JP2019533434A5 - - Google Patents
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- Publication number
- JP2019533434A5 JP2019533434A5 JP2019513876A JP2019513876A JP2019533434A5 JP 2019533434 A5 JP2019533434 A5 JP 2019533434A5 JP 2019513876 A JP2019513876 A JP 2019513876A JP 2019513876 A JP2019513876 A JP 2019513876A JP 2019533434 A5 JP2019533434 A5 JP 2019533434A5
- Authority
- JP
- Japan
- Prior art keywords
- expression cassette
- cells
- sequence
- pharmaceutical composition
- gene delivery
- 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
- 210000004027 cell Anatomy 0.000 claims description 46
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims description 26
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims description 26
- 238000000034 method Methods 0.000 claims description 19
- 239000013598 vector Substances 0.000 claims description 19
- 238000001476 gene delivery Methods 0.000 claims description 16
- 239000012634 fragment Substances 0.000 claims description 15
- 206010053138 Congenital aplastic anaemia Diseases 0.000 claims description 14
- 201000004939 Fanconi anemia Diseases 0.000 claims description 14
- 102000011755 Phosphoglycerate Kinase Human genes 0.000 claims description 12
- 101001099217 Thermotoga maritima (strain ATCC 43589 / DSM 3109 / JCM 10099 / NBRC 100826 / MSB8) Triosephosphate isomerase Proteins 0.000 claims description 12
- 239000008194 pharmaceutical composition Substances 0.000 claims description 12
- 229920001184 polypeptide Polymers 0.000 claims description 12
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 12
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 12
- 102100039619 Granulocyte colony-stimulating factor Human genes 0.000 claims description 8
- 101000914673 Homo sapiens Fanconi anemia group A protein Proteins 0.000 claims description 8
- 102000046452 human FANCA Human genes 0.000 claims description 8
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 7
- 241000700605 Viruses Species 0.000 claims description 6
- 239000012472 biological sample Substances 0.000 claims description 6
- 239000002773 nucleotide Substances 0.000 claims description 6
- 125000003729 nucleotide group Chemical group 0.000 claims description 6
- 108010087740 Fanconi Anemia Complementation Group A protein Proteins 0.000 claims description 4
- 102000009095 Fanconi Anemia Complementation Group A protein Human genes 0.000 claims description 4
- 108010029961 Filgrastim Proteins 0.000 claims description 4
- 108010017080 Granulocyte Colony-Stimulating Factor Proteins 0.000 claims description 4
- 241000713666 Lentivirus Species 0.000 claims description 4
- 229960004177 filgrastim Drugs 0.000 claims description 4
- 230000002489 hematologic effect Effects 0.000 claims description 4
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 4
- 108091033319 polynucleotide Proteins 0.000 claims description 4
- 239000002157 polynucleotide Substances 0.000 claims description 4
- 102000040430 polynucleotide Human genes 0.000 claims description 4
- 239000013603 viral vector Substances 0.000 claims description 4
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims description 3
- 230000001105 regulatory effect Effects 0.000 claims description 3
- 241000282693 Cercopithecidae Species 0.000 claims description 2
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 2
- 206010033661 Pancytopenia Diseases 0.000 claims description 2
- 108091027981 Response element Proteins 0.000 claims description 2
- 208000007502 anemia Diseases 0.000 claims description 2
- 210000001185 bone marrow Anatomy 0.000 claims description 2
- 210000003743 erythrocyte Anatomy 0.000 claims description 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 2
- 201000002364 leukopenia Diseases 0.000 claims description 2
- 231100001022 leukopenia Toxicity 0.000 claims description 2
- 208000004235 neutropenia Diseases 0.000 claims description 2
- 238000004806 packaging method and process Methods 0.000 claims description 2
- 210000005259 peripheral blood Anatomy 0.000 claims description 2
- 239000011886 peripheral blood Substances 0.000 claims description 2
- 230000008488 polyadenylation Effects 0.000 claims description 2
- 208000024891 symptom Diseases 0.000 claims description 2
- 206010043554 thrombocytopenia Diseases 0.000 claims description 2
- 230000002463 transducing effect Effects 0.000 claims description 2
- 238000010361 transduction Methods 0.000 claims description 2
- 230000026683 transduction Effects 0.000 claims description 2
- 238000011144 upstream manufacturing Methods 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022117708A JP2022160505A (ja) | 2016-09-08 | 2022-07-25 | ファンコニ貧血患者に対する遺伝子療法 |
| JP2024228258A JP2025043346A (ja) | 2016-09-08 | 2024-12-25 | ファンコニ貧血患者に対する遺伝子療法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662385185P | 2016-09-08 | 2016-09-08 | |
| US62/385,185 | 2016-09-08 | ||
| US201662412028P | 2016-10-24 | 2016-10-24 | |
| US62/412,028 | 2016-10-24 | ||
| PCT/US2017/050837 WO2018049273A1 (en) | 2016-09-08 | 2017-09-08 | Gene therapy for patients with fanconi anemia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022117708A Division JP2022160505A (ja) | 2016-09-08 | 2022-07-25 | ファンコニ貧血患者に対する遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019533434A JP2019533434A (ja) | 2019-11-21 |
| JP2019533434A5 true JP2019533434A5 (https=) | 2020-10-08 |
| JP7197466B2 JP7197466B2 (ja) | 2022-12-27 |
Family
ID=61561668
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019513876A Active JP7197466B2 (ja) | 2016-09-08 | 2017-09-08 | ファンコニ貧血患者に対する遺伝子療法 |
| JP2022117708A Pending JP2022160505A (ja) | 2016-09-08 | 2022-07-25 | ファンコニ貧血患者に対する遺伝子療法 |
| JP2024228258A Pending JP2025043346A (ja) | 2016-09-08 | 2024-12-25 | ファンコニ貧血患者に対する遺伝子療法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022117708A Pending JP2022160505A (ja) | 2016-09-08 | 2022-07-25 | ファンコニ貧血患者に対する遺伝子療法 |
| JP2024228258A Pending JP2025043346A (ja) | 2016-09-08 | 2024-12-25 | ファンコニ貧血患者に対する遺伝子療法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20190203225A1 (https=) |
| EP (1) | EP3510162A4 (https=) |
| JP (3) | JP7197466B2 (https=) |
| KR (2) | KR102672636B1 (https=) |
| CN (1) | CN110536966A (https=) |
| AU (3) | AU2017322511B2 (https=) |
| CA (1) | CA3035605A1 (https=) |
| IL (2) | IL265196B2 (https=) |
| MX (1) | MX2019002699A (https=) |
| SG (1) | SG11201901718VA (https=) |
| WO (1) | WO2018049273A1 (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3021572A1 (en) | 2016-04-20 | 2017-10-26 | Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas | Compositions and methods for enhanced gene expression of pklr |
| EP3697452A4 (en) | 2017-10-16 | 2021-11-24 | Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. | LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS |
| BR112020020841A2 (pt) * | 2018-04-11 | 2021-01-19 | Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus | Composições e métodos para transplante de célulastronco |
| CA3096293A1 (en) | 2018-04-27 | 2019-10-31 | Rocket Pharmaceuticals, Ltd. | Gene therapy for cns degeneration |
| WO2020014523A1 (en) * | 2018-07-12 | 2020-01-16 | Rocket Pharmaceuticals, Ltd. | Gene therapy vectors for treatment of danon disease |
| KR20210046004A (ko) * | 2018-07-30 | 2021-04-27 | 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. | 조혈 세포의 유전자 변형 방법 |
| CN110904102A (zh) * | 2018-09-18 | 2020-03-24 | 中国科学院上海生命科学研究院 | 一种用于重组蛋白质表达的启动子 |
| CN113454235A (zh) * | 2019-02-21 | 2021-09-28 | 豪夫迈·罗氏有限公司 | 经改进的核酸靶标富集和相关方法 |
| CN113994008A (zh) * | 2019-05-23 | 2022-01-28 | 太空飞船七有限责任公司 | 用于婴儿恶性骨硬化病的基因治疗载体 |
| DE102020111571A1 (de) | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| JP2023538519A (ja) | 2020-08-07 | 2023-09-08 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療 |
| JP2024518770A (ja) * | 2021-04-26 | 2024-05-02 | ストーム バイオ インコーポレイテッド | ファンコニ貧血のための巨核球由来細胞外小胞に関する組成物及び方法 |
| WO2023183909A2 (en) * | 2022-03-25 | 2023-09-28 | Modernatx, Inc. | Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia |
| WO2025137373A1 (en) * | 2023-12-19 | 2025-06-26 | Cellarity, Inc. | Disease detection systems and methods |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5952190A (en) * | 1996-10-04 | 1999-09-14 | Fanconi Anemia Research Fund, Inc. | cDNA for fanconi anemia complementation group A |
| GB0024550D0 (https=) * | 2000-10-06 | 2000-11-22 | Oxford Biomedica Ltd | |
| JP2007054069A (ja) * | 2005-08-24 | 2007-03-08 | Medizinische Hochschule Hannover | 自己不活性化レトロウイルスベクター |
| AU2010241864B2 (en) * | 2009-04-30 | 2014-02-20 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Inducible interleukin-12 |
| DE102009021592A1 (de) * | 2009-05-15 | 2010-11-18 | Medizinische Hochschule Hannover | ASLV-Vektorsystem |
| RU2638802C2 (ru) * | 2011-05-16 | 2017-12-15 | Джензим Корпорейшн | Применение антагонистов cxcr4 |
| KR102530118B1 (ko) * | 2012-07-25 | 2023-05-08 | 더 브로드 인스티튜트, 인코퍼레이티드 | 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용 |
| US20160194660A1 (en) * | 2012-12-21 | 2016-07-07 | Merck Sharp & Dohme Corp. | Expression vectors for recombinant protein production in mammalian cells |
| GB201318347D0 (en) * | 2013-10-16 | 2013-11-27 | Ucl Business Plc | Retroviral vectors |
| WO2015073922A2 (en) * | 2013-11-15 | 2015-05-21 | Northwestern University | Inhibition of oxidative stress in atrial fibrillation |
| CN107405411A (zh) * | 2014-05-01 | 2017-11-28 | 华盛顿大学 | 使用腺病毒载体的体内基因改造 |
| US11030531B2 (en) * | 2014-06-06 | 2021-06-08 | Trustees Of Boston University | DNA recombinase circuits for logical control of gene expression |
| US10350245B2 (en) * | 2015-01-21 | 2019-07-16 | Fred Hutchinson Cancer Research Center | Point-of-care and/or portable platform for gene therapy |
| WO2016183593A2 (en) * | 2015-05-14 | 2016-11-17 | The Regents Of The University Of California | Prenatal therapy |
-
2017
- 2017-09-08 CN CN201780068686.XA patent/CN110536966A/zh active Pending
- 2017-09-08 KR KR1020197009772A patent/KR102672636B1/ko active Active
- 2017-09-08 US US16/331,455 patent/US20190203225A1/en active Pending
- 2017-09-08 KR KR1020247018425A patent/KR20240151726A/ko not_active Ceased
- 2017-09-08 SG SG11201901718VA patent/SG11201901718VA/en unknown
- 2017-09-08 CA CA3035605A patent/CA3035605A1/en active Pending
- 2017-09-08 WO PCT/US2017/050837 patent/WO2018049273A1/en not_active Ceased
- 2017-09-08 IL IL265196A patent/IL265196B2/en unknown
- 2017-09-08 EP EP17849687.3A patent/EP3510162A4/en active Pending
- 2017-09-08 MX MX2019002699A patent/MX2019002699A/es unknown
- 2017-09-08 IL IL320371A patent/IL320371A/en unknown
- 2017-09-08 JP JP2019513876A patent/JP7197466B2/ja active Active
- 2017-09-08 AU AU2017322511A patent/AU2017322511B2/en active Active
-
2021
- 2021-11-22 AU AU2021273525A patent/AU2021273525B2/en active Active
-
2022
- 2022-07-25 JP JP2022117708A patent/JP2022160505A/ja active Pending
-
2024
- 2024-12-24 AU AU2024287211A patent/AU2024287211A1/en active Pending
- 2024-12-25 JP JP2024228258A patent/JP2025043346A/ja active Pending
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