JP2022190081A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2022190081A5 JP2022190081A5 JP2022176371A JP2022176371A JP2022190081A5 JP 2022190081 A5 JP2022190081 A5 JP 2022190081A5 JP 2022176371 A JP2022176371 A JP 2022176371A JP 2022176371 A JP2022176371 A JP 2022176371A JP 2022190081 A5 JP2022190081 A5 JP 2022190081A5
- Authority
- JP
- Japan
- Prior art keywords
- cedna
- vector
- cedna vector
- asymmetric
- itrs
- 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
- 241000238631 Hexapoda Species 0.000 claims 15
- 230000003612 virological effect Effects 0.000 claims 11
- 108091023040 Transcription factor Proteins 0.000 claims 8
- 102000040945 Transcription factor Human genes 0.000 claims 8
- 108091033319 polynucleotide Proteins 0.000 claims 7
- 102000040430 polynucleotide Human genes 0.000 claims 7
- 239000002157 polynucleotide Substances 0.000 claims 7
- 108090000623 proteins and genes Proteins 0.000 claims 7
- 102000004169 proteins and genes Human genes 0.000 claims 7
- 239000002773 nucleotide Substances 0.000 claims 6
- 125000003729 nucleotide group Chemical group 0.000 claims 6
- 108020004414 DNA Proteins 0.000 claims 4
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 4
- 238000012217 deletion Methods 0.000 claims 4
- 230000037430 deletion Effects 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 238000004519 manufacturing process Methods 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 238000003306 harvesting Methods 0.000 claims 2
- 239000002539 nanocarrier Substances 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- 230000010076 replication Effects 0.000 claims 2
- 238000006467 substitution reaction Methods 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 108091023037 Aptamer Proteins 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 241000125945 Protoparvovirus Species 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 239000000427 antigen Substances 0.000 claims 1
- 108091007433 antigens Proteins 0.000 claims 1
- 102000036639 antigens Human genes 0.000 claims 1
- 238000001962 electrophoresis Methods 0.000 claims 1
- 239000000499 gel Substances 0.000 claims 1
- 238000010362 genome editing Methods 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000002105 nanoparticle Substances 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 108091008146 restriction endonucleases Proteins 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
- 241000701447 unidentified baculovirus Species 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024189111A JP2025010253A (ja) | 2017-09-08 | 2024-10-28 | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
Applications Claiming Priority (13)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762556329P | 2017-09-08 | 2017-09-08 | |
| US201762556331P | 2017-09-08 | 2017-09-08 | |
| US201762556324P | 2017-09-08 | 2017-09-08 | |
| US201762556281P | 2017-09-08 | 2017-09-08 | |
| US201762556319P | 2017-09-08 | 2017-09-08 | |
| US201762556335P | 2017-09-08 | 2017-09-08 | |
| US62/556,331 | 2017-09-08 | ||
| US62/556,335 | 2017-09-08 | ||
| US62/556,281 | 2017-09-08 | ||
| US62/556,324 | 2017-09-08 | ||
| US62/556,319 | 2017-09-08 | ||
| US62/556,329 | 2017-09-08 | ||
| JP2020512843A JP2020532981A (ja) | 2017-09-08 | 2018-09-07 | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020512843A Division JP2020532981A (ja) | 2017-09-08 | 2018-09-07 | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024189111A Division JP2025010253A (ja) | 2017-09-08 | 2024-10-28 | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022190081A JP2022190081A (ja) | 2022-12-22 |
| JP2022190081A5 true JP2022190081A5 (https=) | 2023-08-10 |
Family
ID=65635235
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020512843A Pending JP2020532981A (ja) | 2017-09-08 | 2018-09-07 | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
| JP2022176371A Pending JP2022190081A (ja) | 2017-09-08 | 2022-11-02 | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
| JP2024189111A Pending JP2025010253A (ja) | 2017-09-08 | 2024-10-28 | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020512843A Pending JP2020532981A (ja) | 2017-09-08 | 2018-09-07 | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024189111A Pending JP2025010253A (ja) | 2017-09-08 | 2024-10-28 | 修飾型閉端ｄｎａ（ｃｅｄｎａ） |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20200283794A1 (https=) |
| EP (1) | EP3678710A4 (https=) |
| JP (3) | JP2020532981A (https=) |
| KR (1) | KR20200051011A (https=) |
| CN (1) | CN111132699A (https=) |
| AU (1) | AU2018327348A1 (https=) |
| BR (1) | BR112020004151A2 (https=) |
| CA (1) | CA3075168A1 (https=) |
| IL (1) | IL272797A (https=) |
| MA (1) | MA50100A (https=) |
| MX (1) | MX2020002500A (https=) |
| PH (1) | PH12020500465A1 (https=) |
| SG (1) | SG11202000698SA (https=) |
| UA (1) | UA129010C2 (https=) |
| WO (1) | WO2019051255A1 (https=) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| WO2015105955A1 (en) | 2014-01-08 | 2015-07-16 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| NZ762100A (en) | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
| CA3084185A1 (en) * | 2017-12-06 | 2019-06-13 | Generation Bio Co. | Gene editing using a modified closed-ended dna (cedna) |
| KR102439221B1 (ko) | 2017-12-14 | 2022-09-01 | 프로디자인 소닉스, 인크. | 음향 트랜스듀서 구동기 및 제어기 |
| KR20200120649A (ko) * | 2018-02-14 | 2020-10-21 | 제너레이션 바이오 컴퍼니 | 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도 |
| EP3833766A1 (en) | 2018-08-09 | 2021-06-16 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| KR20210090619A (ko) * | 2018-11-09 | 2021-07-20 | 제너레이션 바이오 컴퍼니 | 대칭인 변형된 역말단반복을 포함하는 변형된 폐쇄형 DNA(ceDNA) |
| GB201905651D0 (en) | 2019-04-24 | 2019-06-05 | Lightbio Ltd | Nucleic acid constructs and methods for their manufacture |
| CA3146966A1 (en) * | 2019-07-17 | 2021-01-21 | Generation Bio Co. | Compositions and production of nicked closed-ended dna vectors |
| CA3147414A1 (en) * | 2019-07-17 | 2021-01-21 | Generation Bio Co. | Synthetic production of single-stranded adeno associated viral dna vectors |
| EP4013414A4 (en) * | 2019-08-15 | 2023-09-27 | The Children's Hospital of Philadelphia | COMBINED INTRON-DERIVED MIRNA AND TRANSGENE THERAPY FOR THE TREATMENT OF SCA1 |
| WO2021046265A1 (en) * | 2019-09-06 | 2021-03-11 | Generation Bio Co. | Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof |
| KR20220128607A (ko) | 2019-09-18 | 2022-09-21 | 인터갈락틱 테라퓨틱스, 인크. | 합성 dna 벡터 및 사용 방법 |
| WO2021072031A1 (en) * | 2019-10-11 | 2021-04-15 | Insideoutbio, Inc. | Methods and compositions for the manufacture and use of circular dna encoded therapeutics for genetic disorders and other diseases |
| WO2021169167A1 (en) * | 2020-02-29 | 2021-09-02 | Nanjing GenScript Biotech Co., Ltd. | Method for treating coronavirus infections |
| DE102020111571A1 (de) | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| US20230138409A1 (en) * | 2020-03-24 | 2023-05-04 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing factor ix therapeutics |
| JP2023520763A (ja) * | 2020-03-24 | 2023-05-19 | ジェネレーション バイオ カンパニー | ゴーシェ治療薬を発現するための非ウイルス性ｄｎａベクター及びその使用 |
| IL298241A (en) * | 2020-05-18 | 2023-01-01 | Generation Bio Co | New lipids and their nanoparticle preparations |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Ｄｎａ分子の組成物、その作製方法、及びその使用方法 |
| WO2022046665A1 (en) | 2020-08-23 | 2022-03-03 | Bioverativ Therapeutics Inc. | MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA) |
| US20220243201A1 (en) * | 2020-08-23 | 2022-08-04 | Bioverativ Therapeutics Inc. | Engineered itr sequences and methods of use |
| EP4214309A4 (en) * | 2020-09-16 | 2025-07-16 | Generation Bio Co | CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) |
| CA3191743A1 (en) * | 2020-09-16 | 2022-03-24 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
| GB202014751D0 (en) * | 2020-09-18 | 2020-11-04 | Lightbio Ltd | Targeting vector |
| CA3211687A1 (en) * | 2021-03-19 | 2022-09-22 | Ozan ALKAN | Non-viral dna vectors and uses thereof for expressing pfic therapeutics |
| US20240209393A1 (en) * | 2021-04-26 | 2024-06-27 | University Of Florida Research Foundation, Incorporated | Synthetic aav vectors for repeated delivery of therapeutic genes |
| US20240216535A1 (en) * | 2021-04-27 | 2024-07-04 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
| AU2022264509A1 (en) * | 2021-04-27 | 2023-12-14 | Generation Bio Co. | Non-viral dna vectors expressing therapeutic antibodies and uses thereof |
| CA3218126A1 (en) * | 2021-05-07 | 2022-11-10 | Matthew MANGANIELLO | Lyophilized non-viral dna vector compositions and uses thereof |
| KR20240005903A (ko) * | 2021-05-07 | 2024-01-12 | 제너레이션 바이오 컴퍼니 | 백신 전달을 위한 비바이러스성 dna 벡터 |
| WO2023069948A1 (en) * | 2021-10-18 | 2023-04-27 | Flagship Pioneering Innovations Vii, Llc | Dna compositions and related methods |
| CN119072330A (zh) | 2022-03-14 | 2024-12-03 | 世代生物公司 | 异源初免加强疫苗组合物和使用方法 |
| CN117802161A (zh) * | 2022-06-30 | 2024-04-02 | 苏州吉恒基因科技有限公司 | 精准重组腺相关病毒载体及其用途 |
| US20260055426A1 (en) * | 2022-08-19 | 2026-02-26 | Generation Bio Co. | CLEAVABLE CLOSED-ENDED DNA (ceDNA) AND METHODS OF USE THEREOF |
| AU2024229184A1 (en) | 2023-03-02 | 2025-08-28 | Krystal Biotech, Inc. | Interleukin-2 and interleukin-12 for cancer therapy |
| US11767541B1 (en) * | 2023-03-27 | 2023-09-26 | Genecraft, Inc. | Adeno-associated virus complex with improved expression of RUNX3 gene and uses for preventing or treating KRAS mutated lung cancer |
| US11993783B1 (en) * | 2023-03-27 | 2024-05-28 | Genecraft Inc. | Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and use thereof |
| US11891617B1 (en) * | 2023-03-27 | 2024-02-06 | Genecraft Inc. | Modified complex platform of adeno-associated virus with improved rate of expression of loaded genes and reduced genotoxicity |
| EP4698646A2 (en) * | 2023-04-21 | 2026-02-25 | ARIZONA BOARD OF REGENTS ON BEHALF OF ARIZONA STATE UNIVERSITY 1475 North Scottsdale Road | Novel transmembrane sensors and method of characterization for lysis-free detection of intracellular targets |
| WO2025114932A1 (en) * | 2023-11-30 | 2025-06-05 | Sanofi | Purification of closed-ended dna molecules |
| WO2026085495A1 (en) | 2024-10-18 | 2026-04-23 | Generation Bio Co. | Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050019824A1 (en) * | 1994-03-08 | 2005-01-27 | Human Genome Sciences, Inc. | Fibroblast Growth Factor-10 |
| US20050026838A1 (en) * | 1995-06-05 | 2005-02-03 | Human Genome Sciences, Inc. | Fibroblast Growth Factor-13 |
| DE10044384A1 (de) * | 2000-09-08 | 2002-04-18 | Medigene Ag | Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung |
| US20060166363A1 (en) * | 2004-01-27 | 2006-07-27 | Sergei Zolotukhin | Modified baculovirus expression system for production of pseudotyped rAAV vector |
| WO2008016391A2 (en) * | 2006-01-31 | 2008-02-07 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
| EP2500434A1 (en) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| US9943574B2 (en) * | 2012-06-27 | 2018-04-17 | Arthrogen B.V. | Combination for treating an inflammatory disorder |
| CN104087613B (zh) * | 2014-06-30 | 2017-08-29 | 中国科学院苏州生物医学工程技术研究所 | 基于aav‑itr的基因表达微载体及其构建方法和应用 |
| CN116064678A (zh) * | 2016-01-15 | 2023-05-05 | 美国基因技术国际有限公司 | 用于活化γ-δT细胞的方法和组合物 |
| MX2018010633A (es) * | 2016-03-03 | 2019-06-13 | Univ Massachusetts | Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral. |
-
2018
- 2018-09-07 CA CA3075168A patent/CA3075168A1/en active Pending
- 2018-09-07 US US16/644,568 patent/US20200283794A1/en not_active Abandoned
- 2018-09-07 MX MX2020002500A patent/MX2020002500A/es unknown
- 2018-09-07 WO PCT/US2018/049996 patent/WO2019051255A1/en not_active Ceased
- 2018-09-07 EP EP18854941.4A patent/EP3678710A4/en active Pending
- 2018-09-07 AU AU2018327348A patent/AU2018327348A1/en not_active Abandoned
- 2018-09-07 BR BR112020004151-3A patent/BR112020004151A2/pt not_active Application Discontinuation
- 2018-09-07 MA MA050100A patent/MA50100A/fr unknown
- 2018-09-07 KR KR1020207009737A patent/KR20200051011A/ko not_active Ceased
- 2018-09-07 SG SG11202000698SA patent/SG11202000698SA/en unknown
- 2018-09-07 JP JP2020512843A patent/JP2020532981A/ja active Pending
- 2018-09-07 UA UAA202000594A patent/UA129010C2/uk unknown
- 2018-09-07 CN CN201880058155.7A patent/CN111132699A/zh active Pending
-
2020
- 2020-02-20 IL IL272797A patent/IL272797A/en unknown
- 2020-03-06 PH PH12020500465A patent/PH12020500465A1/en unknown
-
2022
- 2022-11-02 JP JP2022176371A patent/JP2022190081A/ja active Pending
-
2024
- 2024-10-28 JP JP2024189111A patent/JP2025010253A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2022190081A5 (https=) | ||
| JP2022520803A5 (https=) | ||
| JP2024009857A5 (https=) | ||
| JP2020532981A5 (https=) | ||
| JP2022516004A5 (https=) | ||
| JP2021533795A5 (https=) | ||
| JP2022549140A5 (https=) | ||
| JP2020519284A5 (https=) | ||
| JP2022542828A5 (https=) | ||
| US11499141B2 (en) | Adeno-associated virus (AAV) with modified phospholipase domain | |
| JP2022524434A5 (https=) | ||
| IL292264B1 (en) | Aav transfer cassette | |
| JP2022525302A5 (https=) | ||
| JP2022505106A5 (https=) | ||
| JP2022551744A5 (https=) | ||
| JP2023537358A5 (https=) | ||
| JP2023541443A5 (https=) | ||
| JP2023520763A5 (https=) | ||
| JP2025029049A5 (https=) | ||
| CN121925426A (zh) | 肝脏去靶向衣壳 | |
| CN120787264A (zh) | 肝脏去靶向衣壳 | |
| JP2024503895A5 (https=) | ||
| CN120882875A (zh) | 肌肉靶向衣壳 | |
| JP2022527126A5 (https=) | ||
| WO2024121778A1 (en) | Improved rep genes for recombinant aav production |