JPWO2020236351A5 - - Google Patents
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- Publication number
- JPWO2020236351A5 JPWO2020236351A5 JP2021568543A JP2021568543A JPWO2020236351A5 JP WO2020236351 A5 JPWO2020236351 A5 JP WO2020236351A5 JP 2021568543 A JP2021568543 A JP 2021568543A JP 2021568543 A JP2021568543 A JP 2021568543A JP WO2020236351 A5 JPWO2020236351 A5 JP WO2020236351A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- cells
- glycoside hydrolase
- transgene
- gene therapy
- 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
- 102000005744 Glycoside Hydrolases Human genes 0.000 claims description 34
- 108010031186 Glycoside Hydrolases Proteins 0.000 claims description 34
- 108700019146 Transgenes Proteins 0.000 claims description 31
- 238000001415 gene therapy Methods 0.000 claims description 29
- 210000004027 cell Anatomy 0.000 claims description 24
- 239000003814 drug Substances 0.000 claims description 16
- 235000011073 invertase Nutrition 0.000 claims description 16
- 230000002207 retinal effect Effects 0.000 claims description 16
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 15
- 208000035475 disorder Diseases 0.000 claims description 15
- 238000000034 method Methods 0.000 claims description 10
- 239000004382 Amylase Substances 0.000 claims description 8
- 108010065511 Amylases Proteins 0.000 claims description 8
- 102000013142 Amylases Human genes 0.000 claims description 8
- 102100026189 Beta-galactosidase Human genes 0.000 claims description 8
- 108010059892 Cellulase Proteins 0.000 claims description 8
- 108010022172 Chitinases Proteins 0.000 claims description 8
- 102000012286 Chitinases Human genes 0.000 claims description 8
- 108010059881 Lactase Proteins 0.000 claims description 8
- 108010014251 Muramidase Proteins 0.000 claims description 8
- 102000016943 Muramidase Human genes 0.000 claims description 8
- 108010062010 N-Acetylmuramoyl-L-alanine Amidase Proteins 0.000 claims description 8
- 108010006232 Neuraminidase Proteins 0.000 claims description 8
- 102000005348 Neuraminidase Human genes 0.000 claims description 8
- 101710184309 Probable sucrose-6-phosphate hydrolase Proteins 0.000 claims description 8
- 102400000472 Sucrase Human genes 0.000 claims description 8
- 101710112652 Sucrose-6-phosphate hydrolase Proteins 0.000 claims description 8
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims description 8
- 108010028144 alpha-Glucosidases Proteins 0.000 claims description 8
- 102000016679 alpha-Glucosidases Human genes 0.000 claims description 8
- 235000019418 amylase Nutrition 0.000 claims description 8
- 108010051210 beta-Fructofuranosidase Proteins 0.000 claims description 8
- 108010005774 beta-Galactosidase Proteins 0.000 claims description 8
- 229940106157 cellulase Drugs 0.000 claims description 8
- 239000001573 invertase Substances 0.000 claims description 8
- 229940116108 lactase Drugs 0.000 claims description 8
- 229960000274 lysozyme Drugs 0.000 claims description 8
- 235000010335 lysozyme Nutrition 0.000 claims description 8
- 239000004325 lysozyme Substances 0.000 claims description 8
- 230000000007 visual effect Effects 0.000 claims description 8
- 238000007913 intrathecal administration Methods 0.000 claims description 6
- 238000001990 intravenous administration Methods 0.000 claims description 6
- 239000003795 chemical substances by application Substances 0.000 claims description 5
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims description 4
- 206010003694 Atrophy Diseases 0.000 claims description 4
- 206010007747 Cataract congenital Diseases 0.000 claims description 4
- 208000033810 Choroidal dystrophy Diseases 0.000 claims description 4
- 208000006992 Color Vision Defects Diseases 0.000 claims description 4
- 208000025133 Congenital eye disease Diseases 0.000 claims description 4
- 206010018325 Congenital glaucomas Diseases 0.000 claims description 4
- 206010012565 Developmental glaucoma Diseases 0.000 claims description 4
- 206010012688 Diabetic retinal oedema Diseases 0.000 claims description 4
- 208000003098 Ganglion Cysts Diseases 0.000 claims description 4
- 208000010412 Glaucoma Diseases 0.000 claims description 4
- 208000032087 Hereditary Leber Optic Atrophy Diseases 0.000 claims description 4
- 101000829958 Homo sapiens N-acetyllactosaminide beta-1,6-N-acetylglucosaminyl-transferase Proteins 0.000 claims description 4
- 208000032578 Inherited retinal disease Diseases 0.000 claims description 4
- 201000006165 Kuhnt-Junius degeneration Diseases 0.000 claims description 4
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims description 4
- 201000000639 Leber hereditary optic neuropathy Diseases 0.000 claims description 4
- 102100023315 N-acetyllactosaminide beta-1,6-N-acetylglucosaminyl-transferase Human genes 0.000 claims description 4
- 201000007737 Retinal degeneration Diseases 0.000 claims description 4
- 208000032430 Retinal dystrophy Diseases 0.000 claims description 4
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 4
- 208000027073 Stargardt disease Diseases 0.000 claims description 4
- 208000004350 Strabismus Diseases 0.000 claims description 4
- 208000005400 Synovial Cyst Diseases 0.000 claims description 4
- 208000014769 Usher Syndromes Diseases 0.000 claims description 4
- 201000001408 X-linked juvenile retinoschisis 1 Diseases 0.000 claims description 4
- 208000017441 X-linked retinoschisis Diseases 0.000 claims description 4
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 4
- 210000000411 amacrine cell Anatomy 0.000 claims description 4
- 230000037444 atrophy Effects 0.000 claims description 4
- 208000003571 choroideremia Diseases 0.000 claims description 4
- 201000007254 color blindness Diseases 0.000 claims description 4
- 206010012601 diabetes mellitus Diseases 0.000 claims description 4
- 201000011190 diabetic macular edema Diseases 0.000 claims description 4
- 208000011325 dry age related macular degeneration Diseases 0.000 claims description 4
- 201000006321 fundus dystrophy Diseases 0.000 claims description 4
- 210000002287 horizontal cell Anatomy 0.000 claims description 4
- 208000017532 inherited retinal dystrophy Diseases 0.000 claims description 4
- 208000002780 macular degeneration Diseases 0.000 claims description 4
- 230000002025 microglial effect Effects 0.000 claims description 4
- 210000004498 neuroglial cell Anatomy 0.000 claims description 4
- 210000001328 optic nerve Anatomy 0.000 claims description 4
- 210000000964 retinal cone photoreceptor cell Anatomy 0.000 claims description 4
- 230000004258 retinal degeneration Effects 0.000 claims description 4
- 210000000880 retinal rod photoreceptor cell Anatomy 0.000 claims description 4
- 201000007714 retinoschisis Diseases 0.000 claims description 4
- 230000000699 topical effect Effects 0.000 claims description 4
- 208000029257 vision disease Diseases 0.000 claims description 3
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 229940088598 enzyme Drugs 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
- 239000002872 contrast media Substances 0.000 description 2
- AMDBBAQNWSUWGN-UHFFFAOYSA-N Ioversol Chemical compound OCCN(C(=O)CO)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I AMDBBAQNWSUWGN-UHFFFAOYSA-N 0.000 description 1
- 229960004108 iobitridol Drugs 0.000 description 1
- YLPBXIKWXNRACS-UHFFFAOYSA-N iobitridol Chemical compound OCC(O)CN(C)C(=O)C1=C(I)C(NC(=O)C(CO)CO)=C(I)C(C(=O)N(C)CC(O)CO)=C1I YLPBXIKWXNRACS-UHFFFAOYSA-N 0.000 description 1
- 229960001025 iohexol Drugs 0.000 description 1
- NTHXOOBQLCIOLC-UHFFFAOYSA-N iohexol Chemical compound OCC(O)CN(C(=O)C)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I NTHXOOBQLCIOLC-UHFFFAOYSA-N 0.000 description 1
- 229960000780 iomeprol Drugs 0.000 description 1
- NJKDOADNQSYQEV-UHFFFAOYSA-N iomeprol Chemical compound OCC(=O)N(C)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I NJKDOADNQSYQEV-UHFFFAOYSA-N 0.000 description 1
- 229960004647 iopamidol Drugs 0.000 description 1
- XQZXYNRDCRIARQ-LURJTMIESA-N iopamidol Chemical compound C[C@H](O)C(=O)NC1=C(I)C(C(=O)NC(CO)CO)=C(I)C(C(=O)NC(CO)CO)=C1I XQZXYNRDCRIARQ-LURJTMIESA-N 0.000 description 1
- 229960000824 iopentol Drugs 0.000 description 1
- IUNJANQVIJDFTQ-UHFFFAOYSA-N iopentol Chemical compound COCC(O)CN(C(C)=O)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I IUNJANQVIJDFTQ-UHFFFAOYSA-N 0.000 description 1
- 229960002603 iopromide Drugs 0.000 description 1
- DGAIEPBNLOQYER-UHFFFAOYSA-N iopromide Chemical compound COCC(=O)NC1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)N(C)CC(O)CO)=C1I DGAIEPBNLOQYER-UHFFFAOYSA-N 0.000 description 1
- 229960004537 ioversol Drugs 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962849794P | 2019-05-17 | 2019-05-17 | |
| US62/849,794 | 2019-05-17 | ||
| PCT/US2020/028207 WO2020236351A1 (en) | 2019-05-17 | 2020-04-15 | Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022533645A JP2022533645A (ja) | 2022-07-25 |
| JPWO2020236351A5 true JPWO2020236351A5 (https=) | 2023-04-24 |
| JP2022533645A5 JP2022533645A5 (https=) | 2023-04-24 |
Family
ID=70482910
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021568543A Pending JP2022533645A (ja) | 2019-05-17 | 2020-04-15 | グリコシドヒドロラーゼ酵素を使用する網膜細胞への遺伝子治療ベクターの送達の改善 |
| JP2021568813A Active JP7707081B2 (ja) | 2019-05-17 | 2020-04-15 | 網膜細胞を標的とした最適化された遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021568813A Active JP7707081B2 (ja) | 2019-05-17 | 2020-04-15 | 網膜細胞を標的とした最適化された遺伝子治療 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20220233655A1 (https=) |
| EP (2) | EP3969060A1 (https=) |
| JP (2) | JP2022533645A (https=) |
| KR (1) | KR20220009427A (https=) |
| CN (1) | CN114126667A (https=) |
| AR (1) | AR118696A1 (https=) |
| AU (2) | AU2020278960A1 (https=) |
| CA (2) | CA3141020A1 (https=) |
| TW (1) | TW202110486A (https=) |
| WO (2) | WO2020236351A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL294102A (en) * | 2019-12-25 | 2022-08-01 | Lumus Ltd | Optical systems and methods for tracking eyes based on directing light from the eye through an optical arrangement associated with a light-directing optical element |
| CN114516901B (zh) * | 2022-03-11 | 2023-01-17 | 上海勉亦生物科技有限公司 | 一种神经系统高亲和性的aav载体及其应用 |
| CN115323001B (zh) * | 2022-07-28 | 2024-11-26 | 深圳先进技术研究院 | 一种靶向视网膜的基因递送系统及其应用 |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| JP2001514845A (ja) | 1997-09-05 | 2001-09-18 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法 |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| WO2012109214A1 (en) * | 2011-02-07 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Targeted transduction of aav vectors |
| DK2675902T3 (da) * | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| AU2013296425B2 (en) * | 2012-08-01 | 2018-06-07 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| US9730888B2 (en) * | 2013-03-14 | 2017-08-15 | Wayne State University | Method of enhancing delivery of therapeutic compounds to the eye |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| WO2015126972A1 (en) * | 2014-02-21 | 2015-08-27 | University Of Florida Research Foundation, Inc. | Methods and compositions for gene delivery to on bipolar cells |
| GB201403260D0 (en) * | 2014-02-25 | 2014-04-09 | Univ Manchester | Treatment of retinal degeneration using gene therapy |
| JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及びプログラム |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| CN107249646B (zh) * | 2014-12-16 | 2021-06-29 | 内布拉斯加大学董事会 | 用于青少年巴滕病的基因疗法 |
| JP6684343B2 (ja) * | 2015-05-21 | 2020-04-22 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | ニューロン生存因子の相乗組み合わせ及びその使用 |
| CA2994160C (en) * | 2015-07-30 | 2021-08-10 | Massachusetts Eye And Ear Infirmary | Ancestral virus sequences and uses thereof |
| US20180236105A1 (en) * | 2015-10-23 | 2018-08-23 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| RU2764919C2 (ru) * | 2016-06-13 | 2022-01-24 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Оптимизированные гены и экспрессионные кассеты cln1, и их применение |
| JP7048585B2 (ja) * | 2016-09-22 | 2022-04-05 | ソルボンヌ・ユニヴェルシテ | 網膜変性疾患の処置に使用するための光遺伝学的に形質転換した光受容体前駆細胞 |
| AU2018350990A1 (en) * | 2017-10-18 | 2020-05-21 | Regenxbio Inc. | Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap |
-
2020
- 2020-04-15 JP JP2021568543A patent/JP2022533645A/ja active Pending
- 2020-04-15 KR KR1020217040926A patent/KR20220009427A/ko not_active Withdrawn
- 2020-04-15 JP JP2021568813A patent/JP7707081B2/ja active Active
- 2020-04-15 WO PCT/US2020/028207 patent/WO2020236351A1/en not_active Ceased
- 2020-04-15 CA CA3141020A patent/CA3141020A1/en active Pending
- 2020-04-15 WO PCT/US2020/028352 patent/WO2020236352A1/en not_active Ceased
- 2020-04-15 CA CA3141017A patent/CA3141017A1/en active Pending
- 2020-04-15 AU AU2020278960A patent/AU2020278960A1/en active Pending
- 2020-04-15 AU AU2020278499A patent/AU2020278499A1/en active Pending
- 2020-04-15 AR ARP200101063A patent/AR118696A1/es unknown
- 2020-04-15 EP EP20727414.3A patent/EP3969060A1/en active Pending
- 2020-04-15 US US17/611,972 patent/US20220233655A1/en not_active Abandoned
- 2020-04-15 EP EP20723738.9A patent/EP3969059A1/en active Pending
- 2020-04-15 CN CN202080051735.0A patent/CN114126667A/zh active Pending
- 2020-04-15 US US17/611,982 patent/US20220226507A1/en not_active Abandoned
- 2020-04-15 TW TW109112715A patent/TW202110486A/zh unknown
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