JP2020500539A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020500539A5 JP2020500539A5 JP2019530472A JP2019530472A JP2020500539A5 JP 2020500539 A5 JP2020500539 A5 JP 2020500539A5 JP 2019530472 A JP2019530472 A JP 2019530472A JP 2019530472 A JP2019530472 A JP 2019530472A JP 2020500539 A5 JP2020500539 A5 JP 2020500539A5
- Authority
- JP
- Japan
- Prior art keywords
- alkyl
- independently
- carbon
- composition
- 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
- 229910052799 carbon Inorganic materials 0.000 claims description 77
- 125000000217 alkyl group Chemical group 0.000 claims description 63
- 150000001721 carbon Chemical group 0.000 claims description 32
- 108700019146 Transgenes Proteins 0.000 claims description 22
- 238000000034 method Methods 0.000 claims description 20
- 239000008194 pharmaceutical composition Substances 0.000 claims description 20
- 108091033319 polynucleotide Proteins 0.000 claims description 19
- 102000040430 polynucleotide Human genes 0.000 claims description 19
- 239000002157 polynucleotide Substances 0.000 claims description 19
- 101710163270 Nuclease Proteins 0.000 claims description 18
- 239000011203 carbon fibre reinforced carbon Substances 0.000 claims description 18
- 125000002947 alkylene group Chemical group 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 13
- 125000004450 alkenylene group Chemical group 0.000 claims description 11
- 150000002632 lipids Chemical class 0.000 claims description 10
- 239000013603 viral vector Substances 0.000 claims description 10
- 125000003342 alkenyl group Chemical group 0.000 claims description 8
- 238000003776 cleavage reaction Methods 0.000 claims description 8
- 230000007017 scission Effects 0.000 claims description 8
- 108020004414 DNA Proteins 0.000 claims description 6
- 108091023040 Transcription factor Proteins 0.000 claims description 6
- 102000040945 Transcription factor Human genes 0.000 claims description 6
- 150000001875 compounds Chemical class 0.000 claims description 6
- 229910052757 nitrogen Inorganic materials 0.000 claims description 6
- 125000004433 nitrogen atom Chemical group N* 0.000 claims description 6
- -1 cationic lipid Chemical class 0.000 claims description 5
- 125000005724 cycloalkenylene group Chemical group 0.000 claims description 5
- 125000002993 cycloalkylene group Chemical group 0.000 claims description 5
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 4
- 201000010099 disease Diseases 0.000 claims description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 4
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 4
- 150000003431 steroids Chemical class 0.000 claims description 4
- 108700026220 vif Genes Proteins 0.000 claims description 4
- 230000027455 binding Effects 0.000 claims description 3
- 150000003839 salts Chemical class 0.000 claims description 3
- 230000001225 therapeutic effect Effects 0.000 claims description 3
- 125000004070 6 membered heterocyclic group Chemical group 0.000 claims description 2
- 125000003341 7 membered heterocyclic group Chemical group 0.000 claims description 2
- 108010088751 Albumins Proteins 0.000 claims description 2
- 102000009027 Albumins Human genes 0.000 claims description 2
- 101150017501 CCR5 gene Proteins 0.000 claims description 2
- 230000004568 DNA-binding Effects 0.000 claims description 2
- 108020005004 Guide RNA Proteins 0.000 claims description 2
- 101001000998 Homo sapiens Protein phosphatase 1 regulatory subunit 12C Proteins 0.000 claims description 2
- 108010091358 Hypoxanthine Phosphoribosyltransferase Proteins 0.000 claims description 2
- 235000011449 Rosa Nutrition 0.000 claims description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 claims description 2
- 101710185494 Zinc finger protein Proteins 0.000 claims description 2
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 2
- 125000004432 carbon atom Chemical group C* 0.000 claims description 2
- 125000000753 cycloalkyl group Chemical group 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 238000012239 gene modification Methods 0.000 claims description 2
- 230000005017 genetic modification Effects 0.000 claims description 2
- 235000013617 genetically modified food Nutrition 0.000 claims description 2
- 125000000623 heterocyclic group Chemical group 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims description 2
- 238000001727 in vivo Methods 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 230000010354 integration Effects 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 239000002105 nanoparticle Substances 0.000 claims description 2
- 230000007935 neutral effect Effects 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 229920000642 polymer Polymers 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 230000001105 regulatory effect Effects 0.000 claims description 2
- 230000002103 transcriptional effect Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 21
- 241001481833 Coryphaena hippurus Species 0.000 claims 1
- 241001125840 Coryphaenidae Species 0.000 claims 1
- 125000003710 aryl alkyl group Chemical group 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 0 CC(CCOC(C)C(N(*)*)=O)OC Chemical compound CC(CCOC(C)C(N(*)*)=O)OC 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 239000000651 prodrug Substances 0.000 description 1
- 229940002612 prodrug Drugs 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022101160A JP2022121531A (ja) | 2016-12-09 | 2022-06-23 | 標的特異的ヌクレアーゼの送達 |
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022101160A Division JP2022121531A (ja) | 2016-12-09 | 2022-06-23 | 標的特異的ヌクレアーゼの送達 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020500539A JP2020500539A (ja) | 2020-01-16 |
| JP2020500539A5 true JP2020500539A5 (https=) | 2021-01-21 |
Family
ID=62492169
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019530472A Pending JP2020500539A (ja) | 2016-12-09 | 2017-12-08 | 標的特異的ヌクレアーゼの送達 |
| JP2022101160A Pending JP2022121531A (ja) | 2016-12-09 | 2022-06-23 | 標的特異的ヌクレアーゼの送達 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022101160A Pending JP2022121531A (ja) | 2016-12-09 | 2022-06-23 | 標的特異的ヌクレアーゼの送達 |
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 |
|---|---|---|---|---|
| SMT202200502T1 (it) | 2014-06-25 | 2023-01-13 | Acuitas Therapeutics Inc | Nuovi lipidi e formulazioni di nanoparticelle lipidiche per l'erogazione di acidi nucleici |
| LT3313829T (lt) | 2015-06-29 | 2024-08-12 | Acuitas Therapeutics Inc. | Lipidai ir lipidų nanodalelių sudėtys, skirtos nukleorūgščių tiekimui |
| IL307179A (en) | 2015-10-28 | 2023-11-01 | Acuitas Therapeutics Inc | Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| LT3504229T (lt) | 2016-08-24 | 2021-12-10 | Sangamo Therapeutics, Inc. | Genų raiškos reguliavimas, panaudojant rekombinantines nukleazes |
| SG10201913948PA (en) | 2016-08-24 | 2020-03-30 | Sangamo Therapeutics Inc | Engineered target specific nucleases |
| CA3039673A1 (en) | 2016-10-20 | 2018-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| IL314915B1 (en) | 2017-01-23 | 2026-02-01 | Regeneron Pharma | Variants of 17-beta-hydroxysteroid dehydrogenase and their uses |
| 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 |
| WO2018200943A1 (en) * | 2017-04-28 | 2018-11-01 | Acuitas Therapeutics, Inc. | Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| CN111182790A (zh) | 2017-07-31 | 2020-05-19 | 瑞泽恩制药公司 | Crispr报告体非人类动物及其用途 |
| WO2019028032A1 (en) | 2017-07-31 | 2019-02-07 | Regeneron Pharmaceuticals, Inc. | EMBRYONIC STEM CELLS OF TRANSGENIC MOUSE CASES AND MICE AND USES THEREOF |
| CA3073020A1 (en) * | 2017-08-16 | 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 |
| US11524932B2 (en) | 2017-08-17 | 2022-12-13 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| EP3668834B1 (en) | 2017-08-17 | 2024-10-02 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| US10961583B2 (en) | 2017-10-11 | 2021-03-30 | Regeneron Phramaceuticals, 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 |
| JP7550648B2 (ja) | 2018-03-19 | 2024-09-13 | クリスパー セラピューティクス アーゲー | 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用 |
| CN111885915B (zh) | 2018-03-19 | 2023-04-28 | 瑞泽恩制药公司 | 使用crispr/cas系统对动物进行转录调制 |
| JP7507093B2 (ja) | 2018-03-21 | 2024-06-27 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法 |
| DK3835287T3 (da) | 2018-08-10 | 2025-01-02 | Takeda Pharmaceuticals Co | Kationisk lipid |
| JP7436990B2 (ja) * | 2018-08-10 | 2024-02-22 | 国立大学法人京都大学 | カチオン性脂質を用いた心筋細胞へのトランスフェクション方法 |
| WO2020061161A1 (en) * | 2018-09-18 | 2020-03-26 | Sangamo Therapeutics, Inc. | Programmed cell death 1 (pd1) specific nucleases |
| IL281615B2 (en) | 2018-09-21 | 2026-01-01 | Acuitas Therapeutics Inc | Systems and methods for producing lipid nanoparticles and liposomes |
| CN113166783B (zh) | 2018-10-09 | 2024-10-11 | 不列颠哥伦比亚大学 | 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法 |
| JP7578590B2 (ja) | 2018-10-18 | 2024-11-06 | インテリア セラピューティクス，インコーポレーテッド | 第ｉｘ因子を発現するための組成物及び方法 |
| JP7543259B2 (ja) * | 2018-10-18 | 2024-09-02 | アクイタス セラピューティクス インコーポレイテッド | 活性剤の脂質ナノ粒子送達のための脂質 |
| CA3121191A1 (en) * | 2018-11-28 | 2020-06-04 | Crispr Therapeutics Ag | Optimized mrna encoding cas9 for use in lnps |
| MY202837A (en) | 2019-01-11 | 2024-05-24 | Acuitas Therapeutics Inc | Lipids for lipid nanoparticle delivery of active agents |
| US11622547B2 (en) | 2019-06-07 | 2023-04-11 | Regeneran Pharmaceuticals, Inc. | Genetically modified mouse that expresses human albumin |
| 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 |
| US20230097090A1 (en) * | 2019-08-14 | 2023-03-30 | Acuitas Therapeutics, Inc. | Improved lipid nanoparticles for delivery of nucleic acids |
| AU2020355000A1 (en) | 2019-09-23 | 2022-03-17 | 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 |
| CN116096886A (zh) | 2020-03-11 | 2023-05-09 | 欧米茄治疗公司 | 用于调节叉头框p3(foxp3)基因表达的组合物和方法 |
| EP4132576A1 (en) | 2020-04-09 | 2023-02-15 | Suzhou Abogen Biosciences Co., Ltd. | Nucleic acid vaccines for coronavirus |
| US12194157B2 (en) | 2020-04-09 | 2025-01-14 | Finncure Oy | Carrier for targeted delivery to a host |
| EP4132478A1 (en) | 2020-04-09 | 2023-02-15 | Finncure Oy | Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses |
| JP7681617B2 (ja) | 2020-04-09 | 2025-05-22 | スージョウ・アボジェン・バイオサイエンシズ・カンパニー・リミテッド | 脂質ナノ粒子組成物 |
| KR20230017783A (ko) * | 2020-04-28 | 2023-02-06 | 인텔리아 테라퓨틱스, 인크. | 시험관내 세포 전달 방법 |
| 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 |
| WO2022016070A1 (en) | 2020-07-16 | 2022-01-20 | 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 |
| JP2023546175A (ja) | 2020-10-14 | 2023-11-01 | ジョージ・メイソン・リサーチ・ファウンデーション・インコーポレイテッド | 脂質ナノ粒子製造の方法及びそれに由来する組成物 |
| US20240238448A1 (en) * | 2021-05-06 | 2024-07-18 | 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 |
| MX2024002726A (es) | 2021-09-03 | 2024-03-20 | CureVac SE | Nuevas nanoparticulas lipidicas para la administracion de acidos nucleicos. |
| JP2024534915A (ja) | 2021-09-03 | 2024-09-26 | グラクソスミスクライン バイオロジカルズ ソシエテ アノニム | 自己増幅性メッセンジャーリボ核酸におけるヌクレオチド塩基の置換 |
| AR127312A1 (es) | 2021-10-08 | 2024-01-10 | Suzhou Abogen Biosciences Co Ltd | Compuestos lipídicos ycomposiciones de nanopartículas lipídicas |
| US20250027107A1 (en) | 2021-10-18 | 2025-01-23 | Flagship Pioneering Innovations Vii, Llc | Dna compositions and related methods |
| WO2023073228A1 (en) | 2021-10-29 | 2023-05-04 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
| CN118632622A (zh) | 2021-12-08 | 2024-09-10 | 瑞泽恩制药公司 | 突变型肌纤蛋白疾病模型及其用途 |
| 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 |
| WO2023227608A1 (en) | 2022-05-25 | 2023-11-30 | Glaxosmithkline Biologicals Sa | 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 |
| WO2024089638A1 (en) | 2022-10-28 | 2024-05-02 | Glaxosmithkline Biologicals Sa | Nucleic acid based vaccine |
| WO2024133160A1 (en) | 2022-12-19 | 2024-06-27 | Glaxosmithkline Biologicals Sa | Hepatitis b compositions |
| KR20250153298A (ko) | 2023-03-08 | 2025-10-24 | 큐어백 에스이 | 핵산 전달을 위한 신규의 지질 나노입자 제형 |
| WO2024230934A1 (en) | 2023-05-11 | 2024-11-14 | CureVac SE | Therapeutic nucleic acid for the treatment of ophthalmic diseases |
| WO2024243438A2 (en) | 2023-05-23 | 2024-11-28 | Omega Therapeutics, 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 |
| TW202543583A (zh) | 2024-03-01 | 2025-11-16 | 加拿大商阿奎特斯醫療公司 | 用於將治療劑包覆於脂質奈米顆粒中之材料及方法 |
| WO2025186725A2 (en) | 2024-03-06 | 2025-09-12 | Pfizer Inc. | Improved lnp formulations and uses thereof |
| TW202602839A (zh) | 2024-04-10 | 2026-01-16 | 加拿大商阿奎特斯醫療公司 | 用於脂質奈米顆粒之陽離子脂質化合物 |
| 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 |
|---|---|---|---|---|
| AU2012312260B2 (en) * | 2011-09-21 | 2017-08-31 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| CN104114572A (zh) * | 2011-12-16 | 2014-10-22 | 现代治疗公司 | 经修饰的核苷、核苷酸和核酸组合物 |
| WO2013148541A1 (en) * | 2012-03-27 | 2013-10-03 | Merck Sharp & Dohme Corp. | DIETHER BASED BIODEGRADABLE CATIONIC LIPIDS FOR siRNA DELIVERY |
| WO2014089212A1 (en) * | 2012-12-05 | 2014-06-12 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of metabolic disorders |
| DK3080274T3 (da) * | 2013-12-09 | 2020-08-31 | Sangamo Therapeutics Inc | Fremgangsmåder og sammensætninger til genom-manipulation |
| SMT202200502T1 (it) * | 2014-06-25 | 2023-01-13 | Acuitas Therapeutics Inc | Nuovi lipidi e formulazioni di nanoparticelle lipidiche per l'erogazione di acidi nucleici |
| US9816074B2 (en) * | 2014-07-25 | 2017-11-14 | Sangamo Therapeutics, Inc. | Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells |
| US20180303925A1 (en) * | 2015-04-27 | 2018-10-25 | The Trustees Of The University Of Pennsylvania | Nucleoside-Modified RNA For Inducing an Adaptive Immune Response |
| JP2019507579A (ja) * | 2015-10-28 | 2019-03-22 | クリスパー セラピューティクス アーゲー | デュシェンヌ型筋ジストロフィーの処置のための材料および方法 |
-
2017
- 2017-12-08 EP EP17879571.2A patent/EP3551169A4/en not_active Withdrawn
- 2017-12-08 WO PCT/US2017/065303 patent/WO2018107026A1/en not_active Ceased
- 2017-12-08 AU AU2017374042A patent/AU2017374042C1/en not_active Ceased
- 2017-12-08 US US15/835,957 patent/US20180185516A1/en not_active Abandoned
- 2017-12-08 JP JP2019530472A patent/JP2020500539A/ja active Pending
- 2017-12-08 CA CA3045122A patent/CA3045122A1/en active Pending
-
2022
- 2022-06-23 JP JP2022101160A patent/JP2022121531A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020500539A5 (https=) | ||
| US20230355654A1 (en) | Rnai therapy for hepatitis b virus infection | |
| JP7766583B2 (ja) | 強化された薬剤送達のための組成物及び方法 | |
| TWI811238B (zh) | 用於抑制脂蛋白元C-III(APOC3)表現之RNAi試劑及組合物 | |
| ES2765463T3 (es) | Acido nucleico antisentido para usar en el tratamiento de la distrofia muscular de Duchenne | |
| US20200009268A1 (en) | Expression constructs and systems for systemic and non-specific in vivo delivery of a nucleic acid to human cells and transient target cell-specific production of a therapeutic protein | |
| JP6715325B2 (ja) | ｓｉＲＮＡ、ｓｉＲＮＡを含む医薬組成物及び結合体、並びにそれらの応用 | |
| US8309356B2 (en) | Pseudocomplementary oligonucleotides for targeted gene therapy | |
| CA3229270A1 (en) | Rnai agents for hepatitis b virus infection | |
| WO2021113365A1 (en) | Nanomaterials | |
| EP3087988A2 (en) | Use of inhibitors of toll-like receptors in the prevention and treatment of hypercholesterolemia and hyperlipidemia and diseases related thereto | |
| BR112016020618B1 (pt) | Oligômero antissentido, composição farmacêutica, e, uso de um oligômero antissentido | |
| US20220259597A1 (en) | Oligonucleotide antagonists for rna guided genome editing | |
| CN102459301A (zh) | 亲脂性多聚核苷酸缀合物 | |
| TW201907009A (zh) | 用於治療b型肝炎之治療組成物及方法 | |
| JP2021526505A (ja) | 治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法 | |
| KR20240118881A (ko) | 항푸소제닉 폴리펩티드를 인코딩하는 원형 폴리리보뉴클레오티드 | |
| JP2024545602A (ja) | Ｈｓｄ１７ｂ１３関連二本鎖オリゴヌクレオチド組成物及びそれに関連する方法 | |
| KR101685304B1 (ko) | 간 조직 특이적 유전자 발현 억제인자 전달용 나노리포좀 | |
| US20220106608A1 (en) | Fusogenic lipid nanoparticles for the target cell-specific production of rapamycin inducible therapeutic proteins | |
| US20210139870A1 (en) | Anti-hbv combination therapies involving specific endonucleases | |
| WO2019183005A1 (en) | 2'f-ana-let7 mediated utrophin upregulation for dmd therapy | |
| WO2024059878A9 (en) | Drugs targeting telomerase reverse transcriptase (tert) for treatment of cancers | |
| WO2023233290A1 (en) | Rnai agents targeting pd-l1 | |
| WO2020044349A1 (en) | Compounds, cojugates and compositions for use in the methods for trans-membrane delivery of molecules |