JPWO2022076711A5 - - Google Patents
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- JPWO2022076711A5 JPWO2022076711A5 JP2023521279A JP2023521279A JPWO2022076711A5 JP WO2022076711 A5 JPWO2022076711 A5 JP WO2022076711A5 JP 2023521279 A JP2023521279 A JP 2023521279A JP 2023521279 A JP2023521279 A JP 2023521279A JP WO2022076711 A5 JPWO2022076711 A5 JP WO2022076711A5
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- 210000004027 cell Anatomy 0.000 claims description 45
- 239000008194 pharmaceutical composition Substances 0.000 claims description 29
- 210000000234 capsid Anatomy 0.000 claims description 26
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 16
- 241000702421 Dependoparvovirus Species 0.000 claims description 14
- 239000013608 rAAV vector Substances 0.000 claims description 13
- 241000958487 Adeno-associated virus 3B Species 0.000 claims description 10
- 108700019146 Transgenes Proteins 0.000 claims description 10
- 210000001519 tissue Anatomy 0.000 claims description 9
- 208000022873 Ocular disease Diseases 0.000 claims description 8
- 208000030533 eye disease Diseases 0.000 claims description 8
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 6
- 239000003814 drug Substances 0.000 claims description 6
- 230000002207 retinal effect Effects 0.000 claims description 6
- 229940124597 therapeutic agent Drugs 0.000 claims description 6
- 210000001585 trabecular meshwork Anatomy 0.000 claims description 6
- 206010046851 Uveitis Diseases 0.000 claims description 5
- 208000015181 infectious disease Diseases 0.000 claims description 5
- 230000002458 infectious effect Effects 0.000 claims description 5
- 108090000565 Capsid Proteins Proteins 0.000 claims description 4
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 4
- 208000010412 Glaucoma Diseases 0.000 claims description 4
- 102000039446 nucleic acids Human genes 0.000 claims description 4
- 108020004707 nucleic acids Proteins 0.000 claims description 4
- 150000007523 nucleic acids Chemical class 0.000 claims description 4
- 239000002773 nucleotide Substances 0.000 claims description 4
- 125000003729 nucleotide group Chemical group 0.000 claims description 4
- 230000001105 regulatory effect Effects 0.000 claims description 4
- KIUKXJAPPMFGSW-DNGZLQJQSA-N (2S,3S,4S,5R,6R)-6-[(2S,3R,4R,5S,6R)-3-Acetamido-2-[(2S,3S,4R,5R,6R)-6-[(2R,3R,4R,5S,6R)-3-acetamido-2,5-dihydroxy-6-(hydroxymethyl)oxan-4-yl]oxy-2-carboxy-4,5-dihydroxyoxan-3-yl]oxy-5-hydroxy-6-(hydroxymethyl)oxan-4-yl]oxy-3,4,5-trihydroxyoxane-2-carboxylic acid Chemical compound CC(=O)N[C@H]1[C@H](O)O[C@H](CO)[C@@H](O)[C@@H]1O[C@H]1[C@H](O)[C@@H](O)[C@H](O[C@H]2[C@@H]([C@@H](O[C@H]3[C@@H]([C@@H](O)[C@H](O)[C@H](O3)C(O)=O)O)[C@H](O)[C@@H](CO)O2)NC(C)=O)[C@@H](C(O)=O)O1 KIUKXJAPPMFGSW-DNGZLQJQSA-N 0.000 claims description 3
- 210000004240 ciliary body Anatomy 0.000 claims description 3
- 229920002674 hyaluronan Polymers 0.000 claims description 3
- 229960003160 hyaluronic acid Drugs 0.000 claims description 3
- 210000001328 optic nerve Anatomy 0.000 claims description 3
- 239000013607 AAV vector Substances 0.000 claims description 2
- 239000003795 chemical substances by application Substances 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims description 2
- 238000004806 packaging method and process Methods 0.000 claims description 2
- 230000026683 transduction Effects 0.000 claims description 2
- 238000010361 transduction Methods 0.000 claims description 2
- 208000002780 macular degeneration Diseases 0.000 claims 8
- 208000001344 Macular Edema Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 206010012689 Diabetic retinopathy Diseases 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000011325 dry age related macular degeneration Diseases 0.000 claims 3
- 208000036693 Color-vision disease Diseases 0.000 claims 2
- 206010058202 Cystoid macular oedema Diseases 0.000 claims 2
- 206010025415 Macular oedema Diseases 0.000 claims 2
- 206010030113 Oedema Diseases 0.000 claims 2
- 208000002158 Proliferative Vitreoretinopathy Diseases 0.000 claims 2
- 206010038934 Retinopathy proliferative Diseases 0.000 claims 2
- 230000001154 acute effect Effects 0.000 claims 2
- 206010064930 age-related macular degeneration Diseases 0.000 claims 2
- 201000010206 cystoid macular edema Diseases 0.000 claims 2
- 230000007850 degeneration Effects 0.000 claims 2
- 238000002647 laser therapy Methods 0.000 claims 2
- 201000010230 macular retinal edema Diseases 0.000 claims 2
- 208000021971 neovascular inflammatory vitreoretinopathy Diseases 0.000 claims 2
- 208000008795 neuromyelitis optica Diseases 0.000 claims 2
- 238000002428 photodynamic therapy Methods 0.000 claims 2
- 230000006785 proliferative vitreoretinopathy Effects 0.000 claims 2
- 208000004644 retinal vein occlusion Diseases 0.000 claims 2
- 208000036443 AIPL1-related retinopathy Diseases 0.000 claims 1
- 206010003694 Atrophy Diseases 0.000 claims 1
- 201000001321 Bardet-Biedl syndrome Diseases 0.000 claims 1
- 208000009137 Behcet syndrome Diseases 0.000 claims 1
- 208000005590 Choroidal Neovascularization Diseases 0.000 claims 1
- 206010060823 Choroidal neovascularisation Diseases 0.000 claims 1
- 208000006992 Color Vision Defects Diseases 0.000 claims 1
- 206010012667 Diabetic glaucoma Diseases 0.000 claims 1
- 206010012688 Diabetic retinal oedema Diseases 0.000 claims 1
- 206010012692 Diabetic uveitis Diseases 0.000 claims 1
- 208000001351 Epiretinal Membrane Diseases 0.000 claims 1
- 208000032087 Hereditary Leber Optic Atrophy Diseases 0.000 claims 1
- 201000002563 Histoplasmosis Diseases 0.000 claims 1
- 208000010038 Ischemic Optic Neuropathy Diseases 0.000 claims 1
- 206010056715 Laurence-Moon-Bardet-Biedl syndrome Diseases 0.000 claims 1
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 1
- 201000000639 Leber hereditary optic neuropathy Diseases 0.000 claims 1
- 208000031471 Macular fibrosis Diseases 0.000 claims 1
- 208000010164 Multifocal Choroiditis Diseases 0.000 claims 1
- 206010030924 Optic ischaemic neuropathy Diseases 0.000 claims 1
- 206010038848 Retinal detachment Diseases 0.000 claims 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 1
- 206010038933 Retinopathy of prematurity Diseases 0.000 claims 1
- 208000027073 Stargardt disease Diseases 0.000 claims 1
- 206010042742 Sympathetic ophthalmia Diseases 0.000 claims 1
- 206010043189 Telangiectasia Diseases 0.000 claims 1
- 208000031861 Tritanopia Diseases 0.000 claims 1
- 208000014769 Usher Syndromes Diseases 0.000 claims 1
- 208000029977 White Dot Syndromes Diseases 0.000 claims 1
- 208000023564 acute macular neuroretinopathy Diseases 0.000 claims 1
- 201000007058 anterior ischemic optic neuropathy Diseases 0.000 claims 1
- 230000037444 atrophy Effects 0.000 claims 1
- 201000010018 blue color blindness Diseases 0.000 claims 1
- 201000005667 central retinal vein occlusion Diseases 0.000 claims 1
- 201000006754 cone-rod dystrophy Diseases 0.000 claims 1
- 208000006623 congenital stationary night blindness Diseases 0.000 claims 1
- 206010012601 diabetes mellitus Diseases 0.000 claims 1
- 201000011190 diabetic macular edema Diseases 0.000 claims 1
- 238000009792 diffusion process Methods 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 208000024519 eye neoplasm Diseases 0.000 claims 1
- 208000014674 injury Diseases 0.000 claims 1
- 201000008106 ocular cancer Diseases 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 201000000763 red color blindness Diseases 0.000 claims 1
- 210000001927 retinal artery Anatomy 0.000 claims 1
- 230000004264 retinal detachment Effects 0.000 claims 1
- 230000004283 retinal dysfunction Effects 0.000 claims 1
- 201000007714 retinoschisis Diseases 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 208000009056 telangiectasis Diseases 0.000 claims 1
- 230000008733 trauma Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 24
- 125000003275 alpha amino acid group Chemical group 0.000 description 10
- 238000006467 substitution reaction Methods 0.000 description 9
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 4
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 3
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 3
- 230000001413 cellular effect Effects 0.000 description 1
- 239000011159 matrix material Substances 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063088982P | 2020-10-07 | 2020-10-07 | |
| US63/088,982 | 2020-10-07 | ||
| US202163187197P | 2021-05-11 | 2021-05-11 | |
| US63/187,197 | 2021-05-11 | ||
| PCT/US2021/054008 WO2022076711A2 (en) | 2020-10-07 | 2021-10-07 | Adeno-associated viruses for ocular delivery of gene therapy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023545722A JP2023545722A (ja) | 2023-10-31 |
| JPWO2022076711A5 true JPWO2022076711A5 (https=) | 2024-10-17 |
| JP2023545722A5 JP2023545722A5 (https=) | 2024-10-17 |
Family
ID=78483550
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023521279A Pending JP2023545722A (ja) | 2020-10-07 | 2021-10-07 | 遺伝子治療剤の眼送達のためのアデノ随伴ウイルス |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230381341A1 (https=) |
| EP (1) | EP4225777A2 (https=) |
| JP (1) | JP2023545722A (https=) |
| KR (1) | KR20230082614A (https=) |
| AU (1) | AU2021356667A1 (https=) |
| CA (1) | CA3193697A1 (https=) |
| IL (1) | IL301647A (https=) |
| MX (1) | MX2023003699A (https=) |
| WO (1) | WO2022076711A2 (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023060272A2 (en) * | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns tropic delivery |
| TW202345913A (zh) | 2022-04-06 | 2023-12-01 | 美商銳進科斯生物股份有限公司 | 用於脈絡膜上投與之調配物諸如凝膠調配物 |
| TW202404651A (zh) | 2022-04-06 | 2024-02-01 | 美商銳進科斯生物股份有限公司 | 用於脈絡膜上投與之調配物諸如形成聚集體之調配物 |
| EP4518972A2 (en) * | 2022-05-03 | 2025-03-12 | RegenxBio Inc. | Vectorized anti-complement antibodies and complement agents and administration thereof |
| US20250288697A1 (en) * | 2022-05-03 | 2025-09-18 | Regenxbio Inc. | Vectorized anti-tnf-alpha inhibitors for ocular indications |
| AU2023282121A1 (en) * | 2022-05-31 | 2025-01-09 | Mount Spec Investments Pty Ltd | Modified adeno-associated virus capsid proteins and methods thereof |
| WO2023242633A2 (en) * | 2022-06-14 | 2023-12-21 | Vectory B.V. | Recombinant aav capsid proteins |
| JP2025527658A (ja) * | 2022-08-24 | 2025-08-22 | リジェネックスバイオ インコーポレイテッド | 組換えアデノ随伴ウイルス及びその使用 |
| WO2024148179A2 (en) * | 2023-01-05 | 2024-07-11 | Emugen Therapeutics Llc | Retro-aav and use in treating neurodegenerative diseases |
| AU2024271004A1 (en) * | 2023-05-16 | 2026-01-15 | Regenxbio Inc. | Vectorized anti-complement antibodies and administration thereof |
| WO2024238853A1 (en) * | 2023-05-16 | 2024-11-21 | Regenxbio Inc. | Adeno-associated viruses for ocular delivery of gene therapy |
| WO2025217230A1 (en) * | 2024-04-08 | 2025-10-16 | Regenxbio Inc. | Vectorized anti-complement antibodies and complement agents and administration thereof |
| CN119039401B (zh) * | 2024-08-19 | 2025-04-11 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2026047120A1 (en) * | 2024-08-28 | 2026-03-05 | Medizinische Hochschule Hannover | Aav viral vectors targeting the inner ear and medical treatment of the inner ear using said vectors |
Family Cites Families (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE37983T1 (de) | 1982-04-22 | 1988-11-15 | Ici Plc | Mittel mit verzoegerter freigabe. |
| US5128326A (en) | 1984-12-06 | 1992-07-07 | Biomatrix, Inc. | Drug delivery systems based on hyaluronans derivatives thereof and their salts and methods of producing same |
| WO1991005548A1 (en) | 1989-10-10 | 1991-05-02 | Pitman-Moore, Inc. | Sustained release composition for macromolecular proteins |
| WO1991006287A1 (en) | 1989-11-06 | 1991-05-16 | Enzytech, Inc. | Protein microspheres and methods of using them |
| US5912015A (en) | 1992-03-12 | 1999-06-15 | Alkermes Controlled Therapeutics, Inc. | Modulated release from biocompatible polymers |
| EP0805678B1 (en) | 1995-01-05 | 2003-10-29 | THE BOARD OF REGENTS acting for and on behalf of THE UNIVERSITY OF MICHIGAN | Surface-modified nanoparticles and method of making and using same |
| EP0850051A2 (en) | 1995-08-31 | 1998-07-01 | Alkermes Controlled Therapeutics, Inc. | Composition for sustained release of an agent |
| US6156303A (en) * | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US5989463A (en) | 1997-09-24 | 1999-11-23 | Alkermes Controlled Therapeutics, Inc. | Methods for fabricating polymer-based controlled release devices |
| SE512663C2 (sv) | 1997-10-23 | 2000-04-17 | Biogram Ab | Inkapslingsförfarande för aktiv substans i en bionedbrytbar polymer |
| PT1916258E (pt) | 1999-08-09 | 2014-07-29 | Genzyme Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| US7183969B2 (en) | 2004-12-22 | 2007-02-27 | Raytheon Company | System and technique for calibrating radar arrays |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| EP3257937B1 (en) | 2008-02-19 | 2022-08-03 | uniQure IP B.V. | Optimisation of expression of parvoviral rep and cap proteins in insect cells |
| ES2724122T3 (es) | 2009-04-30 | 2019-09-06 | Univ Pennsylvania | Composiciones para dirigir células de las vías respiratorias de conducción que comprenden construcciones de virus adenoasociado |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| WO2013170078A1 (en) | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| EP2970946A4 (en) | 2013-03-13 | 2016-09-07 | Philadelphia Children Hospital | ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF |
| KR102413498B1 (ko) | 2013-04-20 | 2022-06-24 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달 |
| KR20240090694A (ko) | 2013-07-22 | 2024-06-21 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| HUE052676T2 (hu) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015138628A1 (en) * | 2014-03-11 | 2015-09-17 | University Of Florida Research Foundation, Inc. | Use of aav-expressed m013 protein as an anti-inflammatory therapeutic |
| WO2015164757A1 (en) | 2014-04-25 | 2015-10-29 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| PT4012035T (pt) * | 2014-09-16 | 2025-02-11 | Genzyme Corp | Vetores virais adenoassociados para tratar o glaucoma de miocilina (mioc) |
| BR112017005892A2 (pt) | 2014-09-24 | 2017-12-12 | Hope City | variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma |
| JP6665466B2 (ja) | 2015-09-26 | 2020-03-13 | 日亜化学工業株式会社 | 半導体発光素子及びその製造方法 |
| WO2017070491A1 (en) | 2015-10-23 | 2017-04-27 | Applied Genetic Technologies Corporation | Ophthalmic formulations |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| TW202102526A (zh) * | 2019-04-04 | 2021-01-16 | 美商銳進科斯生物股份有限公司 | 重組腺相關病毒及其用途 |
| AU2020308909A1 (en) * | 2019-06-27 | 2022-01-27 | University Of Florida Research Foundation, Incorporated | Enhancing AAV-mediated transduction of ocular tissues with hyaluronic acid |
-
2021
- 2021-10-07 US US18/030,706 patent/US20230381341A1/en active Pending
- 2021-10-07 KR KR1020237010842A patent/KR20230082614A/ko active Pending
- 2021-10-07 EP EP21801765.5A patent/EP4225777A2/en active Pending
- 2021-10-07 AU AU2021356667A patent/AU2021356667A1/en not_active Abandoned
- 2021-10-07 WO PCT/US2021/054008 patent/WO2022076711A2/en not_active Ceased
- 2021-10-07 IL IL301647A patent/IL301647A/en unknown
- 2021-10-07 JP JP2023521279A patent/JP2023545722A/ja active Pending
- 2021-10-07 MX MX2023003699A patent/MX2023003699A/es unknown
- 2021-10-07 CA CA3193697A patent/CA3193697A1/en active Pending
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