JP2024038327A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2024038327A5 JP2024038327A5 JP2024002319A JP2024002319A JP2024038327A5 JP 2024038327 A5 JP2024038327 A5 JP 2024038327A5 JP 2024002319 A JP2024002319 A JP 2024002319A JP 2024002319 A JP2024002319 A JP 2024002319A JP 2024038327 A5 JP2024038327 A5 JP 2024038327A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- seq
- composition
- protein
- nucleotide
- 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
- 125000003729 nucleotide group Chemical group 0.000 claims 79
- 239000002773 nucleotide Substances 0.000 claims 66
- 108090000623 proteins and genes Proteins 0.000 claims 47
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 32
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims 28
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 26
- 102000004169 proteins and genes Human genes 0.000 claims 25
- 125000006850 spacer group Chemical group 0.000 claims 25
- 102100025668 Angiopoietin-related protein 3 Human genes 0.000 claims 22
- 101710085848 Angiopoietin-related protein 3 Proteins 0.000 claims 22
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims 20
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 18
- 108020004999 messenger RNA Proteins 0.000 claims 18
- 230000004568 DNA-binding Effects 0.000 claims 17
- 229940113082 thymine Drugs 0.000 claims 16
- MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 claims 14
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 claims 14
- 229940029575 guanosine Drugs 0.000 claims 14
- UHDGCWIWMRVCDJ-UHFFFAOYSA-N 1-beta-D-Xylofuranosyl-NH-Cytosine Natural products O=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 UHDGCWIWMRVCDJ-UHFFFAOYSA-N 0.000 claims 13
- UHDGCWIWMRVCDJ-PSQAKQOGSA-N Cytidine Natural products O=C1N=C(N)C=CN1[C@@H]1[C@@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-PSQAKQOGSA-N 0.000 claims 13
- UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-ZAKLUEHWSA-N 0.000 claims 13
- 229940035893 uracil Drugs 0.000 claims 13
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 12
- 108091033319 polynucleotide Proteins 0.000 claims 12
- 102000040430 polynucleotide Human genes 0.000 claims 12
- 239000002157 polynucleotide Substances 0.000 claims 12
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 claims 10
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 10
- 102000039446 nucleic acids Human genes 0.000 claims 10
- 108020004707 nucleic acids Proteins 0.000 claims 10
- 150000007523 nucleic acids Chemical class 0.000 claims 10
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 10
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims 10
- 229940045145 uridine Drugs 0.000 claims 10
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims 9
- 229960005305 adenosine Drugs 0.000 claims 9
- 108020005067 RNA Splice Sites Proteins 0.000 claims 6
- 238000012986 modification Methods 0.000 claims 6
- 230000004048 modification Effects 0.000 claims 6
- 229930024421 Adenine Natural products 0.000 claims 5
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 5
- 229960000643 adenine Drugs 0.000 claims 5
- 229940104302 cytosine Drugs 0.000 claims 5
- 238000010459 TALEN Methods 0.000 claims 4
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 4
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims 3
- 102000055025 Adenosine deaminases Human genes 0.000 claims 3
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 3
- 108091036066 Three prime untranslated region Proteins 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 3
- 102000008682 Argonaute Proteins Human genes 0.000 claims 2
- 108010088141 Argonaute Proteins Proteins 0.000 claims 2
- 108091033409 CRISPR Proteins 0.000 claims 2
- 108700004991 Cas12a Proteins 0.000 claims 2
- 102100026846 Cytidine deaminase Human genes 0.000 claims 2
- 108010031325 Cytidine deaminase Proteins 0.000 claims 2
- 208000030673 Homozygous familial hypercholesterolemia Diseases 0.000 claims 2
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims 2
- 238000001802 infusion Methods 0.000 claims 2
- 238000001990 intravenous administration Methods 0.000 claims 2
- 230000030648 nucleus localization Effects 0.000 claims 2
- 108010052418 (N-(2-((4-((2-((4-(9-acridinylamino)phenyl)amino)-2-oxoethyl)amino)-4-oxobutyl)amino)-1-(1H-imidazol-4-ylmethyl)-1-oxoethyl)-6-(((-2-aminoethyl)amino)methyl)-2-pyridinecarboxamidato) iron(1+) Proteins 0.000 claims 1
- UVBYMVOUBXYSFV-XUTVFYLZSA-N 1-methylpseudouridine Chemical group O=C1NC(=O)N(C)C=C1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 UVBYMVOUBXYSFV-XUTVFYLZSA-N 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims 1
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
Applications Claiming Priority (12)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063007797P | 2020-04-09 | 2020-04-09 | |
| US202063007803P | 2020-04-09 | 2020-04-09 | |
| US63/007,803 | 2020-04-09 | ||
| US63/007,797 | 2020-04-09 | ||
| US202063045033P | 2020-06-26 | 2020-06-26 | |
| US202063045032P | 2020-06-26 | 2020-06-26 | |
| US63/045,033 | 2020-06-26 | ||
| US63/045,032 | 2020-06-26 | ||
| US202163136087P | 2021-01-11 | 2021-01-11 | |
| US63/136,087 | 2021-01-11 | ||
| PCT/US2021/026729 WO2021207710A2 (en) | 2020-04-09 | 2021-04-09 | Base editing of angptl3 and methods of using same for treatment of disease |
| JP2022562030A JP7457833B2 (ja) | 2020-04-09 | 2021-04-09 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022562030A Division JP7457833B2 (ja) | 2020-04-09 | 2021-04-09 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2026081981A Division JP2026133532A (ja) | 2020-04-09 | 2026-05-14 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024038327A JP2024038327A (ja) | 2024-03-19 |
| JP2024038327A5 true JP2024038327A5 (enExample) | 2025-05-14 |
Family
ID=78022701
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022562027A Active JP7457832B2 (ja) | 2020-04-09 | 2021-04-09 | Ｐｃｓｋ９の塩基編集および疾患の処置のためにこれを使用する方法 |
| JP2022562030A Active JP7457833B2 (ja) | 2020-04-09 | 2021-04-09 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
| JP2024002319A Pending JP2024038327A (ja) | 2020-04-09 | 2024-01-11 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
| JP2024002316A Pending JP2024038326A (ja) | 2020-04-09 | 2024-01-11 | Ｐｃｓｋ９の塩基編集および疾患の処置のためにこれを使用する方法 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022562027A Active JP7457832B2 (ja) | 2020-04-09 | 2021-04-09 | Ｐｃｓｋ９の塩基編集および疾患の処置のためにこれを使用する方法 |
| JP2022562030A Active JP7457833B2 (ja) | 2020-04-09 | 2021-04-09 | Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024002316A Pending JP2024038326A (ja) | 2020-04-09 | 2024-01-11 | Ｐｃｓｋ９の塩基編集および疾患の処置のためにこれを使用する方法 |
Country Status (13)
| Country | Link |
|---|---|
| US (5) | US12115230B2 (enExample) |
| EP (3) | EP4133072A4 (enExample) |
| JP (4) | JP7457832B2 (enExample) |
| KR (2) | KR20230016176A (enExample) |
| CN (2) | CN116096429A (enExample) |
| AU (4) | AU2021252991B2 (enExample) |
| BR (2) | BR112022020407A2 (enExample) |
| CA (2) | CA3179863A1 (enExample) |
| GB (8) | GB2623891B (enExample) |
| IL (2) | IL297193A (enExample) |
| MX (1) | MX2022012683A (enExample) |
| PH (2) | PH12022553033A1 (enExample) |
| WO (3) | WO2021207712A2 (enExample) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018224387B2 (en) | 2017-02-22 | 2024-08-08 | Crispr Therapeutics Ag | Compositions and methods for gene editing |
| US12115230B2 (en) * | 2020-04-09 | 2024-10-15 | Verve Therapeutics, Inc. | Base editing of ANGPTL3 and methods of using same for treatment of disease |
| 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 |
| GB2702733A (en) * | 2021-09-22 | 2026-06-24 | Verve Therapeutics Inc | Gene editing of PCSK9 or ANGPTL3 and compositions and methods of using same for treatment of disease |
| CA3235827A1 (en) * | 2021-10-21 | 2023-04-27 | Wei Hsi Yeh | Genome editing compositions and methods for treatment of retinitis pigmentosa |
| WO2023166425A1 (en) * | 2022-03-01 | 2023-09-07 | Crispr Therapeutics Ag | Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions |
| US20230293646A1 (en) * | 2022-03-21 | 2023-09-21 | Crispr Therapeutics Ag | Methods and compositions for treating lipoprotein-related diseases |
| WO2023230587A2 (en) | 2022-05-25 | 2023-11-30 | Akagera Medicines, Inc. | Lipid nanoparticles for delivery of nucleic acids and methods of use thereof |
| CN119998446A (zh) | 2022-06-07 | 2025-05-13 | 斯克里贝治疗公司 | 用于靶向pcsk9的组合物和方法 |
| EP4590820A2 (en) * | 2022-09-22 | 2025-07-30 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease |
| JP2026506287A (ja) * | 2022-12-21 | 2026-02-24 | インテリア セラピューティクス，インコーポレイテッド | プロタンパク質転換酵素サブチリシンケキシン９（ｐｃｓｋ９）編集のための組成物及び方法 |
| WO2024152058A1 (en) * | 2023-01-13 | 2024-07-18 | The Trustees Of The University Of Pennsylvania | Compositions and methods for the treatment of hereditary tyrosinemia type 1 |
| WO2024175550A1 (en) * | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| WO2024197065A2 (en) * | 2023-03-20 | 2024-09-26 | Verve Therapeutics, Inc. | In vivo nickase-based editing of the lpa gene for treatment of cardiovascular disease |
| CN118813611B (zh) * | 2023-04-19 | 2025-09-16 | 清华大学 | 一种基于rna核酶的dear核酸操纵系统及其应用 |
| EP4735603A2 (en) * | 2023-06-30 | 2026-05-06 | nChroma Bio, Inc. | Guide rna compositions |
| WO2025045247A1 (en) * | 2023-08-31 | 2025-03-06 | Geneditbio Limited | Nucleic acids encoding crispr-associated proteins and uses thereof |
| WO2025054363A1 (en) * | 2023-09-05 | 2025-03-13 | Cornell University | Minigene cassettes, recombinant polynucleotides, and methods of their use |
| WO2025064656A1 (en) * | 2023-09-19 | 2025-03-27 | Beam Therapeutics Inc. | Compositions and methods for altering a nucleobase in a lipoprotein(a) polynucleotide |
| CN117587013B (zh) * | 2023-11-27 | 2024-06-14 | 湖南师范大学 | 一种血管发育异常的斑马鱼模型的构建方法 |
| CN117568313B (zh) * | 2024-01-15 | 2024-04-26 | 上海贝斯昂科生物科技有限公司 | 基因编辑组合物及其用途 |
| CN118956837B (zh) * | 2024-02-26 | 2025-09-23 | 深圳信立泰药业股份有限公司 | 用于pcsk9基因修饰或编辑的组合物及其使用方法 |
| WO2025184520A1 (en) * | 2024-02-29 | 2025-09-04 | Intellia Therapeutics, Inc. | Compositions and methods for angiopoietin like 3 (angptl3) editing |
| JP2025139413A (ja) * | 2024-03-12 | 2025-09-26 | サミー株式会社 | 遊技機 |
| WO2026020120A1 (en) * | 2024-07-19 | 2026-01-22 | University Of Massachusetts | Increasing cas9 genome editing fidelity through attenuation of guide rna watson-crick base pairing potential |
| WO2026143185A1 (en) | 2024-12-23 | 2026-07-02 | Ensoma Aps | Engineered rna-guided dna-binding polypeptides |
| CN120130446B (zh) * | 2025-03-10 | 2026-01-20 | 苏州系统医学研究所 | 一种制备疾病动物模型的方法及其应用 |
Family Cites Families (75)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2001034768A2 (en) | 1999-11-09 | 2001-05-17 | Human Genome Sciences, Inc. | 15 human secreted proteins |
| CA2289702C (en) | 1997-05-14 | 2008-02-19 | Inex Pharmaceuticals Corp. | High efficiency encapsulation of charged therapeutic agents in lipid vesicles |
| EP1514933A1 (en) | 1999-07-08 | 2005-03-16 | Research Association for Biotechnology | Secretory protein or membrane protein |
| US20030119038A1 (en) | 1999-09-09 | 2003-06-26 | Bingham Brendan William | NARC1, novel subtilase-like homologs |
| US7029895B2 (en) | 1999-09-27 | 2006-04-18 | Millennium Pharmaceuticals, Inc. | 27411, a novel human PGP synthase |
| AU1099601A (en) | 1999-10-22 | 2001-05-08 | Millennium Pharmaceuticals, Inc. | Nucleic acid molecules derived from rat brain and programmed cell death models |
| JP2003514564A (ja) | 1999-11-24 | 2003-04-22 | エムシーエス マイクロ キャリア システムズ ゲーエムベーハー | 核局在化シグナルまたはタンパク質導入領域の多量体を含むポリペプチド、および分子を細胞内へ移入するためのその使用法 |
| AU2001241461A1 (en) | 2000-02-07 | 2001-08-14 | Millennium Pharmaceuticals, Inc. | Narc-1, novel subtilase-like homologs |
| US6926898B2 (en) | 2000-04-12 | 2005-08-09 | Human Genome Sciences, Inc. | Albumin fusion proteins |
| CA2411971A1 (en) | 2000-06-16 | 2001-12-27 | Incyte Genomics, Inc. | Proteases |
| AU2001280471A1 (en) | 2000-08-11 | 2002-02-25 | Eli Lilly And Company | Novel secreted proteins and their uses |
| JP2005500005A (ja) | 2000-12-08 | 2005-01-06 | インサイト・ゲノミックス・インコーポレイテッド | タンパク質修飾分子とメンテナンス分子 |
| DE10109897A1 (de) | 2001-02-21 | 2002-11-07 | Novosom Ag | Fakultativ kationische Liposomen und Verwendung dieser |
| EP1423134A2 (en) | 2001-03-21 | 2004-06-02 | Human Genome Sciences | Human secreted proteins |
| ATE291925T1 (de) * | 2001-06-05 | 2005-04-15 | Curevac Gmbh | Stabilisierte mrna mit erhöhtem g/c-gehalt und optimierter codon usage für die gentherapie |
| US7858117B2 (en) | 2002-02-21 | 2010-12-28 | Novosom Ag | Amphoteric liposomes and their use |
| EP1440981A3 (en) | 2003-01-21 | 2005-11-23 | Research Association for Biotechnology | Full-length human cdna |
| EP1471152A1 (en) | 2003-04-25 | 2004-10-27 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Mutations in the human PCSK9 gene associated to hypercholesterolemia |
| DE102004054730A1 (de) | 2004-03-28 | 2006-05-11 | Novosom Ag | Serumstabile amphotere Liposomen |
| WO2006007712A1 (en) | 2004-07-19 | 2006-01-26 | Protiva Biotherapeutics, Inc. | Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents |
| US20070087045A1 (en) | 2005-10-14 | 2007-04-19 | Industrial Technology Research Institute | Lipid carrier and method of preparing the same |
| WO2008133647A2 (en) | 2006-11-07 | 2008-11-06 | Merck & Co., Inc. | Antagonists of pcsk9 |
| TWI445716B (zh) * | 2008-09-12 | 2014-07-21 | Rinat Neuroscience Corp | Ｐｃｓｋ９拮抗劑類 |
| WO2013143555A1 (en) | 2012-03-26 | 2013-10-03 | Biontech Ag | Rna formulation for immunotherapy |
| US9283287B2 (en) | 2012-04-02 | 2016-03-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of nuclear proteins |
| CN105408483A (zh) * | 2013-03-15 | 2016-03-16 | 通用医疗公司 | 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向 |
| CA2923029A1 (en) | 2013-09-03 | 2015-03-12 | Moderna Therapeutics, Inc. | Chimeric polynucleotides |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| FR3012370B1 (fr) | 2013-10-30 | 2017-04-14 | Michelin & Cie | Armature de sommet pour pneumatique d'avion |
| US10059655B2 (en) | 2013-12-19 | 2018-08-28 | Novartis Ag | Lipids and lipid compositions for the delivery of active agents |
| PL3087183T3 (pl) * | 2013-12-24 | 2021-01-11 | Ionis Pharmaceuticals, Inc. | Modulacja ekspresji białka angiopoetynopodobnego 3 |
| CA2953341C (en) | 2014-06-25 | 2023-01-24 | Acuitas Therapeutics Inc. | Lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| BR112017007923B1 (pt) * | 2014-10-17 | 2023-12-12 | The Penn State Research Foundation | Método para produzir manipulação genética mediada por reações multiplex com rna em uma célula receptora, construção de ácido nucleico,cassete de expressão, vetor, célula receptora e célula geneticamente modificada |
| MA41123A (fr) * | 2014-12-02 | 2017-10-10 | Oncomed Pharm Inc | Polythérapie pour le traitement du cancer |
| HUE067372T2 (hu) | 2015-06-29 | 2024-10-28 | Acuitas Therapeutics Inc | Lipidek és nanoszemcsés lipid formulázások nukleinsavak beadására |
| US11279928B2 (en) * | 2015-06-29 | 2022-03-22 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
| ES2898340T3 (es) | 2015-07-09 | 2022-03-07 | Insmed Inc | Composiciones y métodos para tratar neumopatías y lesión pulmonar |
| KR102405695B1 (ko) * | 2015-08-31 | 2022-06-03 | 엘지디스플레이 주식회사 | 유기 발광 표시 장치 및 그 제조 방법 |
| CN108368028B (zh) | 2015-10-28 | 2021-09-03 | 爱康泰生治疗公司 | 用于递送核酸的新型脂质和脂质纳米颗粒制剂 |
| EP3373979A1 (en) * | 2015-11-12 | 2018-09-19 | Pfizer Inc | Tissue-specific genome engineering using crispr-cas9 |
| WO2017093804A2 (en) * | 2015-12-01 | 2017-06-08 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| DK3386484T3 (da) | 2015-12-10 | 2022-07-04 | Modernatx Inc | Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler |
| WO2017136794A1 (en) * | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| ES3030658T3 (en) | 2016-03-30 | 2025-07-01 | Intellia Therapeutics Inc | Lipid nanoparticle formulations for crispr/cas components |
| US10767175B2 (en) * | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
| US10927383B2 (en) | 2016-06-30 | 2021-02-23 | Ethris Gmbh | Cas9 mRNAs |
| WO2018119514A1 (en) | 2016-12-28 | 2018-07-05 | Precision Nanosystems Inc. | Compositions for transfecting resistant cell types |
| KR20240007715A (ko) * | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| EP3551757A1 (en) * | 2016-12-08 | 2019-10-16 | Intellia Therapeutics, Inc. | Modified guide rnas |
| KR102569848B1 (ko) * | 2016-12-23 | 2023-08-25 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | Pcsk9의 유전자 편집 |
| MX2019009070A (es) * | 2017-02-01 | 2019-10-30 | Modernatx Inc | Composiciones de arnm terapeuticas inmunomoduladoras que codifican peptidos de mutacion de oncogenes de activacion. |
| TW201839136A (zh) * | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| JP7277052B2 (ja) | 2017-02-22 | 2023-05-18 | クリスパー セラピューティクス アーゲー | プロタンパク質転換酵素サブチリシン／ケキシン９型（ｐｃｓｋ９）関連障害の処置のための組成物および方法 |
| AU2018224387B2 (en) * | 2017-02-22 | 2024-08-08 | Crispr Therapeutics Ag | Compositions and methods for gene editing |
| WO2018183808A1 (en) * | 2017-03-31 | 2018-10-04 | Agenovir Corporation | Antiviral therapeutic |
| WO2018191719A1 (en) | 2017-04-13 | 2018-10-18 | Acuitas Therapeutics, Inc. | Lipid delivery of therapeutic agents to adipose tissue |
| CN111328290A (zh) | 2017-06-26 | 2020-06-23 | 博德研究所 | 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法 |
| EP3658573A1 (en) * | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| EP3437650A1 (en) | 2017-07-31 | 2019-02-06 | Accanis Biotech F&E GmbH & Co KG | Treatment of local skin hypotrophy conditions |
| WO2019036028A1 (en) | 2017-08-17 | 2019-02-21 | Acuitas Therapeutics, Inc. | LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS |
| EP3688162B1 (en) | 2017-09-29 | 2024-03-06 | Intellia Therapeutics, Inc. | Formulations |
| IL311278B2 (en) | 2017-09-29 | 2026-05-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing |
| CA3077391A1 (en) | 2017-09-29 | 2019-04-04 | Intellia Therapeutics, Inc. | In vitro method of mrna delivery using lipid nanoparticles |
| US12636381B2 (en) | 2018-04-30 | 2026-05-26 | The Trustees Of The University Of Pennsylvania | In utero CRISPR-mediated therapeutic editing of genes |
| KR102959497B1 (ko) | 2018-05-11 | 2026-05-04 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 돌연변이를 억제하는 방법 |
| WO2019226953A1 (en) * | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| JP7832761B2 (ja) * | 2018-06-08 | 2026-03-18 | インテリア セラピューティクス，インコーポレイテッド | 遺伝子編集のための修飾されたガイドｒｎａ |
| KR20210055733A (ko) * | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
| EP3852911B1 (en) | 2018-09-21 | 2025-01-22 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| CA3116576A1 (en) | 2018-10-18 | 2020-04-23 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| WO2021056302A1 (en) * | 2019-09-26 | 2021-04-01 | Syngenta Crop Protection Ag | Methods and compositions for dna base editing |
| CA3174708A1 (en) | 2020-03-04 | 2021-09-10 | Verve Therapeutics, Inc. | Compositions and methods for targeted rna delivery |
| US12115230B2 (en) * | 2020-04-09 | 2024-10-15 | Verve Therapeutics, Inc. | Base editing of ANGPTL3 and methods of using same for treatment of disease |
| WO2022060871A1 (en) | 2020-09-15 | 2022-03-24 | Verve Therapeutics, Inc. | Lipid formulations for gene editing |
-
2021
- 2021-04-09 US US17/918,092 patent/US12115230B2/en active Active
- 2021-04-09 MX MX2022012683A patent/MX2022012683A/es unknown
- 2021-04-09 GB GB2316054.2A patent/GB2623891B/en active Active
- 2021-04-09 WO PCT/US2021/026732 patent/WO2021207712A2/en not_active Ceased
- 2021-04-09 KR KR1020227038947A patent/KR20230016176A/ko active Pending
- 2021-04-09 EP EP21785068.4A patent/EP4133072A4/en active Pending
- 2021-04-09 GB GB2413218.5A patent/GB2632563B/en active Active
- 2021-04-09 CA CA3179863A patent/CA3179863A1/en active Pending
- 2021-04-09 US US17/918,057 patent/US12029795B2/en active Active
- 2021-04-09 GB GB2216693.8A patent/GB2613457B/en active Active
- 2021-04-09 BR BR112022020407A patent/BR112022020407A2/pt unknown
- 2021-04-09 CN CN202180043135.4A patent/CN116096429A/zh active Pending
- 2021-04-09 GB GB2413231.8A patent/GB2632564B/en active Active
- 2021-04-09 GB GB2413235.9A patent/GB2632566B/en active Active
- 2021-04-09 WO PCT/US2021/026729 patent/WO2021207710A2/en not_active Ceased
- 2021-04-09 AU AU2021252991A patent/AU2021252991B2/en active Active
- 2021-04-09 CN CN202180041917.4A patent/CN116096883A/zh active Pending
- 2021-04-09 IL IL297193A patent/IL297193A/en unknown
- 2021-04-09 KR KR1020227038926A patent/KR20230016175A/ko active Pending
- 2021-04-09 CA CA3179862A patent/CA3179862A1/en active Pending
- 2021-04-09 GB GB2413232.6A patent/GB2632565B/en active Active
- 2021-04-09 GB GB2216687.0A patent/GB2612452C/en active Active
- 2021-04-09 PH PH1/2022/553033A patent/PH12022553033A1/en unknown
- 2021-04-09 BR BR112022020428A patent/BR112022020428A2/pt unknown
- 2021-04-09 AU AU2021253959A patent/AU2021253959B2/en active Active
- 2021-04-09 JP JP2022562027A patent/JP7457832B2/ja active Active
- 2021-04-09 IL IL297194A patent/IL297194A/en unknown
- 2021-04-09 EP EP21784686.4A patent/EP4133071A4/en active Pending
- 2021-04-09 EP EP21783997.6A patent/EP4132591A4/en active Pending
- 2021-04-09 JP JP2022562030A patent/JP7457833B2/ja active Active
- 2021-04-09 WO PCT/US2021/026731 patent/WO2021207711A2/en not_active Ceased
- 2021-04-09 PH PH1/2022/553015A patent/PH12022553015A1/en unknown
- 2021-04-09 US US17/918,069 patent/US20230158174A1/en active Pending
- 2021-04-09 GB GB2317179.6A patent/GB2625902B/en active Active
-
2024
- 2024-01-11 JP JP2024002319A patent/JP2024038327A/ja active Pending
- 2024-01-11 JP JP2024002316A patent/JP2024038326A/ja active Pending
- 2024-02-20 US US18/582,406 patent/US20240390523A1/en active Pending
- 2024-10-02 US US18/904,423 patent/US20250152746A1/en active Pending
-
2025
- 2025-06-03 AU AU2025204143A patent/AU2025204143A1/en active Pending
- 2025-06-04 AU AU2025204175A patent/AU2025204175A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2024038326A5 (enExample) | ||
| JP6799058B2 (ja) | アレル選択的な遺伝子編集およびその使用 | |
| CN113994000B (zh) | 包括胞苷类似物的反义rna编辑寡核苷酸 | |
| JP7698881B2 (ja) | Ｒｎａ編集のための化学修飾したオリゴヌクレオチド | |
| CA2968336C (en) | Construct for site directed editing of an adenosine nucleotide in target rna | |
| Patanjali et al. | Construction of a uniform-abundance (normalized) cDNA library. | |
| TuRCHI et al. | Enzymatic completion of mammalian lagging-strand DNA replication. | |
| DE69331524T2 (de) | Verfahren zum spezifischen schneiden von rna-strängen | |
| US20180016601A1 (en) | Methods for Modulating Genome Editing | |
| CN114174510A (zh) | 添加了功能性碱基序列的靶标编辑指导rna | |
| KR102501980B1 (ko) | 화학적으로 변형된 단일 가닥 rna-편집 올리고뉴클레오타이드 | |
| JP2020512845A5 (enExample) | ||
| KR20260007576A (ko) | 변형된 가이드 rna | |
| JP2022519882A5 (enExample) | ||
| CN105916983A (zh) | 用于高效且特异性靶向包含高度重复基序的dna序列的稀有切割核酸内切酶的设计 | |
| AU2020263804A1 (en) | RNA editing inhibitors and methods of use | |
| WO2024196779A2 (en) | Optimized antisense sequences that enhance targeted rna editing by adar proteins | |
| JP2024534639A5 (enExample) | ||
| Herman et al. | Vesicular stomatitis virus RNA polymerase can read through the boundary between the leader and N genes in vitro | |
| JP2024529400A5 (enExample) | ||
| JP2024519922A5 (enExample) | ||
| Chaix et al. | 3′-3′-linked oligonucleotides: Synthesis and stability studies | |
| WO2026019837A1 (en) | Compositions and methods for overcoming t-cell exhaustion | |
| Lewin | First success with reverse genetics | |
| CN117821506A (zh) | 一种转基因质粒及其构建方法 |