JP2014519335A5 - - Google Patents
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- JP2014519335A5 JP2014519335A5 JP2014514899A JP2014514899A JP2014519335A5 JP 2014519335 A5 JP2014519335 A5 JP 2014519335A5 JP 2014514899 A JP2014514899 A JP 2014514899A JP 2014514899 A JP2014514899 A JP 2014514899A JP 2014519335 A5 JP2014519335 A5 JP 2014519335A5
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- JP
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- Prior art keywords
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- vector
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- flap
- 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.)
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- 239000013598 vector Substances 0.000 claims description 52
- 108091033319 polynucleotide Proteins 0.000 claims description 17
- 102000040430 polynucleotide Human genes 0.000 claims description 17
- 239000002157 polynucleotide Substances 0.000 claims description 17
- 230000008488 polyadenylation Effects 0.000 claims description 14
- 229920001184 polypeptide Polymers 0.000 claims description 14
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 14
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 14
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims description 12
- 210000004027 cell Anatomy 0.000 claims description 10
- 101000760602 Homo sapiens ATP-binding cassette sub-family D member 1 Proteins 0.000 claims description 8
- 239000002299 complementary DNA Substances 0.000 claims description 8
- 102000047168 human ABCD1 Human genes 0.000 claims description 8
- 230000001105 regulatory effect Effects 0.000 claims description 8
- 241001430294 unidentified retrovirus Species 0.000 claims description 8
- 238000004806 packaging method and process Methods 0.000 claims description 7
- 241000701022 Cytomegalovirus Species 0.000 claims description 5
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 5
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 5
- 241000713666 Lentivirus Species 0.000 claims description 5
- 241000283973 Oryctolagus cuniculus Species 0.000 claims description 5
- 210000000274 microglia Anatomy 0.000 claims description 5
- 241000829100 Macaca mulatta polyomavirus 1 Species 0.000 claims description 4
- 241000714474 Rous sarcoma virus Species 0.000 claims description 4
- 238000012217 deletion Methods 0.000 claims description 3
- 230000037430 deletion Effects 0.000 claims description 3
- 238000004519 manufacturing process Methods 0.000 claims description 3
- 230000001177 retroviral effect Effects 0.000 claims description 3
- 241000725303 Human immunodeficiency virus Species 0.000 claims description 2
- 241000713883 Myeloproliferative sarcoma virus Species 0.000 claims description 2
- 108010006025 bovine growth hormone Proteins 0.000 claims description 2
- 239000003623 enhancer Substances 0.000 claims description 2
- 239000012634 fragment Substances 0.000 claims description 2
- 210000004962 mammalian cell Anatomy 0.000 claims description 2
- 239000002245 particle Substances 0.000 claims description 2
- 230000002103 transcriptional effect Effects 0.000 claims 5
- 241000283923 Marmota monax Species 0.000 description 6
- 230000001124 posttranscriptional effect Effects 0.000 description 6
- 230000001919 adrenal effect Effects 0.000 description 4
- 238000000034 method Methods 0.000 description 4
- 108020004414 DNA Proteins 0.000 description 2
- 230000002490 cerebral effect Effects 0.000 description 2
- 210000001671 embryonic stem cell Anatomy 0.000 description 2
- 238000001415 gene therapy Methods 0.000 description 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 description 2
- 201000001119 neuropathy Diseases 0.000 description 2
- 230000007823 neuropathy Effects 0.000 description 2
- 208000033808 peripheral neuropathy Diseases 0.000 description 2
- 210000001988 somatic stem cell Anatomy 0.000 description 2
- 210000000130 stem cell Anatomy 0.000 description 2
- 210000004885 white matter Anatomy 0.000 description 2
- KDCGOANMDULRCW-UHFFFAOYSA-N Purine Natural products N1=CNC2=NC=NC2=C1 KDCGOANMDULRCW-UHFFFAOYSA-N 0.000 description 1
- 108091027981 Response element Proteins 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 125000000561 purinyl group Chemical group N1=C(N=C2N=CNC2=C1)* 0.000 description 1
- 239000013603 viral vector Substances 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161495857P | 2011-06-10 | 2011-06-10 | |
| US61/495,857 | 2011-06-10 | ||
| PCT/US2012/041693 WO2012170911A2 (en) | 2011-06-10 | 2012-06-08 | Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015042141A Division JP2015107134A (ja) | 2011-06-10 | 2015-03-04 | 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014519335A JP2014519335A (ja) | 2014-08-14 |
| JP2014519335A5 true JP2014519335A5 (cg-RX-API-DMAC7.html) | 2014-12-11 |
| JP6143231B2 JP6143231B2 (ja) | 2017-06-07 |
Family
ID=47296778
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014514899A Expired - Fee Related JP6143231B2 (ja) | 2011-06-10 | 2012-06-08 | 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター |
| JP2015042141A Pending JP2015107134A (ja) | 2011-06-10 | 2015-03-04 | 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015042141A Pending JP2015107134A (ja) | 2011-06-10 | 2015-03-04 | 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US8858928B2 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3492108A1 (cg-RX-API-DMAC7.html) |
| JP (2) | JP6143231B2 (cg-RX-API-DMAC7.html) |
| KR (2) | KR20150029756A (cg-RX-API-DMAC7.html) |
| CN (1) | CN103717240A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2012267512B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2838749A1 (cg-RX-API-DMAC7.html) |
| DK (1) | DK2717922T3 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2710452T3 (cg-RX-API-DMAC7.html) |
| MX (1) | MX2013014544A (cg-RX-API-DMAC7.html) |
| PL (1) | PL2717922T3 (cg-RX-API-DMAC7.html) |
| PT (1) | PT2717922T (cg-RX-API-DMAC7.html) |
| RU (1) | RU2014100160A (cg-RX-API-DMAC7.html) |
| TR (1) | TR201819015T4 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2012170911A2 (cg-RX-API-DMAC7.html) |
| ZA (1) | ZA201309515B (cg-RX-API-DMAC7.html) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2717922T3 (en) | 2011-06-10 | 2019-01-07 | Bluebird Bio Inc | GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE |
| ES2708948T3 (es) | 2012-11-27 | 2019-04-12 | Childrens Medical Ct Corp | Elementos reguladores distales de bcl11a como dianas para la reinducción de la hemoglobina fetal |
| GB201318804D0 (en) † | 2013-10-24 | 2013-12-11 | Adaptimmune Ltd | Vectors for transgene expression |
| CN106794260B (zh) | 2014-04-25 | 2021-02-23 | 儿童医疗中心有限公司 | 治疗血红蛋白病的组合物和方法 |
| WO2015164759A2 (en) * | 2014-04-25 | 2015-10-29 | Bluebird Bio, Inc. | Mnd promoter chimeric antigen receptors |
| CN106456670B (zh) | 2014-04-25 | 2020-01-07 | 蓝鸟生物公司 | 制备过继性细胞疗法的改善方法 |
| RS61406B1 (sr) | 2014-06-06 | 2021-03-31 | Bluebird Bio Inc | Poboljšane kompozicije t ćelija |
| EP3172231B1 (en) | 2014-07-24 | 2021-05-05 | Bluebird Bio, Inc. | Bcma chimeric antigen receptors |
| US10383929B2 (en) | 2014-12-12 | 2019-08-20 | Bluebird Bio, Inc. | BCMA chimeric antigen receptors |
| EP3270960A4 (en) * | 2015-03-20 | 2018-08-08 | Bluebird Bio, Inc. | Vector formulations |
| CN104857014B (zh) * | 2015-04-21 | 2017-12-08 | 武汉大学 | 2‑羟丙基‑β‑环化糊精在制备治疗X‑连锁肾上腺脑白质退化症的药物中的应用 |
| EP3763814A1 (en) | 2015-05-08 | 2021-01-13 | The Children's Medical Center Corporation | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
| IL246634B (en) * | 2015-07-23 | 2020-08-31 | Brett Buno | A method for creating a high-titer hepatitis e virus stock and detecting calibration for hepatitis e virus |
| PL3370705T3 (pl) * | 2015-11-05 | 2022-08-01 | The General Hospital Corporation | Dooponowe dostarczanie sekwencji kwasów nukleinowych do kodowania abcd1 w leczeniu adrenomieloneuropatii |
| WO2017099712A1 (en) | 2015-12-07 | 2017-06-15 | Bluebird Bio, Inc. | Improved t cell compositions |
| CN108883136A (zh) | 2016-02-12 | 2018-11-23 | 蓝鸟生物公司 | Vcn增强子组合物及其使用方法 |
| KR20250134722A (ko) | 2016-02-12 | 2025-09-11 | 블루버드 바이오, 인코포레이티드. | Vcn 인핸서 조성물 및 이의 사용 방법 |
| JP7215905B2 (ja) * | 2016-03-18 | 2023-01-31 | プレシゲン，インコーポレイテッド | Ｖｉｉ型コラーゲン欠損症の治療のための組成物及び方法 |
| US20220323612A1 (en) * | 2016-06-13 | 2022-10-13 | Bluebird Bio, Inc. | Gene therapy of neuronal ceroid lipofuscinoses |
| US20190345450A1 (en) * | 2016-06-17 | 2019-11-14 | Fred Hutchinson Cancer Research Center | Strategies to assess and/or produce cell populations with predictive engraftment potential |
| MX2019005277A (es) | 2016-11-04 | 2019-09-27 | Bluebird Bio Inc | Composiciones de celulas t con car anti-bcma. |
| US20180193270A1 (en) | 2016-11-29 | 2018-07-12 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| CN110199028A (zh) * | 2016-12-06 | 2019-09-03 | 蓝鸟生物公司 | 用于ii型黏多糖贮积症的基因治疗 |
| MX2019006552A (es) * | 2016-12-06 | 2019-10-15 | Bluebird Bio Inc | Terapia génica para mucopolisacaridosis de tipo i. |
| SG11201908844YA (en) | 2017-03-29 | 2019-10-30 | Bluebird Bio Inc | Vectors and compositions for treating hemoglobinopathies |
| US11261441B2 (en) * | 2017-03-29 | 2022-03-01 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
| GB201706121D0 (en) * | 2017-04-18 | 2017-05-31 | Glaxosmithkline Ip Dev Ltd | Stable cell lines for retroviral production |
| US11788087B2 (en) | 2017-05-25 | 2023-10-17 | The Children's Medical Center Corporation | BCL11A guide delivery |
| WO2019070740A2 (en) | 2017-10-02 | 2019-04-11 | Fred Hutchinson Cancer Research Center | LUTENING HORMONE RECEPTOR BINDING AGENTS AND LUTINISING HORMONE AGONISTS FOR IDENTIFICATION, MULTIPLICATION, REMOVAL AND MODIFICATION OF PRIMITIVE STEM CELL POPULATIONS |
| CN108456697B (zh) * | 2018-03-29 | 2022-05-27 | 成都优娃生物科技有限公司 | 应用慢病毒载体EF1α启动子优化表达ABCD1基因治疗肾上腺脑白质营养不良症 |
| US12522811B2 (en) | 2018-05-01 | 2026-01-13 | The Children's Medical Center Corporation | Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9 |
| US12350284B2 (en) | 2018-05-02 | 2025-07-08 | The Children's Medical Center Corporation | BCL11A microRNAs for treating hemoglobinopathies |
| CN112367973A (zh) * | 2018-05-15 | 2021-02-12 | 旗舰先锋创新V股份有限公司 | 融合剂脂质体组合物和其用途 |
| CA3105953A1 (en) | 2018-07-09 | 2020-01-16 | Flagship Pioneering Innovations V, Inc. | Fusosome compositions and uses thereof |
| MY204234A (en) | 2018-08-27 | 2024-08-16 | Regeneron Pharma | Use of raman spectroscopy in downstream purification |
| KR20210131991A (ko) | 2018-11-14 | 2021-11-03 | 플래그쉽 파이어니어링 이노베이션스 브이, 인크. | 구획-특이적 카고 전달을 위한 조성물 및 방법 |
| EP3880179A2 (en) * | 2018-11-14 | 2021-09-22 | Flagship Pioneering Innovations V, Inc. | Fusosome compositions for cns delivery |
| KR102163889B1 (ko) * | 2019-01-11 | 2020-10-12 | 동의대학교 산학협력단 | 해죽순 추출물을 포함하는 신경병증성 통증 예방 및 치료용 조성물 |
| CN116096904A (zh) * | 2019-12-31 | 2023-05-09 | 天鹅生物疗法有限公司 | 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途 |
| JP2024532963A (ja) | 2021-04-08 | 2024-09-11 | サナ バイオテクノロジー，インコーポレイテッド | Ｃｄ８特異的抗体コンストラクト及びその組成物 |
| CN115838765A (zh) * | 2021-09-18 | 2023-03-24 | 济南赛尔生物科技股份有限公司 | 一种治疗异染性脑白质营养不良的慢病毒载体 |
| CN114478713B (zh) * | 2022-02-21 | 2023-03-28 | 江苏蒙彼利生物科技有限公司 | 一种cmv包膜蛋白包装慢病毒载体及其应用 |
| CN121620592A (zh) * | 2023-05-01 | 2026-03-06 | 西雅图儿童医院d/b/a西雅图儿童研究所 | 表达突变体白细胞介素15的免疫细胞 |
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| DK2717922T3 (en) | 2011-06-10 | 2019-01-07 | Bluebird Bio Inc | GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE |
-
2012
- 2012-06-08 DK DK12796252.0T patent/DK2717922T3/en active
- 2012-06-08 ES ES12796252T patent/ES2710452T3/es active Active
- 2012-06-08 TR TR2018/19015T patent/TR201819015T4/tr unknown
- 2012-06-08 AU AU2012267512A patent/AU2012267512B2/en not_active Ceased
- 2012-06-08 WO PCT/US2012/041693 patent/WO2012170911A2/en not_active Ceased
- 2012-06-08 EP EP18193359.9A patent/EP3492108A1/en not_active Withdrawn
- 2012-06-08 US US13/492,553 patent/US8858928B2/en active Active
- 2012-06-08 PT PT12796252T patent/PT2717922T/pt unknown
- 2012-06-08 KR KR1020157004059A patent/KR20150029756A/ko not_active Withdrawn
- 2012-06-08 PL PL12796252T patent/PL2717922T3/pl unknown
- 2012-06-08 MX MX2013014544A patent/MX2013014544A/es active IP Right Grant
- 2012-06-08 US US14/125,220 patent/US9789139B2/en active Active
- 2012-06-08 JP JP2014514899A patent/JP6143231B2/ja not_active Expired - Fee Related
- 2012-06-08 EP EP12796252.0A patent/EP2717922B1/en active Active
- 2012-06-08 CA CA2838749A patent/CA2838749A1/en not_active Abandoned
- 2012-06-08 CN CN201280038832.1A patent/CN103717240A/zh active Pending
- 2012-06-08 KR KR1020147000344A patent/KR101528440B1/ko not_active Expired - Fee Related
- 2012-06-08 RU RU2014100160/10A patent/RU2014100160A/ru not_active Application Discontinuation
-
2013
- 2013-12-17 ZA ZA2013/09515A patent/ZA201309515B/en unknown
-
2014
- 2014-09-16 US US14/488,058 patent/US9061031B2/en active Active
-
2015
- 2015-03-04 JP JP2015042141A patent/JP2015107134A/ja active Pending
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