JP2024019727A5 - - Google Patents
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- JP2024019727A5 JP2024019727A5 JP2023223638A JP2023223638A JP2024019727A5 JP 2024019727 A5 JP2024019727 A5 JP 2024019727A5 JP 2023223638 A JP2023223638 A JP 2023223638A JP 2023223638 A JP2023223638 A JP 2023223638A JP 2024019727 A5 JP2024019727 A5 JP 2024019727A5
- Authority
- JP
- Japan
- Prior art keywords
- sgrna
- nme
- nucleotides
- length
- seq
- 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
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- 108091027544 Subgenomic mRNA Proteins 0.000 claims 83
- 239000002773 nucleotide Substances 0.000 claims 56
- 125000003729 nucleotide group Chemical group 0.000 claims 56
- 239000013607 AAV vector Substances 0.000 claims 24
- 108091033409 CRISPR Proteins 0.000 claims 18
- 101710163270 Nuclease Proteins 0.000 claims 18
- 241000588650 Neisseria meningitidis Species 0.000 claims 15
- 125000006850 spacer group Chemical group 0.000 claims 12
- 108090000623 proteins and genes Proteins 0.000 claims 8
- 238000007385 chemical modification Methods 0.000 claims 6
- 229920002477 rna polymer Polymers 0.000 claims 6
- 238000000034 method Methods 0.000 claims 5
- 241000606766 Haemophilus parainfluenzae Species 0.000 claims 4
- 241000863010 Simonsiella muelleri Species 0.000 claims 4
- 150000007523 nucleic acids Chemical group 0.000 claims 4
- 208000024891 symptom Diseases 0.000 claims 4
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 238000000338 in vitro Methods 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 239000013598 vector Substances 0.000 claims 3
- HMFHBZSHGGEWLO-SOOFDHNKSA-N D-ribofuranose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H]1O HMFHBZSHGGEWLO-SOOFDHNKSA-N 0.000 claims 2
- 101150065592 NME2 gene Proteins 0.000 claims 2
- 101150001372 Nme1 gene Proteins 0.000 claims 2
- 101150094724 PCSK9 gene Proteins 0.000 claims 2
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 claims 2
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 claims 2
- 230000001747 exhibiting effect Effects 0.000 claims 2
- 238000007069 methylation reaction Methods 0.000 claims 2
- 239000011859 microparticle Substances 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- 101001098868 Homo sapiens Proprotein convertase subtilisin/kexin type 9 Proteins 0.000 claims 1
- 208000035150 Hypercholesterolemia Diseases 0.000 claims 1
- 208000031226 Hyperlipidaemia Diseases 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 102100038955 Proprotein convertase subtilisin/kexin type 9 Human genes 0.000 claims 1
- 238000010362 genome editing Methods 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 239000004005 microsphere Substances 0.000 claims 1
- 239000002105 nanoparticle Substances 0.000 claims 1
- 201000011296 tyrosinemia Diseases 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762584310P | 2017-11-10 | 2017-11-10 | |
| US62/584,310 | 2017-11-10 | ||
| US201762596375P | 2017-12-08 | 2017-12-08 | |
| US62/596,375 | 2017-12-08 | ||
| US201862667084P | 2018-05-04 | 2018-05-04 | |
| US62/667,084 | 2018-05-04 | ||
| JP2020525971A JP2021502097A (ja) | 2017-11-10 | 2018-11-09 | 標的化ｃｒｉｓｐｒ送達プラットフォーム |
| PCT/US2018/060126 WO2019094791A2 (en) | 2017-11-10 | 2018-11-09 | Targeted crispr delivery platforms |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020525971A Division JP2021502097A (ja) | 2017-11-10 | 2018-11-09 | 標的化ｃｒｉｓｐｒ送達プラットフォーム |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024019727A JP2024019727A (ja) | 2024-02-09 |
| JP2024019727A5 true JP2024019727A5 (enExample) | 2025-04-17 |
Family
ID=66438139
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020525971A Withdrawn JP2021502097A (ja) | 2017-11-10 | 2018-11-09 | 標的化ｃｒｉｓｐｒ送達プラットフォーム |
| JP2023223638A Pending JP2024019727A (ja) | 2017-11-10 | 2023-12-28 | 標的化ｃｒｉｓｐｒ送達プラットフォーム |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020525971A Withdrawn JP2021502097A (ja) | 2017-11-10 | 2018-11-09 | 標的化ｃｒｉｓｐｒ送達プラットフォーム |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US20220389447A9 (enExample) |
| EP (1) | EP3707254A4 (enExample) |
| JP (2) | JP2021502097A (enExample) |
| KR (1) | KR20200080314A (enExample) |
| CN (1) | CN111868240A (enExample) |
| AU (2) | AU2018364993B2 (enExample) |
| BR (1) | BR112020009268A2 (enExample) |
| CA (1) | CA3082370A1 (enExample) |
| CO (1) | CO2020007046A2 (enExample) |
| IL (2) | IL274526B2 (enExample) |
| MX (1) | MX2020004777A (enExample) |
| SG (1) | SG11202005103RA (enExample) |
| WO (1) | WO2019094791A2 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3868887A4 (en) * | 2018-09-25 | 2022-12-07 | Microbial Chemistry Research Foundation | NOVEL VIRAL VECTOR AND METHODS OF PRODUCTION AND USE THEREOF |
| CA3129055A1 (en) | 2019-02-05 | 2020-08-13 | AMMR Joint Venture | Enhanced selection of efficient targeted genome manipulating agents |
| EP3924477A4 (en) * | 2019-02-14 | 2023-03-29 | Metagenomi, Inc. | ENZYMES WITH RUVC DOMAINS |
| CN110257406B (zh) * | 2019-06-21 | 2022-07-22 | 安徽省农业科学院水稻研究所 | 密码子植物化改造的Plant Nme2Cas9基因及其应用 |
| US12297426B2 (en) | 2019-10-01 | 2025-05-13 | The Broad Institute, Inc. | DNA damage response signature guided rational design of CRISPR-based systems and therapies |
| US20230054569A1 (en) | 2019-12-18 | 2023-02-23 | Alia Therapeutics Srl | Compositions and methods for treating retinitis pigmentosa |
| CN113652411B (zh) * | 2021-07-30 | 2025-01-10 | 复旦大学 | Cas9蛋白、含有Cas9蛋白的基因编辑系统及应用 |
| WO2023028348A1 (en) | 2021-08-27 | 2023-03-02 | Metagenomi, Inc. | Enzymes with ruvc domains |
| US20230183687A1 (en) * | 2021-10-13 | 2023-06-15 | University Of Massachusetts | Modified guide rnas for neisseria meningitidis cas9 |
| MX2024005241A (es) * | 2021-11-03 | 2024-07-02 | Intellia Therapeutics Inc | Arn guia modificados para la edicion genica. |
| CA3237303A1 (en) | 2021-11-03 | 2023-05-11 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing |
| KR20250124819A (ko) | 2022-12-21 | 2025-08-20 | 인텔리아 테라퓨틱스, 인크. | 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법 |
| TW202436622A (zh) | 2023-03-06 | 2024-09-16 | 美商英特利亞醫療公司 | 用於ｂ型肝炎病毒（ｈｂｖ）基因體編輯之組合物及方法 |
| WO2025038648A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2) |
| WO2025038646A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Cd70 car-t compositions and methods for cell-based therapy |
| WO2025038637A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2) |
| WO2025038642A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Compositions and methods for genetically modifying cd70 |
| WO2025255308A1 (en) | 2024-06-07 | 2025-12-11 | Intellia Therapeutics, Inc. | Cd8 co-receptor chimeric polypeptides in tcr cell therapy |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB2537000C (en) * | 2012-05-25 | 2019-10-09 | Univ California | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| AU2014281028B2 (en) * | 2013-06-17 | 2020-09-10 | Massachusetts Institute Of Technology | Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy |
| JP6718813B2 (ja) * | 2013-07-10 | 2020-07-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | Ｒｎａ誘導性遺伝子制御および編集のための直交性ｃａｓ９タンパク質 |
| EP3071695A2 (en) * | 2013-11-18 | 2016-09-28 | Crispr Therapeutics AG | Crispr-cas system materials and methods |
| WO2015079057A2 (en) * | 2013-11-28 | 2015-06-04 | Haplogen Genomics Gmbh | Somatic haploid human cell line |
| EP3080259B1 (en) * | 2013-12-12 | 2023-02-01 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| WO2015089486A2 (en) * | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
| WO2016049258A2 (en) * | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
| US10190106B2 (en) * | 2014-12-22 | 2019-01-29 | Univesity Of Massachusetts | Cas9-DNA targeting unit chimeras |
| US20160346359A1 (en) * | 2015-05-01 | 2016-12-01 | Spark Therapeutics, Inc. | Adeno-associated Virus-Mediated CRISPR-Cas9 Treatment of Ocular Disease |
| US11732258B2 (en) * | 2016-11-02 | 2023-08-22 | President And Fellows Of Harvard College | Engineered guide RNA sequences for in situ detection and sequencing |
| AU2018311695A1 (en) * | 2017-07-31 | 2020-01-16 | Sigma-Aldrich Co. Llc | Synthetic guide RNA for CRISPR/Cas activator systems |
-
2018
- 2018-11-09 MX MX2020004777A patent/MX2020004777A/es unknown
- 2018-11-09 IL IL274526A patent/IL274526B2/en unknown
- 2018-11-09 SG SG11202005103RA patent/SG11202005103RA/en unknown
- 2018-11-09 AU AU2018364993A patent/AU2018364993B2/en active Active
- 2018-11-09 CN CN201880082218.2A patent/CN111868240A/zh active Pending
- 2018-11-09 JP JP2020525971A patent/JP2021502097A/ja not_active Withdrawn
- 2018-11-09 EP EP18876344.5A patent/EP3707254A4/en active Pending
- 2018-11-09 KR KR1020207016690A patent/KR20200080314A/ko active Pending
- 2018-11-09 IL IL317505A patent/IL317505A/en unknown
- 2018-11-09 WO PCT/US2018/060126 patent/WO2019094791A2/en not_active Ceased
- 2018-11-09 US US16/186,352 patent/US20220389447A9/en not_active Abandoned
- 2018-11-09 BR BR112020009268-1A patent/BR112020009268A2/pt unknown
- 2018-11-09 CA CA3082370A patent/CA3082370A1/en active Pending
-
2020
- 2020-06-10 CO CONC2020/0007046A patent/CO2020007046A2/es unknown
-
2023
- 2023-01-06 AU AU2023200084A patent/AU2023200084B2/en active Active
- 2023-12-28 JP JP2023223638A patent/JP2024019727A/ja active Pending
-
2024
- 2024-03-21 US US18/612,111 patent/US20250223611A1/en active Pending
- 2024-12-13 US US18/980,456 patent/US20250197889A1/en active Pending
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