moderna Therapeutics, Inc. | Polynucleotide formulations for use in the treatment of renal diseases |
| CA3006618A1 (en) * | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| BR112018012894A2 (pt) * | 2015-12-23 | 2018-12-04 | Crispr Therapeutics Ag | materiais e métodos para tratamento de esclerose lateral amiotrófica e/ou degeneração lobular frontotemporal |
| EP3405579A1 (en) * | 2016-01-22 | 2018-11-28 | Modernatx, Inc. | Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof |
| TWI773666B (zh) * | 2016-03-30 | 2022-08-11 | 美商英特利亞醫療公司 | Crispr/cas 組分之脂質奈米粒子調配物 |
| KR102551664B1 (ko) * | 2016-12-22 | 2023-07-05 | 인텔리아 테라퓨틱스, 인크. | 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법 |
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| CN111406108A (zh) | 2020-07-10 |
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| BR112020006300A2 (pt) | 2020-10-20 |
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| EP4385514A2 (en) | 2024-06-19 |
| MX2020007148A (es) | 2020-10-08 |
| KR20200079497A (ko) | 2020-07-03 |
| TWI833708B (zh) | 2024-03-01 |
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