CA2704383A1 - Rationally-designed single-chain meganucleases with non-palindromic recognition sequences - Google Patents
Rationally-designed single-chain meganucleases with non-palindromic recognition sequences Download PDFInfo
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- CA2704383A1 CA2704383A1 CA2704383A CA2704383A CA2704383A1 CA 2704383 A1 CA2704383 A1 CA 2704383A1 CA 2704383 A CA2704383 A CA 2704383A CA 2704383 A CA2704383 A CA 2704383A CA 2704383 A1 CA2704383 A1 CA 2704383A1
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- meganuclease
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| PCT/US2008/082072 WO2009059195A2 (en) | 2007-10-31 | 2008-10-31 | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
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| US8206965B2 (en) † | 2002-03-15 | 2012-06-26 | Cellectis S.A. | Hybrid and single chain meganucleases and use thereof |
| WO2009095742A1 (en) | 2008-01-31 | 2009-08-06 | Cellectis | New i-crei derived single-chain meganuclease and uses thereof |
| EP3578646A3 (en) | 2007-10-31 | 2020-03-18 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| WO2011064750A1 (en) | 2009-11-27 | 2011-06-03 | Basf Plant Science Company Gmbh | Chimeric endonucleases and uses thereof |
| AU2010325549B2 (en) | 2009-11-27 | 2017-04-20 | Basf Plant Science Company Gmbh | Optimized endonucleases and uses thereof |
| JP2013511979A (ja) | 2009-11-27 | 2013-04-11 | ビーエーエスエフ プラント サイエンス カンパニー ゲーエムベーハー | キメラエンドヌクレアーゼおよびその使用 |
| WO2011117249A1 (en) | 2010-03-22 | 2011-09-29 | Philip Morris Products S.A. | Modifying enzyme activity in plants |
| WO2012138901A1 (en) * | 2011-04-05 | 2012-10-11 | Cellectis Sa | Method for enhancing rare-cutting endonuclease efficiency and uses thereof |
| WO2012167192A2 (en) | 2011-06-01 | 2012-12-06 | Precision Biosciences, Inc. | Methods and products for producing engineered mammalian cell lines with amplified transgenes |
| EP2612918A1 (en) | 2012-01-06 | 2013-07-10 | BASF Plant Science Company GmbH | In planta recombination |
| WO2013166113A1 (en) * | 2012-05-04 | 2013-11-07 | E. I. Du Pont De Nemours And Company | Compositions and methods comprising sequences having meganuclease activity |
| AU2015229299A1 (en) | 2014-03-12 | 2016-09-22 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
| WO2016037161A2 (en) | 2014-09-07 | 2016-03-10 | Selecta Biosciences, Inc. | Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses |
| DK3289076T3 (da) | 2015-05-01 | 2022-01-17 | Prec Biosciences Inc | Præcis sletning af kromosomale sekvenser in vivo |
| AU2016263198C1 (en) | 2015-05-15 | 2023-10-05 | The General Hospital Corporation | Antagonistic anti-tumor necrosis factor receptor superfamily antibodies |
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| CA3033617A1 (en) | 2015-08-11 | 2017-02-16 | Anie Philip | Peptidic tgf-beta antagonists |
| EP4530354A3 (en) | 2015-09-08 | 2025-09-10 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| DK3359660T3 (en) | 2015-10-05 | 2020-02-17 | Prec Biosciences Inc | Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene |
| DK3359184T3 (da) | 2015-10-05 | 2020-06-15 | Prec Biosciences Inc | Genetisk modificerede celler, der omfatter et modificeret gen fra konstant alfaregion i human t-cellereceptor |
| ES2901000T3 (es) | 2015-12-23 | 2022-03-21 | Prec Biosciences Inc | Meganucleasas de ingeniería con secuencias de reconocimiento encontradas en el gen de la microglobulina beta-2 humana |
| WO2017142923A1 (en) * | 2016-02-16 | 2017-08-24 | Emendobio Inc. | Compositions and methods for promoting homology directed repair mediated gene editing |
| WO2017161001A1 (en) | 2016-03-15 | 2017-09-21 | Children's Medical Center Corporation | Methods and compositions relating to hematopoietic stem cell expansion |
| CA3017645C (en) | 2016-04-27 | 2021-07-13 | University Of Puerto Rico | 1,5-disubstituted 1,2,3-triazoles are inhibitors of rac/cdc42 gtpases |
| EP4019628A1 (en) | 2016-05-03 | 2022-06-29 | Precision Biosciences, Inc. | Engineered nucleases useful for treatment of hemophilia a |
| MX2018015798A (es) | 2016-06-22 | 2019-07-12 | Univ North Carolina State | Método para incrementar la eficiencia de uso de nitrógeno y/o la eficiencia de utilización de nitrógeno en plantas. |
| WO2018022619A1 (en) * | 2016-07-25 | 2018-02-01 | Bluebird Bio, Inc. | Bcl11a homing endonuclease variants, compositions, and methods of use |
| MA46059A (fr) | 2016-08-23 | 2019-07-03 | Bluebird Bio Inc | Variants de l'endonucléase homing tim3, compositions et procédés d'utilisation |
| ES2952525T3 (es) | 2016-09-08 | 2023-11-02 | 2Seventy Bio Inc | Variantes de endonucleasa homing PD-1, composiciones y métodos de uso |
| ES2811500T3 (es) | 2016-10-04 | 2021-03-12 | Prec Biosciences Inc | Dominios coestimuladores para su uso en células genéticamente modificadas |
| MA46535A (fr) | 2016-10-14 | 2019-08-21 | Prec Biosciences Inc | Méganucléases modifiées spécifiques de séquences de reconnaissance dans le génome du virus de l'hépatite b |
| EP3526334A4 (en) * | 2016-10-17 | 2020-09-30 | Bluebird Bio, Inc. | TGFBETA R2 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE |
| WO2018073393A2 (en) | 2016-10-19 | 2018-04-26 | Cellectis | Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy |
| TW201841916A (zh) | 2017-04-12 | 2018-12-01 | 美商麥珍塔治療學股份有限公司 | 芳香烴受體拮抗劑及其用途 |
| MX2019012452A (es) * | 2017-04-21 | 2020-08-03 | Prec Biosciences Inc | Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9. |
| WO2018201144A1 (en) | 2017-04-28 | 2018-11-01 | Precision Biosciences, Inc. | Methods for reducing dna-induced cytotoxicity and enhancing gene editing in primary cells |
| WO2018208837A1 (en) | 2017-05-08 | 2018-11-15 | Precision Biosciences, Inc. | Nucleic acid molecules encoding an engineered antigen receptor and an inhibitory nucleic acid molecule and methods of use thereof |
| KR102742817B1 (ko) * | 2017-05-25 | 2024-12-17 | 리제너론 파마슈티칼스 인코포레이티드 | Cblb 엔도뉴클레아제 변이체, 조성물 및 사용 방법 |
| BR112019027710A2 (pt) | 2017-06-23 | 2020-08-18 | University Of Kentucky Research Foundation | métodos de modulação do teor de alcaloides e do teor de uma nitrosamina específica do tabaco (tsna), uso de pelo menos um gene nic1 erf, método para produzir uma planta, planta modificada, material de propagação vegetal, uso de uma planta, folha colhida de uma planta, folha processada, material de tabaco curado, mistura de tabaco, produto da indústria do tabaco, produto de tabaco, uso de uma célula de tabaco, artigo de fumo combustível, uso de uma sequência de nucleotídeos, mutante de uma planta, progênie ou semente |
| CA3068465A1 (en) | 2017-06-30 | 2019-01-03 | Precision Biosciences, Inc. | Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene |
| WO2019027861A1 (en) | 2017-07-31 | 2019-02-07 | R. J. Reynolds Tobacco Company | METHODS AND COMPOSITIONS FOR EDITING VIRUS-BASED GENES IN PLANTS |
| US20200239544A1 (en) | 2017-10-03 | 2020-07-30 | Precision Biosciences, Inc. | Modified epidermal growth factor receptor peptides for use in genetically-modified cells |
| WO2019070974A1 (en) * | 2017-10-04 | 2019-04-11 | Bluebird Bio, Inc. | PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE |
| AU2018347583A1 (en) | 2017-10-13 | 2020-05-21 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector IgM responses |
| WO2019089826A1 (en) | 2017-10-31 | 2019-05-09 | Magenta Therapeutics Inc. | Compositions and methods for the expansion of hematopoietic stem and progenitor cells |
| CN111683669A (zh) | 2017-10-31 | 2020-09-18 | 美真达治疗公司 | 用于造血干细胞和祖细胞移植疗法的组合物和方法 |
| WO2019089913A1 (en) | 2017-11-01 | 2019-05-09 | Precision Biosciences, Inc. | Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a |
| WO2019113375A2 (en) | 2017-12-06 | 2019-06-13 | Magenta Therapeutics, Inc. | Dosing regimens for the mobilization of hematopoietic stem and progenitor cells |
| US20210002621A1 (en) * | 2017-12-20 | 2021-01-07 | Bluebird Bio, Inc. | Ctla4 homing endonuclease variants, compositions, and methods of use |
| WO2019136159A1 (en) | 2018-01-03 | 2019-07-11 | Magenta Therapeutics Inc. | Compositions and methods for the expansion of hematopoietic stem and progenitor cells and treatment of inherited metabolic disorders |
| WO2019140009A1 (en) | 2018-01-09 | 2019-07-18 | Cibus Us Llc | Shatterproof genes and mutations |
| US12178908B2 (en) | 2018-02-26 | 2024-12-31 | AnTolRx, Inc. | Tolerogenic liposomes and methods of use thereof |
| AU2019247403B2 (en) | 2018-04-04 | 2025-04-10 | Cibus Europe, B.V. | FAD2 genes and mutations |
| IT201800004253A1 (it) | 2018-04-05 | 2019-10-05 | Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria. | |
| TW201945388A (zh) | 2018-04-12 | 2019-12-01 | 美商精密生物科學公司 | 對b型肝炎病毒基因體中之識別序列具有特異性之最佳化之經工程化巨核酸酶 |
| US11786554B2 (en) | 2018-04-12 | 2023-10-17 | Precision Biosciences, Inc. | Optimized engineered nucleases having specificity for the human T cell receptor alpha constant region gene |
| EP3784790A4 (en) * | 2018-04-27 | 2022-02-09 | Seattle Children's Hospital d/b/a Seattle Children's Research Institute | BRUTON TYROSINE KINASE HOMING ENDONUCLEAR VARIANTS, COMPOSITIONS AND METHOD OF USE |
| US12060419B2 (en) | 2018-06-14 | 2024-08-13 | Regeneron Pharmaceuticals, Inc. | CD79A chimeric antigen receptors |
| GB201812603D0 (en) | 2018-08-02 | 2018-09-19 | British American Tobacco Investments Ltd | Method |
| WO2020051207A2 (en) | 2018-09-04 | 2020-03-12 | Magenta Therapeutics Inc. | Aryl hydrocarbon receptor antagonists and methods of use |
| WO2020072059A1 (en) | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| GB201817971D0 (en) | 2018-11-02 | 2018-12-19 | British American Tobacco Investments Ltd | Method |
| GB201818715D0 (en) | 2018-11-16 | 2019-01-02 | British American Tobacco Investments Ltd | Method |
| AU2019395334A1 (en) | 2018-12-10 | 2021-06-24 | Regeneron Pharmaceuticals, Inc. | PDCD-1 homing endonuclease variants |
| US12404500B2 (en) | 2018-12-10 | 2025-09-02 | Novo Nordisk A/S | Homing endonuclease variants |
| EP3898661A1 (en) | 2018-12-21 | 2021-10-27 | Precision BioSciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| GB201900940D0 (en) | 2019-01-23 | 2019-03-13 | British American Tobacco Investments Ltd | Method |
| KR20240122580A (ko) | 2019-04-03 | 2024-08-12 | 프리시젼 바이오사이언시스 인코포레이티드 | 마이크로RNA-적응 shRNA(shRNAmiR)를 포함하는 유전자-변형 면역 세포 |
| US20220204994A1 (en) | 2019-04-05 | 2022-06-30 | Precision Biosciences, Inc. | Methods of preparing populations of genetically-modified immune cells |
| US20220195407A1 (en) | 2019-05-07 | 2022-06-23 | Precision Biosciences, Inc. | Optimization of engineered meganucleases for recognition sequences |
| GB201906768D0 (en) | 2019-05-14 | 2019-06-26 | British American Tobacco Investments Ltd | Method |
| WO2020243261A1 (en) | 2019-05-28 | 2020-12-03 | Selecta Biosciences, Inc. | Methods and compositions for attenuated anti-viral transfer vector immune response |
| GB201909563D0 (en) | 2019-07-03 | 2019-08-14 | British American Tobacco Investments Ltd | Method |
| GB201909562D0 (en) | 2019-07-03 | 2019-08-14 | British American Tobacco Investments Ltd | Method |
| CA3148179A1 (en) | 2019-08-20 | 2021-02-25 | Bruce J. Mccreedy Jr. | Lymphodepletion dosing regimens for cellular immunotherapies |
| US20220411479A1 (en) | 2019-10-30 | 2022-12-29 | Precision Biosciences, Inc. | Cd20 chimeric antigen receptors and methods of use for immunotherapy |
| US20220401481A1 (en) | 2019-11-01 | 2022-12-22 | Magenta Therapeutics, Inc. | Dosing regimens for the mobilization of hematopoietic stem and progenitor cells |
| DK4069729T3 (da) | 2019-12-06 | 2025-04-07 | Prec Biosciences Inc | Optimerede, modificerede meganukleaser med specificitet for en genkendelsessekvens i hepatitis b-virusgenomet |
| EP3835309A1 (en) | 2019-12-13 | 2021-06-16 | KWS SAAT SE & Co. KGaA | Method for increasing cold or frost tolerance in a plant |
| WO2021158915A1 (en) | 2020-02-06 | 2021-08-12 | Precision Biosciences, Inc. | Recombinant adeno-associated virus compositions and methods for producing and using the same |
| US20230159945A1 (en) | 2020-04-09 | 2023-05-25 | R.J. Reynolds Tobacco Company | Method |
| AU2021263754A1 (en) | 2020-04-27 | 2022-12-01 | Ensoma, Inc. | Methods and compositions for transducing hematopoietic stem and progenitor cells in vivo |
| US20230183664A1 (en) | 2020-05-11 | 2023-06-15 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| US20230193230A1 (en) | 2020-05-12 | 2023-06-22 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using improved engineered meganucleases |
| EP4162059A1 (en) | 2020-05-12 | 2023-04-12 | The Trustees of The University of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20260028412A1 (en) | 2020-08-10 | 2026-01-29 | Precision Biosciences, Inc. | Antibodies and fragments specific for b-cell maturation antigen and uses thereof |
| AU2021329403A1 (en) | 2020-08-21 | 2023-05-04 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for a recognition sequence in the transthyretin gene |
| WO2022076547A1 (en) | 2020-10-07 | 2022-04-14 | Precision Biosciences, Inc. | Lipid nanoparticle compositions |
| CN117042787A (zh) | 2020-10-29 | 2023-11-10 | 宾夕法尼亚州大学信托人 | Aav衣壳和含有aav衣壳的组合物 |
| WO2022104062A1 (en) | 2020-11-12 | 2022-05-19 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| WO2022150616A1 (en) | 2021-01-08 | 2022-07-14 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for a recognition sequence in the hydroxyacid oxidase 1 gene |
| WO2022165111A1 (en) | 2021-01-28 | 2022-08-04 | Precision Biosciences, Inc. | Modulation of tgf beta signaling in genetically-modified eukaryotic cells |
| EP4308694A1 (en) | 2021-03-16 | 2024-01-24 | Magenta Therapeutics, Inc. | Dosing regimens for hematopoietic stem cell mobilization for stem cell transplants in multiple myeloma patients |
| US20240141311A1 (en) | 2021-04-22 | 2024-05-02 | North Carolina State University | Compositions and methods for generating male sterile plants |
| CA3173051A1 (en) | 2021-04-22 | 2022-10-22 | Precision Biosciences, Inc. | Engineered meganucleases that target human mitochondrial genomes |
| JP2024514939A (ja) | 2021-04-22 | 2024-04-03 | プレシジョン バイオサイエンシズ，インク． | ヒトミトコンドリアゲノムを標的化する遺伝子操作メガヌクレアーゼ |
| WO2022232267A1 (en) | 2021-04-27 | 2022-11-03 | The Trustees Of The University Of Pennsylvania | Porcine-derived adeno-associated virus capsids and uses thereof |
| WO2023064367A1 (en) | 2021-10-12 | 2023-04-20 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector igm responses |
| MX2024004840A (es) | 2021-10-19 | 2024-07-09 | Prec Biosciences Inc | Método de edición de genes para tratar la deficiencia de alfa-1 antitripsina (aat). |
| US20240408235A1 (en) | 2021-10-19 | 2024-12-12 | Precision Biosciences, Inc. | Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20250215443A1 (en) | 2022-02-03 | 2025-07-03 | Nicoventures Trading Limited | Method of modulating alkaloid content in tobacco plants |
| WO2023148476A1 (en) | 2022-02-03 | 2023-08-10 | Nicoventures Trading Limited | Method of modulating alkaloid content in tobacco plants |
| EP4472405A1 (en) | 2022-02-04 | 2024-12-11 | Nicoventures Trading Limited | Method of modulating alkaloid content in tobacco plants |
| WO2023172624A1 (en) | 2022-03-09 | 2023-09-14 | Selecta Biosciences, Inc. | Immunosuppressants in combination with anti-igm agents and related dosing |
| GB202205149D0 (en) | 2022-04-07 | 2022-05-25 | Nicoventures Trading Ltd | Method |
| GB202205148D0 (en) | 2022-04-07 | 2022-05-25 | Nicoventures Trading Ltd | Method |
| GB202205562D0 (en) | 2022-04-14 | 2022-06-01 | Nicoventures Trading Ltd | Method |
| GB202205561D0 (en) | 2022-04-14 | 2022-06-01 | Nicoventures Trading Ltd | Method |
| GB202206109D0 (en) | 2022-04-27 | 2022-06-08 | Nicoventures Trading Ltd | Method |
| GB202206107D0 (en) | 2022-04-27 | 2022-06-08 | Nicoventures Trading Ltd | Method |
| WO2023215498A2 (en) | 2022-05-05 | 2023-11-09 | Modernatx, Inc. | Compositions and methods for cd28 antagonism |
| US20260021205A1 (en) | 2022-07-15 | 2026-01-22 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| AU2023342734A1 (en) | 2022-09-16 | 2025-03-27 | King's College London | Compositions and methods for treating neurological diseases |
| WO2024097639A1 (en) | 2022-10-31 | 2024-05-10 | Modernatx, Inc. | Hsa-binding antibodies and binding proteins and uses thereof |
| WO2024118866A1 (en) | 2022-12-01 | 2024-06-06 | Modernatx, Inc. | Gpc3-specific antibodies, binding domains, and related proteins and uses thereof |
| WO2024126696A1 (en) | 2022-12-14 | 2024-06-20 | King's College London | Compositions and methods for treating neurological diseases |
| AU2024206812A1 (en) | 2023-01-05 | 2025-08-14 | Precision Biosciences, Inc. | Optimized engineered meganucleases having specificity for the human t cell receptor alpha constant region gene |
| GB202300905D0 (en) | 2023-01-20 | 2023-03-08 | Nicoventures Trading Ltd | Method |
| WO2024161022A2 (en) | 2023-02-03 | 2024-08-08 | King's College London | Compositions and methods for treating neurological diseases |
| JP2026504479A (ja) | 2023-02-06 | 2026-02-05 | ブルーロック セラピューティクス エルピー | デグロン融合タンパク質並びにその産生及び使用の方法 |
| GB202303077D0 (en) | 2023-03-02 | 2023-04-19 | Univ Oslo | Brassica plants with improved seed retention |
| TW202448928A (zh) | 2023-03-02 | 2024-12-16 | 美商艾羅伊治療公司 | 抗cd22抗體及其用途 |
| CN121311497A (zh) | 2023-04-14 | 2026-01-09 | 精密生物科学公司 | 肌肉特异性表达盒 |
| WO2024216116A1 (en) | 2023-04-14 | 2024-10-17 | Precision Biosciences, Inc. | Muscle-specific expression cassettes |
| WO2024236547A1 (en) | 2023-05-18 | 2024-11-21 | Inceptor Bio, Llc | Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof |
| WO2025046513A1 (en) | 2023-08-29 | 2025-03-06 | Inceptor Bio, Llc | Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof |
| WO2025083169A1 (en) | 2023-10-17 | 2025-04-24 | Tenpoint Therapeutics Limited | Combination of a vegf inhibitor and a complement pathway inhibitor for treating ocular disorders |
| WO2025101946A1 (en) * | 2023-11-08 | 2025-05-15 | Precision Biosciences, Inc. | Polypeptide linkers for use in engineered meganucleases |
| WO2025126153A2 (en) | 2023-12-14 | 2025-06-19 | Aviadobio Ltd. | Compositions and methods for treating sod1-mediated neurological diseases |
| WO2025233894A1 (en) | 2024-05-09 | 2025-11-13 | University Of Miami | Engineered meganucleases that target human mitochondrial genomes |
| WO2026006492A2 (en) | 2024-06-25 | 2026-01-02 | Ypsilon Therapeutics, Inc. | Anti-prame/hla-a2 antibodies and uses thereof |
| WO2026006495A1 (en) | 2024-06-25 | 2026-01-02 | Alloy Therapeutics, Inc. | Anti-wt1/hla-a2 antibody and uses thereof |
| WO2026028165A1 (en) | 2024-08-01 | 2026-02-05 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the c9orf72 gene |
| WO2026047626A1 (en) | 2024-08-30 | 2026-03-05 | Inceptor Bio, Llc | Compositions and methods involving immune cells engineered for binding bispecific antibodies for use in cell therapy |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE122007000007I1 (de) | 1986-04-09 | 2007-05-16 | Genzyme Corp | Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern |
| US4873316A (en) | 1987-06-23 | 1989-10-10 | Biogen, Inc. | Isolation of exogenous recombinant proteins from the milk of transgenic mammals |
| US6156304A (en) | 1990-12-20 | 2000-12-05 | University Of Pittsburgh Of The Commonwealth System Of Higher Education | Gene transfer for studying and treating a connective tissue of a mammalian host |
| US5384253A (en) | 1990-12-28 | 1995-01-24 | Dekalb Genetics Corporation | Genetic transformation of maize cells by electroporation of cells pretreated with pectin degrading enzymes |
| US5997861A (en) | 1994-10-31 | 1999-12-07 | Burstein Laboratories, Inc. | Antiviral supramolecules containing target-binding molecules and therapeutic molecules bound to spectrin |
| US6056973A (en) | 1996-10-11 | 2000-05-02 | Sequus Pharmaceuticals, Inc. | Therapeutic liposome composition and method of preparation |
| US6060082A (en) | 1997-04-18 | 2000-05-09 | Massachusetts Institute Of Technology | Polymerized liposomes targeted to M cells and useful for oral or mucosal drug delivery |
| US6511676B1 (en) | 1999-11-05 | 2003-01-28 | Teni Boulikas | Therapy for human cancers using cisplatin and other drugs or genes encapsulated into liposomes |
| US6593308B2 (en) | 1999-12-03 | 2003-07-15 | The Regents Of The University Of California | Targeted drug delivery with a hyaluronan ligand |
| AU2002317771A1 (en) * | 2001-06-05 | 2002-12-16 | Cellectis | Methods for modifying the cpg content of polynucleotides |
| EP1483298A4 (en) * | 2002-02-11 | 2007-03-14 | Zymogenetics Inc | MATERIALS AND METHODS FOR PREPARING DIMERED GROWTH FACTORS |
| US8206965B2 (en) * | 2002-03-15 | 2012-06-26 | Cellectis S.A. | Hybrid and single chain meganucleases and use thereof |
| WO2009095742A1 (en) | 2008-01-31 | 2009-08-06 | Cellectis | New i-crei derived single-chain meganuclease and uses thereof |
| WO2004067753A2 (en) * | 2003-01-28 | 2004-08-12 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof. |
| JP4095622B2 (ja) * | 2004-03-31 | 2008-06-04 | キヤノン株式会社 | 金結合性複合タンパク質 |
| EP1591521A1 (en) * | 2004-04-30 | 2005-11-02 | Cellectis | I-Dmo I derivatives with enhanced activity at 37 degrees C and use thereof |
| ES2347684T3 (es) * | 2005-03-15 | 2010-11-03 | Cellectis | Variantes de la meganucleasa i-crei con especificidad modifica, metodo de preparacion y usos de las mismas. |
| WO2006097784A1 (en) | 2005-03-15 | 2006-09-21 | Cellectis | I-crei meganuclease variants with modified specificity, method of preparation and uses thereof |
| WO2007034262A1 (en) | 2005-09-19 | 2007-03-29 | Cellectis | Heterodimeric meganucleases and use thereof |
| EP1764414A1 (en) * | 2005-09-17 | 2007-03-21 | Icon Genetics AG | Plant viral particles comprising a plurality of fusion proteins consisting of a plant viral coat protein, a peptide linker and a recombinant protein and use of such plant viral particles for protein purification |
| CA2626262C (en) * | 2005-10-18 | 2015-09-08 | Homme W. Hellinga | Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity |
| WO2007060495A1 (en) | 2005-10-25 | 2007-05-31 | Cellectis | I-crei homing endonuclease variants having novel cleavage specificity and use thereof |
| WO2007049095A1 (en) | 2005-10-25 | 2007-05-03 | Cellectis | Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof |
| WO2007065098A2 (en) * | 2005-11-29 | 2007-06-07 | Virginia Commonwealth University | Polyvalent chimeric ospc vaccinogen and diagnostic antigen |
| WO2007093836A1 (en) | 2006-02-13 | 2007-08-23 | Cellectis | Meganuclease variants cleaving a dna target sequence from a xp gene and uses thereof |
| GB0603099D0 (en) * | 2006-02-16 | 2006-03-29 | Lontra Environmental Technolog | Rotary piston and cylinder devices |
| WO2008010009A1 (en) | 2006-07-18 | 2008-01-24 | Cellectis | Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof |
| BRPI0718747A2 (pt) | 2006-11-14 | 2013-12-03 | Cellectis | Variantes meganuclease que clavam uma ou sequência alvo de dna a partir do gene hprt e usos das mesmas. |
| WO2008059317A1 (en) | 2006-11-14 | 2008-05-22 | Cellectis | Meganuclease variants cleaving a dna target sequence from the hprt gene and uses thereof |
| WO2008093152A1 (en) | 2007-02-01 | 2008-08-07 | Cellectis | Obligate heterodimer meganucleases and uses thereof |
| CN101679959A (zh) | 2007-02-19 | 2010-03-24 | 赛莱克蒂斯公司 | 具有新的底物特异性的laglidadg归巢核酸内切酶变体及其用途 |
| WO2008102199A1 (en) * | 2007-02-20 | 2008-08-28 | Cellectis | Meganuclease variants cleaving a dna target sequence from the beta-2-microglobulin gene and uses thereof |
| WO2008149176A1 (en) * | 2007-06-06 | 2008-12-11 | Cellectis | Meganuclease variants cleaving a dna target sequence from the mouse rosa26 locus and uses thereof |
| WO2009006297A2 (en) | 2007-06-29 | 2009-01-08 | Pioneer Hi-Bred International, Inc. | Methods for altering the genome of a monocot plant cell |
| EP3578646A3 (en) | 2007-10-31 | 2020-03-18 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| WO2009074842A1 (en) | 2007-12-13 | 2009-06-18 | Cellectis | Improved chimeric meganuclease enzymes and uses thereof |
-
2008
- 2008-10-31 EP EP19167904.2A patent/EP3578646A3/en active Pending
- 2008-10-31 EP EP08845549.8A patent/EP2215223B1/en active Active
- 2008-10-31 DK DK13165733.0T patent/DK2660317T3/en active
- 2008-10-31 ES ES13165733.0T patent/ES2575412T3/es active Active
- 2008-10-31 WO PCT/US2008/082072 patent/WO2009059195A2/en not_active Ceased
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