JP2022190081A5 - - Google Patents
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- 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 |
|---|---|---|---|
| US201762556319P | 2017-09-08 | 2017-09-08 | |
| US201762556324P | 2017-09-08 | 2017-09-08 | |
| US201762556329P | 2017-09-08 | 2017-09-08 | |
| US201762556335P | 2017-09-08 | 2017-09-08 | |
| US201762556281P | 2017-09-08 | 2017-09-08 | |
| US201762556331P | 2017-09-08 | 2017-09-08 | |
| US62/556,329 | 2017-09-08 | ||
| US62/556,335 | 2017-09-08 | ||
| US62/556,331 | 2017-09-08 | ||
| US62/556,319 | 2017-09-08 | ||
| US62/556,324 | 2017-09-08 | ||
| US62/556,281 | 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 (44)
| 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 |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| TW202545984A (zh) | 2017-08-09 | 2025-12-01 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| KR20200093635A (ko) * | 2017-12-06 | 2020-08-05 | 제너레이션 바이오 컴퍼니 | 변형된 폐쇄된 말단 dna (cedna)를 사용한 유전자 편집 |
| WO2019118921A1 (en) | 2017-12-14 | 2019-06-20 | Flodesign Sonics, Inc. | Acoustic transducer drive and controller |
| MA51842A (fr) * | 2018-02-14 | 2020-12-23 | Generation Bio Co | Vecteurs d'adn non viraux et utilisations associées pour la production d'anticorps et de protéines de fusion |
| WO2020033863A1 (en) | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| CN113316640A (zh) * | 2018-11-09 | 2021-08-27 | 世代生物公司 | 包含对称的被修饰的反向末端重复序列的被修饰的封闭端dna(cedna) |
| GB201905651D0 (en) * | 2019-04-24 | 2019-06-05 | Lightbio Ltd | Nucleic acid constructs and methods for their manufacture |
| EP3999646A4 (en) * | 2019-07-17 | 2023-08-30 | Generation Bio Co. | COMPOSITIONS AND PRODUCTION OF NICKEL-CLOSED CLOSED-END DNA VECTOR |
| AU2020315444A1 (en) * | 2019-07-17 | 2021-12-23 | Generation Bio Co. | Synthetic production of single-stranded adeno associated viral DNA vectors |
| BR112022002794A2 (pt) * | 2019-08-15 | 2022-08-09 | Childrens Hospital Philadelphia | Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1 |
| JP7700101B2 (ja) * | 2019-09-06 | 2025-06-30 | ジェネレーション バイオ カンパニー | 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法 |
| GB2700048B (en) | 2019-09-18 | 2026-03-04 | Aldevron Llc | synthetic dna vectors and method of use |
| 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 |
| MX2022011805A (es) * | 2020-03-24 | 2023-01-11 | Generation Bio Co | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix. |
| AU2021244559A1 (en) * | 2020-03-24 | 2022-11-17 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics |
| US20230181764A1 (en) * | 2020-05-18 | 2023-06-15 | Generation Bio Co. | Novel lipids and nanoparticle compositions thereof |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| US20220243201A1 (en) * | 2020-08-23 | 2022-08-04 | Bioverativ Therapeutics Inc. | Engineered itr sequences and methods of use |
| US20220090130A1 (en) | 2020-08-23 | 2022-03-24 | Bioverativ Therapeutics Inc. | MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA) |
| AU2021342503A1 (en) * | 2020-09-16 | 2023-04-06 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing FVIII therapeutics |
| US20240026374A1 (en) * | 2020-09-16 | 2024-01-25 | Generation Bio Co. | Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) |
| 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 |
| WO2022232029A2 (en) * | 2021-04-26 | 2022-11-03 | University Of Florida Research Foundation, Incorporated | Synthetic aav vectors for repeated delivery of therapeutic genes |
| EP4329884A1 (en) * | 2021-04-27 | 2024-03-06 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
| CN117881786A (zh) * | 2021-04-27 | 2024-04-12 | 世代生物公司 | 表达治疗性抗体的非病毒dna载体及其用途 |
| CN117729934A (zh) * | 2021-05-07 | 2024-03-19 | 世代生物公司 | 用于疫苗递送的非病毒dna载体 |
| WO2022236016A1 (en) * | 2021-05-07 | 2022-11-10 | Generation Bio Co. | Lyophilized non-viral dna vector compositions and uses thereof |
| US20250027107A1 (en) * | 2021-10-18 | 2025-01-23 | Flagship Pioneering Innovations Vii, Llc | Dna compositions and related methods |
| WO2023177655A1 (en) | 2022-03-14 | 2023-09-21 | Generation Bio Co. | Heterologous prime boost vaccine compositions and methods of use |
| 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 |
| WO2024040222A1 (en) * | 2022-08-19 | 2024-02-22 | 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 |
| 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 |
| 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 |
| 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 |
| 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 |
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 |
| WO2005072364A2 (en) * | 2004-01-27 | 2005-08-11 | University Of Florida | A 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 |
| AU2013281328B2 (en) * | 2012-06-27 | 2017-11-23 | MeiraGTx Gene Regulation Limited | 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 |
| DK3402483T3 (da) * | 2016-01-15 | 2024-01-02 | American Gene Tech Int Inc | Fremgangsmåder og sammensætninger til aktivering af gamma-delta-T-celler |
| WO2017152149A1 (en) * | 2016-03-03 | 2017-09-08 | University Of Massachusetts | Closed-ended linear duplex dna for non-viral gene transfer |
-
2018
- 2018-09-07 JP JP2020512843A patent/JP2020532981A/ja active Pending
- 2018-09-07 CA CA3075168A patent/CA3075168A1/en active Pending
- 2018-09-07 UA UAA202000594A patent/UA129010C2/uk unknown
- 2018-09-07 KR KR1020207009737A patent/KR20200051011A/ko not_active Ceased
- 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 SG SG11202000698SA patent/SG11202000698SA/en unknown
- 2018-09-07 EP EP18854941.4A patent/EP3678710A4/en active Pending
- 2018-09-07 MA MA050100A patent/MA50100A/fr unknown
- 2018-09-07 WO PCT/US2018/049996 patent/WO2019051255A1/en not_active Ceased
- 2018-09-07 US US16/644,568 patent/US20200283794A1/en not_active Abandoned
- 2018-09-07 CN CN201880058155.7A patent/CN111132699A/zh active Pending
- 2018-09-07 MX MX2020002500A patent/MX2020002500A/es unknown
-
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