JPWO2021214282A5 - - Google Patents
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- Publication number
- JPWO2021214282A5 JPWO2021214282A5 JP2022563882A JP2022563882A JPWO2021214282A5 JP WO2021214282 A5 JPWO2021214282 A5 JP WO2021214282A5 JP 2022563882 A JP2022563882 A JP 2022563882A JP 2022563882 A JP2022563882 A JP 2022563882A JP WO2021214282 A5 JPWO2021214282 A5 JP WO2021214282A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- transgene
- peptide
- derived
- transgene product
- 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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- 238000000034 method Methods 0.000 claims 23
- 108700019146 Transgenes Proteins 0.000 claims 16
- 239000013598 vector Substances 0.000 claims 15
- 108090000765 processed proteins & peptides Proteins 0.000 claims 13
- 208000002780 macular degeneration Diseases 0.000 claims 5
- 208000017442 Retinal disease Diseases 0.000 claims 4
- 206010064930 age-related macular degeneration Diseases 0.000 claims 4
- 206010012689 Diabetic retinopathy Diseases 0.000 claims 3
- 239000013603 viral vector Substances 0.000 claims 3
- 239000013607 AAV vector Substances 0.000 claims 2
- 108090000565 Capsid Proteins Proteins 0.000 claims 2
- 102100023321 Ceruloplasmin Human genes 0.000 claims 2
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 2
- 201000001408 X-linked juvenile retinoschisis 1 Diseases 0.000 claims 2
- 208000017441 X-linked retinoschisis Diseases 0.000 claims 2
- 238000001415 gene therapy Methods 0.000 claims 2
- 230000028993 immune response Effects 0.000 claims 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims 2
- 201000007914 proliferative diabetic retinopathy Diseases 0.000 claims 2
- 201000007714 retinoschisis Diseases 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 208000037663 Best vitelliform macular dystrophy Diseases 0.000 claims 1
- 201000004569 Blindness Diseases 0.000 claims 1
- 208000002691 Choroiditis Diseases 0.000 claims 1
- 208000006992 Color Vision Defects Diseases 0.000 claims 1
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 206010058202 Cystoid macular oedema Diseases 0.000 claims 1
- 102000004533 Endonucleases Human genes 0.000 claims 1
- 108010042407 Endonucleases Proteins 0.000 claims 1
- 208000010412 Glaucoma Diseases 0.000 claims 1
- 208000032087 Hereditary Leber Optic Atrophy Diseases 0.000 claims 1
- 206010061218 Inflammation Diseases 0.000 claims 1
- 208000032578 Inherited retinal disease Diseases 0.000 claims 1
- 201000000639 Leber hereditary optic neuropathy Diseases 0.000 claims 1
- 208000001344 Macular Edema Diseases 0.000 claims 1
- 208000035719 Maculopathy Diseases 0.000 claims 1
- 208000003971 Posterior uveitis Diseases 0.000 claims 1
- 208000034461 Progressive cone dystrophy Diseases 0.000 claims 1
- 201000007737 Retinal degeneration Diseases 0.000 claims 1
- 206010038848 Retinal detachment Diseases 0.000 claims 1
- 206010038923 Retinopathy Diseases 0.000 claims 1
- 208000027073 Stargardt disease Diseases 0.000 claims 1
- 208000014769 Usher Syndromes Diseases 0.000 claims 1
- 239000000872 buffer Substances 0.000 claims 1
- 230000003915 cell function Effects 0.000 claims 1
- 201000007254 color blindness Diseases 0.000 claims 1
- 201000008615 cone dystrophy Diseases 0.000 claims 1
- 201000010206 cystoid macular edema Diseases 0.000 claims 1
- 230000007402 cytotoxic response Effects 0.000 claims 1
- 206010012601 diabetes mellitus Diseases 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000006870 function Effects 0.000 claims 1
- 230000006698 induction Effects 0.000 claims 1
- 230000004054 inflammatory process Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 210000001525 retina Anatomy 0.000 claims 1
- 230000004258 retinal degeneration Effects 0.000 claims 1
- 230000004264 retinal detachment Effects 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- 230000004393 visual impairment Effects 0.000 claims 1
- 201000007790 vitelliform macular dystrophy Diseases 0.000 claims 1
- 208000020938 vitelliform macular dystrophy 2 Diseases 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP20315217.8 | 2020-04-24 | ||
| EP20315217 | 2020-04-24 | ||
| EP20305407 | 2020-04-27 | ||
| EP20305407.7 | 2020-04-27 | ||
| PCT/EP2021/060647 WO2021214282A1 (en) | 2020-04-24 | 2021-04-23 | Methods for preventing induction of immune responses to the transduced cells expressing a transgene product after ocular gene therapy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023523211A JP2023523211A (ja) | 2023-06-02 |
| JPWO2021214282A5 true JPWO2021214282A5 (https=) | 2024-02-08 |
| JP2023523211A5 JP2023523211A5 (https=) | 2024-02-08 |
Family
ID=75625591
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022563882A Pending JP2023523211A (ja) | 2020-04-24 | 2021-04-23 | 眼科遺伝子治療後の、導入遺伝子産物を発現する形質転換細胞に対する免疫応答の誘導を防止するための方法 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20230190956A1 (https=) |
| EP (1) | EP4138929A1 (https=) |
| JP (1) | JP2023523211A (https=) |
| CA (1) | CA3179895A1 (https=) |
| WO (1) | WO2021214282A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2026005189A1 (ko) * | 2024-06-27 | 2026-01-02 | 이엔셀 주식회사 | 안구 세포 또는 조직을 표적하는 펩타이드 및 이를 포함하는 바이러스 캡시드 단백질 및 바이러스 벡터 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5639872A (en) | 1993-07-27 | 1997-06-17 | Hybridon, Inc. | Human VEGF-specific oligonucleotides |
| WO1997020925A1 (en) | 1995-12-08 | 1997-06-12 | Hybridon, Inc. | Modified vegf antisense oligonucleotides for treatment of skin disorders |
| US20030144221A1 (en) | 2001-07-17 | 2003-07-31 | Isis Pharmaceuticals Inc. | Antisense modulation of BCL2-associated X protein expression |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| US7148342B2 (en) | 2002-07-24 | 2006-12-12 | The Trustees Of The University Of Pennyslvania | Compositions and methods for sirna inhibition of angiogenesis |
| AU2005222902B2 (en) | 2004-03-12 | 2010-06-10 | Alnylam Pharmaceuticals, Inc. | iRNA agents targeting VEGF |
| EP1919497B1 (en) | 2005-07-22 | 2020-02-12 | The Board of Trustees of the Leland Stanford Junior University | Light-activated cation channel and uses thereof |
| EP2019588A4 (en) | 2006-05-04 | 2010-11-24 | Univ Wayne State | RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN |
| US7872118B2 (en) | 2006-09-08 | 2011-01-18 | Opko Ophthalmics, Llc | siRNA and methods of manufacture |
| US20100081707A1 (en) | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| 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 |
| JP6348064B2 (ja) * | 2011-11-22 | 2018-06-27 | ザ チルドレンズ ホスピタル オブ フィラデルフィア | 効率の高いトランスジーン送達のためのウイルスベクター |
| ES2908467T3 (es) * | 2016-04-29 | 2022-04-29 | Adverum Biotechnologies Inc | Evasión de anticuerpos neutralizantes por un virus adenoasociado recombinante |
-
2021
- 2021-04-23 CA CA3179895A patent/CA3179895A1/en active Pending
- 2021-04-23 WO PCT/EP2021/060647 patent/WO2021214282A1/en not_active Ceased
- 2021-04-23 JP JP2022563882A patent/JP2023523211A/ja active Pending
- 2021-04-23 EP EP21720493.2A patent/EP4138929A1/en not_active Withdrawn
- 2021-04-23 US US17/996,987 patent/US20230190956A1/en active Pending
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