JP2017505626A5 - - Google Patents
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- JP2017505626A5 JP2017505626A5 JP2016549495A JP2016549495A JP2017505626A5 JP 2017505626 A5 JP2017505626 A5 JP 2017505626A5 JP 2016549495 A JP2016549495 A JP 2016549495A JP 2016549495 A JP2016549495 A JP 2016549495A JP 2017505626 A5 JP2017505626 A5 JP 2017505626A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- subject
- expression vector
- cells
- lentiviral
- 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
Links
- 239000013604 expression vector Substances 0.000 claims description 15
- 108091005886 Hemoglobin subunit gamma Proteins 0.000 claims description 10
- 241000713666 Lentivirus Species 0.000 claims description 10
- 239000013598 vector Substances 0.000 claims description 10
- 210000004027 cell Anatomy 0.000 claims description 9
- 208000018020 Sickle cell-beta-thalassemia disease syndrome Diseases 0.000 claims description 7
- 206010043391 Thalassaemia beta Diseases 0.000 claims description 7
- 208000007056 sickle cell anemia Diseases 0.000 claims description 7
- 230000003612 virological effect Effects 0.000 claims description 6
- 108700028146 Genetic Enhancer Elements Proteins 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
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 5
- 238000012217 deletion Methods 0.000 claims description 5
- 230000037430 deletion Effects 0.000 claims description 5
- 230000000925 erythroid effect Effects 0.000 claims description 5
- 239000012212 insulator Substances 0.000 claims description 5
- 208000024891 symptom Diseases 0.000 claims description 5
- 238000002054 transplantation Methods 0.000 claims description 5
- 108010044495 Fetal Hemoglobin Proteins 0.000 claims description 4
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 4
- 210000003995 blood forming stem cell Anatomy 0.000 claims description 3
- 108090000623 proteins and genes Proteins 0.000 claims description 3
- 102000004169 proteins and genes Human genes 0.000 claims description 3
- 230000006490 viral transcription Effects 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
- 108010054147 Hemoglobins Proteins 0.000 claims description 2
- 102000001554 Hemoglobins Human genes 0.000 claims description 2
- 210000001185 bone marrow Anatomy 0.000 claims description 2
- 230000003750 conditioning effect Effects 0.000 claims description 2
- 210000003743 erythrocyte Anatomy 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 9
- 108060003196 globin Proteins 0.000 claims 1
- 238000002513 implantation Methods 0.000 claims 1
- 238000001890 transfection Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 9
- 101100455521 Arabidopsis thaliana LSD1 gene Proteins 0.000 description 2
- 101150079814 CHS4 gene Proteins 0.000 description 2
- 101100203325 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) SKT5 gene Proteins 0.000 description 2
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461933788P | 2014-01-30 | 2014-01-30 | |
| US61/933,788 | 2014-01-30 | ||
| PCT/US2015/013960 WO2015117027A1 (en) | 2014-01-30 | 2015-01-30 | An improved fetal hemoglobin for genetic correction of sickle cell disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017505626A JP2017505626A (ja) | 2017-02-23 |
| JP2017505626A5 true JP2017505626A5 (enExample) | 2018-03-08 |
| JP6509884B2 JP6509884B2 (ja) | 2019-05-08 |
Family
ID=53757790
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016549495A Active JP6509884B2 (ja) | 2014-01-30 | 2015-01-30 | 鎌状赤血球病の遺伝子的是正のための改善された胎児ヘモグロビン |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US10072067B2 (enExample) |
| EP (1) | EP3099334B1 (enExample) |
| JP (1) | JP6509884B2 (enExample) |
| CN (1) | CN106456802B (enExample) |
| AU (2) | AU2015210775B2 (enExample) |
| CA (1) | CA2938419C (enExample) |
| ES (1) | ES2747817T3 (enExample) |
| WO (1) | WO2015117027A1 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10072067B2 (en) | 2014-01-30 | 2018-09-11 | Children's Hospital Medical Center | Fetal hemoglobin for genetic correction of sickle cell disease |
| BR112017004349A2 (pt) * | 2014-09-04 | 2017-12-05 | Memorial Sloan Kettering Cancer Center | terapia com gene de globina para o tratamento de hemoglobinopatias |
| DK3368052T3 (da) | 2015-10-27 | 2022-03-14 | Childrens Hospital Med Ct | Anvendelse af mapk-hæmmere for at nedbringe tabet af hæmatopoietiske stamceller under ex vivo-dyrkning og -genmanipulation |
| CN106497976A (zh) * | 2016-10-31 | 2017-03-15 | 广东铱科基因科技有限公司 | 一种用于矫正重症β‑地中海贫血患者自体造血干细胞的HBB基因试剂盒 |
| CA3070242A1 (en) * | 2017-07-18 | 2019-01-24 | Calimmune, Inc. | Compositions and methods for treating beta-hemoglobinopathies |
| US11970707B2 (en) | 2017-09-18 | 2024-04-30 | Children's Hospital Medical Center | Strong insulator and uses thereof in gene delivery |
| CN109722414A (zh) * | 2017-10-27 | 2019-05-07 | 博雅辑因（北京）生物科技有限公司 | 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基 |
| JP7470119B2 (ja) * | 2018-08-24 | 2024-04-17 | シーエスエル ベーリング ジーン セラピー インコーポレイテッド | 無血清培地におけるベクター産生 |
| WO2020091797A1 (en) * | 2018-11-01 | 2020-05-07 | Children's Hospital Medical Center | Reduced intensity conditioning with melphalan |
| WO2020209934A1 (en) | 2019-04-11 | 2020-10-15 | Massachusetts Institute Of Technology | Novel chimeric antigen receptors and libraries |
| US20220243224A1 (en) * | 2019-04-15 | 2022-08-04 | Children's Hospital Medical Center | Viral vector manufacturing methods |
| CN114127305A (zh) | 2019-05-23 | 2022-03-01 | 麻省理工学院 | 经由逆转录病毒表面展示的配体发现和基因递送 |
| EP3790545A4 (en) | 2019-07-17 | 2021-07-28 | Emmaus Medical, Inc. | METHODS AND COMPOSITIONS FOR TREATMENT OF SICKLE CLEANEMIA AND THALASSEMIA |
| CA3212006A1 (en) | 2021-02-26 | 2022-09-01 | Kelonia Therapeutics, Inc. | Lymphocyte targeted lentiviral vectors |
| WO2024050450A1 (en) | 2022-08-31 | 2024-03-07 | Gigamune, Inc. | Engineered enveloped vectors and methods of use thereof |
| EP4784626A1 (en) | 2023-09-25 | 2026-08-05 | Kelonia Therapeutics, Inc. | Antigen binding polypeptides |
| IL327310A (en) | 2023-09-25 | 2026-05-01 | Kelonia Therapeutics Inc | Cancer treatment vehicles |
| WO2025151744A1 (en) * | 2024-01-10 | 2025-07-17 | University Of Tennessee Research Foundation | Improved globin and other lentiviral vectors for gene therapy |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6172039B1 (en) | 1990-04-16 | 2001-01-09 | Apex Bioscience, Inc. | Expression of recombinant hemoglobin and hemoglobin variants in yeast |
| CA2080589A1 (en) * | 1990-04-16 | 1991-10-17 | Joseph De Angelo | Expression of recombinant hemoglobin in yeast |
| CA2246005A1 (en) | 1998-10-01 | 2000-04-01 | Hsc Research And Development Limited Partnership | Hybrid genes for gene therapy in erythroid cells |
| EP1852511B1 (en) * | 1999-12-14 | 2010-02-17 | Novartis AG | Bovine immunodeficiency virus (biv) based vectors |
| JP2004537539A (ja) * | 2001-06-29 | 2004-12-16 | スローン − ケッタリング インスティチュート フォー キャンサー リサーチ | ヒトグロビン遺伝子をコードするベクターとその異常ヘモグロビン血症の治療におけるその使用 |
| CA2506798C (en) | 2002-11-22 | 2013-01-22 | Institut Clayton De La Recherche | Compositions and systems for the regulation of genes |
| WO2004083383A2 (en) * | 2003-03-14 | 2004-09-30 | Albert Einstein College Of Medicine Of Yeshiva University, A Division Of Yeshiva University | Globin variant gene methods and compositions |
| WO2005030999A1 (en) | 2003-09-25 | 2005-04-07 | Dana-Farber Cancer Institute, Inc | Methods to detect lineage-specific cells |
| US7276340B1 (en) * | 2004-09-22 | 2007-10-02 | University Of Central Florida Research Foundation, Inc. | Age determination from biological stains using messenger RNA profiling analysis |
| US20090156534A1 (en) * | 2007-09-13 | 2009-06-18 | Memorial Sloan-Kettering Cancer Center | Globin lentiviral vectors for treatment of disease |
| WO2010127195A2 (en) | 2009-05-01 | 2010-11-04 | Curna, Inc. | Antisense oligonucleotides of hemoglobins |
| US20110294114A1 (en) | 2009-12-04 | 2011-12-01 | Cincinnati Children's Hospital Medical Center | Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells |
| AU2011377617B2 (en) * | 2011-09-23 | 2018-03-08 | Bluebird Bio, Inc. | Improved gene therapy methods |
| US10072067B2 (en) | 2014-01-30 | 2018-09-11 | Children's Hospital Medical Center | Fetal hemoglobin for genetic correction of sickle cell disease |
-
2015
- 2015-01-30 US US15/115,530 patent/US10072067B2/en active Active
- 2015-01-30 JP JP2016549495A patent/JP6509884B2/ja active Active
- 2015-01-30 AU AU2015210775A patent/AU2015210775B2/en not_active Ceased
- 2015-01-30 CN CN201580018389.5A patent/CN106456802B/zh active Active
- 2015-01-30 EP EP15744021.5A patent/EP3099334B1/en active Active
- 2015-01-30 WO PCT/US2015/013960 patent/WO2015117027A1/en not_active Ceased
- 2015-01-30 CA CA2938419A patent/CA2938419C/en active Active
- 2015-01-30 ES ES15744021T patent/ES2747817T3/es active Active
-
2018
- 2018-06-01 US US15/995,675 patent/US11345740B2/en active Active
-
2020
- 2020-09-30 AU AU2020244473A patent/AU2020244473B2/en not_active Ceased
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