JP2018506982A5 - - Google Patents
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- Publication number
- JP2018506982A5 JP2018506982A5 JP2017544639A JP2017544639A JP2018506982A5 JP 2018506982 A5 JP2018506982 A5 JP 2018506982A5 JP 2017544639 A JP2017544639 A JP 2017544639A JP 2017544639 A JP2017544639 A JP 2017544639A JP 2018506982 A5 JP2018506982 A5 JP 2018506982A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- pharmaceutical composition
- photoreceptor
- composition according
- rod
- 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.)
- Granted
Links
- 239000013598 vector Substances 0.000 claims description 40
- 102000039446 nucleic acids Human genes 0.000 claims description 15
- 108020004707 nucleic acids Proteins 0.000 claims description 15
- 150000007523 nucleic acids Chemical class 0.000 claims description 15
- 210000000880 retinal rod photoreceptor cell Anatomy 0.000 claims description 15
- 208000002780 macular degeneration Diseases 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 11
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 9
- 108091008695 photoreceptors Proteins 0.000 claims description 7
- 238000000034 method Methods 0.000 claims description 6
- 210000001525 retina Anatomy 0.000 claims description 5
- 230000011664 signaling Effects 0.000 claims description 5
- 239000013607 AAV vector Substances 0.000 claims description 4
- 101000609949 Homo sapiens Rod cGMP-specific 3',5'-cyclic phosphodiesterase subunit beta Proteins 0.000 claims description 4
- 102100039174 Rod cGMP-specific 3',5'-cyclic phosphodiesterase subunit beta Human genes 0.000 claims description 4
- 230000004377 improving vision Effects 0.000 claims description 4
- 101001079096 Homo sapiens Regulator of G-protein signaling 9-binding protein Proteins 0.000 claims description 3
- 206010034972 Photosensitivity reaction Diseases 0.000 claims description 3
- 102100028148 Regulator of G-protein signaling 9-binding protein Human genes 0.000 claims description 3
- 210000004027 cell Anatomy 0.000 claims description 3
- 230000004064 dysfunction Effects 0.000 claims description 3
- 210000000608 photoreceptor cell Anatomy 0.000 claims description 3
- 230000036211 photosensitivity Effects 0.000 claims description 3
- 239000013603 viral vector Substances 0.000 claims description 3
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical compound O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 claims description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
- 206010007747 Cataract congenital Diseases 0.000 claims description 2
- 208000006992 Color Vision Defects Diseases 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 208000034461 Progressive cone dystrophy Diseases 0.000 claims description 2
- 108090000820 Rhodopsin Proteins 0.000 claims description 2
- 102100040756 Rhodopsin Human genes 0.000 claims description 2
- 230000004913 activation Effects 0.000 claims description 2
- 210000000234 capsid Anatomy 0.000 claims description 2
- 201000007254 color blindness Diseases 0.000 claims description 2
- 201000008615 cone dystrophy Diseases 0.000 claims description 2
- 230000007850 degeneration Effects 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 230000004438 eyesight Effects 0.000 claims description 2
- 238000005286 illumination Methods 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims description 2
- 230000002779 inactivation Effects 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 230000004310 photopic vision Effects 0.000 claims description 2
- 210000000964 retinal cone photoreceptor cell Anatomy 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 17
- 102100034268 Neural retina-specific leucine zipper protein Human genes 0.000 claims 2
- 208000008069 Geographic Atrophy Diseases 0.000 claims 1
- 108050003956 Neural retina-specific leucine zipper proteins Proteins 0.000 claims 1
- 206010047571 Visual impairment Diseases 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 208000029257 vision disease Diseases 0.000 claims 1
- 230000004393 visual impairment Effects 0.000 claims 1
- 102100030814 Regulator of G-protein signaling 9 Human genes 0.000 description 2
- 102000004169 proteins and genes Human genes 0.000 description 2
- 108010064950 regulator of g-protein signaling 9 Proteins 0.000 description 2
- 206010003694 Atrophy Diseases 0.000 description 1
- 102100026398 Cyclic AMP-responsive element-binding protein 3 Human genes 0.000 description 1
- 101000855520 Homo sapiens Cyclic AMP-responsive element-binding protein 3 Proteins 0.000 description 1
- 108090000862 Ion Channels Proteins 0.000 description 1
- 102000004310 Ion Channels Human genes 0.000 description 1
- 108010083687 Ion Pumps Proteins 0.000 description 1
- 108091006671 Ion Transporter Proteins 0.000 description 1
- 102000037862 Ion Transporter Human genes 0.000 description 1
- 108010052285 Membrane Proteins Proteins 0.000 description 1
- 102000018697 Membrane Proteins Human genes 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- 230000037444 atrophy Effects 0.000 description 1
- 230000002102 hyperpolarization Effects 0.000 description 1
- 239000012528 membrane Substances 0.000 description 1
- 230000007727 signaling mechanism Effects 0.000 description 1
- 230000000638 stimulation Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB201503008A GB201503008D0 (en) | 2015-02-23 | 2015-02-23 | Treatment |
| GB1503008.3 | 2015-02-23 | ||
| PCT/GB2016/050419 WO2016135457A1 (en) | 2015-02-23 | 2016-02-19 | Gene therapy to improve vision |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018506982A JP2018506982A (ja) | 2018-03-15 |
| JP2018506982A5 true JP2018506982A5 (enExample) | 2019-04-04 |
| JP6771474B2 JP6771474B2 (ja) | 2020-10-21 |
Family
ID=52822045
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017544639A Active JP6771474B2 (ja) | 2015-02-23 | 2016-02-19 | 視覚改善のための遺伝子治療 |
Country Status (29)
| Country | Link |
|---|---|
| US (3) | US20180030477A1 (enExample) |
| EP (1) | EP3261679B1 (enExample) |
| JP (1) | JP6771474B2 (enExample) |
| KR (1) | KR102272466B1 (enExample) |
| CN (1) | CN107530449A (enExample) |
| AU (2) | AU2016225277B2 (enExample) |
| CA (1) | CA2977464C (enExample) |
| CY (1) | CY1126098T1 (enExample) |
| DK (1) | DK3261679T3 (enExample) |
| EA (1) | EA201791900A1 (enExample) |
| ES (1) | ES2947308T3 (enExample) |
| FI (1) | FI3261679T3 (enExample) |
| GB (1) | GB201503008D0 (enExample) |
| HK (1) | HK1248594A1 (enExample) |
| HR (1) | HRP20230775T1 (enExample) |
| HU (1) | HUE062319T2 (enExample) |
| IL (1) | IL254066B (enExample) |
| LT (1) | LT3261679T (enExample) |
| MX (1) | MX2017010772A (enExample) |
| MY (1) | MY186444A (enExample) |
| PH (1) | PH12017501525B1 (enExample) |
| PL (1) | PL3261679T3 (enExample) |
| PT (1) | PT3261679T (enExample) |
| RS (1) | RS64372B1 (enExample) |
| SG (2) | SG11201706822XA (enExample) |
| SI (1) | SI3261679T1 (enExample) |
| SM (1) | SMT202300200T1 (enExample) |
| WO (1) | WO2016135457A1 (enExample) |
| ZA (1) | ZA201706440B (enExample) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018055131A1 (en) * | 2016-09-22 | 2018-03-29 | Universite Pierre Et Marie Curie (Paris 6) | Optogenetically transformed photoreceptor precursor cells for the use in the treatment of retinal degenerative diseases |
| JP2024524600A (ja) * | 2021-07-06 | 2024-07-05 | インスティテュート オブ モレキュラー アンド クリニカル オフサルモロジー バーゼル | 桿体光受容体における遺伝子の特異的発現のためのプロモーター |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2002082904A2 (en) * | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| EP2019588A4 (en) * | 2006-05-04 | 2010-11-24 | Univ Wayne State | RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN |
| PT2271369E (pt) * | 2008-04-18 | 2014-07-10 | Novartis Forschungsstiftung | Novas ferramentas terapêuticas e métodos para tratar a cegueira |
| WO2012051599A2 (en) * | 2010-10-15 | 2012-04-19 | Eos Neuroscience, Inc. | Modulation neural pathways |
| US8957028B2 (en) * | 2010-11-13 | 2015-02-17 | Massachusetts Institute Of Technology | Red-shifted opsin molecules and uses thereof |
| WO2012167109A2 (en) * | 2011-06-03 | 2012-12-06 | Massachusetts Eye & Ear Infirmary | Rpgrip1 gene therapy for leber congenital amaurosis |
| WO2013124477A1 (en) * | 2012-02-24 | 2013-08-29 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and compositions for treatment of retinal degenerative diseases |
| EP3174600A4 (en) * | 2014-07-29 | 2018-03-14 | Circuit Therapeutics, Inc. | System and method for optogenetic therapy |
-
2015
- 2015-02-23 GB GB201503008A patent/GB201503008D0/en not_active Ceased
-
2016
- 2016-02-19 PT PT167064138T patent/PT3261679T/pt unknown
- 2016-02-19 EP EP16706413.8A patent/EP3261679B1/en active Active
- 2016-02-19 KR KR1020177026938A patent/KR102272466B1/ko active Active
- 2016-02-19 PL PL16706413.8T patent/PL3261679T3/pl unknown
- 2016-02-19 RS RS20230560A patent/RS64372B1/sr unknown
- 2016-02-19 CN CN201680023264.6A patent/CN107530449A/zh active Pending
- 2016-02-19 LT LTEPPCT/GB2016/050419T patent/LT3261679T/lt unknown
- 2016-02-19 DK DK16706413.8T patent/DK3261679T3/da active
- 2016-02-19 EA EA201791900A patent/EA201791900A1/ru unknown
- 2016-02-19 WO PCT/GB2016/050419 patent/WO2016135457A1/en not_active Ceased
- 2016-02-19 MY MYPI2017001247A patent/MY186444A/en unknown
- 2016-02-19 HR HRP20230775TT patent/HRP20230775T1/hr unknown
- 2016-02-19 AU AU2016225277A patent/AU2016225277B2/en active Active
- 2016-02-19 MX MX2017010772A patent/MX2017010772A/es unknown
- 2016-02-19 PH PH1/2017/501525A patent/PH12017501525B1/en unknown
- 2016-02-19 FI FIEP16706413.8T patent/FI3261679T3/fi active
- 2016-02-19 US US15/552,737 patent/US20180030477A1/en not_active Abandoned
- 2016-02-19 SG SG11201706822XA patent/SG11201706822XA/en unknown
- 2016-02-19 ES ES16706413T patent/ES2947308T3/es active Active
- 2016-02-19 HK HK18108485.5A patent/HK1248594A1/zh unknown
- 2016-02-19 JP JP2017544639A patent/JP6771474B2/ja active Active
- 2016-02-19 SM SM20230200T patent/SMT202300200T1/it unknown
- 2016-02-19 CA CA2977464A patent/CA2977464C/en active Active
- 2016-02-19 HU HUE16706413A patent/HUE062319T2/hu unknown
- 2016-02-19 SI SI201631710T patent/SI3261679T1/sl unknown
- 2016-02-19 SG SG10202005923WA patent/SG10202005923WA/en unknown
-
2017
- 2017-08-20 IL IL254066A patent/IL254066B/en unknown
- 2017-09-22 ZA ZA2017/06440A patent/ZA201706440B/en unknown
-
2020
- 2020-02-21 US US16/797,527 patent/US20200377907A1/en not_active Abandoned
-
2022
- 2022-03-07 AU AU2022201553A patent/AU2022201553B2/en active Active
-
2023
- 2023-07-21 CY CY20231100352T patent/CY1126098T1/el unknown
-
2025
- 2025-06-04 US US19/228,304 patent/US20250290096A1/en active Pending
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