JPWO2021183779A5 - - Google Patents
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- Publication number
- JPWO2021183779A5 JPWO2021183779A5 JP2022554440A JP2022554440A JPWO2021183779A5 JP WO2021183779 A5 JPWO2021183779 A5 JP WO2021183779A5 JP 2022554440 A JP2022554440 A JP 2022554440A JP 2022554440 A JP2022554440 A JP 2022554440A JP WO2021183779 A5 JPWO2021183779 A5 JP WO2021183779A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- promoter
- nmnat1
- composition according
- eye
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims description 23
- 239000013598 vector Substances 0.000 claims description 20
- 101710143608 Nicotinamide/nicotinic acid mononucleotide adenylyltransferase 1 Proteins 0.000 claims description 19
- 102100023742 Rhodopsin kinase GRK1 Human genes 0.000 claims description 12
- 238000000034 method Methods 0.000 claims description 11
- 101000996052 Homo sapiens Nicotinamide/nicotinic acid mononucleotide adenylyltransferase 1 Proteins 0.000 claims description 10
- 102000048894 human NMNAT1 Human genes 0.000 claims description 10
- 108091004242 G-Protein-Coupled Receptor Kinase 1 Proteins 0.000 claims description 6
- 101000829506 Homo sapiens Rhodopsin kinase GRK1 Proteins 0.000 claims description 6
- 201000007737 Retinal degeneration Diseases 0.000 claims description 6
- 102100040756 Rhodopsin Human genes 0.000 claims description 6
- 108090000799 Rhodopsin kinases Proteins 0.000 claims description 6
- 238000002347 injection Methods 0.000 claims description 6
- 239000007924 injection Substances 0.000 claims description 6
- 230000035772 mutation Effects 0.000 claims description 6
- 230000004258 retinal degeneration Effects 0.000 claims description 6
- 108010041788 rho-Associated Kinases Proteins 0.000 claims description 6
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 4
- 210000000608 photoreceptor cell Anatomy 0.000 claims description 3
- 210000001525 retina Anatomy 0.000 claims description 3
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 230000002207 retinal effect Effects 0.000 claims description 2
- 239000013607 AAV vector Substances 0.000 claims 2
- 108020004705 Codon Proteins 0.000 claims 2
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 108091008695 photoreceptors Proteins 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062988260P | 2020-03-11 | 2020-03-11 | |
| US62/988,260 | 2020-03-11 | ||
| PCT/US2021/021936 WO2021183779A1 (en) | 2020-03-11 | 2021-03-11 | Gene therapy for nmnat1-associated retinal degeneration |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023517929A JP2023517929A (ja) | 2023-04-27 |
| JPWO2021183779A5 true JPWO2021183779A5 (https=) | 2024-03-19 |
| JP2023517929A5 JP2023517929A5 (https=) | 2024-03-19 |
Family
ID=77672442
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022554440A Pending JP2023517929A (ja) | 2020-03-11 | 2021-03-11 | Ｎｍｎａｔ１関連網膜変性に対する遺伝子療法 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US12257320B2 (https=) |
| EP (1) | EP4117644A4 (https=) |
| JP (1) | JP2023517929A (https=) |
| KR (1) | KR20220152550A (https=) |
| CN (1) | CN115379834A (https=) |
| AU (1) | AU2021236233A1 (https=) |
| BR (1) | BR112022017970A2 (https=) |
| CA (1) | CA3174781A1 (https=) |
| IL (1) | IL296262A (https=) |
| MX (1) | MX2022011177A (https=) |
| WO (1) | WO2021183779A1 (https=) |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2008501343A (ja) * | 2004-06-04 | 2008-01-24 | ワシントン・ユニバーシティ | 神経障害を治療するための方法および組成物 |
| EP3030665B9 (en) | 2013-08-05 | 2023-10-04 | Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum | Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp |
| KR102537394B1 (ko) * | 2014-03-17 | 2023-05-30 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| PT3191139T (pt) | 2014-07-24 | 2020-11-05 | Massachusetts Eye & Ear Infirmary | Terapia do gene rpgr para retinite pigmentosa |
| PL3265568T3 (pl) * | 2015-03-06 | 2020-11-30 | Massachusetts Eye & Ear Infirmary | Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31 |
| EP3364973A1 (en) * | 2015-10-23 | 2018-08-29 | The Jackson Laboratory | Nicotinamide for use in the treatment and prevention of ocular neurodegenerative disorder (e.g. glaucoma) |
| US11260081B2 (en) | 2016-06-29 | 2022-03-01 | Ramot At Tel-Aviv University Ltd. | Engineered parasites for delivering protein to the central nervous system (CNS) |
| CN116509885A (zh) | 2016-12-21 | 2023-08-01 | 新南创新私人有限公司 | 增加血管密度的方法 |
| KR20200036912A (ko) * | 2017-07-31 | 2020-04-07 | 리플렉션 바이오테크놀러지스 리미티드 | 안과 질환을 위한 세포 모델 및 치료요법 |
-
2021
- 2021-03-11 US US17/199,115 patent/US12257320B2/en active Active
- 2021-03-11 KR KR1020227034618A patent/KR20220152550A/ko active Pending
- 2021-03-11 WO PCT/US2021/021936 patent/WO2021183779A1/en not_active Ceased
- 2021-03-11 AU AU2021236233A patent/AU2021236233A1/en active Pending
- 2021-03-11 CA CA3174781A patent/CA3174781A1/en active Pending
- 2021-03-11 BR BR112022017970A patent/BR112022017970A2/pt not_active Application Discontinuation
- 2021-03-11 CN CN202180027825.0A patent/CN115379834A/zh active Pending
- 2021-03-11 EP EP21767865.5A patent/EP4117644A4/en active Pending
- 2021-03-11 JP JP2022554440A patent/JP2023517929A/ja active Pending
- 2021-03-11 MX MX2022011177A patent/MX2022011177A/es unknown
- 2021-03-11 IL IL296262A patent/IL296262A/en unknown
-
2025
- 2025-02-21 US US19/060,033 patent/US20250269068A1/en active Pending
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