JP2019533434A5 - - Google Patents
Download PDFInfo
- 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) | KR20240151726A (https=) |
| CN (1) | CN110536966A (https=) |
| AU (3) | AU2017322511B2 (https=) |
| CA (1) | CA3035605A1 (https=) |
| IL (2) | IL320371A (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 |
|---|---|---|---|---|
| HUE055862T2 (hu) | 2016-04-20 | 2021-12-28 | Centro De Investig Energeticas | Készítmények és eljárások PKLR fokozott génexpressziójára |
| SG11202002263TA (en) | 2017-10-16 | 2020-04-29 | Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas | Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency |
| WO2019200167A1 (en) * | 2018-04-11 | 2019-10-17 | Rocket Pharmaceuticals, Ltd. | Compositions and methods for stem cell transplant |
| MX2020011386A (es) | 2018-04-27 | 2021-01-29 | Spacecraft Seven Llc | Terapia genica para la degeneracion del snc. |
| KR102918544B1 (ko) * | 2018-07-12 | 2026-01-27 | 로켓 파마슈티컬스, 리미티드 | 다논병을 치료하기 위한 유전자 요법 벡터 |
| 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 |
| EP3927841A1 (en) * | 2019-02-21 | 2021-12-29 | F. Hoffmann-La Roche AG | Improved nucleic acid target enrichment and related methods |
| SG11202112347RA (en) * | 2019-05-23 | 2021-12-30 | Spacecraft Seven Llc | Gene therapy vectors for infantile malignant osteopetrosis |
| DE102020111571A1 (de) | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| KR20230043869A (ko) | 2020-08-07 | 2023-03-31 | 스페이스크래프트 세븐, 엘엘씨 | Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법 |
| AU2022264498A1 (en) * | 2021-04-26 | 2023-09-28 | Strm.Bio Incorporated | Compositions and methods related to megakaryocyte-derived extracellular vesicles for fanconi anemia |
| US20250221931A1 (en) * | 2022-03-25 | 2025-07-10 | 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 | |
| US20070048285A1 (en) | 2005-08-24 | 2007-03-01 | Christopher Baum | Self-inactivating retroviral vector |
| WO2010126766A1 (en) * | 2009-04-30 | 2010-11-04 | 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 |
| KR20140045411A (ko) | 2011-05-16 | 2014-04-16 | 젠자임 코포레이션 | Ｃｘｃｒ４ 길항제의 용도 |
| AU2013293270B2 (en) * | 2012-07-25 | 2018-08-16 | Massachusetts Institute Of Technology | Inducible DNA binding proteins and genome perturbation tools and applications thereof |
| WO2014100073A2 (en) * | 2012-12-21 | 2014-06-26 | 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 |
| US20170037431A1 (en) | 2014-05-01 | 2017-02-09 | University Of Washington | In vivo Gene Engineering with Adenoviral Vectors |
| WO2015188191A1 (en) * | 2014-06-06 | 2015-12-10 | Wong Wilson W | Dna recombinase circuits for logical control of gene expression |
| WO2016118780A1 (en) | 2015-01-21 | 2016-07-28 | 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 KR KR1020247018425A patent/KR20240151726A/ko not_active Ceased
- 2017-09-08 IL IL320371A patent/IL320371A/en unknown
- 2017-09-08 SG SG11201901718VA patent/SG11201901718VA/en unknown
- 2017-09-08 CA CA3035605A patent/CA3035605A1/en active Pending
- 2017-09-08 KR KR1020197009772A patent/KR102672636B1/ko active Active
- 2017-09-08 EP EP17849687.3A patent/EP3510162A4/en active Pending
- 2017-09-08 JP JP2019513876A patent/JP7197466B2/ja active Active
- 2017-09-08 IL IL265196A patent/IL265196B2/en unknown
- 2017-09-08 MX MX2019002699A patent/MX2019002699A/es unknown
- 2017-09-08 CN CN201780068686.XA patent/CN110536966A/zh active Pending
- 2017-09-08 US US16/331,455 patent/US20190203225A1/en active Pending
- 2017-09-08 AU AU2017322511A patent/AU2017322511B2/en active Active
- 2017-09-08 WO PCT/US2017/050837 patent/WO2018049273A1/en not_active Ceased
-
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
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019533434A5 (https=) | ||
| IL320371A (en) | Gene therapy for patients with fanconi anemia | |
| CA3076270C (en) | Retroviral vectors | |
| CN112040988A (zh) | 修饰的aav衣壳多肽用于治疗肌肉疾病 | |
| US20210222178A1 (en) | MICROBIAL SYSTEM FOR PRODUCTION AND DELIVERY OF EUKARYOTE-TRANSLATABLE mRNA TO EUKARYA | |
| JP2017505626A5 (https=) | ||
| JP2019501882A5 (https=) | ||
| JP2024521811A5 (https=) | ||
| JP2020533969A5 (https=) | ||
| JP2022514465A5 (https=) | ||
| CN112442514B (zh) | 慢病毒包装载体系统、慢病毒及其构建方法、试剂盒 | |
| JP5123922B2 (ja) | 融合糖タンパク質から成るエンベロープを有する逆行性輸送ウィルスベクター系 | |
| JP2021528407A5 (https=) | ||
| JP2011115077A5 (https=) | ||
| AU2021206846A1 (en) | Lentivirus packaging system, method for improving lentivirus production in a host cell, and method for treating cancer | |
| CN101532031A (zh) | 非整合慢病毒载体系统及其制备与应用 | |
| US20230416777A1 (en) | Moloney murine leukemia virus-based self-inactivating vector and applications thereof | |
| JP2020517289A5 (https=) | ||
| CN116555351B (zh) | 一种核酸构建体 | |
| CN117866107A (zh) | 靶向cd5分子的嵌合抗原受体t细胞及其抗肿瘤应用 | |
| RU2023119872A (ru) | Генная терапия пациентов с анемией фанкони | |
| CN121342932B (zh) | 许氏平鲉内源性逆转录病毒包膜蛋白P_env、慢病毒载体及其应用 | |
| CN115197969A (zh) | 一种治疗乙型肝炎的慢病毒载体、慢病毒颗粒及其制备方法和应用 | |
| JP2012110290A5 (https=) | ||
| JP2024516115A5 (https=) |