JPWO2020061581A5 - - Google Patents
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- JPWO2020061581A5 JPWO2020061581A5 JP2021515515A JP2021515515A JPWO2020061581A5 JP WO2020061581 A5 JPWO2020061581 A5 JP WO2020061581A5 JP 2021515515 A JP2021515515 A JP 2021515515A JP 2021515515 A JP2021515515 A JP 2021515515A JP WO2020061581 A5 JPWO2020061581 A5 JP WO2020061581A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- item
- sequence encoding
- sequences
- plasmid vector
- 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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- 235000018102 proteins Nutrition 0.000 claims description 62
- 102000004169 proteins and genes Human genes 0.000 claims description 62
- 108090000623 proteins and genes Proteins 0.000 claims description 62
- 230000003612 virological Effects 0.000 claims description 62
- 239000002245 particle Substances 0.000 claims description 56
- 239000000203 mixture Substances 0.000 claims description 49
- 210000004027 cells Anatomy 0.000 claims description 39
- 238000004191 hydrophobic interaction chromatography Methods 0.000 claims description 34
- 239000002609 media Substances 0.000 claims description 34
- 229920002873 Polyethylenimine Polymers 0.000 claims description 28
- 238000005277 cation exchange chromatography Methods 0.000 claims description 28
- 238000009295 crossflow filtration Methods 0.000 claims description 27
- 102100020499 GRK1 Human genes 0.000 claims description 25
- 229920001850 Nucleic acid sequence Polymers 0.000 claims description 24
- 239000011780 sodium chloride Substances 0.000 claims description 24
- 101710028887 CKL_0015 Proteins 0.000 claims description 22
- 101710029785 DA26 Proteins 0.000 claims description 22
- 101710028938 M1627_0030 Proteins 0.000 claims description 22
- 101710011281 MDV056 Proteins 0.000 claims description 22
- 101700028338 ORF15 Proteins 0.000 claims description 22
- 101710030183 ORF161 Proteins 0.000 claims description 22
- 101710043875 Vi01_157c Proteins 0.000 claims description 22
- 101700019905 YD2M Proteins 0.000 claims description 22
- 238000005349 anion exchange Methods 0.000 claims description 22
- 101700030621 ycbP Proteins 0.000 claims description 22
- FAPWRFPIFSIZLT-UHFFFAOYSA-M sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 20
- 230000000875 corresponding Effects 0.000 claims description 17
- 101700001406 RPGR Proteins 0.000 claims description 16
- 102100009234 RPGR Human genes 0.000 claims description 16
- 238000001890 transfection Methods 0.000 claims description 16
- 150000003839 salts Chemical class 0.000 claims description 14
- 108090000799 EC 2.7.11.14 Proteins 0.000 claims description 13
- 108091004285 G-Protein-Coupled Receptor Kinase 1 Proteins 0.000 claims description 13
- 101700083645 GRK1 Proteins 0.000 claims description 12
- UIIMBOGNXHQVGW-UHFFFAOYSA-M NaHCO3 Chemical compound [Na+].OC([O-])=O UIIMBOGNXHQVGW-UHFFFAOYSA-M 0.000 claims description 12
- 239000003795 chemical substances by application Substances 0.000 claims description 12
- 239000012537 formulation buffer Substances 0.000 claims description 12
- 238000003306 harvesting Methods 0.000 claims description 12
- 108010006025 bovine growth hormone Proteins 0.000 claims description 11
- 229920003013 deoxyribonucleic acid Polymers 0.000 claims description 8
- 239000012510 hollow fiber Substances 0.000 claims description 8
- WCUXLLCKKVVCTQ-UHFFFAOYSA-M potassium chloride Chemical compound [Cl-].[K+] WCUXLLCKKVVCTQ-UHFFFAOYSA-M 0.000 claims description 8
- 229960002743 Glutamine Drugs 0.000 claims description 6
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical compound OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 claims description 6
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L MgCl2 Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 claims description 6
- 108020004412 RNA 3' Polyadenylation Signals Proteins 0.000 claims description 6
- QORWJWZARLRLPR-UHFFFAOYSA-H Tricalcium phosphate Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 claims description 6
- 241000700605 Viruses Species 0.000 claims description 6
- 239000001506 calcium phosphate Substances 0.000 claims description 6
- 229910000389 calcium phosphate Inorganic materials 0.000 claims description 6
- 235000011010 calcium phosphates Nutrition 0.000 claims description 6
- 238000004587 chromatography analysis Methods 0.000 claims description 6
- CSNNHWWHGAXBCP-UHFFFAOYSA-L mgso4 Chemical compound [Mg+2].[O-][S+2]([O-])([O-])[O-] CSNNHWWHGAXBCP-UHFFFAOYSA-L 0.000 claims description 6
- 239000002105 nanoparticle Substances 0.000 claims description 6
- 229920000642 polymer Polymers 0.000 claims description 6
- 239000000243 solution Substances 0.000 claims description 6
- 238000010361 transduction Methods 0.000 claims description 5
- 230000026683 transduction Effects 0.000 claims description 5
- HJCMDXDYPOUFDY-WHFBIAKZSA-N Ala-Gln Chemical compound C[C@H](N)C(=O)N[C@H](C(O)=O)CCC(N)=O HJCMDXDYPOUFDY-WHFBIAKZSA-N 0.000 claims description 4
- 210000000234 Capsid Anatomy 0.000 claims description 4
- 210000000170 Cell Membrane Anatomy 0.000 claims description 4
- 239000006144 Dulbecco’s modified Eagle's medium Substances 0.000 claims description 4
- 229960001031 Glucose Drugs 0.000 claims description 4
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 claims description 4
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 claims description 4
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 claims description 4
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 claims description 4
- MTCFGRXMJLQNBG-REOHCLBHSA-N L-serine Chemical compound OC[C@H](N)C(O)=O MTCFGRXMJLQNBG-REOHCLBHSA-N 0.000 claims description 4
- AYFVYJQAPQTCCC-GBXIJSLDSA-N L-threonine Chemical compound C[C@@H](O)[C@H](N)C(O)=O AYFVYJQAPQTCCC-GBXIJSLDSA-N 0.000 claims description 4
- QIVBCDIJIAJPQS-VIFPVBQESA-N L-tryptophane Chemical compound C1=CC=C2C(C[C@H](N)C(O)=O)=CNC2=C1 QIVBCDIJIAJPQS-VIFPVBQESA-N 0.000 claims description 4
- KZSNJWFQEVHDMF-BYPYZUCNSA-N L-valine Chemical compound CC(C)[C@H](N)C(O)=O KZSNJWFQEVHDMF-BYPYZUCNSA-N 0.000 claims description 4
- 239000004695 Polyether sulfone Substances 0.000 claims description 4
- GSEJCLTVZPLZKY-UHFFFAOYSA-N Tris Chemical compound OCCN(CCO)CCO GSEJCLTVZPLZKY-UHFFFAOYSA-N 0.000 claims description 4
- 239000007983 Tris buffer Substances 0.000 claims description 4
- 230000015572 biosynthetic process Effects 0.000 claims description 4
- 238000010790 dilution Methods 0.000 claims description 4
- 230000000694 effects Effects 0.000 claims description 4
- 238000001914 filtration Methods 0.000 claims description 4
- OVBPIULPVIDEAO-LBPRGKRZSA-N folic acid Chemical compound C=1N=C2NC(N)=NC(=O)C2=NC=1CNC1=CC=C(C(=O)N[C@@H](CCC(O)=O)C(O)=O)C=C1 OVBPIULPVIDEAO-LBPRGKRZSA-N 0.000 claims description 4
- 238000005755 formation reaction Methods 0.000 claims description 4
- VCJMYUPGQJHHFU-UHFFFAOYSA-N iron(3+);trinitrate Chemical compound [Fe+3].[O-][N+]([O-])=O.[O-][N+]([O-])=O.[O-][N+]([O-])=O VCJMYUPGQJHHFU-UHFFFAOYSA-N 0.000 claims description 4
- 229910052943 magnesium sulfate Inorganic materials 0.000 claims description 4
- 235000019341 magnesium sulphate Nutrition 0.000 claims description 4
- 239000003550 marker Substances 0.000 claims description 4
- 239000011159 matrix material Substances 0.000 claims description 4
- 230000003472 neutralizing Effects 0.000 claims description 4
- 229920001993 poloxamer 188 Polymers 0.000 claims description 4
- 229920002496 poly(ether sulfone) Polymers 0.000 claims description 4
- 230000001402 polyadenylating Effects 0.000 claims description 4
- 239000001103 potassium chloride Substances 0.000 claims description 4
- 235000011164 potassium chloride Nutrition 0.000 claims description 4
- 235000017557 sodium bicarbonate Nutrition 0.000 claims description 4
- 229910000030 sodium bicarbonate Inorganic materials 0.000 claims description 4
- WQZGKKKJIJFFOK-VFUOTHLCSA-N β-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 claims description 4
- 150000001413 amino acids Chemical group 0.000 claims description 3
- UXVMQQNJUSDDNG-UHFFFAOYSA-L cacl2 Chemical compound [Cl-].[Cl-].[Ca+2] UXVMQQNJUSDDNG-UHFFFAOYSA-L 0.000 claims description 3
- 239000001110 calcium chloride Substances 0.000 claims description 3
- 229910001628 calcium chloride Inorganic materials 0.000 claims description 3
- DHMQDGOQFOQNFH-UHFFFAOYSA-N glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims description 3
- HHGZUQPEIHGQST-RGVONZFCSA-N (2R)-2-amino-3-[[(2R)-2-amino-2-carboxyethyl]disulfanyl]propanoic acid;dihydrochloride Chemical compound Cl.Cl.OC(=O)[C@@H](N)CSSC[C@H](N)C(O)=O HHGZUQPEIHGQST-RGVONZFCSA-N 0.000 claims description 2
- KWTQSFXGGICVPE-WCCKRBBISA-N (2S)-2-amino-5-(diaminomethylideneamino)pentanoic acid;hydron;chloride Chemical compound Cl.OC(=O)[C@@H](N)CCCN=C(N)N KWTQSFXGGICVPE-WCCKRBBISA-N 0.000 claims description 2
- 239000001763 2-hydroxyethyl(trimethyl)azanium Substances 0.000 claims description 2
- 229960003178 Choline Chloride Drugs 0.000 claims description 2
- SGMZJAMFUVOLNK-UHFFFAOYSA-M Choline chloride Chemical compound [Cl-].C[N+](C)(C)CCO SGMZJAMFUVOLNK-UHFFFAOYSA-M 0.000 claims description 2
- 235000019743 Choline chloride Nutrition 0.000 claims description 2
- WQZGKKKJIJFFOK-GASJEMHNSA-N D-Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 claims description 2
- 229960000304 Folic Acid Drugs 0.000 claims description 2
- 239000004471 Glycine Substances 0.000 claims description 2
- 229960002885 Histidine Drugs 0.000 claims description 2
- 229960000310 ISOLEUCINE Drugs 0.000 claims description 2
- CDAISMWEOUEBRE-GPIVLXJGSA-N Inositol Chemical compound O[C@H]1[C@H](O)[C@@H](O)[C@H](O)[C@H](O)[C@@H]1O CDAISMWEOUEBRE-GPIVLXJGSA-N 0.000 claims description 2
- AGPKZVBTJJNPAG-WHFBIAKZSA-N L-isoleucine Chemical compound CC[C@H](C)[C@H](N)C(O)=O AGPKZVBTJJNPAG-WHFBIAKZSA-N 0.000 claims description 2
- 239000004395 L-leucine Substances 0.000 claims description 2
- 235000019454 L-leucine Nutrition 0.000 claims description 2
- BVHLGVCQOALMSV-JEDNCBNOSA-N L-lysine hydrochloride Chemical compound Cl.NCCCC[C@H](N)C(O)=O BVHLGVCQOALMSV-JEDNCBNOSA-N 0.000 claims description 2
- 229960004452 Methionine Drugs 0.000 claims description 2
- 229940053487 Niacinamide Drugs 0.000 claims description 2
- 229960005190 Phenylalanine Drugs 0.000 claims description 2
- 229940044519 Poloxamer 188 Drugs 0.000 claims description 2
- 229960004172 Pyridoxine Hydrochloride Drugs 0.000 claims description 2
- 229960002477 Riboflavin Drugs 0.000 claims description 2
- AUNGANRZJHBGPY-SCRDCRAPSA-N Riboflavin Chemical compound OC[C@@H](O)[C@@H](O)[C@@H](O)CN1C=2C=C(C)C(C)=CC=2N=C2C1=NC(=O)NC2=O AUNGANRZJHBGPY-SCRDCRAPSA-N 0.000 claims description 2
- AUNGANRZJHBGPY-OUCADQQQSA-N Riboflavin Natural products OC[C@@H](O)[C@H](O)[C@@H](O)CN1C=2C=C(C)C(C)=CC=2N=C2C1=NC(=O)NC2=O AUNGANRZJHBGPY-OUCADQQQSA-N 0.000 claims description 2
- 229960000344 Thiamine hydrochloride Drugs 0.000 claims description 2
- 239000004473 Threonine Substances 0.000 claims description 2
- 229960004799 Tryptophan Drugs 0.000 claims description 2
- FAPWYRCQGJNNSJ-UBKPKTQASA-L calcium D-pantothenic acid Chemical compound [Ca+2].OCC(C)(C)[C@@H](O)C(=O)NCCC([O-])=O.OCC(C)(C)[C@@H](O)C(=O)NCCC([O-])=O FAPWYRCQGJNNSJ-UBKPKTQASA-L 0.000 claims description 2
- 235000011148 calcium chloride Nutrition 0.000 claims description 2
- OKTJSMMVPCPJKN-UHFFFAOYSA-N carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims description 2
- 229910052799 carbon Inorganic materials 0.000 claims description 2
- 238000005341 cation exchange Methods 0.000 claims description 2
- 238000004113 cell culture Methods 0.000 claims description 2
- 239000008121 dextrose Substances 0.000 claims description 2
- 238000011026 diafiltration Methods 0.000 claims description 2
- 238000007865 diluting Methods 0.000 claims description 2
- ASIYFCYUCMQNGK-JZGIKJSDSA-L disodium L-tyrosinate Chemical compound [Na+].[Na+].[O-]C(=O)[C@@H](N)CC1=CC=C([O-])C=C1 ASIYFCYUCMQNGK-JZGIKJSDSA-L 0.000 claims description 2
- 235000019152 folic acid Nutrition 0.000 claims description 2
- 239000011724 folic acid Substances 0.000 claims description 2
- PCHJSUWPFVWCPO-UHFFFAOYSA-N gold Chemical compound [Au] PCHJSUWPFVWCPO-UHFFFAOYSA-N 0.000 claims description 2
- 229910052737 gold Inorganic materials 0.000 claims description 2
- 239000010931 gold Substances 0.000 claims description 2
- 239000012145 high-salt buffer Substances 0.000 claims description 2
- SZQUEWJRBJDHSM-UHFFFAOYSA-N iron(3+);trinitrate;nonahydrate Chemical compound O.O.O.O.O.O.O.O.O.[Fe+3].[O-][N+]([O-])=O.[O-][N+]([O-])=O.[O-][N+]([O-])=O SZQUEWJRBJDHSM-UHFFFAOYSA-N 0.000 claims description 2
- 229960003136 leucine Drugs 0.000 claims description 2
- 150000002632 lipids Chemical class 0.000 claims description 2
- 239000002502 liposome Substances 0.000 claims description 2
- 239000000693 micelle Substances 0.000 claims description 2
- DFPAKSUCGFBDDF-UHFFFAOYSA-N nicotinamide Chemical compound NC(=O)C1=CC=CN=C1 DFPAKSUCGFBDDF-UHFFFAOYSA-N 0.000 claims description 2
- 229960003966 nicotinamide Drugs 0.000 claims description 2
- 235000005152 nicotinamide Nutrition 0.000 claims description 2
- 239000011570 nicotinamide Substances 0.000 claims description 2
- ZUFQODAHGAHPFQ-UHFFFAOYSA-N pyridoxine hydrochloride Chemical compound Cl.CC1=NC=C(CO)C(CO)=C1O ZUFQODAHGAHPFQ-UHFFFAOYSA-N 0.000 claims description 2
- 235000019171 pyridoxine hydrochloride Nutrition 0.000 claims description 2
- 239000011764 pyridoxine hydrochloride Substances 0.000 claims description 2
- 235000019192 riboflavin Nutrition 0.000 claims description 2
- 239000002151 riboflavin Substances 0.000 claims description 2
- 229960001153 serine Drugs 0.000 claims description 2
- 229910052710 silicon Inorganic materials 0.000 claims description 2
- 239000010703 silicon Substances 0.000 claims description 2
- XUIMIQQOPSSXEZ-UHFFFAOYSA-N silicon Chemical compound [Si] XUIMIQQOPSSXEZ-UHFFFAOYSA-N 0.000 claims description 2
- DPJRMOMPQZCRJU-UHFFFAOYSA-M thiamine hydrochloride Chemical compound Cl.[Cl-].CC1=C(CCO)SC=[N+]1CC1=CN=C(C)N=C1N DPJRMOMPQZCRJU-UHFFFAOYSA-M 0.000 claims description 2
- 235000019190 thiamine hydrochloride Nutrition 0.000 claims description 2
- 239000011747 thiamine hydrochloride Substances 0.000 claims description 2
- 229960002898 threonine Drugs 0.000 claims description 2
- 229960004295 valine Drugs 0.000 claims description 2
- 229910001629 magnesium chloride Inorganic materials 0.000 claims 2
- 230000003750 conditioning Effects 0.000 claims 1
- 239000007788 liquid Substances 0.000 claims 1
- 229910000403 monosodium phosphate Inorganic materials 0.000 claims 1
- 235000019799 monosodium phosphate Nutrition 0.000 claims 1
- AJPJDKMHJJGVTQ-UHFFFAOYSA-M sodium dihydrogen phosphate Chemical compound [Na+].OP(O)([O-])=O AJPJDKMHJJGVTQ-UHFFFAOYSA-M 0.000 claims 1
- 239000001488 sodium phosphate Substances 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 description 4
- 125000003729 nucleotide group Chemical group 0.000 description 4
- 102100011550 ACTB Human genes 0.000 description 2
- 101700033661 ACTB Proteins 0.000 description 2
- 101710032514 ACTI Proteins 0.000 description 2
- ZKHQWZAMYRWXGA-KQYNXXCUSA-N Adenosine triphosphate Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](COP(O)(=O)OP(O)(=O)OP(O)(O)=O)[C@@H](O)[C@H]1O ZKHQWZAMYRWXGA-KQYNXXCUSA-N 0.000 description 2
- 241000287828 Gallus gallus Species 0.000 description 2
- 241001135555 Sandfly fever Sicilian virus Species 0.000 description 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-N phosphoric acid Chemical compound OP(O)(O)=O NBIIXXVUZAFLBC-UHFFFAOYSA-N 0.000 description 2
- 229910000147 aluminium phosphate Inorganic materials 0.000 description 1
- 125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 description 1
- KEAYESYHFKHZAL-UHFFFAOYSA-N sodium Chemical compound [Na] KEAYESYHFKHZAL-UHFFFAOYSA-N 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862734505P | 2018-09-21 | 2018-09-21 | |
| US62/734,505 | 2018-09-21 | ||
| PCT/US2019/052501 WO2020061581A1 (fr) | 2018-09-21 | 2019-09-23 | Compositions et procédés permettant la fabrication de vecteurs de thérapie génique |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022501037A JP2022501037A (ja) | 2022-01-06 |
| JPWO2020061581A5 true JPWO2020061581A5 (fr) | 2022-10-04 |
Family
ID=69887990
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021515515A Withdrawn JP2022501037A (ja) | 2018-09-21 | 2019-09-23 | 遺伝子治療用ベクターを製造するための組成物及び方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20210355503A1 (fr) |
| EP (1) | EP3853357A4 (fr) |
| JP (1) | JP2022501037A (fr) |
| KR (1) | KR20210093862A (fr) |
| CN (1) | CN113227362A (fr) |
| AU (1) | AU2019344073A1 (fr) |
| BR (1) | BR112021005110A2 (fr) |
| CA (1) | CA3112824A1 (fr) |
| EA (1) | EA202190512A1 (fr) |
| IL (1) | IL281586A (fr) |
| MX (1) | MX2021003188A (fr) |
| WO (1) | WO2020061581A1 (fr) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2020211990A1 (en) * | 2019-01-22 | 2021-08-12 | 2Seventy Bio, Inc. | Methods and systems for manufacturing viral vectors |
| BR112022018658A2 (pt) * | 2020-03-16 | 2022-12-20 | Ultragenyx Pharmaceutical Inc | Métodos para potencializar o rendimento de vírus adenoassociado recombinante |
| AU2021375404A1 (en) * | 2020-11-03 | 2023-06-08 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| GB202104611D0 (en) * | 2021-03-31 | 2021-05-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| KR20240054958A (ko) * | 2021-06-17 | 2024-04-26 | 메이라지티엑스 유케이 Ii 리미티드 | Aav 제조 방법 |
| AU2022299552A1 (en) * | 2021-06-25 | 2024-01-04 | Oxford Biomedica (Us) Llc | Adeno-associated virus packaging systems |
| GB202117844D0 (en) * | 2021-12-09 | 2022-01-26 | Oxford Biomedica Ltd | Purification method |
| US20230323395A1 (en) * | 2021-12-15 | 2023-10-12 | Homology Medicines, Inc. | Methods and compositions for the production of adeno-associated virus |
| EP4363597A1 (fr) * | 2022-01-20 | 2024-05-08 | Sartorius Xell GmbH | Procédé de détection et de quantification de virus adéno-associés (aav) à l'aide d'une matrice d'affinité |
| WO2024011203A2 (fr) * | 2022-07-07 | 2024-01-11 | Intergalactic Therapeutics, Inc. | Vecteurs oculaires et leurs utilisations |
| WO2024056561A1 (fr) | 2022-09-12 | 2024-03-21 | F. Hoffmann-La Roche Ag | Procédé de séparation de particules de vaa pleines et vides |
| US20240156988A1 (en) * | 2022-11-11 | 2024-05-16 | Eli Lilly And Company | Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3077531C (fr) * | 2009-06-16 | 2022-09-20 | Genzyme Corporation | Procedes ameliores pour la purification de vecteurs aav recombinants |
| US11098286B2 (en) * | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| GB201704192D0 (en) * | 2017-03-16 | 2017-05-03 | Nightstarx Ltd | Treatment of Retinitis Pigmentosa |
-
2019
- 2019-09-23 CA CA3112824A patent/CA3112824A1/fr not_active Withdrawn
- 2019-09-23 EA EA202190512A patent/EA202190512A1/ru unknown
- 2019-09-23 MX MX2021003188A patent/MX2021003188A/es unknown
- 2019-09-23 CN CN201980077121.7A patent/CN113227362A/zh not_active Withdrawn
- 2019-09-23 BR BR112021005110-4A patent/BR112021005110A2/pt not_active Application Discontinuation
- 2019-09-23 US US17/277,877 patent/US20210355503A1/en not_active Abandoned
- 2019-09-23 AU AU2019344073A patent/AU2019344073A1/en not_active Withdrawn
- 2019-09-23 KR KR1020217011785A patent/KR20210093862A/ko unknown
- 2019-09-23 WO PCT/US2019/052501 patent/WO2020061581A1/fr unknown
- 2019-09-23 JP JP2021515515A patent/JP2022501037A/ja not_active Withdrawn
- 2019-09-23 EP EP19862998.2A patent/EP3853357A4/fr not_active Withdrawn
-
2021
- 2021-03-17 IL IL281586A patent/IL281586A/en unknown
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