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 (cg-RX-API-DMAC7.html) | 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 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3510162A4 (cg-RX-API-DMAC7.html) |
| JP (3) | JP7197466B2 (cg-RX-API-DMAC7.html) |
| KR (2) | KR102672636B1 (cg-RX-API-DMAC7.html) |
| CN (1) | CN110536966A (cg-RX-API-DMAC7.html) |
| AU (3) | AU2017322511B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3035605A1 (cg-RX-API-DMAC7.html) |
| IL (2) | IL265196B2 (cg-RX-API-DMAC7.html) |
| MX (1) | MX2019002699A (cg-RX-API-DMAC7.html) |
| SG (1) | SG11201901718VA (cg-RX-API-DMAC7.html) |
| WO (1) | WO2018049273A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX382997B (es) | 2016-04-20 | 2025-03-13 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Composiciones y metodos para mejorar la expresion genetica de pklr. |
| KR102726267B1 (ko) | 2017-10-16 | 2024-11-11 | 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. | 피루베이트 키나제 결핍증을 치료하기 위한 pklr 전달용 렌티바이러스 벡터 |
| CA3093708A1 (en) * | 2018-04-11 | 2019-10-17 | Rocket Pharmaceuticals, Ltd. | Compositions and methods for treating fanconi anemia |
| WO2019210325A1 (en) | 2018-04-27 | 2019-10-31 | Rocket Pharmaceuticals, Ltd. | Gene therapy for cns degeneration |
| SG11202100022SA (en) * | 2018-07-12 | 2021-01-28 | Rocket Pharmaceuticals Ltd | Gene therapy vectors for treatment of danon disease |
| IL280465B2 (en) * | 2018-07-30 | 2025-03-01 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Methods for gene modification of hematopoietic cells |
| 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 |
| WO2020169666A1 (en) * | 2019-02-21 | 2020-08-27 | F. Hoffmann-La Roche Ag | Improved nucleic acid target enrichment and related methods |
| AU2020278046A1 (en) * | 2019-05-23 | 2022-01-27 | 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 |
| EP4192487A4 (en) | 2020-08-07 | 2024-10-02 | Spacecraft Seven, LLC | GENE THERAPY FOR PLAKOPHILIN-2 (PKP2) INVOLVING AAV VECTOR |
| WO2022232193A1 (en) * | 2021-04-26 | 2022-11-03 | Strm.Bio Incorporated | Compositions and methods related to megakaryocyte-derived extracellular vesicles for fanconi anemia |
| EP4499153A2 (en) * | 2022-03-25 | 2025-02-05 | 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 (cg-RX-API-DMAC7.html) | 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 |
| US8556882B2 (en) * | 2009-04-30 | 2013-10-15 | 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 |
| JP2014513727A (ja) | 2011-05-16 | 2014-06-05 | ジェンザイム・コーポレーション | Ｃｘｃｒ４拮抗薬の使用 |
| EP3808844A1 (en) * | 2012-07-25 | 2021-04-21 | The Broad Institute, Inc. | 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 |
| US9932588B2 (en) | 2013-11-15 | 2018-04-03 | 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 |
| US11030531B2 (en) * | 2014-06-06 | 2021-06-08 | Trustees Of Boston University | DNA recombinase circuits for logical control of gene expression |
| EP3247808B1 (en) | 2015-01-21 | 2021-05-05 | 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 SG SG11201901718VA patent/SG11201901718VA/en unknown
- 2017-09-08 CA CA3035605A patent/CA3035605A1/en active Pending
- 2017-09-08 MX MX2019002699A patent/MX2019002699A/es unknown
- 2017-09-08 IL IL265196A patent/IL265196B2/en unknown
- 2017-09-08 CN CN201780068686.XA patent/CN110536966A/zh active Pending
- 2017-09-08 KR KR1020197009772A patent/KR102672636B1/ko active Active
- 2017-09-08 IL IL320371A patent/IL320371A/en unknown
- 2017-09-08 JP JP2019513876A patent/JP7197466B2/ja active Active
- 2017-09-08 US US16/331,455 patent/US20190203225A1/en active Pending
- 2017-09-08 EP EP17849687.3A patent/EP3510162A4/en active Pending
- 2017-09-08 KR KR1020247018425A patent/KR20240151726A/ko not_active Ceased
- 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
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