CA3045122A1 - Delivery of target specific nucleases - Google Patents
Delivery of target specific nucleases Download PDFInfo
- Publication number
- CA3045122A1 CA3045122A1 CA3045122A CA3045122A CA3045122A1 CA 3045122 A1 CA3045122 A1 CA 3045122A1 CA 3045122 A CA3045122 A CA 3045122A CA 3045122 A CA3045122 A CA 3045122A CA 3045122 A1 CA3045122 A1 CA 3045122A1
- Authority
- CA
- Canada
- Prior art keywords
- formula
- alkyl
- cell
- independently
- lnp
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- 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
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted 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
- A61K9/48—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
- 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
-
- 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]
- C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
-
- 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
- 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)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Mycology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Dispersion Chemistry (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662432042P | 2016-12-09 | 2016-12-09 | |
| US62/432,042 | 2016-12-09 | ||
| US201762458373P | 2017-02-13 | 2017-02-13 | |
| US62/458,373 | 2017-02-13 | ||
| US201762503470P | 2017-05-09 | 2017-05-09 | |
| US62/503,470 | 2017-05-09 | ||
| US201762559186P | 2017-09-15 | 2017-09-15 | |
| US62/559,186 | 2017-09-15 | ||
| PCT/US2017/065303 WO2018107026A1 (en) | 2016-12-09 | 2017-12-08 | Delivery of target specific nucleases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3045122A1 true CA3045122A1 (en) | 2018-06-14 |
Family
ID=62492169
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3045122A Pending CA3045122A1 (en) | 2016-12-09 | 2017-12-08 | Delivery of target specific nucleases |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20180185516A1 (https=) |
| EP (1) | EP3551169A4 (https=) |
| JP (2) | JP2020500539A (https=) |
| AU (1) | AU2017374042C1 (https=) |
| CA (1) | CA3045122A1 (https=) |
| WO (1) | WO2018107026A1 (https=) |
Families Citing this family (79)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HUE060907T2 (hu) | 2014-06-25 | 2023-04-28 | Acuitas Therapeutics Inc | Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére |
| PT3313829T (pt) | 2015-06-29 | 2024-07-08 | Acuitas Therapeutics Inc | Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos |
| HRP20230209T1 (hr) | 2015-10-28 | 2023-04-14 | Acuitas Therapeutics Inc. | Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina |
| EP3995574A1 (en) | 2016-08-24 | 2022-05-11 | Sangamo Therapeutics, Inc. | Regulation of gene expression using engineered nucleases |
| KR102455249B1 (ko) | 2016-08-24 | 2022-10-17 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
| KR102712926B1 (ko) | 2016-10-20 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 파브리병의 치료를 위한 방법 및 조성물 |
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| RU2019126483A (ru) | 2017-01-23 | 2021-02-24 | Ридженерон Фармасьютикалз, Инк. | Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| WO2018191657A1 (en) | 2017-04-13 | 2018-10-18 | Acuitas Therapeutics, Inc. | Lipids for delivery of active agents |
| CN110799492B (zh) * | 2017-04-28 | 2023-06-27 | 爱康泰生治疗公司 | 用于递送核酸的新型羰基脂质和脂质纳米颗粒制剂 |
| KR20200033259A (ko) | 2017-07-31 | 2020-03-27 | 리제너론 파마슈티칼스 인코포레이티드 | 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물 |
| CN110891420B (zh) | 2017-07-31 | 2022-06-03 | 瑞泽恩制药公司 | Cas转基因小鼠胚胎干细胞和小鼠及其应用 |
| CA3073020A1 (en) | 2017-08-16 | 2019-02-21 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| EP4501322A3 (en) | 2017-08-17 | 2025-04-16 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| WO2019036030A1 (en) | 2017-08-17 | 2019-02-21 | Acuitas Therapeutics, Inc. | LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS |
| WO2019036028A1 (en) | 2017-08-17 | 2019-02-21 | Acuitas Therapeutics, Inc. | LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS |
| AU2018348195B2 (en) | 2017-10-11 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| CA3089587A1 (en) | 2018-02-08 | 2019-08-15 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
| EP3592140A1 (en) | 2018-03-19 | 2020-01-15 | Regeneron Pharmaceuticals, Inc. | Transcription modulation in animals using crispr/cas systems |
| MA52074A (fr) | 2018-03-19 | 2021-01-27 | Bayer Healthcare Llc | Nouveaux systèmes d'endonucléase à arn programmable et leurs utilisations |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| US12479791B2 (en) | 2018-08-10 | 2025-11-25 | Takeda Pharmaceutical Company Limited | Cationic lipid |
| EP3842538A4 (en) | 2018-08-10 | 2022-06-08 | Kyoto University | METHOD OF TRANSFECTION INTO CARDIOMYOCYTES USING CATIONIC LIPID |
| KR20210060533A (ko) * | 2018-09-18 | 2021-05-26 | 상가모 테라퓨틱스, 인코포레이티드 | 프로그램화된 세포 사멸 1 (pd1) 특이적 뉴클레아제 |
| IL281615B2 (en) | 2018-09-21 | 2026-01-01 | Acuitas Therapeutics Inc | Systems and methods for producing lipid nanoparticles and liposomes |
| HRP20240586T1 (hr) | 2018-10-09 | 2024-07-19 | The University Of British Columbia | Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima |
| CA3116576A1 (en) * | 2018-10-18 | 2020-04-23 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| CN113710799B (zh) * | 2018-11-28 | 2024-11-12 | 克里斯珀医疗股份公司 | 用于在LNP中使用的编码CAS9的优化mRNA |
| PT3908568T (pt) | 2019-01-11 | 2024-09-30 | Acuitas Therapeutics Inc | Lípidos para a administração de agentes ativos por nanopartículas lipídicas |
| MX2021015122A (es) | 2019-06-07 | 2022-04-06 | Regeneron Pharma | Animales no humanos que comprenden un locus de albumina humanizado. |
| JP7630904B2 (ja) * | 2019-07-23 | 2025-02-18 | 株式会社東芝 | 核酸導入キャリア、核酸導入キャリアセット、核酸導入組成物及び核酸導入方法 |
| CN114901253A (zh) * | 2019-08-14 | 2022-08-12 | 爱康泰生治疗公司 | 用于递送核酸的改进的脂质纳米颗粒 |
| EP4048807A1 (en) | 2019-09-23 | 2022-08-31 | Omega Therapeutics, Inc. | Compositions and methods for modulating apolipoprotein b (apob) gene expression |
| CN114729376A (zh) | 2019-09-23 | 2022-07-08 | 欧米茄治疗公司 | 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法 |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| CA3173528A1 (en) | 2020-03-11 | 2021-09-16 | Omega Therapeutics, Inc. | Compositions and methods for modulating forkhead box p3 (foxp3) gene expression |
| US12194157B2 (en) | 2020-04-09 | 2025-01-14 | Finncure Oy | Carrier for targeted delivery to a host |
| FI20215508A1 (en) | 2020-04-09 | 2021-10-10 | Niemelae Erik Johan | Mimetic nanoparticles to prevent the spread of new coronaviruses and reduce the rate of infection |
| WO2021204175A1 (en) | 2020-04-09 | 2021-10-14 | Suzhou Abogen Biosciences Co., Ltd. | Lipid nanoparticle composition |
| EP4132576A1 (en) | 2020-04-09 | 2023-02-15 | Suzhou Abogen Biosciences Co., Ltd. | Nucleic acid vaccines for coronavirus |
| CA3181340A1 (en) * | 2020-04-28 | 2021-11-04 | Intellia Therapeutics, Inc. | Methods of in vitro cell delivery |
| JP2023534035A (ja) * | 2020-07-15 | 2023-08-07 | ザ ジャクソン ラボラトリー | ＳＡＲＳ－ＣｏＶ－２感染のためのヒト化マウスモデル |
| ES3054438T3 (en) | 2020-07-16 | 2026-02-03 | Acuitas Therapeutics Inc | Cationic lipids for use in lipid nanoparticles |
| 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 |
| EP4228658A4 (en) | 2020-10-14 | 2025-02-19 | George Mason Research Foundation, Inc. | IONIZABLE LIPIDS AND METHODS FOR THE PRODUCTION AND USE THEREOF |
| WO2022236010A1 (en) * | 2021-05-06 | 2022-11-10 | The University Of North Carolina At Chapel Hill | Use of chemical epigenetic modifiers to modulate gene expression from vectors |
| EP4367242A2 (en) | 2021-07-07 | 2024-05-15 | Omega Therapeutics, Inc. | Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression |
| US20240398940A1 (en) | 2021-09-03 | 2024-12-05 | CureVac SE | Novel lipid nanoparticles for delivery of nucleic acids |
| CA3229889A1 (en) | 2021-09-03 | 2023-03-09 | Glaxosmithkline Biologicals Sa | Substitution of nucleotide bases in self-amplifying messenger ribonucleic acids |
| AR127312A1 (es) * | 2021-10-08 | 2024-01-10 | Suzhou Abogen Biosciences Co Ltd | Compuestos lipídicos ycomposiciones de nanopartículas lipídicas |
| WO2023069948A1 (en) | 2021-10-18 | 2023-04-27 | Flagship Pioneering Innovations Vii, Llc | Dna compositions and related methods |
| EP4422698A1 (en) | 2021-10-29 | 2024-09-04 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
| KR20240117571A (ko) | 2021-12-08 | 2024-08-01 | 리제너론 파마슈티칼스 인코포레이티드 | 돌연변이 마이오실린 질환 모델 및 이의 용도 |
| CA3242402A1 (en) | 2021-12-16 | 2023-06-22 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| US20250099614A1 (en) | 2022-01-28 | 2025-03-27 | CureVac SE | Nucleic acid encoded transcription factor inhibitors |
| US20250345407A1 (en) | 2022-05-25 | 2025-11-13 | CureVac SE | Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide |
| WO2023242817A2 (en) | 2022-06-18 | 2023-12-21 | Glaxosmithkline Biologicals Sa | Recombinant rna molecules comprising untranslated regions or segments encoding spike protein from the omicron strain of severe acute respiratory coronavirus-2 |
| CN114989182B (zh) * | 2022-06-23 | 2023-06-23 | 尧唐(上海)生物科技有限公司 | 脂质化合物、包含其的组合物及应用 |
| EP4608442A1 (en) | 2022-10-28 | 2025-09-03 | GlaxoSmithKline Biologicals S.A. | Nucleic acid based vaccine |
| WO2024133160A1 (en) | 2022-12-19 | 2024-06-27 | Glaxosmithkline Biologicals Sa | Hepatitis b compositions |
| WO2024184500A1 (en) | 2023-03-08 | 2024-09-12 | CureVac SE | Novel lipid nanoparticle formulations for delivery of nucleic acids |
| WO2024230934A1 (en) | 2023-05-11 | 2024-11-14 | CureVac SE | Therapeutic nucleic acid for the treatment of ophthalmic diseases |
| EP4716741A2 (en) | 2023-05-23 | 2026-04-01 | Flagship Labs 114, Inc. | Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression |
| WO2025010554A1 (en) * | 2023-07-07 | 2025-01-16 | Yoltech Therapeutics Co., Ltd. | Lipid compounds for delivery of therapeutic agents and preparation method and its use thereof |
| US20250161227A1 (en) | 2023-11-17 | 2025-05-22 | Acuitas Therapeutics, Inc. | Pegylated lipids |
| WO2025128696A1 (en) | 2023-12-12 | 2025-06-19 | Acuitas Therapeutics, Inc. | Cationic lipid compounds for use in lipid nanoparticles |
| WO2025132122A1 (en) | 2023-12-13 | 2025-06-26 | Berlin Institute Of Health | Methods of delivering therapeutics using lipid nanoparticles |
| WO2025126071A1 (en) | 2023-12-14 | 2025-06-19 | Pfizer Inc. | Rna molecules |
| WO2025129128A1 (en) | 2023-12-15 | 2025-06-19 | Vivasor, Inc. | Compositions and methods for non-viral delivery of therapeutic compounds |
| WO2025174858A1 (en) | 2024-02-15 | 2025-08-21 | Acuitas Therapeutics, Inc. | Cationic lipid compounds for use in lipid nanoparticles |
| WO2025184508A1 (en) | 2024-03-01 | 2025-09-04 | Acuitas Therapeutics, Inc. | Materials and methods for encapsulating therapeutics in lipid nanoparticles |
| WO2025186725A2 (en) | 2024-03-06 | 2025-09-12 | Pfizer Inc. | Improved lnp formulations and uses thereof |
| WO2025217264A1 (en) | 2024-04-10 | 2025-10-16 | Acuitas Therapeutics, Inc. | Cationic lipid compounds for use in lipid nanoparticles |
| WO2025231114A1 (en) | 2024-05-01 | 2025-11-06 | Acuitas Therapeutics, Inc. | Method of using lipid nanoparticles for intramuscular delivery |
| WO2025229572A1 (en) | 2024-05-01 | 2025-11-06 | Glaxosmithkline Biologicals Sa | Epstein-barr virus antigen-encoding messenger ribonucleic acid and antigen protein vaccines |
| WO2025240833A1 (en) | 2024-05-17 | 2025-11-20 | Acuitas Therapeutics, Inc. | Galnac lipid compounds for use in lipid nanoparticles |
| WO2026018181A1 (en) | 2024-07-17 | 2026-01-22 | Pfizer Inc. | Immunogenic compositions and uses thereof |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2848417C (en) * | 2011-09-21 | 2023-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| CA3018046A1 (en) * | 2011-12-16 | 2013-06-20 | Moderna Therapeutics, Inc. | Modified nucleoside, nucleotide, and nucleic acid compositions |
| AU2013240051B2 (en) * | 2012-03-27 | 2017-11-30 | Sirna Therapeutics, Inc. | Diether based biodegradable cationic lipids for siRNA delivery |
| US9255250B2 (en) * | 2012-12-05 | 2016-02-09 | Sangamo Bioscience, Inc. | Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene |
| CN105940013B (zh) * | 2013-12-09 | 2020-03-27 | 桑格摩生物科学股份有限公司 | 用于治疗血友病的方法和组合物 |
| HUE060907T2 (hu) * | 2014-06-25 | 2023-04-28 | Acuitas Therapeutics Inc | Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére |
| WO2016014794A1 (en) * | 2014-07-25 | 2016-01-28 | Sangamo Biosciences, Inc. | Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells |
| AU2016253972B2 (en) * | 2015-04-27 | 2020-01-02 | Acuitas Therapeutics Inc. | Nucleoside-modified RNA for inducing an adaptive immune response |
| AU2016344609B2 (en) * | 2015-10-28 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of duchenne muscular dystrophy |
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2017
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- 2017-12-08 US US15/835,957 patent/US20180185516A1/en not_active Abandoned
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- 2017-12-08 EP EP17879571.2A patent/EP3551169A4/en not_active Withdrawn
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2022
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| EP3551169A4 (en) | 2020-09-09 |
| AU2017374042B2 (en) | 2024-02-15 |
| JP2022121531A (ja) | 2022-08-19 |
| WO2018107026A1 (en) | 2018-06-14 |
| EP3551169A1 (en) | 2019-10-16 |
| JP2020500539A (ja) | 2020-01-16 |
| AU2017374042C1 (en) | 2024-07-11 |
| US20180185516A1 (en) | 2018-07-05 |
| AU2017374042A1 (en) | 2019-06-13 |
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