JPWO2022051633A5 - - Google Patents
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- JPWO2022051633A5 JPWO2022051633A5 JP2023514976A JP2023514976A JPWO2022051633A5 JP WO2022051633 A5 JPWO2022051633 A5 JP WO2022051633A5 JP 2023514976 A JP2023514976 A JP 2023514976A JP 2023514976 A JP2023514976 A JP 2023514976A JP WO2022051633 A5 JPWO2022051633 A5 JP WO2022051633A5
- Authority
- JP
- Japan
- Prior art keywords
- growth factor
- aav vector
- engineered
- vector
- engineered aav
- 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
Links
- 239000013607 AAV vector Substances 0.000 claims 9
- 239000000203 mixture Substances 0.000 claims 5
- 108010025020 Nerve Growth Factor Proteins 0.000 claims 4
- 208000012902 Nervous system disease Diseases 0.000 claims 4
- 208000025966 Neurological disease Diseases 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 230000001105 regulatory effect Effects 0.000 claims 4
- QZAYGJVTTNCVMB-UHFFFAOYSA-N serotonin Chemical compound C1=C(O)C=C2C(CCN)=CNC2=C1 QZAYGJVTTNCVMB-UHFFFAOYSA-N 0.000 claims 4
- 238000006467 substitution reaction Methods 0.000 claims 4
- 108090000723 Insulin-Like Growth Factor I Proteins 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 210000001519 tissue Anatomy 0.000 claims 3
- 239000013598 vector Substances 0.000 claims 3
- 102100029470 Apolipoprotein E Human genes 0.000 claims 2
- 101710095339 Apolipoprotein E Proteins 0.000 claims 2
- CJLHTKGWEUGORV-UHFFFAOYSA-N Artemin Chemical compound C1CC2(C)C(O)CCC(=C)C2(O)C2C1C(C)C(=O)O2 CJLHTKGWEUGORV-UHFFFAOYSA-N 0.000 claims 2
- 102000007350 Bone Morphogenetic Proteins Human genes 0.000 claims 2
- 108010007726 Bone Morphogenetic Proteins Proteins 0.000 claims 2
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 claims 2
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims 2
- 102100025903 C-Jun-amino-terminal kinase-interacting protein 3 Human genes 0.000 claims 2
- 101710105203 C-Jun-amino-terminal kinase-interacting protein 3 Proteins 0.000 claims 2
- 108010005939 Ciliary Neurotrophic Factor Proteins 0.000 claims 2
- 102100031614 Ciliary neurotrophic factor Human genes 0.000 claims 2
- 108090000368 Fibroblast growth factor 8 Proteins 0.000 claims 2
- 102000034615 Glial cell line-derived neurotrophic factor Human genes 0.000 claims 2
- 108091010837 Glial cell line-derived neurotrophic factor Proteins 0.000 claims 2
- 102000004547 Glucosylceramidase Human genes 0.000 claims 2
- 108010017544 Glucosylceramidase Proteins 0.000 claims 2
- 102000003693 Hedgehog Proteins Human genes 0.000 claims 2
- 108090000031 Hedgehog Proteins Proteins 0.000 claims 2
- 102000003745 Hepatocyte Growth Factor Human genes 0.000 claims 2
- 108090000100 Hepatocyte Growth Factor Proteins 0.000 claims 2
- 102000014429 Insulin-like growth factor Human genes 0.000 claims 2
- 102000015696 Interleukins Human genes 0.000 claims 2
- 108010063738 Interleukins Proteins 0.000 claims 2
- 102000004058 Leukemia inhibitory factor Human genes 0.000 claims 2
- 108090000581 Leukemia inhibitory factor Proteins 0.000 claims 2
- 102000002233 Myelin-Oligodendrocyte Glycoprotein Human genes 0.000 claims 2
- 108010000123 Myelin-Oligodendrocyte Glycoprotein Proteins 0.000 claims 2
- 102000015336 Nerve Growth Factor Human genes 0.000 claims 2
- 102000007072 Nerve Growth Factors Human genes 0.000 claims 2
- 108700018419 Niemann-Pick C1 Proteins 0.000 claims 2
- 108010038512 Platelet-Derived Growth Factor Proteins 0.000 claims 2
- 102000010780 Platelet-Derived Growth Factor Human genes 0.000 claims 2
- 102100026263 Sphingomyelin phosphodiesterase Human genes 0.000 claims 2
- 101710201924 Sphingomyelin phosphodiesterase 1 Proteins 0.000 claims 2
- 101710095280 Sphingomyelinase C 1 Proteins 0.000 claims 2
- 108010009583 Transforming Growth Factors Proteins 0.000 claims 2
- 102000009618 Transforming Growth Factors Human genes 0.000 claims 2
- 108700019146 Transgenes Proteins 0.000 claims 2
- 108010073929 Vascular Endothelial Growth Factor A Proteins 0.000 claims 2
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 claims 2
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 claims 2
- 108010003205 Vasoactive Intestinal Peptide Proteins 0.000 claims 2
- 102400000015 Vasoactive intestinal peptide Human genes 0.000 claims 2
- 229940112869 bone morphogenetic protein Drugs 0.000 claims 2
- 229940077737 brain-derived neurotrophic factor Drugs 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- VBUWHHLIZKOSMS-RIWXPGAOSA-N invicorp Chemical compound C([C@@H](C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CO)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CC(N)=O)C(O)=O)NC(=O)[C@H](CCCCN)NC(=O)[C@H](CCCCN)NC(=O)[C@@H](NC(=O)[C@H](C)NC(=O)[C@H](CCSC)NC(=O)[C@H](CCC(N)=O)NC(=O)[C@H](CCCCN)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@@H](NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CC(O)=O)NC(=O)[C@@H](NC(=O)[C@H](CC=1C=CC=CC=1)NC(=O)[C@@H](NC(=O)[C@H](C)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CO)NC(=O)[C@@H](N)CC=1NC=NC=1)C(C)C)[C@@H](C)O)[C@@H](C)O)C(C)C)C1=CC=C(O)C=C1 VBUWHHLIZKOSMS-RIWXPGAOSA-N 0.000 claims 2
- 229940053128 nerve growth factor Drugs 0.000 claims 2
- 230000001537 neural effect Effects 0.000 claims 2
- 102000005162 pleiotrophin Human genes 0.000 claims 2
- 229940124597 therapeutic agent Drugs 0.000 claims 2
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 102000003823 Aromatic-L-amino-acid decarboxylases Human genes 0.000 claims 1
- 108090000121 Aromatic-L-amino-acid decarboxylases Proteins 0.000 claims 1
- 102100026376 Artemin Human genes 0.000 claims 1
- 101710205806 Artemin Proteins 0.000 claims 1
- 102100026189 Beta-galactosidase Human genes 0.000 claims 1
- 101710195294 Beta-galactosidase 1 Proteins 0.000 claims 1
- 101710124976 Beta-hexosaminidase A Proteins 0.000 claims 1
- 108091033409 CRISPR Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 102100031361 Fibroblast growth factor 20 Human genes 0.000 claims 1
- 101100412102 Haemophilus influenzae (strain ATCC 51907 / DSM 11121 / KW20 / Rd) rec2 gene Proteins 0.000 claims 1
- HTTJABKRGRZYRN-UHFFFAOYSA-N Heparin Chemical compound OC1C(NC(=O)C)C(O)OC(COS(O)(=O)=O)C1OC1C(OS(O)(=O)=O)C(O)C(OC2C(C(OS(O)(=O)=O)C(OC3C(C(O)C(O)C(O3)C(O)=O)OS(O)(=O)=O)C(CO)O2)NS(O)(=O)=O)C(C(O)=O)O1 HTTJABKRGRZYRN-UHFFFAOYSA-N 0.000 claims 1
- 101000765010 Homo sapiens Beta-galactosidase Proteins 0.000 claims 1
- 101000846532 Homo sapiens Fibroblast growth factor 20 Proteins 0.000 claims 1
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 claims 1
- 102000048143 Insulin-Like Growth Factor II Human genes 0.000 claims 1
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 claims 1
- 241000283923 Marmota monax Species 0.000 claims 1
- 102100033857 Neurotrophin-4 Human genes 0.000 claims 1
- 102100021584 Neurturin Human genes 0.000 claims 1
- 108010015406 Neurturin Proteins 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 102100036660 Persephin Human genes 0.000 claims 1
- 102100021947 Survival motor neuron protein Human genes 0.000 claims 1
- 102000006747 Transforming Growth Factor alpha Human genes 0.000 claims 1
- 108090001012 Transforming Growth Factor beta Proteins 0.000 claims 1
- 102000004887 Transforming Growth Factor beta Human genes 0.000 claims 1
- 101800004564 Transforming growth factor alpha Proteins 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 1
- 229940126864 fibroblast growth factor Drugs 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 229960002897 heparin Drugs 0.000 claims 1
- 229920000669 heparin Polymers 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 108091070501 miRNA Proteins 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 210000002161 motor neuron Anatomy 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 108010070453 persephin Proteins 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 230000006798 recombination Effects 0.000 claims 1
- 238000005215 recombination Methods 0.000 claims 1
- 230000004083 survival effect Effects 0.000 claims 1
- 230000010415 tropism Effects 0.000 claims 1
- VBEQCZHXXJYVRD-GACYYNSASA-N uroanthelone Chemical compound C([C@@H](C(=O)N[C@H](C(=O)N[C@@H](CS)C(=O)N[C@@H](CC(N)=O)C(=O)N[C@@H](CS)C(=O)N[C@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)NCC(=O)N[C@@H](CC=1C=CC(O)=CC=1)C(=O)N[C@@H](CO)C(=O)NCC(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CS)C(=O)N[C@@H](CCC(N)=O)C(=O)N[C@@H]([C@@H](C)O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CC=1C2=CC=CC=C2NC=1)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CC(C)C)C(=O)N[C@@H](CCCNC(N)=N)C(O)=O)C(C)C)[C@@H](C)O)NC(=O)[C@H](CO)NC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CO)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@@H](NC(=O)[C@H](CC=1NC=NC=1)NC(=O)[C@H](CCSC)NC(=O)[C@H](CS)NC(=O)[C@@H](NC(=O)CNC(=O)CNC(=O)[C@H](CC(N)=O)NC(=O)[C@H](CC(C)C)NC(=O)[C@H](CS)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)CNC(=O)[C@H](CC(O)=O)NC(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CO)NC(=O)[C@H](CO)NC(=O)[C@H]1N(CCC1)C(=O)[C@H](CS)NC(=O)CNC(=O)[C@H]1N(CCC1)C(=O)[C@H](CC=1C=CC(O)=CC=1)NC(=O)[C@H](CO)NC(=O)[C@@H](N)CC(N)=O)C(C)C)[C@@H](C)CC)C1=CC=C(O)C=C1 VBEQCZHXXJYVRD-GACYYNSASA-N 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063074548P | 2020-09-04 | 2020-09-04 | |
| US63/074,548 | 2020-09-04 | ||
| PCT/US2021/049081 WO2022051633A2 (en) | 2020-09-04 | 2021-09-03 | Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023540756A JP2023540756A (ja) | 2023-09-26 |
| JPWO2022051633A5 true JPWO2022051633A5 (https=) | 2024-09-11 |
| JP2023540756A5 JP2023540756A5 (https=) | 2024-09-11 |
Family
ID=80491526
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023514976A Pending JP2023540756A (ja) | 2020-09-04 | 2021-09-03 | 向上した形質導入効率及び脳内における広範な分布を有する組換えアデノ随伴ウイルスベクターの新規の操作されたキャプシド血清型 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20230330267A1 (https=) |
| EP (1) | EP4208557A4 (https=) |
| JP (1) | JP2023540756A (https=) |
| CN (1) | CN116249541A (https=) |
| AU (1) | AU2021335601A1 (https=) |
| CA (1) | CA3191543A1 (https=) |
| WO (1) | WO2022051633A2 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20240158861A1 (en) * | 2021-04-23 | 2024-05-16 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and compositions for treating cell senescence accumulation related disease |
| WO2025059678A1 (en) * | 2023-09-15 | 2025-03-20 | Ohio State Innovation Foundation | Novel engineered capsid serotypes of recombinant adeno-associated viral vectors with reduced liver tropism |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1486567A1 (en) * | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| WO2009120978A2 (en) * | 2008-03-27 | 2009-10-01 | The Ohio State University | Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| MX2020002148A (es) * | 2017-08-25 | 2020-07-20 | Ovid Therapeutics Inc | Vectores adenoasociados recombinantes. |
| EP3793615A2 (en) * | 2018-05-16 | 2021-03-24 | Voyager Therapeutics, Inc. | Directed evolution of aav to improve tropism for cns |
-
2021
- 2021-09-03 AU AU2021335601A patent/AU2021335601A1/en not_active Abandoned
- 2021-09-03 JP JP2023514976A patent/JP2023540756A/ja active Pending
- 2021-09-03 CA CA3191543A patent/CA3191543A1/en active Pending
- 2021-09-03 EP EP21865201.4A patent/EP4208557A4/en active Pending
- 2021-09-03 WO PCT/US2021/049081 patent/WO2022051633A2/en not_active Ceased
- 2021-09-03 US US18/024,806 patent/US20230330267A1/en active Pending
- 2021-09-03 CN CN202180054939.4A patent/CN116249541A/zh active Pending
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