AR113031A1 - Composiciones de nanopartículas lipídicas (lnp) que comprende arn - Google Patents
Composiciones de nanopartículas lipídicas (lnp) que comprende arnInfo
- Publication number
- AR113031A1 AR113031A1 ARP180102822A ARP180102822A AR113031A1 AR 113031 A1 AR113031 A1 AR 113031A1 AR P180102822 A ARP180102822 A AR P180102822A AR P180102822 A ARP180102822 A AR P180102822A AR 113031 A1 AR113031 A1 AR 113031A1
- Authority
- AR
- Argentina
- Prior art keywords
- lnp
- lipid nanoparticle
- nanoparticle compositions
- including rna
- administration
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- 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
-
- 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
-
- 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
- C12N15/87—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
-
- 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
-
- 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]
-
- 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
- C12N2310/34—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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Dispersion Chemistry (AREA)
- Optics & Photonics (AREA)
- Dermatology (AREA)
- Nanotechnology (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)
Abstract
Composiciones basadas en nanopartículas lipídicas con propiedades mejoradas para la administración de agentes biológicamente activos, células manipuladas por ingeniería, métodos para la administración de los agentes.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762566240P | 2017-09-29 | 2017-09-29 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR113031A1 true AR113031A1 (es) | 2020-01-15 |
Family
ID=64267891
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP180102822A AR113031A1 (es) | 2017-09-29 | 2018-09-28 | Composiciones de nanopartículas lipídicas (lnp) que comprende arn |
Country Status (21)
Country | Link |
---|---|
US (1) | US20230140670A1 (es) |
EP (2) | EP3688162B1 (es) |
JP (2) | JP7284179B2 (es) |
KR (1) | KR20200079497A (es) |
CN (1) | CN111406108A (es) |
AR (1) | AR113031A1 (es) |
AU (1) | AU2018338915A1 (es) |
BR (1) | BR112020006300A2 (es) |
CA (1) | CA3077413A1 (es) |
DK (1) | DK3688162T3 (es) |
EA (1) | EA202090868A1 (es) |
FI (1) | FI3688162T3 (es) |
IL (1) | IL273541A (es) |
LT (1) | LT3688162T (es) |
MX (1) | MX2020007148A (es) |
PL (1) | PL3688162T3 (es) |
PT (1) | PT3688162T (es) |
RS (1) | RS65493B1 (es) |
SG (1) | SG11202002653UA (es) |
TW (1) | TWI833708B (es) |
WO (1) | WO2019067992A1 (es) |
Families Citing this family (58)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EA202192636A1 (ru) | 2019-03-28 | 2022-03-17 | Интеллиа Терапьютикс, Инк. | Композиции и способы, содержащие гидовую рнк ttr и полинуклеотид, кодирующий днк-связывающий агент, гидируемый рнк |
WO2020198706A1 (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 |
WO2021016075A1 (en) | 2019-07-19 | 2021-01-28 | Flagship Pioneering Innovations Vi, Llc | Recombinase compositions and methods of use |
WO2021119275A1 (en) * | 2019-12-11 | 2021-06-17 | 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 |
JP2023526423A (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 | 免疫原性組成物及びその使用 |
CA3179420A1 (en) | 2020-05-20 | 2021-11-25 | Avak Kahvejian | Coronavirus antigen compositions and their uses |
WO2021243301A2 (en) | 2020-05-29 | 2021-12-02 | Flagship Pioneering Innovations Vi, Llc. | Trem compositions and methods relating thereto |
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 |
WO2022051629A1 (en) | 2020-09-03 | 2022-03-10 | Flagship Pioneering Innovations Vi, Llc | Immunogenic compositions and uses thereof |
CA3200234A1 (en) | 2020-11-25 | 2022-06-02 | Daryl C. Drummond | Lipid nanoparticles for delivery of nucleic acids, and related methods of use |
WO2022125982A1 (en) | 2020-12-11 | 2022-06-16 | Intellia Therapeutics, Inc. | Compositions and methods for reducing mhc class ii in a cell |
TW202237845A (zh) | 2020-12-11 | 2022-10-01 | 美商英特利亞醫療公司 | 用於涉及去胺作用之基因體編輯之多核苷酸、組合物及方法 |
TW202239959A (zh) | 2020-12-23 | 2022-10-16 | 美商英特利亞醫療公司 | 用於減少細胞中hla-a之組合物及方法 |
KR20230135068A (ko) | 2020-12-23 | 2023-09-22 | 인텔리아 테라퓨틱스, 인크. | 세포에서 ciita를 유전적으로 변형시키기 위한 조성물및 방법 |
JP2024501288A (ja) | 2020-12-23 | 2024-01-11 | 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 | 修飾tremの組成物及びその使用 |
CA3206484A1 (en) | 2020-12-30 | 2022-07-07 | Intellia Therapeutics, Inc. | Engineered t cells |
US20220325287A1 (en) | 2021-03-31 | 2022-10-13 | Flagship Pioneering Innovations V, Inc. | Thanotransmission polypeptides and their use in treating cancer |
JP2024516376A (ja) | 2021-04-17 | 2024-04-15 | インテリア セラピューティクス,インコーポレーテッド | Dna依存性プロテインキナーゼの阻害剤ならびにその組成物及び使用 |
BR112023025724A2 (pt) | 2021-06-10 | 2024-02-27 | Intellia Therapeutics Inc | Rnas-guia modificados que compreendem um ligante interno para edição de gene |
TW202327626A (zh) | 2021-06-22 | 2023-07-16 | 美商英特利亞醫療公司 | 用於體內編輯肝臟基因之方法 |
EP4377457A1 (en) | 2021-07-26 | 2024-06-05 | Flagship Pioneering Innovations VI, LLC | Trem compositions and uses thereof |
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 |
AU2022370530A1 (en) | 2021-10-18 | 2024-05-02 | 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 |
WO2023081689A2 (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 |
AU2022397292A1 (en) | 2021-11-24 | 2024-05-30 | Flagship Pioneering Innovations Vi, Llc | Varicella-zoster virus immunogen compositions and their uses |
CA3238735A1 (en) | 2021-11-24 | 2023-06-01 | Jennifer A. Nelson | Immunogenic compositions and their uses |
WO2023102550A2 (en) | 2021-12-03 | 2023-06-08 | The Broad Institute, Inc. | Compositions and methods for efficient in vivo delivery |
WO2023122745A1 (en) | 2021-12-22 | 2023-06-29 | Flagship Pioneering Innovations Vi, Llc | Compositions and methods for purifying polyribonucleotides |
WO2023122789A1 (en) | 2021-12-23 | 2023-06-29 | Flagship Pioneering Innovations Vi, Llc | Circular polyribonucleotides encoding antifusogenic polypeptides |
TW202332767A (zh) | 2022-02-02 | 2023-08-16 | 美商雷傑納榮製藥公司 | 用於治療龐貝氏症之抗TfR:GAA及抗CD63:GAA插入 |
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 |
WO2023220729A2 (en) | 2022-05-13 | 2023-11-16 | Flagship Pioneering Innovations Vii, Llc | Double stranded dna compositions and related methods |
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 |
WO2023245113A1 (en) | 2022-06-16 | 2023-12-21 | Intellia Therapeutics, Inc. | Methods and compositions for genetically modifying a cell |
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 |
US20240042021A1 (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 |
US20240174732A1 (en) | 2022-10-05 | 2024-05-30 | Flagship Pioneering Innovations V, Inc. | Nucleic acid molecules encoding trif and additional polypeptides 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 |
WO2024107765A2 (en) | 2022-11-14 | 2024-05-23 | 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 |
Family Cites Families (21)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20110200582A1 (en) | 2009-12-23 | 2011-08-18 | 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 |
US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
EP3553174A1 (en) | 2012-12-17 | 2019-10-16 | President and Fellows of Harvard College | Rna-guided human genome engineering |
BR112015021791B1 (pt) | 2013-03-08 | 2022-08-30 | Novartis Ag | Compostos de lipídio catiônico e composições de lipídios e farmacêuticas |
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 |
US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
CN106536729A (zh) * | 2013-12-12 | 2017-03-22 | 布罗德研究所有限公司 | 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用 |
EP3872066A1 (en) * | 2013-12-19 | 2021-09-01 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
US10342761B2 (en) | 2014-07-16 | 2019-07-09 | Novartis Ag | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
EP3265559B1 (en) | 2015-03-03 | 2021-01-06 | 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 |
EP3967758A1 (en) | 2015-12-01 | 2022-03-16 | CRISPR Therapeutics AG | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
SG11201805320XA (en) | 2015-12-23 | 2018-07-30 | Crispr Therapeutics Ag | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
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 |
JP7245651B2 (ja) * | 2016-03-30 | 2023-03-24 | インテリア セラピューティクス,インコーポレイテッド | Crispr/cas構成成分のための脂質ナノ粒子製剤 |
CN117737062A (zh) * | 2016-12-22 | 2024-03-22 | 因特利亚治疗公司 | 用于治疗α-1抗胰蛋白酶缺乏症的组合物及方法 |
-
2018
- 2018-09-28 TW TW107134481A patent/TWI833708B/zh active
- 2018-09-28 PT PT188003552T patent/PT3688162T/pt unknown
- 2018-09-28 AU AU2018338915A patent/AU2018338915A1/en active Pending
- 2018-09-28 CA CA3077413A patent/CA3077413A1/en active Pending
- 2018-09-28 SG SG11202002653UA patent/SG11202002653UA/en unknown
- 2018-09-28 RS RS20240516A patent/RS65493B1/sr unknown
- 2018-09-28 EP EP18800355.2A patent/EP3688162B1/en active Active
- 2018-09-28 BR BR112020006300-2A patent/BR112020006300A2/pt unknown
- 2018-09-28 WO PCT/US2018/053559 patent/WO2019067992A1/en active Application Filing
- 2018-09-28 EP EP24159518.0A patent/EP4385514A2/en active Pending
- 2018-09-28 MX MX2020007148A patent/MX2020007148A/es unknown
- 2018-09-28 CN CN201880070589.9A patent/CN111406108A/zh active Pending
- 2018-09-28 PL PL18800355.2T patent/PL3688162T3/pl unknown
- 2018-09-28 JP JP2020540244A patent/JP7284179B2/ja active Active
- 2018-09-28 DK DK18800355.2T patent/DK3688162T3/da active
- 2018-09-28 AR ARP180102822A patent/AR113031A1/es unknown
- 2018-09-28 US US16/651,911 patent/US20230140670A1/en active Pending
- 2018-09-28 FI FIEP18800355.2T patent/FI3688162T3/fi active
- 2018-09-28 KR KR1020207012309A patent/KR20200079497A/ko not_active Application Discontinuation
- 2018-09-28 LT LTEPPCT/US2018/053559T patent/LT3688162T/lt unknown
- 2018-09-28 EA EA202090868A patent/EA202090868A1/ru unknown
-
2020
- 2020-03-24 IL IL273541A patent/IL273541A/en unknown
-
2023
- 2023-05-18 JP JP2023082312A patent/JP2023103421A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
IL273541A (en) | 2020-05-31 |
MX2020007148A (es) | 2020-10-08 |
BR112020006300A2 (pt) | 2020-10-20 |
JP2020536125A (ja) | 2020-12-10 |
DK3688162T3 (da) | 2024-05-13 |
CN111406108A (zh) | 2020-07-10 |
JP7284179B2 (ja) | 2023-05-30 |
JP2023103421A (ja) | 2023-07-26 |
EA202090868A1 (ru) | 2020-09-16 |
SG11202002653UA (en) | 2020-04-29 |
CA3077413A1 (en) | 2019-04-04 |
FI3688162T3 (fi) | 2024-05-15 |
PL3688162T3 (pl) | 2024-06-10 |
EP3688162A1 (en) | 2020-08-05 |
RS65493B1 (sr) | 2024-05-31 |
TWI833708B (zh) | 2024-03-01 |
LT3688162T (lt) | 2024-05-27 |
US20230140670A1 (en) | 2023-05-04 |
EP3688162B1 (en) | 2024-03-06 |
AU2018338915A1 (en) | 2020-04-23 |
PT3688162T (pt) | 2024-04-23 |
KR20200079497A (ko) | 2020-07-03 |
TW201924724A (zh) | 2019-07-01 |
WO2019067992A1 (en) | 2019-04-04 |
EP4385514A2 (en) | 2024-06-19 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
AR113031A1 (es) | Composiciones de nanopartículas lipídicas (lnp) que comprende arn | |
CL2017003436A1 (es) | Compuestos de benzoxacepina oxazolidinona y métodos de uso | |
CY1121843T1 (el) | Σκευασματα ετανερσεπτης σταθεροποιημενα με ιοντα μαγνησιου | |
MX2019003735A (es) | Formulaciones diluibles de cannabinoides y procesos para su preparacion. | |
CY1124380T1 (el) | Συστηματα διαβλεννογονιας και διαδερμικης χορηγησης | |
EA202191559A1 (ru) | Модифицированные аминовые липиды | |
UY36554A (es) | ARNi VARIANTE | |
CL2015001984A1 (es) | (divisional de la sol. 1567-2013) combinaciones de principios activos que comprenden piridiletilbenzamidas y otros principios activos. | |
CL2018000786A1 (es) | Nuevos derivados de pirrolo[2,3-d]pirimidina, un proceso para su preparación y composiciones farmacéuticas que los contienen. | |
BR112013006341A2 (pt) | formulações agrícolas líquidas com estabilidade aperfeiçoada | |
CL2023001550A1 (es) | Sistemas activos dinámicos acromosómicos. | |
BR112019000693A2 (pt) | composições de muc1- car e métodos para uso | |
CR20160418A (es) | Nuevos compuestos biciclicos de 7 eslabones | |
UY34605A (es) | Derivados de arilalquiloxipirimidina e insecticidas agrohortícolas que comprenden dichos derivados como principios activos, y método de uso de los mismos | |
ECSP10010548A (es) | Composiciones y metodos de preparacion y uso de las mismas | |
CO2017004735A2 (es) | Moléculas que tienen utilidad plaguicida e intermediarios, composiciones y procedimientos relacionados con las mismas | |
CR20170425A (es) | Nuevas proteínas específicas para pioverdina y pioquelina | |
BR112017025711A2 (pt) | ?composições, processo para preparação de siloxanos modificados por poliéter e uso das composições inovadoras? | |
CL2017002427A1 (es) | Producción de ácido ribonucleico libre de células. | |
MX2023007137A (es) | Nanomateriales que comprenden acetales unidos a ésteres. | |
CL2018000158A1 (es) | Composiciones y métodos para inhibir la expresión del gen alas1 (divisional de solicitud 20160772) | |
CR20150310A (es) | Solución para preservar conductos vasculares | |
BR112014006608A2 (pt) | formulações com base em dispersões sólidas | |
BR112015018047A8 (pt) | método para preparar composições estáveis que compreendem heparinoide, anestésico de ação aguda e tampão e as mesmas | |
MX2018005785A (es) | Conjugados de anticuerpo anti-cd22-maytansina y metodos de uso de los mismos. |