JP2020511991A5 - - Google Patents
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- JP2020511991A5 JP2020511991A5 JP2019553350A JP2019553350A JP2020511991A5 JP 2020511991 A5 JP2020511991 A5 JP 2020511991A5 JP 2019553350 A JP2019553350 A JP 2019553350A JP 2019553350 A JP2019553350 A JP 2019553350A JP 2020511991 A5 JP2020511991 A5 JP 2020511991A5
- Authority
- JP
- Japan
- Prior art keywords
- lentiviral vector
- item
- vector according
- cell
- hiv
- 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.)
- Granted
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- 210000004027 cell Anatomy 0.000 claims description 45
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims description 27
- 239000002773 nucleotide Substances 0.000 claims description 10
- 125000003729 nucleotide group Chemical group 0.000 claims description 10
- 108091033319 polynucleotide Proteins 0.000 claims description 10
- 102000040430 polynucleotide Human genes 0.000 claims description 10
- 239000002157 polynucleotide Substances 0.000 claims description 10
- 206010020751 Hypersensitivity Diseases 0.000 claims description 9
- 239000000203 mixture Substances 0.000 claims description 9
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 8
- 208000026935 allergic disease Diseases 0.000 claims description 8
- 210000003743 erythrocyte Anatomy 0.000 claims description 8
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 8
- 230000009610 hypersensitivity Effects 0.000 claims description 8
- 101000899111 Homo sapiens Hemoglobin subunit beta Proteins 0.000 claims description 7
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims description 4
- 108010054147 Hemoglobins Proteins 0.000 claims description 4
- 102000001554 Hemoglobins Human genes 0.000 claims description 4
- 101000903703 Homo sapiens B-cell lymphoma/leukemia 11A Proteins 0.000 claims description 4
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims description 4
- 230000000692 anti-sense effect Effects 0.000 claims description 4
- 102000052096 human BCL11A Human genes 0.000 claims description 4
- 108020004999 messenger RNA Proteins 0.000 claims description 4
- 230000025308 nuclear transport Effects 0.000 claims description 4
- 238000004806 packaging method and process Methods 0.000 claims description 4
- 101710177291 Gag polyprotein Proteins 0.000 claims description 3
- 101710125418 Major capsid protein Proteins 0.000 claims description 3
- 208000005980 beta thalassemia Diseases 0.000 claims description 3
- DWNBPRRXEVJMPO-RNGYDEEPSA-N hepoxilin B3 Chemical compound CCCCC\C=C/C[C@@H]1O[C@H]1C(O)\C=C/C\C=C/CCCC(O)=O DWNBPRRXEVJMPO-RNGYDEEPSA-N 0.000 claims description 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 2
- 102100031585 ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 Human genes 0.000 claims description 2
- 101000777636 Homo sapiens ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 Proteins 0.000 claims description 2
- 101000800116 Homo sapiens Thy-1 membrane glycoprotein Proteins 0.000 claims description 2
- 102000008731 Nuclear RNA export factor Human genes 0.000 claims description 2
- 108050000506 Nuclear RNA export factor Proteins 0.000 claims description 2
- 108091034057 RNA (poly(A)) Proteins 0.000 claims description 2
- 102100033523 Thy-1 membrane glycoprotein Human genes 0.000 claims description 2
- 201000006288 alpha thalassemia Diseases 0.000 claims description 2
- 208000007056 sickle cell anemia Diseases 0.000 claims description 2
- 210000000130 stem cell Anatomy 0.000 claims description 2
- 108091081024 Start codon Proteins 0.000 claims 1
- 230000005856 abnormality Effects 0.000 claims 1
- 208000034737 hemoglobinopathy Diseases 0.000 claims 1
- 208000018337 inherited hemoglobinopathy Diseases 0.000 claims 1
- 230000030147 nuclear export Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 25
- 108091005904 Hemoglobin subunit beta Proteins 0.000 description 11
- 102100021519 Hemoglobin subunit beta Human genes 0.000 description 11
- RVGRUAULSDPKGF-UHFFFAOYSA-N Poloxamer Chemical compound C1CO1.CC1CO1 RVGRUAULSDPKGF-UHFFFAOYSA-N 0.000 description 9
- 108700028369 Alleles Proteins 0.000 description 8
- 229920001983 poloxamer Polymers 0.000 description 7
- 239000008194 pharmaceutical composition Substances 0.000 description 6
- 229960000502 poloxamer Drugs 0.000 description 6
- 239000000018 receptor agonist Substances 0.000 description 6
- 229940044601 receptor agonist Drugs 0.000 description 5
- 241000713666 Lentivirus Species 0.000 description 4
- 239000002253 acid Substances 0.000 description 4
- QAOBBBBDJSWHMU-WMBBNPMCSA-N 16,16-dimethylprostaglandin E2 Chemical compound CCCCC(C)(C)[C@H](O)\C=C\[C@H]1[C@H](O)CC(=O)[C@@H]1C\C=C/CCCC(O)=O QAOBBBBDJSWHMU-WMBBNPMCSA-N 0.000 description 3
- 229920002517 Poloxamer 338 Polymers 0.000 description 3
- -1 Ratanoprost Chemical compound 0.000 description 3
- 208000002903 Thalassemia Diseases 0.000 description 3
- 230000004064 dysfunction Effects 0.000 description 3
- 229940106032 poloxamer 335 Drugs 0.000 description 3
- 229920001992 poloxamer 407 Polymers 0.000 description 3
- 229940044476 poloxamer 407 Drugs 0.000 description 3
- KXDAEFPNCMNJSK-UHFFFAOYSA-N Benzamide Chemical compound NC(=O)C1=CC=CC=C1 KXDAEFPNCMNJSK-UHFFFAOYSA-N 0.000 description 2
- VJGGHXVGBSZVMZ-QIZQQNKQSA-N Cloprostenol Chemical compound C([C@H](O)\C=C\[C@@H]1[C@H]([C@@H](O)C[C@H]1O)C\C=C/CCCC(O)=O)OC1=CC=CC(Cl)=C1 VJGGHXVGBSZVMZ-QIZQQNKQSA-N 0.000 description 2
- 239000002245 particle Substances 0.000 description 2
- LLVVDFDWPQHXBA-QEJIITRLSA-N 11-Deoxy-16,16-dimethyl-PGE2 Chemical compound CCCCC(C)(C)[C@H](O)\C=C\[C@H]1CCC(=O)[C@@H]1C\C=C/CCCC(O)=O LLVVDFDWPQHXBA-QEJIITRLSA-N 0.000 description 1
- TUXFWOHFPFBNEJ-GJGHEGAFSA-N 13,14-dihydro-Delta(12)-prostaglandin J2 Chemical compound CCCCC[C@H](O)C\C=C1/[C@@H](C\C=C/CCCC(O)=O)C=CC1=O TUXFWOHFPFBNEJ-GJGHEGAFSA-N 0.000 description 1
- XSGQFHNPNWBVPT-DSFPJDRCSA-N 15-Methyl-15S-PGE2 Chemical compound CCCCC[C@](C)(O)\C=C\[C@H]1[C@H](O)CC(=O)[C@@H]1C\C=C/CCCC(O)=O XSGQFHNPNWBVPT-DSFPJDRCSA-N 0.000 description 1
- XSGQFHNPNWBVPT-VFXMVCAWSA-N 15-methyl-15R-PGE2 Chemical compound CCCCC[C@@](C)(O)\C=C\[C@H]1[C@H](O)CC(=O)[C@@H]1C\C=C/CCCC(O)=O XSGQFHNPNWBVPT-VFXMVCAWSA-N 0.000 description 1
- DXWJXQSQVUJRNS-YYWARMNLSA-N 16-Phenyl-tetranor-PGE2 Chemical compound C([C@H](O)\C=C\[C@@H]1[C@H](C(=O)C[C@H]1O)C\C=C/CCCC(O)=O)C1=CC=CC=C1 DXWJXQSQVUJRNS-YYWARMNLSA-N 0.000 description 1
- WTJYDBMHYPQFNJ-ZUVVJKHESA-N 19R-Hydroxy-PGE2 Chemical compound C[C@@H](O)CCC[C@H](O)\C=C\[C@H]1[C@H](O)CC(=O)[C@@H]1C\C=C/CCCC(O)=O WTJYDBMHYPQFNJ-ZUVVJKHESA-N 0.000 description 1
- WMLGLMGSFIXSGO-KTXJXPLISA-N 9-Deoxy-9-methylene-16,16-dimethyl -PGE2 Chemical compound CCCCC(C)(C)[C@H](O)\C=C\[C@H]1[C@H](O)CC(=C)[C@@H]1C\C=C/CCCC(O)=O WMLGLMGSFIXSGO-KTXJXPLISA-N 0.000 description 1
- VKEJXDXJUFQESA-DLMPNJEASA-N 9-Deoxy-9-methylene-PGE2 Chemical compound CCCCC[C@H](O)\C=C\[C@H]1[C@H](O)CC(=C)[C@@H]1C\C=C/CCCC(O)=O VKEJXDXJUFQESA-DLMPNJEASA-N 0.000 description 1
- OYHQOLUKZRVURQ-HZJYTTRNSA-N Linoleic acid Chemical compound CCCCC\C=C/C\C=C/CCCCCCCC(O)=O OYHQOLUKZRVURQ-HZJYTTRNSA-N 0.000 description 1
- FQYUMYWMJTYZTK-UHFFFAOYSA-N Phenyl glycidyl ether Chemical compound C1OC1COC1=CC=CC=C1 FQYUMYWMJTYZTK-UHFFFAOYSA-N 0.000 description 1
- XNRNNGPBEPRNAR-UHFFFAOYSA-N Thromboxane B2 Natural products CCCCCC(O)C=CC1OC(O)CC(O)C1CC=CCCCC(O)=O XNRNNGPBEPRNAR-UHFFFAOYSA-N 0.000 description 1
- 229960002470 bimatoprost Drugs 0.000 description 1
- AQOKCDNYWBIDND-FTOWTWDKSA-N bimatoprost Chemical compound CCNC(=O)CCC\C=C/C[C@H]1[C@@H](O)C[C@@H](O)[C@@H]1\C=C\[C@@H](O)CCC1=CC=CC=C1 AQOKCDNYWBIDND-FTOWTWDKSA-N 0.000 description 1
- 229950008654 butaprost Drugs 0.000 description 1
- 229960005296 carboprost tromethamine Drugs 0.000 description 1
- UMMADZJLZAPZAW-OVXHCKHTSA-N carboprost tromethamine Chemical compound OCC([NH3+])(CO)CO.CCCCC[C@](C)(O)\C=C\[C@H]1[C@@H](O)C[C@H](O)[C@@H]1C\C=C/CCCC([O-])=O UMMADZJLZAPZAW-OVXHCKHTSA-N 0.000 description 1
- 229960004409 cloprostenol Drugs 0.000 description 1
- 238000012258 culturing Methods 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 102000018146 globin Human genes 0.000 description 1
- 108060003196 globin Proteins 0.000 description 1
- 229960002240 iloprost Drugs 0.000 description 1
- HIFJCPQKFCZDDL-ACWOEMLNSA-N iloprost Chemical compound C1\C(=C/CCCC(O)=O)C[C@@H]2[C@@H](/C=C/[C@@H](O)C(C)CC#CC)[C@H](O)C[C@@H]21 HIFJCPQKFCZDDL-ACWOEMLNSA-N 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 125000001449 isopropyl group Chemical group [H]C([H])([H])C([H])(*)C([H])([H])[H] 0.000 description 1
- 229960004232 linoleic acid Drugs 0.000 description 1
- OJLOPKGSLYJEMD-URPKTTJQSA-N methyl 7-[(1r,2r,3r)-3-hydroxy-2-[(1e)-4-hydroxy-4-methyloct-1-en-1-yl]-5-oxocyclopentyl]heptanoate Chemical compound CCCCC(C)(O)C\C=C\[C@H]1[C@H](O)CC(=O)[C@@H]1CCCCCCC(=O)OC OJLOPKGSLYJEMD-URPKTTJQSA-N 0.000 description 1
- XRISENIKJUKIHD-LHQZMKCDSA-N methyl 7-[(1r,2r,3r)-3-hydroxy-2-[(e,4r)-4-hydroxy-4-(1-propylcyclobutyl)but-1-enyl]-5-oxocyclopentyl]heptanoate Chemical compound CCCC1([C@H](O)C\C=C\[C@@H]2[C@H](C(=O)C[C@H]2O)CCCCCCC(=O)OC)CCC1 XRISENIKJUKIHD-LHQZMKCDSA-N 0.000 description 1
- 150000004702 methyl esters Chemical class 0.000 description 1
- 229960005249 misoprostol Drugs 0.000 description 1
- 229930182884 serinolamide Natural products 0.000 description 1
- 229960003400 sulprostone Drugs 0.000 description 1
- UQZVCDCIMBLVNR-TWYODKAFSA-N sulprostone Chemical compound O[C@@H]1CC(=O)[C@H](C\C=C/CCCC(=O)NS(=O)(=O)C)[C@H]1\C=C\[C@@H](O)COC1=CC=CC=C1 UQZVCDCIMBLVNR-TWYODKAFSA-N 0.000 description 1
- 208000024891 symptom Diseases 0.000 description 1
- 229960004458 tafluprost Drugs 0.000 description 1
- WSNODXPBBALQOF-VEJSHDCNSA-N tafluprost Chemical compound CC(C)OC(=O)CCC\C=C/C[C@H]1[C@@H](O)C[C@@H](O)[C@@H]1\C=C\C(F)(F)COC1=CC=CC=C1 WSNODXPBBALQOF-VEJSHDCNSA-N 0.000 description 1
- DSNBHJFQCNUKMA-SCKDECHMSA-N thromboxane A2 Chemical compound OC(=O)CCC\C=C/C[C@@H]1[C@@H](/C=C/[C@@H](O)CCCCC)O[C@@H]2O[C@H]1C2 DSNBHJFQCNUKMA-SCKDECHMSA-N 0.000 description 1
- XNRNNGPBEPRNAR-JQBLCGNGSA-N thromboxane B2 Chemical compound CCCCC[C@H](O)\C=C\[C@H]1OC(O)C[C@H](O)[C@@H]1C\C=C/CCCC(O)=O XNRNNGPBEPRNAR-JQBLCGNGSA-N 0.000 description 1
- 238000013518 transcription Methods 0.000 description 1
- 230000035897 transcription Effects 0.000 description 1
- 230000002463 transducing effect Effects 0.000 description 1
- 229960002368 travoprost Drugs 0.000 description 1
- MKPLKVHSHYCHOC-AHTXBMBWSA-N travoprost Chemical compound CC(C)OC(=O)CCC\C=C/C[C@H]1[C@@H](O)C[C@@H](O)[C@@H]1\C=C\[C@@H](O)COC1=CC=CC(C(F)(F)F)=C1 MKPLKVHSHYCHOC-AHTXBMBWSA-N 0.000 description 1
- 229960005032 treprostinil Drugs 0.000 description 1
- PAJMKGZZBBTTOY-ZFORQUDYSA-N treprostinil Chemical compound C1=CC=C(OCC(O)=O)C2=C1C[C@@H]1[C@@H](CC[C@@H](O)CCCCC)[C@H](O)C[C@@H]1C2 PAJMKGZZBBTTOY-ZFORQUDYSA-N 0.000 description 1
- 150000005671 trienes Chemical class 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023005869A JP2023033551A (ja) | 2017-03-29 | 2023-01-18 | ヘモグロビン異常症を治療するためのベクターおよび組成物 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762478375P | 2017-03-29 | 2017-03-29 | |
| US62/478,375 | 2017-03-29 | ||
| US201762489149P | 2017-04-24 | 2017-04-24 | |
| US62/489,149 | 2017-04-24 | ||
| PCT/US2018/025165 WO2018183692A1 (en) | 2017-03-29 | 2018-03-29 | Vectors and compositions for treating hemoglobinopathies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023005869A Division JP2023033551A (ja) | 2017-03-29 | 2023-01-18 | ヘモグロビン異常症を治療するためのベクターおよび組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020511991A JP2020511991A (ja) | 2020-04-23 |
| JP2020511991A5 true JP2020511991A5 (enExample) | 2021-04-30 |
| JP7228523B2 JP7228523B2 (ja) | 2023-02-24 |
Family
ID=63677097
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019553350A Active JP7228523B2 (ja) | 2017-03-29 | 2018-03-29 | ヘモグロビン異常症を治療するためのベクターおよび組成物 |
| JP2023005869A Withdrawn JP2023033551A (ja) | 2017-03-29 | 2023-01-18 | ヘモグロビン異常症を治療するためのベクターおよび組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023005869A Withdrawn JP2023033551A (ja) | 2017-03-29 | 2023-01-18 | ヘモグロビン異常症を治療するためのベクターおよび組成物 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20220259594A1 (enExample) |
| EP (1) | EP3600448A4 (enExample) |
| JP (2) | JP7228523B2 (enExample) |
| CN (1) | CN110582305A (enExample) |
| AU (1) | AU2018243293A1 (enExample) |
| BR (1) | BR112019020322A2 (enExample) |
| CA (1) | CA3057862A1 (enExample) |
| IL (1) | IL269551A (enExample) |
| SG (1) | SG11201908844YA (enExample) |
| WO (1) | WO2018183692A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016004113A1 (en) | 2014-06-30 | 2016-01-07 | Biogen Ma Inc. | Optimized factor ix gene |
| WO2018183692A1 (en) * | 2017-03-29 | 2018-10-04 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
| US20220160788A1 (en) * | 2019-03-22 | 2022-05-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Bifunctional vectors allowing bcl11a silencing and expression of an anti-sickling hbb and uses thereof for gene therapy of b-hemoglobinopathies |
| EP3980549A4 (en) * | 2019-06-05 | 2023-07-26 | Orchard Therapeutics (Europe) Limited | COMPOSITIONS AND METHODS FOR MODIFICATION OF EUKARYOTIC CELLS |
| US12403164B2 (en) | 2019-09-30 | 2025-09-02 | Bioverativ Therapeutics Inc. | Lentiviral vector formulations |
| CN117384967A (zh) * | 2020-04-24 | 2024-01-12 | 康霖生物科技（杭州）有限公司 | 一种核酸构建体 |
| WO2022107058A1 (en) * | 2020-11-23 | 2022-05-27 | Intas Pharmaceuticals Ltd. | Gene therapy based administration of lentivirus vector for treating hemoglobinopathies |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2334794T3 (en) * | 2008-09-15 | 2017-02-20 | Children's Medical Center Corp | MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES |
| SG10201602423TA (en) * | 2011-09-30 | 2016-05-30 | Bluebird Bio Inc | Compounds For Improved Viral Transduction |
| WO2014093965A1 (en) * | 2012-12-14 | 2014-06-19 | Case Western Reserve University | Genomic rna packaging enhancer element |
| AU2015249381B2 (en) * | 2014-04-25 | 2020-04-30 | Children's Medical Center Corporation | Compositions and methods to treating hemoglobinopathies |
| EP3134434A4 (en) * | 2014-04-25 | 2017-10-25 | Bluebird Bio, Inc. | Kappa/lambda chimeric antigen receptors |
| MX382565B (es) * | 2014-04-25 | 2025-03-13 | 2Seventy Bio Inc | Receptores de antigenos quimericos del promotor mnd. |
| JP6710680B2 (ja) | 2014-09-04 | 2020-06-17 | メモリアル スローン ケタリング キャンサー センター | ヘモグロビン異常症を処置するためのグロビン遺伝子療法 |
| WO2016094304A2 (en) * | 2014-12-12 | 2016-06-16 | Bluebird Bio, Inc. | Bcma chimeric antigen receptors |
| AU2016239324B2 (en) | 2015-03-31 | 2022-04-21 | Glycotope Gmbh | Eukaryotic expression vectors comprising regulatory elements of the globin gene clusters |
| CA2996893A1 (en) | 2015-08-31 | 2017-03-09 | Bluebird Bio, Inc. | Anti-sialyl tn chimeric antigen receptors |
| US11261441B2 (en) * | 2017-03-29 | 2022-03-01 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
| WO2018183692A1 (en) * | 2017-03-29 | 2018-10-04 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
-
2018
- 2018-03-29 WO PCT/US2018/025165 patent/WO2018183692A1/en not_active Ceased
- 2018-03-29 CA CA3057862A patent/CA3057862A1/en active Pending
- 2018-03-29 EP EP18775163.1A patent/EP3600448A4/en active Pending
- 2018-03-29 JP JP2019553350A patent/JP7228523B2/ja active Active
- 2018-03-29 AU AU2018243293A patent/AU2018243293A1/en not_active Abandoned
- 2018-03-29 BR BR112019020322-2A patent/BR112019020322A2/pt not_active Application Discontinuation
- 2018-03-29 CN CN201880027694.4A patent/CN110582305A/zh active Pending
- 2018-03-29 SG SG11201908844Y patent/SG11201908844YA/en unknown
-
2019
- 2019-09-23 IL IL26955119A patent/IL269551A/en unknown
-
2022
- 2022-01-28 US US17/587,999 patent/US20220259594A1/en active Pending
-
2023
- 2023-01-18 JP JP2023005869A patent/JP2023033551A/ja not_active Withdrawn
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