JPWO2020041773A5 - - Google Patents
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- JPWO2020041773A5 JPWO2020041773A5 JP2021510015A JP2021510015A JPWO2020041773A5 JP WO2020041773 A5 JPWO2020041773 A5 JP WO2020041773A5 JP 2021510015 A JP2021510015 A JP 2021510015A JP 2021510015 A JP2021510015 A JP 2021510015A JP WO2020041773 A5 JPWO2020041773 A5 JP WO2020041773A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nucleic acid
- cpg
- modified
- expression cassette
- 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.)
- Pending
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- 102000004169 proteins and genes Human genes 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 14
- 102100026735 Coagulation factor VIII Human genes 0.000 claims description 8
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims description 8
- 238000012217 deletion Methods 0.000 claims description 6
- 230000037430 deletion Effects 0.000 claims description 6
- 102000002233 Myelin-Oligodendrocyte Glycoprotein Human genes 0.000 claims description 3
- 108010000123 Myelin-Oligodendrocyte Glycoprotein Proteins 0.000 claims description 3
- 239000000427 antigen Substances 0.000 claims description 3
- 108091007433 antigens Proteins 0.000 claims description 3
- 102000036639 antigens Human genes 0.000 claims description 3
- 208000023275 Autoimmune disease Diseases 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 claims description 2
- 150000007523 nucleic acids Chemical group 0.000 claims 94
- CTMZLDSMFCVUNX-VMIOUTBZSA-N cytidylyl-(3'->5')-guanosine Chemical group O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@H](OP(O)(=O)OC[C@@H]2[C@H]([C@@H](O)[C@@H](O2)N2C3=C(C(N=C(N)N3)=O)N=C2)O)[C@@H](CO)O1 CTMZLDSMFCVUNX-VMIOUTBZSA-N 0.000 claims 66
- 102000039446 nucleic acids Human genes 0.000 claims 55
- 108020004707 nucleic acids Proteins 0.000 claims 55
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 39
- 102000015081 Blood Coagulation Factors Human genes 0.000 claims 30
- 108010039209 Blood Coagulation Factors Proteins 0.000 claims 30
- 239000002773 nucleotide Substances 0.000 claims 30
- 125000003729 nucleotide group Chemical group 0.000 claims 30
- 239000003114 blood coagulation factor Substances 0.000 claims 28
- 239000008194 pharmaceutical composition Substances 0.000 claims 19
- 108091033319 polynucleotide Proteins 0.000 claims 19
- 102000040430 polynucleotide Human genes 0.000 claims 19
- 239000002157 polynucleotide Substances 0.000 claims 19
- 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 17
- 108010054218 Factor VIII Proteins 0.000 claims 15
- 102000001690 Factor VIII Human genes 0.000 claims 15
- 230000023555 blood coagulation Effects 0.000 claims 15
- 239000013607 AAV vector Substances 0.000 claims 14
- 230000001105 regulatory effect Effects 0.000 claims 13
- 230000001225 therapeutic effect Effects 0.000 claims 12
- 229960000301 factor viii Drugs 0.000 claims 11
- 108700019146 Transgenes Proteins 0.000 claims 8
- 210000000234 capsid Anatomy 0.000 claims 8
- 238000013518 transcription Methods 0.000 claims 6
- 230000035897 transcription Effects 0.000 claims 6
- 241000282412 Homo Species 0.000 claims 5
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 5
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 4
- 208000009292 Hemophilia A Diseases 0.000 claims 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 3
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 3
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 3
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 3
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 3
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 3
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 3
- 102100022641 Coagulation factor IX Human genes 0.000 claims 3
- 102100023804 Coagulation factor VII Human genes 0.000 claims 2
- 108090000790 Enzymes Proteins 0.000 claims 2
- 102000004190 Enzymes Human genes 0.000 claims 2
- 108010076282 Factor IX Proteins 0.000 claims 2
- 108010023321 Factor VII Proteins 0.000 claims 2
- 206010019860 Hereditary angioedema Diseases 0.000 claims 2
- 108010029485 Protein Isoforms Proteins 0.000 claims 2
- 102000001708 Protein Isoforms Human genes 0.000 claims 2
- 230000004071 biological effect Effects 0.000 claims 2
- 229940009550 c1 esterase inhibitor Drugs 0.000 claims 2
- 210000003169 central nervous system Anatomy 0.000 claims 2
- 229940088598 enzyme Drugs 0.000 claims 2
- 229960004222 factor ix Drugs 0.000 claims 2
- 229940012413 factor vii Drugs 0.000 claims 2
- 208000027866 inflammatory disease Diseases 0.000 claims 2
- 230000002401 inhibitory effect Effects 0.000 claims 2
- 230000002132 lysosomal effect Effects 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 239000013598 vector Substances 0.000 claims 2
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 241000958487 Adeno-associated virus 3B Species 0.000 claims 1
- 108010060219 Apolipoprotein E2 Proteins 0.000 claims 1
- 108010025628 Apolipoproteins E Proteins 0.000 claims 1
- 102000013918 Apolipoproteins E Human genes 0.000 claims 1
- 102000055157 Complement C1 Inhibitor Human genes 0.000 claims 1
- 108091029430 CpG site Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 208000032843 Hemorrhage Diseases 0.000 claims 1
- 108091092195 Intron Proteins 0.000 claims 1
- 102100024295 Maltase-glucoamylase Human genes 0.000 claims 1
- 108050007539 Plasma protease C1 inhibitor Proteins 0.000 claims 1
- 101800004937 Protein C Proteins 0.000 claims 1
- 102000017975 Protein C Human genes 0.000 claims 1
- 101800001700 Saposin-D Proteins 0.000 claims 1
- 230000002378 acidificating effect Effects 0.000 claims 1
- 208000026935 allergic disease Diseases 0.000 claims 1
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 1
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 1
- 108010028144 alpha-Glucosidases Proteins 0.000 claims 1
- 238000003556 assay Methods 0.000 claims 1
- 230000000740 bleeding effect Effects 0.000 claims 1
- 230000037396 body weight Effects 0.000 claims 1
- 238000007820 coagulation assay Methods 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 108020001507 fusion proteins Proteins 0.000 claims 1
- 102000037865 fusion proteins Human genes 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 210000005229 liver cell Anatomy 0.000 claims 1
- 230000004770 neurodegeneration Effects 0.000 claims 1
- 208000015122 neurodegenerative disease Diseases 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 230000001681 protective effect Effects 0.000 claims 1
- 229960000856 protein c Drugs 0.000 claims 1
- 230000028327 secretion Effects 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
- 239000004094 surface-active agent Substances 0.000 claims 1
- 230000001839 systemic circulation Effects 0.000 claims 1
- 206010067584 Type 1 diabetes mellitus Diseases 0.000 description 7
- 201000006417 multiple sclerosis Diseases 0.000 description 3
- 108091022930 Glutamate decarboxylase Proteins 0.000 description 2
- 208000003807 Graves Disease Diseases 0.000 description 2
- 208000015023 Graves' disease Diseases 0.000 description 2
- 102000011923 Thyrotropin Human genes 0.000 description 2
- 108010061174 Thyrotropin Proteins 0.000 description 2
- NOESYZHRGYRDHS-UHFFFAOYSA-N insulin Chemical compound N1C(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(NC(=O)CN)C(C)CC)CSSCC(C(NC(CO)C(=O)NC(CC(C)C)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CCC(N)=O)C(=O)NC(CC(C)C)C(=O)NC(CCC(O)=O)C(=O)NC(CC(N)=O)C(=O)NC(CC=2C=CC(O)=CC=2)C(=O)NC(CSSCC(NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2C=CC(O)=CC=2)NC(=O)C(CC(C)C)NC(=O)C(C)NC(=O)C(CCC(O)=O)NC(=O)C(C(C)C)NC(=O)C(CC(C)C)NC(=O)C(CC=2NC=NC=2)NC(=O)C(CO)NC(=O)CNC2=O)C(=O)NCC(=O)NC(CCC(O)=O)C(=O)NC(CCCNC(N)=N)C(=O)NCC(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC=CC=3)C(=O)NC(CC=3C=CC(O)=CC=3)C(=O)NC(C(C)O)C(=O)N3C(CCC3)C(=O)NC(CCCCN)C(=O)NC(C)C(O)=O)C(=O)NC(CC(N)=O)C(O)=O)=O)NC(=O)C(C(C)CC)NC(=O)C(CO)NC(=O)C(C(C)O)NC(=O)C1CSSCC2NC(=O)C(CC(C)C)NC(=O)C(NC(=O)C(CCC(N)=O)NC(=O)C(CC(N)=O)NC(=O)C(NC(=O)C(N)CC=1C=CC=CC=1)C(C)C)CC1=CN=CN1 NOESYZHRGYRDHS-UHFFFAOYSA-N 0.000 description 2
- 102000005962 receptors Human genes 0.000 description 2
- 108020003175 receptors Proteins 0.000 description 2
- 206010039073 rheumatoid arthritis Diseases 0.000 description 2
- KISWVXRQTGLFGD-UHFFFAOYSA-N 2-[[2-[[6-amino-2-[[2-[[2-[[5-amino-2-[[2-[[1-[2-[[6-amino-2-[(2,5-diamino-5-oxopentanoyl)amino]hexanoyl]amino]-5-(diaminomethylideneamino)pentanoyl]pyrrolidine-2-carbonyl]amino]-3-hydroxypropanoyl]amino]-5-oxopentanoyl]amino]-5-(diaminomethylideneamino)p Chemical compound C1CCN(C(=O)C(CCCN=C(N)N)NC(=O)C(CCCCN)NC(=O)C(N)CCC(N)=O)C1C(=O)NC(CO)C(=O)NC(CCC(N)=O)C(=O)NC(CCCN=C(N)N)C(=O)NC(CO)C(=O)NC(CCCCN)C(=O)NC(C(=O)NC(CC(C)C)C(O)=O)CC1=CC=C(O)C=C1 KISWVXRQTGLFGD-UHFFFAOYSA-N 0.000 description 1
- 108010009685 Cholinergic Receptors Proteins 0.000 description 1
- 102000005598 Chondroitin Sulfate Proteoglycans Human genes 0.000 description 1
- 108010059480 Chondroitin Sulfate Proteoglycans Proteins 0.000 description 1
- 102100036255 Glucose-6-phosphatase 2 Human genes 0.000 description 1
- 102000008214 Glutamate decarboxylase Human genes 0.000 description 1
- 102000003886 Glycoproteins Human genes 0.000 description 1
- 108090000288 Glycoproteins Proteins 0.000 description 1
- 101000930907 Homo sapiens Glucose-6-phosphatase 2 Proteins 0.000 description 1
- 108090001061 Insulin Proteins 0.000 description 1
- 102000004877 Insulin Human genes 0.000 description 1
- OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 description 1
- 206010028372 Muscular weakness Diseases 0.000 description 1
- 102000047918 Myelin Basic Human genes 0.000 description 1
- 102000006386 Myelin Proteins Human genes 0.000 description 1
- 108010083674 Myelin Proteins Proteins 0.000 description 1
- 101710107068 Myelin basic protein Proteins 0.000 description 1
- 102100023302 Myelin-oligodendrocyte glycoprotein Human genes 0.000 description 1
- 102000016611 Proteoglycans Human genes 0.000 description 1
- 108010067787 Proteoglycans Proteins 0.000 description 1
- 102000016202 Proteolipids Human genes 0.000 description 1
- 108010010974 Proteolipids Proteins 0.000 description 1
- 210000001744 T-lymphocyte Anatomy 0.000 description 1
- 102000034337 acetylcholine receptors Human genes 0.000 description 1
- 125000000539 amino acid group Chemical group 0.000 description 1
- 150000001413 amino acids Chemical group 0.000 description 1
- 206010003246 arthritis Diseases 0.000 description 1
- 229940125396 insulin Drugs 0.000 description 1
- 206010022498 insulinoma Diseases 0.000 description 1
- 210000004153 islets of langerhan Anatomy 0.000 description 1
- 230000036473 myasthenia Effects 0.000 description 1
- 210000005012 myelin Anatomy 0.000 description 1
- 208000021255 pancreatic insulinoma Diseases 0.000 description 1
- 208000033808 peripheral neuropathy Diseases 0.000 description 1
- 108010066381 preproinsulin Proteins 0.000 description 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 description 1
- NGVDGCNFYWLIFO-UHFFFAOYSA-N pyridoxal 5'-phosphate Chemical compound CC1=NC=C(COP(O)(O)=O)C(C=O)=C1O NGVDGCNFYWLIFO-UHFFFAOYSA-N 0.000 description 1
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024060919A JP2024102065A (ja) | 2018-08-24 | 2024-04-04 | 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862722547P | 2018-08-24 | 2018-08-24 | |
| US62/722,547 | 2018-08-24 | ||
| US201862725096P | 2018-08-30 | 2018-08-30 | |
| US62/725,096 | 2018-08-30 | ||
| US201862784116P | 2018-12-21 | 2018-12-21 | |
| US62/784,116 | 2018-12-21 | ||
| PCT/US2019/048032 WO2020041773A1 (en) | 2018-08-24 | 2019-08-23 | Optimized promoter sequences, intron-free expression constructs and methods of use |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024060919A Division JP2024102065A (ja) | 2018-08-24 | 2024-04-04 | 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021533805A JP2021533805A (ja) | 2021-12-09 |
| JPWO2020041773A5 true JPWO2020041773A5 (enExample) | 2022-04-26 |
Family
ID=69591143
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021510015A Pending JP2021533805A (ja) | 2018-08-24 | 2019-08-23 | 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法 |
| JP2024060919A Pending JP2024102065A (ja) | 2018-08-24 | 2024-04-04 | 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024060919A Pending JP2024102065A (ja) | 2018-08-24 | 2024-04-04 | 最適化されたプロモーター配列、イントロンを含まない発現構築物及び使用方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12290574B2 (enExample) |
| EP (1) | EP3840775A4 (enExample) |
| JP (2) | JP2021533805A (enExample) |
| CN (1) | CN113226352B (enExample) |
| AU (2) | AU2019325688B2 (enExample) |
| BR (1) | BR112021003399A2 (enExample) |
| CA (1) | CA3111047A1 (enExample) |
| MX (1) | MX2021002198A (enExample) |
| TW (1) | TW202012425A (enExample) |
| WO (1) | WO2020041773A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MA50546A (fr) | 2017-06-07 | 2020-09-16 | Regeneron Pharma | Compositions et méthodes pour l'internalisation d'enzymes |
| AU2019218892B2 (en) | 2018-02-07 | 2025-08-14 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| AU2019269685B2 (en) | 2018-05-17 | 2025-12-04 | Regeneron Pharmaceuticals, Inc. | Anti-CD63 antibodies, conjugates, and uses thereof |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| CA3218126A1 (en) * | 2021-05-07 | 2022-11-10 | Matthew MANGANIELLO | Lyophilized non-viral dna vector compositions and uses thereof |
| EP4333886A4 (en) * | 2021-05-07 | 2025-11-12 | Generation Bio Co | NON-VIRAL DNA VECTORS FOR VACCINE ADMINISTRATION |
| CR20240524A (es) * | 2022-04-28 | 2025-05-05 | Biocad Joint Stock Co | Ácido nucleico de codón optimizado que codifica la proteína del factor viii de coagulación de dominio b suprimido, y uso del mismo |
| CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
| WO2024074143A1 (en) * | 2022-10-08 | 2024-04-11 | Lingyi Biotech Co., Ltd. | Construct for enhancing gene expression |
| CN119242712A (zh) * | 2024-09-27 | 2025-01-03 | 南京师范大学 | 特异性靶向骨骼肌细胞的携带miRNA-486基因的腺相关病毒载体、药物组合物 |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20020065236A1 (en) | 1998-09-09 | 2002-05-30 | Yew Nelson S. | CpG reduced plasmids and viral vectors |
| WO2006060641A2 (en) * | 2004-12-01 | 2006-06-08 | Genzyme Corporation | Methods for targeted delivery of genetic material to the liver |
| US8008468B2 (en) * | 2005-02-16 | 2011-08-30 | Benitec, Inc. | RNAi expression constructs with liver-specific enhancer/promoter |
| AU2011223820B2 (en) | 2010-03-01 | 2016-01-14 | The Children's Hospital Of Philadelphia | Nucleic acids for targeting multiple regions of the HCV genome |
| US20140271550A1 (en) | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| DK3133923T3 (da) * | 2014-04-24 | 2020-08-10 | Univ Florida | Aav-based gene therapy for multiple sclerosis |
| CA2987103A1 (en) * | 2015-05-28 | 2016-12-01 | Cornell University | Adeno-associated virus mediated delivery of c1ei as a therapy for angioedema |
| PE20180675A1 (es) | 2015-06-23 | 2018-04-19 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos |
| RU2020126034A (ru) * | 2015-09-24 | 2020-09-02 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| EP3368664A4 (en) * | 2015-10-28 | 2019-08-28 | Sangamo Therapeutics, Inc. | LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF |
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| EP3293260A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| BR112019004785A2 (pt) | 2016-09-12 | 2019-06-04 | Genethon | variantes de alfa-glicosidase ácida e usos das mesmas |
| EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| CN120665951A (zh) | 2018-02-07 | 2025-09-19 | 吉尼松公司 | 杂合调控元件 |
| RU2020129448A (ru) | 2018-02-08 | 2022-03-09 | Сангамо Терапьютикс, Инк. | Сконструированные специфические для мишени нуклеазы |
| EP3794043A4 (en) | 2018-05-16 | 2022-02-23 | Spark Therapeutics, Inc. | CODON-ENHANCED ACID ALPHA-GLUCOSIDASE EXPRESSION CASSETTES AND METHODS OF USE THEREOF |
| EP3829619A4 (en) | 2018-07-27 | 2022-05-18 | REGENXBIO Inc. | TREATMENT OF IVA MUCOPOLYSACCHARIDOSIS |
| CA3128875A1 (en) | 2019-02-04 | 2020-08-13 | Freeline Therapeutics Limited | Polynucleotides |
| AU2019428629A1 (en) | 2019-02-06 | 2021-01-28 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type I |
| KR20210153069A (ko) | 2019-04-19 | 2021-12-16 | 제네똥 | 섬유아세포 성장 인자 23 관련 저인산혈증성 질환의 유전자 요법 |
| CA3137284A1 (en) | 2019-04-24 | 2020-10-29 | Regenxbio Inc. | Fully-human post-translationally modified antibody therapeutics |
| US20230042103A1 (en) | 2019-07-26 | 2023-02-09 | Regenxbio Inc. | Engineered nucleic acid regulatory element and methods of uses thereof |
| WO2021078833A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric polypeptides and uses thereof |
| WO2021078834A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric acid-alpha glucosidase polypeptides and uses thereof |
| TW202130812A (zh) | 2019-11-01 | 2021-08-16 | 美商聖加莫治療股份有限公司 | 用於基因體工程之組合物及方法 |
| WO2021084277A2 (en) | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Transcription regulatory elements |
| EP3913060A1 (en) | 2020-05-22 | 2021-11-24 | Genethon | Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy |
| GB202009741D0 (en) | 2020-06-25 | 2020-08-12 | Freeline Therapeutics Ltd | Polynucleotide |
| AU2022227005B2 (en) | 2021-02-26 | 2025-09-11 | Logicbio Therapeutics, Inc. | Manufacturing and use of recombinant aav vectors |
-
2019
- 2019-08-23 WO PCT/US2019/048032 patent/WO2020041773A1/en not_active Ceased
- 2019-08-23 CA CA3111047A patent/CA3111047A1/en active Pending
- 2019-08-23 JP JP2021510015A patent/JP2021533805A/ja active Pending
- 2019-08-23 AU AU2019325688A patent/AU2019325688B2/en active Active
- 2019-08-23 MX MX2021002198A patent/MX2021002198A/es unknown
- 2019-08-23 BR BR112021003399-8A patent/BR112021003399A2/pt unknown
- 2019-08-23 US US17/250,712 patent/US12290574B2/en active Active
- 2019-08-23 CN CN201980070435.4A patent/CN113226352B/zh active Active
- 2019-08-23 EP EP19852321.9A patent/EP3840775A4/en active Pending
- 2019-08-26 TW TW108130505A patent/TW202012425A/zh unknown
-
2024
- 2024-04-04 JP JP2024060919A patent/JP2024102065A/ja active Pending
-
2025
- 2025-06-04 AU AU2025204161A patent/AU2025204161A1/en active Pending
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