JPWO2020102499A5 - - Google Patents
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- JPWO2020102499A5 JPWO2020102499A5 JP2021526394A JP2021526394A JPWO2020102499A5 JP WO2020102499 A5 JPWO2020102499 A5 JP WO2020102499A5 JP 2021526394 A JP2021526394 A JP 2021526394A JP 2021526394 A JP2021526394 A JP 2021526394A JP WO2020102499 A5 JPWO2020102499 A5 JP WO2020102499A5
- Authority
- JP
- Japan
- Prior art keywords
- fusosome
- cell
- cns
- target cell
- specific
- 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.)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025075701A JP2025111747A (ja) | 2018-11-14 | 2025-04-30 | Cns送達のためのフソソーム組成物 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862767358P | 2018-11-14 | 2018-11-14 | |
| US62/767,358 | 2018-11-14 | ||
| US201962900064P | 2019-09-13 | 2019-09-13 | |
| US62/900,064 | 2019-09-13 | ||
| PCT/US2019/061424 WO2020102499A2 (en) | 2018-11-14 | 2019-11-14 | Fusosome compositions for cns delivery |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025075701A Division JP2025111747A (ja) | 2018-11-14 | 2025-04-30 | Cns送達のためのフソソーム組成物 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022507454A JP2022507454A (ja) | 2022-01-18 |
| JP2022507454A5 JP2022507454A5 (https=) | 2022-11-22 |
| JPWO2020102499A5 true JPWO2020102499A5 (https=) | 2022-11-22 |
| JP7676305B2 JP7676305B2 (ja) | 2025-05-14 |
Family
ID=68808599
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021526394A Active JP7676305B2 (ja) | 2018-11-14 | 2019-11-14 | Cns送達のためのフソソーム組成物 |
| JP2025075701A Pending JP2025111747A (ja) | 2018-11-14 | 2025-04-30 | Cns送達のためのフソソーム組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025075701A Pending JP2025111747A (ja) | 2018-11-14 | 2025-04-30 | Cns送達のためのフソソーム組成物 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20220008557A1 (https=) |
| EP (1) | EP3880179A2 (https=) |
| JP (2) | JP7676305B2 (https=) |
| KR (1) | KR20210133948A (https=) |
| CN (1) | CN113613633A (https=) |
| AU (1) | AU2019378881A1 (https=) |
| CA (1) | CA3120082A1 (https=) |
| IL (1) | IL283179A (https=) |
| SG (1) | SG11202105079QA (https=) |
| WO (1) | WO2020102499A2 (https=) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3235908A1 (en) | 2016-04-21 | 2017-10-25 | Ecole Normale Superieure De Lyon | Methods for selectively modulating the activity of distinct subtypes of cells |
| US11624061B2 (en) | 2017-04-28 | 2023-04-11 | Agrospheres, Inc. | Compositions and methods for enzyme immobilization |
| AU2018256925B2 (en) | 2017-04-28 | 2024-11-21 | Agrospheres, Inc. | Compositions and methods for the encapsulation and scalable delivery of agrochemicals |
| BR112020005803A2 (pt) | 2017-09-25 | 2020-12-01 | Agrospheres, Inc. | composições e métodos para produção e administração moduláveis de produtos biológicos |
| EP3820509A1 (en) | 2018-07-09 | 2021-05-19 | Flagship Pioneering Innovations V, Inc. | Fusosome compositions and uses thereof |
| US12496274B2 (en) | 2018-11-14 | 2025-12-16 | Flagship Pioneering Innovations V, Inc. | Compositions and methods for compartment-specific cargo delivery |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| JP7657726B2 (ja) | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート,インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| WO2020252455A1 (en) | 2019-06-13 | 2020-12-17 | The General Hospital Corporation | Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells |
| AU2020341454A1 (en) | 2019-09-03 | 2022-03-10 | Sana Biotechnology, Inc. | CD24-associated particles and related methods and uses thereof |
| JP2023521663A (ja) * | 2020-03-31 | 2023-05-25 | サナ バイオテクノロジー,インコーポレイテッド | 標的化脂質粒子及び組成物ならびにその使用 |
| EP4185600A4 (en) | 2020-07-24 | 2024-12-04 | The General Hospital Corporation | Enhanced virus-like particles and methods of use thereof for delivery to cells |
| EP4274901A1 (en) | 2021-01-11 | 2023-11-15 | Sana Biotechnology, Inc. | Use of cd8-targeted viral vectors |
| IL307544A (en) | 2021-04-08 | 2023-12-01 | Sana Biotechnology Inc | Cd8-specific antibody constructs and compositions thereof |
| AU2022280957A1 (en) | 2021-05-28 | 2023-11-30 | Sana Biotechnology, Inc. | Lipid particles containing a truncated baboon endogenous retrovirus (baev) envelope glycoprotein and related methods and uses |
| WO2023015217A1 (en) | 2021-08-04 | 2023-02-09 | Sana Biotechnology, Inc. | Use of cd4-targeted viral vectors |
| WO2023039242A2 (en) * | 2021-09-13 | 2023-03-16 | Achelois Biopharma, Inc. | Multivalent interferon particles compositions and methods of use |
| EP4448775A1 (en) | 2021-12-17 | 2024-10-23 | Sana Biotechnology, Inc. | Modified paramyxoviridae attachment glycoproteins |
| US20250059239A1 (en) | 2021-12-17 | 2025-02-20 | Sana Biotechnology, Inc. | Modified paramyxoviridae fusion glycoproteins |
| EP4472646A1 (en) | 2022-02-01 | 2024-12-11 | Sana Biotechnology, Inc. | Cd3-targeted lentiviral vectors and uses thereof |
| US20250144235A1 (en) | 2022-02-02 | 2025-05-08 | Sana Biotechnology, Inc. | Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses |
| US20250222027A1 (en) | 2022-04-01 | 2025-07-10 | Sana Biotechnology, Inc. | Cytokine receptor agonist and viral vector combination therapies |
| US20260055146A1 (en) | 2022-08-24 | 2026-02-26 | Sana Biotechnology, Inc. | Delivery of heterologous proteins |
| US20260098062A1 (en) | 2022-09-21 | 2026-04-09 | Sana Biotechnology, Inc. | Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof |
| JP2025532963A (ja) * | 2022-09-30 | 2025-10-03 | ヴェサイジェン,インコーポレーテッド | 眼の細胞および組織への治療用薬剤のarrdc1介在性の微小小胞体に基づく送達 |
| WO2024081820A1 (en) | 2022-10-13 | 2024-04-18 | Sana Biotechnology, Inc. | Viral particles targeting hematopoietic stem cells |
| WO2024119157A1 (en) | 2022-12-02 | 2024-06-06 | Sana Biotechnology, Inc. | Lipid particles with cofusogens and methods of producing and using the same |
| EP4698665A1 (en) | 2023-04-18 | 2026-02-25 | Sana Biotechnology, Inc. | Engineered protein g fusogens and related lipid particles and methods thereof |
| EP4698666A1 (en) | 2023-04-18 | 2026-02-25 | Sana Biotechnology, Inc. | Universal protein g fusogens and adapter systems thereof and related lipid particles and uses |
| KR20260016952A (ko) | 2023-05-23 | 2026-02-04 | 사나 바이오테크놀로지, 인크. | 탠덤 퓨소젠 및 관련 지질 입자 |
| WO2025184529A1 (en) | 2024-03-01 | 2025-09-04 | Sana Biotechnology, Inc. | Viral particles with fusogen display and related compositions and methods |
Family Cites Families (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL78703A (en) | 1985-05-10 | 1993-02-21 | Benzon Alfred | Method of producing extracellular enzymes, the enzymes produced thereby and compositions containing them; a hybrid plasmid and a dna fragment comprising dna encoding the enzymes; a microorganism harbouring the plasmid; and a method for removing nucleic acids from biological materials |
| US6534051B1 (en) * | 1992-11-20 | 2003-03-18 | University Of Medicine And Dentistry Of New Jersey | Cell type specific gene transfer using retroviral vectors containing antibody-envelope fusion proteins and wild-type envelope fusion proteins |
| JPH10501403A (ja) * | 1994-03-04 | 1998-02-10 | ユニバーシティ オブ メディシン アンド デンティストリー オブ ニュージャージー | 抗体−エンベロープ融合タンパク質および野生型エンベロープ融合タンパク質を含むレトロウィルスベクターを用いる細胞型特異的遺伝子移入 |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| PL332875A1 (en) | 1996-10-17 | 1999-10-25 | Oxford Biomedica Ltd | Retroviral vectors |
| EP0969876A2 (en) * | 1997-04-17 | 2000-01-12 | Paola Leone | Delivery system for gene therapy to the brain |
| JPH10313865A (ja) * | 1997-05-15 | 1998-12-02 | Deinabetsuku Kenkyusho:Kk | ヒト補体制御因子が呈示されたベクター |
| WO1999013905A1 (en) | 1997-09-18 | 1999-03-25 | The Trustees Of The University Of Pennsylvania | Receptor-binding pocket mutants of influenza a virus hemagglutinin for use in targeted gene delivery |
| GB9720465D0 (en) | 1997-09-25 | 1997-11-26 | Oxford Biomedica Ltd | Dual-virus vectors |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| ES2373406T3 (es) | 1997-12-22 | 2012-02-03 | Oxford Biomedica (Uk) Limited | Vectores basados en el virus de la anemia infecciosa equina (vaie). |
| JP2003532368A (ja) * | 1998-02-09 | 2003-11-05 | ジェンザイム・コーポレイション | 核酸デリバリービヒクル |
| GB9803351D0 (en) | 1998-02-17 | 1998-04-15 | Oxford Biomedica Ltd | Anti-viral vectors |
| FR2777909B1 (fr) | 1998-04-24 | 2002-08-02 | Pasteur Institut | Utilisation de sequences d'adn de structure triplex pour le tranfert de sequences de nucleotides dans des cellules, vecteurs recombinants contenant ces sequences triplex |
| DE60132436T2 (de) * | 2000-03-30 | 2008-04-17 | Oxford Biomedica (Uk) Ltd. | Retinolsäure-rezeptor beta-2 und gentherapievektoren für die behandlung von neurologischen erkrankungen |
| GB0009760D0 (en) | 2000-04-19 | 2000-06-07 | Oxford Biomedica Ltd | Method |
| ATE452972T1 (de) | 2001-05-01 | 2010-01-15 | Ca Nat Research Council | Induzierbares expressionssystem in eukaryotischen zellen |
| CA2464887A1 (en) * | 2001-10-30 | 2003-05-08 | Biogen, Inc. | Methods and compositions for treating parkinson's disease |
| US20040028687A1 (en) | 2002-01-15 | 2004-02-12 | Waelti Ernst Rudolf | Methods and compositions for the targeted delivery of therapeutic substances to specific cells and tissues |
| AU2003258060B2 (en) | 2002-03-27 | 2007-07-12 | Baylor College Of Medicine | Potent oncolytic herpes simplex virus for cancer therapy |
| ES2627445T3 (es) | 2002-05-01 | 2017-07-28 | Miltenyi Biotec Technology, Inc. | Partículas de vector de lentivirus resistentes a la inactivación por el complemento |
| DE60335672D1 (de) | 2002-05-14 | 2011-02-17 | Merck Sharp & Dohme | Verfahren zur reinigung von adenovirus |
| PL377161A1 (pl) | 2002-11-21 | 2006-01-23 | Pevion Biotech Ltd. | Wysoce efektywne pęcherzyki fuzogenne, sposób ich wytwarzania oraz kompozycje farmaceutyczne zawierające te pęcherzyki |
| WO2006027202A1 (en) | 2004-09-06 | 2006-03-16 | Unite De Recherche En Biotherapie Et Oncologie (Rubio) | Generation of multiparent cell hybrids |
| JP2008518630A (ja) | 2004-11-08 | 2008-06-05 | イェール ユニバーシティ | リボスイッチ、リボスイッチによる、構造を基礎とする化合物設計、ならびにリボスイッチの使用方法およびリボスイッチを伴う組成物 |
| US8034331B2 (en) * | 2005-01-20 | 2011-10-11 | Agency For Science, Technology And Research | Method of delivery of nucleic acids to peripheral neurons |
| PL2002003T3 (pl) * | 2005-05-27 | 2016-06-30 | Ospedale San Raffaele Srl | Wektor genetyczny zawierający mi-RNA |
| WO2007099387A1 (en) | 2006-03-03 | 2007-09-07 | Mymetics Corporation | Virosome-like vesicles comprising gp41-derived antigens |
| WO2008071959A1 (en) * | 2006-12-12 | 2008-06-19 | Oxford Biomedica (Uk) Limited | Lentiviral vectors comprising micrornas |
| US20100040650A1 (en) * | 2008-05-30 | 2010-02-18 | Crowe Jr James E | Virus-Like paramyxovirus particles and vaccines |
| CN102264349A (zh) * | 2008-11-07 | 2011-11-30 | 儿童医院医疗中心 | 用于跨膜药物递送系统的鞘脂激活蛋白c以及相关蛋白和肽促融合性质 |
| US9050269B2 (en) | 2009-03-10 | 2015-06-09 | The Trustees Of The University Of Pennsylvania | Protection of virus particles from phagocytosis by expression of CD47 |
| ES2693167T3 (es) * | 2009-11-13 | 2018-12-07 | Inserm - Institut National De La Santé Et De La Recherche Médicale | Administración directa de proteínas con microvesículas modificadas por ingeniería |
| WO2012156839A2 (en) | 2011-05-19 | 2012-11-22 | Ospedale San Raffaele S.R.L. | New generation of splice-less lentiviral vectors for safer gene therapy applications |
| RU2014100160A (ru) * | 2011-06-10 | 2015-07-20 | Блуберд Байо, Инк. | Векторы генной терапии адренолейкодистрофии и адреномиелонейропатии |
| JP2013034401A (ja) * | 2011-08-04 | 2013-02-21 | Gunma Univ | アストロサイトで遺伝子を発現させるためのベクター |
| DE112012007250B4 (de) | 2012-12-20 | 2024-10-24 | Mitsubishi Electric Corp. | Fahrzeuginterne Vorrichtung und Programm |
| US9108442B2 (en) | 2013-08-20 | 2015-08-18 | Ricoh Company, Ltd. | Image forming apparatus |
| GB201412494D0 (en) | 2014-07-14 | 2014-08-27 | Ospedale San Raffaele And Fond Telethon | Vector production |
| WO2017004381A1 (en) * | 2015-06-30 | 2017-01-05 | St. Jude Children's Research Hospital, Inc. | Method for treating schizophrenia |
| JP7097070B2 (ja) * | 2015-10-01 | 2022-07-07 | ゴレイニ インコーポレイテッド | クロライドチャネルの標的発現及びその使用方法 |
| US11248238B2 (en) * | 2015-10-22 | 2022-02-15 | Juno Therapeutics Gmbh | Methods, kits, agents and apparatuses for transduction |
| US20170112773A1 (en) | 2015-10-23 | 2017-04-27 | Board Of Regents, The University Of Texas System | Plasma membrane vesicles comprising functional transmembrane proteins |
| RU2021102893A (ru) * | 2015-11-05 | 2021-03-03 | Бамбу Терапьютикс, Инк. | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
| US11185555B2 (en) * | 2016-04-11 | 2021-11-30 | Noah James Harrison | Method to kill pathogenic microbes in a patient |
| EP3235908A1 (en) * | 2016-04-21 | 2017-10-25 | Ecole Normale Superieure De Lyon | Methods for selectively modulating the activity of distinct subtypes of cells |
| EP3472318B1 (en) * | 2016-07-08 | 2023-09-06 | Exuma Biotech Corp. | Methods and compositions for transducing lymphocytes and regulating the activity thereof |
| JP7483373B2 (ja) | 2016-07-29 | 2024-05-15 | ジュノー セラピューティクス インコーポレイテッド | 複製可能ウイルスの存在または非存在を評価するための方法 |
| WO2019077149A1 (en) * | 2017-10-20 | 2019-04-25 | Genethon | USE OF SYNCYTINE FOR TARGETING DRUG AND GENE ADMINISTRATION TO REGENERATE MUSCLE TISSUE |
| WO2019204666A1 (en) * | 2018-04-18 | 2019-10-24 | Oisin Biotechnologies, Inc. | Fusogenic lipid nanoparticles and methods for the manufacture and use thereof for the target cell-specific production of a therapeutic protein and for the treatment of a disease, condition, or disorder associated with a target cell |
| GB201807945D0 (en) * | 2018-05-16 | 2018-06-27 | Ospedale San Raffaele Srl | Vector production |
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2019
- 2019-11-14 SG SG11202105079QA patent/SG11202105079QA/en unknown
- 2019-11-14 CA CA3120082A patent/CA3120082A1/en active Pending
- 2019-11-14 CN CN201980088528.XA patent/CN113613633A/zh active Pending
- 2019-11-14 JP JP2021526394A patent/JP7676305B2/ja active Active
- 2019-11-14 US US17/293,842 patent/US20220008557A1/en active Pending
- 2019-11-14 KR KR1020217018175A patent/KR20210133948A/ko not_active Ceased
- 2019-11-14 AU AU2019378881A patent/AU2019378881A1/en active Pending
- 2019-11-14 EP EP19817064.9A patent/EP3880179A2/en active Pending
- 2019-11-14 WO PCT/US2019/061424 patent/WO2020102499A2/en not_active Ceased
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2021
- 2021-05-13 IL IL283179A patent/IL283179A/en unknown
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2025
- 2025-04-30 JP JP2025075701A patent/JP2025111747A/ja active Pending
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