JPWO2020237217A5 - - Google Patents
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- Publication number
- JPWO2020237217A5 JPWO2020237217A5 JP2022515977A JP2022515977A JPWO2020237217A5 JP WO2020237217 A5 JPWO2020237217 A5 JP WO2020237217A5 JP 2022515977 A JP2022515977 A JP 2022515977A JP 2022515977 A JP2022515977 A JP 2022515977A JP WO2020237217 A5 JPWO2020237217 A5 JP WO2020237217A5
- Authority
- JP
- Japan
- Prior art keywords
- grna
- cell population
- cell
- targeting domain
- domain
- 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
- 210000004027 cell Anatomy 0.000 claims 17
- 108020005004 Guide RNA Proteins 0.000 claims 11
- 238000000034 method Methods 0.000 claims 8
- 230000008685 targeting Effects 0.000 claims 8
- 101000934338 Homo sapiens Myeloid cell surface antigen CD33 Proteins 0.000 claims 6
- 102100025243 Myeloid cell surface antigen CD33 Human genes 0.000 claims 5
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 5
- 102000004533 Endonucleases Human genes 0.000 claims 3
- 108010042407 Endonucleases Proteins 0.000 claims 3
- 210000000130 stem cell Anatomy 0.000 claims 3
- 101710160107 Outer membrane protein A Proteins 0.000 claims 2
- 108010081734 Ribonucleoproteins Proteins 0.000 claims 2
- 102000004389 Ribonucleoproteins Human genes 0.000 claims 2
- 239000003795 chemical substances by application Substances 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 102100026094 C-type lectin domain family 12 member A Human genes 0.000 claims 1
- 101710188619 C-type lectin domain family 12 member A Proteins 0.000 claims 1
- 101710172824 CRISPR-associated endonuclease Cas9 Proteins 0.000 claims 1
- 208000002250 Hematologic Neoplasms Diseases 0.000 claims 1
- 101000910035 Streptococcus pyogenes serotype M1 CRISPR-associated endonuclease Cas9/Csn1 Proteins 0.000 claims 1
- 239000000427 antigen Substances 0.000 claims 1
- 108091007433 antigens Proteins 0.000 claims 1
- 102000036639 antigens Human genes 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 238000004520 electroporation Methods 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 230000009033 hematopoietic malignancy Effects 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962852238P | 2019-05-23 | 2019-05-23 | |
| US62/852,238 | 2019-05-23 | ||
| US202062962127P | 2020-01-16 | 2020-01-16 | |
| US62/962,127 | 2020-01-16 | ||
| PCT/US2020/034391 WO2020237217A1 (en) | 2019-05-23 | 2020-05-22 | Compositions and methods for cd33 modification |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022534813A JP2022534813A (ja) | 2022-08-03 |
| JP2022534813A5 JP2022534813A5 (https=) | 2023-05-24 |
| JPWO2020237217A5 true JPWO2020237217A5 (https=) | 2023-05-24 |
Family
ID=71078652
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022515977A Pending JP2022534813A (ja) | 2019-05-23 | 2020-05-22 | Ｃｄ３３改変のための組成物および方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220228153A1 (https=) |
| EP (1) | EP3973057A1 (https=) |
| JP (1) | JP2022534813A (https=) |
| KR (1) | KR20220035877A (https=) |
| CN (1) | CN114423865A (https=) |
| AU (1) | AU2020280103A1 (https=) |
| CA (1) | CA3141732A1 (https=) |
| MX (1) | MX2021014306A (https=) |
| WO (1) | WO2020237217A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201907870VA (en) | 2017-02-28 | 2019-09-27 | Vor Biopharma Inc | Compositions and methods for inhibition of lineage specific proteins |
| CA3110837A1 (en) * | 2018-08-28 | 2020-03-05 | Vor Biopharma Inc. | Genetically engineered hematopoietic stem cells and uses thereof |
| US20230407337A1 (en) * | 2020-12-08 | 2023-12-21 | The Regents if the University of California | Crispr ribonucleoprotein complex genome editing of human innate immune cells |
| US20240384304A1 (en) | 2021-07-06 | 2024-11-21 | Vor Biopharma Inc. | Inhibitor oligonucleotides and methods of use thereof |
| AU2022324093A1 (en) | 2021-08-02 | 2024-02-08 | Vor Biopharma Inc. | Compositions and methods for gene modification |
| WO2024015925A2 (en) | 2022-07-13 | 2024-01-18 | Vor Biopharma Inc. | Compositions and methods for artificial protospacer adjacent motif (pam) generation |
| WO2024073751A1 (en) | 2022-09-29 | 2024-04-04 | Vor Biopharma Inc. | Methods and compositions for gene modification and enrichment |
| WO2025030010A1 (en) | 2023-08-01 | 2025-02-06 | Vor Biopharma Inc. | Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| EP3556858A3 (en) | 2014-04-09 | 2020-01-22 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating cystic fibrosis |
| US10900034B2 (en) * | 2014-12-03 | 2021-01-26 | Agilent Technologies, Inc. | Guide RNA with chemical modifications |
| EP3280803B1 (en) | 2015-04-06 | 2021-05-26 | The Board of Trustees of the Leland Stanford Junior University | Chemically modified guide rnas for crispr/cas-mediated gene regulation |
| WO2017053729A1 (en) * | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
| CN108290939B (zh) * | 2015-10-16 | 2023-01-13 | 纽约市哥伦比亚大学理事会 | 用于抑制谱系特异性抗原的组合物和方法 |
| JP7109784B2 (ja) | 2015-10-23 | 2022-08-01 | 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 | 遺伝子編集のための進化したＣａｓ９蛋白質 |
| US10767175B2 (en) | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
| AU2017388753A1 (en) | 2016-12-30 | 2019-07-11 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| SG11201907870VA (en) | 2017-02-28 | 2019-09-27 | Vor Biopharma Inc | Compositions and methods for inhibition of lineage specific proteins |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| US11718659B2 (en) * | 2017-08-28 | 2023-08-08 | The Trustees Of Columbia University In The City Of New York | CD33 exon 2 deficient donor stem cells for use with CD33 targeting agents |
| CA3110837A1 (en) * | 2018-08-28 | 2020-03-05 | Vor Biopharma Inc. | Genetically engineered hematopoietic stem cells and uses thereof |
| SG11202103832SA (en) * | 2018-11-07 | 2021-05-28 | Crispr Therapeutics Ag | Anti-cd33 immune cell cancer therapy |
| MX2021008490A (es) * | 2019-01-16 | 2021-09-28 | Univ Columbia | Composiciones y metodos para la inhibicion de antigenos con especificidad de linaje. |
-
2020
- 2020-05-22 CN CN202080050633.7A patent/CN114423865A/zh active Pending
- 2020-05-22 CA CA3141732A patent/CA3141732A1/en active Pending
- 2020-05-22 EP EP20732076.3A patent/EP3973057A1/en not_active Withdrawn
- 2020-05-22 WO PCT/US2020/034391 patent/WO2020237217A1/en not_active Ceased
- 2020-05-22 JP JP2022515977A patent/JP2022534813A/ja active Pending
- 2020-05-22 KR KR1020217041969A patent/KR20220035877A/ko not_active Withdrawn
- 2020-05-22 MX MX2021014306A patent/MX2021014306A/es unknown
- 2020-05-22 AU AU2020280103A patent/AU2020280103A1/en not_active Abandoned
- 2020-05-22 US US17/613,387 patent/US20220228153A1/en active Pending
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