JP2022141670A5 - - Google Patents
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- Publication number
- JP2022141670A5 JP2022141670A5 JP2022103103A JP2022103103A JP2022141670A5 JP 2022141670 A5 JP2022141670 A5 JP 2022141670A5 JP 2022103103 A JP2022103103 A JP 2022103103A JP 2022103103 A JP2022103103 A JP 2022103103A JP 2022141670 A5 JP2022141670 A5 JP 2022141670A5
- Authority
- JP
- Japan
- Prior art keywords
- aav vector
- cell
- pharmaceutical composition
- subject
- use according
- 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
- 239000013607 AAV vector Substances 0.000 claims 23
- 239000008194 pharmaceutical composition Substances 0.000 claims 17
- 238000002347 injection Methods 0.000 claims 10
- 239000007924 injection Substances 0.000 claims 10
- 206010064930 age-related macular degeneration Diseases 0.000 claims 9
- 208000002780 macular degeneration Diseases 0.000 claims 9
- 239000002773 nucleotide Substances 0.000 claims 7
- 125000003729 nucleotide group Chemical group 0.000 claims 7
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims 6
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 5
- 108090000565 Capsid Proteins Proteins 0.000 claims 3
- 102100023321 Ceruloplasmin Human genes 0.000 claims 3
- 239000002245 particle Substances 0.000 claims 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 206010012689 Diabetic retinopathy Diseases 0.000 claims 2
- 208000022873 Ocular disease Diseases 0.000 claims 2
- -1 cell Substances 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 208000011325 dry age related macular degeneration Diseases 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- BTCSSZJGUNDROE-UHFFFAOYSA-N gamma-aminobutyric acid Chemical compound NCCCC(O)=O BTCSSZJGUNDROE-UHFFFAOYSA-N 0.000 claims 2
- 230000001404 mediated effect Effects 0.000 claims 2
- 210000002467 pigment epithelium of eye Anatomy 0.000 claims 2
- 230000002207 retinal effect Effects 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 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 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024112812A JP2024156706A (ja) | 2015-10-28 | 2024-07-12 | 遺伝子治療 |
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1519086.1A GB201519086D0 (en) | 2015-10-28 | 2015-10-28 | Gene Therapy |
| GB1519086.1 | 2015-10-28 | ||
| JP2018541571A JP7126944B2 (ja) | 2015-10-28 | 2016-10-27 | 遺伝子治療 |
| PCT/GB2016/053343 WO2017072515A1 (en) | 2015-10-28 | 2016-10-27 | Gene therapy |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018541571A Division JP7126944B2 (ja) | 2015-10-28 | 2016-10-27 | 遺伝子治療 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024112812A Division JP2024156706A (ja) | 2015-10-28 | 2024-07-12 | 遺伝子治療 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022141670A JP2022141670A (ja) | 2022-09-29 |
| JP2022141670A5 true JP2022141670A5 (enExample) | 2022-10-07 |
Family
ID=55130363
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018541571A Active JP7126944B2 (ja) | 2015-10-28 | 2016-10-27 | 遺伝子治療 |
| JP2022103103A Pending JP2022141670A (ja) | 2015-10-28 | 2022-06-28 | 遺伝子治療 |
| JP2024112812A Pending JP2024156706A (ja) | 2015-10-28 | 2024-07-12 | 遺伝子治療 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018541571A Active JP7126944B2 (ja) | 2015-10-28 | 2016-10-27 | 遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024112812A Pending JP2024156706A (ja) | 2015-10-28 | 2024-07-12 | 遺伝子治療 |
Country Status (19)
| Country | Link |
|---|---|
| US (3) | US20190255193A1 (enExample) |
| EP (3) | EP3262066B2 (enExample) |
| JP (3) | JP7126944B2 (enExample) |
| KR (1) | KR20180066247A (enExample) |
| CN (4) | CN116370655A (enExample) |
| AU (3) | AU2016344523B2 (enExample) |
| CA (1) | CA3002125A1 (enExample) |
| DK (2) | DK3360890T3 (enExample) |
| ES (2) | ES2828050T3 (enExample) |
| GB (1) | GB201519086D0 (enExample) |
| HK (1) | HK1248723B (enExample) |
| IL (3) | IL284577B2 (enExample) |
| MX (1) | MX366343B (enExample) |
| NO (1) | NO3262066T3 (enExample) |
| PL (2) | PL3360890T3 (enExample) |
| RU (1) | RU2740038C2 (enExample) |
| SG (1) | SG11201802934WA (enExample) |
| WO (1) | WO2017072515A1 (enExample) |
| ZA (1) | ZA201802462B (enExample) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL258024B2 (en) | 2015-09-24 | 2023-03-01 | Univ Pennsylvania | A preparation and method for the treatment of a complement-mediated disease |
| GB201519086D0 (en) * | 2015-10-28 | 2015-12-09 | Syncona Partners Llp | Gene Therapy |
| IL262207B1 (en) | 2016-04-15 | 2025-09-01 | Univ Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| GB201608046D0 (en) * | 2016-05-09 | 2016-06-22 | Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The | Treatment of complement-mediated disorders |
| TWI820034B (zh) * | 2017-07-31 | 2023-11-01 | 香港商映像生物有限公司 | 眼部疾病之細胞模式及用於眼部疾病的療法 |
| CA3079553A1 (en) * | 2017-10-20 | 2019-04-25 | Gemini Therapeutics Inc. | Compositions and methods for treating age-related macular degeneration |
| AU2019357602A1 (en) * | 2018-10-10 | 2021-05-13 | Wisconsin Alumni Research Foundation | Kir 7.1 gene therapy vectors and methods of using the same |
| US20210371480A1 (en) * | 2018-10-23 | 2021-12-02 | Gemini Therapeutics Inc. | Compositions and methods for treating age-related macular degeneration and other diseases |
| GB201821089D0 (en) * | 2018-12-21 | 2019-02-06 | Gyroscope Therapeutics Ltd | Codon-optimised complement factor I |
| GB201821082D0 (en) * | 2018-12-21 | 2019-02-06 | Gyroscope Therapeutics Ltd | Combination of complement factors i and h, and vector encoding thereof |
| CN113966236A (zh) * | 2019-04-03 | 2022-01-21 | 再生生物股份有限公司 | 眼睛病状的基因疗法 |
| CN114828857B (zh) * | 2019-09-09 | 2024-11-12 | 联邦高等教育系统-匹兹堡大学 | 通过激活tfeb恢复视网膜色素上皮细胞的溶酶体功能的方法 |
| IL299048A (en) | 2020-06-14 | 2023-02-01 | Vertex Pharma | Variants of complement factor - 1 fusion structures and preparations containing them and their uses |
| GB202009741D0 (en) | 2020-06-25 | 2020-08-12 | Freeline Therapeutics Ltd | Polynucleotide |
| EP4211252A1 (en) * | 2020-09-09 | 2023-07-19 | Homology Medicines, Inc. | Vectorized antibodies and uses thereof |
| US20230390296A1 (en) | 2020-10-30 | 2023-12-07 | Keio University | Novel treatment and prevention of sarcopenia-related diseases |
| GB202018320D0 (en) * | 2020-11-20 | 2021-01-06 | Univ Newcastle | Methods of producing recombinant complement proteins |
| WO2023079301A1 (en) | 2021-11-04 | 2023-05-11 | Freeline Therapeutics Limited | Assays using mutant complement factor i variants with decreased activity |
| CN118973613A (zh) | 2022-01-25 | 2024-11-15 | 早野元词 | 抑制包括肌肉减少症的与衰老相关的异常的新型治疗与预防 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB9803351D0 (en) | 1998-02-17 | 1998-04-15 | Oxford Biomedica Ltd | Anti-viral vectors |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| GB0009760D0 (en) | 2000-04-19 | 2000-06-07 | Oxford Biomedica Ltd | Method |
| RU2005138368A (ru) * | 2003-06-10 | 2006-07-27 | ЭнЭсДЖЕНЕ А/С (DK) | Улучшенная секреция нейбластина |
| EP2377951A1 (en) * | 2005-02-14 | 2011-10-19 | University of Iowa Research Foundation | Methods and reagents for treatment and diagnosis of age-related macular degeneration |
| AU2006304804B2 (en) * | 2005-10-21 | 2011-06-02 | Vertex Pharmaceuticals Incorporated | Modified proteases that inhibit complement activation |
| EP2826788B1 (en) * | 2006-06-21 | 2017-12-13 | MUSC Foundation for Research Development | Targeting complement factor h for treatment of diseases by the use of cr2-fh molecules |
| CN101657097A (zh) * | 2007-03-01 | 2010-02-24 | 先进视觉疗法公司 | 以炎症为特征的疾病的治疗 |
| CA2678774A1 (en) * | 2007-03-01 | 2008-09-04 | Advanced Vision Therapies, Inc. | Treatment of diseases characterized by inflammation |
| EP3492596A1 (en) | 2007-04-09 | 2019-06-05 | University of Florida Research Foundation, Inc. | Raav vector compositions having tyrosine-modified capsid proteins and methods for use |
| GB0816702D0 (en) * | 2008-09-12 | 2008-10-22 | Trinity College Dublin | Complement proteins |
| GB0904427D0 (en) * | 2009-03-13 | 2009-04-29 | Lachmann Peter | Treatment of diseases related to hyperactivity of the complement system |
| TWI702955B (zh) * | 2012-05-15 | 2020-09-01 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒（ａａｖ）ｓｆｌｔ-１治療老年性黃斑部退化（ａｍｄ） |
| AU2014209350B8 (en) * | 2013-01-23 | 2019-04-18 | Department Of Veterans Affairs (Us) | Targeting constructs based on natural antibodies and uses thereof |
| US20140234275A1 (en) * | 2013-02-15 | 2014-08-21 | Jason Williams | Method for treating als via the increased production of factor h |
| CA2909706C (en) * | 2013-04-17 | 2023-02-14 | Genzyme Corporation | Use of an il17 inhibitor for treating and preventing macular degeneration |
| ES2716615T3 (es) * | 2013-06-28 | 2019-06-13 | Inst Nat Sante Rech Med | Métodos para expresar un polinucleótido de interés en la retina de un sujeto |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| GB201519086D0 (en) * | 2015-10-28 | 2015-12-09 | Syncona Partners Llp | Gene Therapy |
| GB201608046D0 (en) * | 2016-05-09 | 2016-06-22 | Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The | Treatment of complement-mediated disorders |
-
2015
- 2015-10-28 GB GBGB1519086.1A patent/GB201519086D0/en not_active Ceased
-
2016
- 2016-10-27 PL PL17210269T patent/PL3360890T3/pl unknown
- 2016-10-27 JP JP2018541571A patent/JP7126944B2/ja active Active
- 2016-10-27 CA CA3002125A patent/CA3002125A1/en active Pending
- 2016-10-27 NO NO16788764A patent/NO3262066T3/no unknown
- 2016-10-27 CN CN202310132380.3A patent/CN116370655A/zh active Pending
- 2016-10-27 PL PL16788764.5T patent/PL3262066T5/pl unknown
- 2016-10-27 HK HK18108313.3A patent/HK1248723B/en active IP Right Maintenance
- 2016-10-27 CN CN202311083674.8A patent/CN117343960A/zh active Pending
- 2016-10-27 IL IL284577A patent/IL284577B2/en unknown
- 2016-10-27 KR KR1020187015024A patent/KR20180066247A/ko active Pending
- 2016-10-27 EP EP16788764.5A patent/EP3262066B2/en active Active
- 2016-10-27 EP EP20187535.8A patent/EP3786176A1/en active Pending
- 2016-10-27 CN CN202310134734.8A patent/CN116271104A/zh active Pending
- 2016-10-27 RU RU2018118954A patent/RU2740038C2/ru active
- 2016-10-27 ES ES17210269T patent/ES2828050T3/es active Active
- 2016-10-27 EP EP17210269.1A patent/EP3360890B1/en active Active
- 2016-10-27 US US15/771,849 patent/US20190255193A1/en not_active Abandoned
- 2016-10-27 DK DK17210269.1T patent/DK3360890T3/da active
- 2016-10-27 SG SG11201802934WA patent/SG11201802934WA/en unknown
- 2016-10-27 MX MX2018005145A patent/MX366343B/es active IP Right Grant
- 2016-10-27 CN CN201680073421.4A patent/CN108431030B/zh active Active
- 2016-10-27 DK DK16788764.5T patent/DK3262066T4/da active
- 2016-10-27 AU AU2016344523A patent/AU2016344523B2/en active Active
- 2016-10-27 ES ES16788764T patent/ES2666204T5/es active Active
- 2016-10-27 WO PCT/GB2016/053343 patent/WO2017072515A1/en not_active Ceased
-
2018
- 2018-04-13 ZA ZA2018/02462A patent/ZA201802462B/en unknown
- 2018-04-18 IL IL258806A patent/IL258806B/en unknown
-
2022
- 2022-05-30 AU AU2022203675A patent/AU2022203675B2/en active Active
- 2022-06-28 JP JP2022103103A patent/JP2022141670A/ja active Pending
- 2022-12-16 US US18/067,283 patent/US20230277689A1/en not_active Abandoned
-
2023
- 2023-06-27 IL IL304084A patent/IL304084B1/en unknown
-
2024
- 2024-07-12 JP JP2024112812A patent/JP2024156706A/ja active Pending
-
2025
- 2025-02-13 AU AU2025200979A patent/AU2025200979A1/en active Pending
- 2025-03-27 US US19/093,102 patent/US20250319210A1/en active Pending
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