JP2018531023A5 - - Google Patents

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JP2018531023A5
JP2018531023A5 JP2018520155A JP2018520155A JP2018531023A5 JP 2018531023 A5 JP2018531023 A5 JP 2018531023A5 JP 2018520155 A JP2018520155 A JP 2018520155A JP 2018520155 A JP2018520155 A JP 2018520155A JP 2018531023 A5 JP2018531023 A5 JP 2018531023A5
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virus
derived
nucleic acid
vector particles
cas
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JP2018531023A (ja
JP7059179B2 (ja
JP2018531023A6 (ja
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Priority claimed from PCT/EP2016/075289 external-priority patent/WO2017068077A1/en
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Priority to JP2021212300A priority Critical patent/JP7621930B2/ja
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Priority to JP2025005188A priority patent/JP2025063185A/ja
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JP2018520155A 2015-10-20 2016-10-20 遺伝子操作のための方法及び製品 Active JP7059179B2 (ja)

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Application Number Priority Date Filing Date Title
JP2021212300A JP7621930B2 (ja) 2015-10-20 2021-12-27 遺伝子操作のための方法及び製品
JP2025005188A JP2025063185A (ja) 2015-10-20 2025-01-15 遺伝子操作のための方法及び製品

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Application Number Priority Date Filing Date Title
EP15306678 2015-10-20
EP15306678.2 2015-10-20
PCT/EP2016/075289 WO2017068077A1 (en) 2015-10-20 2016-10-20 Methods and products for genetic engineering

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JP2018531023A JP2018531023A (ja) 2018-10-25
JP2018531023A6 JP2018531023A6 (ja) 2018-12-13
JP2018531023A5 true JP2018531023A5 (https=) 2019-11-28
JP7059179B2 JP7059179B2 (ja) 2022-04-25

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JP2021212300A Active JP7621930B2 (ja) 2015-10-20 2021-12-27 遺伝子操作のための方法及び製品
JP2025005188A Pending JP2025063185A (ja) 2015-10-20 2025-01-15 遺伝子操作のための方法及び製品

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JP2025005188A Pending JP2025063185A (ja) 2015-10-20 2025-01-15 遺伝子操作のための方法及び製品

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US (6) US10968253B2 (https=)
JP (3) JP7059179B2 (https=)
WO (1) WO2017068077A1 (https=)

Families Citing this family (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP6890831B2 (ja) 2015-07-08 2021-06-18 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド Ｈｉｖ予備免疫化および免疫療法
WO2017068077A1 (en) 2015-10-20 2017-04-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
DK3402483T3 (da) 2016-01-15 2024-01-02 American Gene Tech Int Inc Fremgangsmåder og sammensætninger til aktivering af gamma-delta-T-celler
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
US10888613B2 (en) 2016-02-08 2021-01-12 American Gene Technologies International Inc. Method of producing cells resistant to HIV infection
DK3426777T3 (da) 2016-03-09 2022-04-25 American Gene Tech Int Inc Kombinationsvektorer og fremgangsmåder til behandling af cancer
US11976292B2 (en) 2016-06-08 2024-05-07 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
IL284348B2 (en) 2016-07-08 2025-06-01 American Gene Tech Int Inc Hiv pre-immunization and immunotherapy
JP7176756B2 (ja) 2016-07-21 2022-11-22 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド パーキンソン病を処置するためのウイルスベクター
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
JP2020510038A (ja) 2017-03-09 2020-04-02 プレジデント アンド フェローズ オブ ハーバード カレッジ がんワクチン
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
CA3057142A1 (en) 2017-04-03 2018-10-11 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
US11643671B2 (en) * 2017-04-21 2023-05-09 Seattle Children's Hospital Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CA3064442A1 (en) 2017-06-16 2018-12-20 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019121491A1 (en) * 2017-12-22 2019-06-27 Asml Netherlands B.V. Patterning process improvement involving optical aberration
JP2019198250A (ja) * 2018-05-14 2019-11-21 石原産業株式会社 ゲノム編集用キット
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020012335A1 (en) * 2018-07-10 2020-01-16 Alia Therapeutics S.R.L. Vesicles for traceless delivery of guide rna molecules and/or guide rna molecule/rna-guided nuclease complex(es) and a production method thereof
WO2020013317A1 (ja) * 2018-07-13 2020-01-16 国立大学法人京都大学 ウイルス様粒子及びその使用
KR20200011899A (ko) * 2018-07-25 2020-02-04 주식회사 툴젠 인위적인 유전자 조작을 통한 자가면역질환 치료
JP7344300B2 (ja) 2018-09-18 2023-09-13 ブイエヌブイ ニューコ インク． Ａｒｃベースのカプシドおよびその使用
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US11352646B2 (en) 2018-11-05 2022-06-07 American Gene Technologies International Inc. Vector system for expressing regulatory RNA
US11884925B2 (en) 2018-11-08 2024-01-30 Arizona Board Of Regents On Behalf Of Arizona State University Synthetic immunomodulation with a CRISPR super-repressor in vivo
WO2020102709A1 (en) * 2018-11-16 2020-05-22 The Regents Of The University Of California Compositions and methods for delivering crispr/cas effector polypeptides
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20220194990A1 (en) * 2019-01-31 2022-06-23 Board Of Regents Of The University Of Nebraska Virus-like particles and methods of use 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
US20220280644A1 (en) 2019-08-09 2022-09-08 University Of Utah Research Foundation "TRPswitch" - A STEP FUNCTION CHEMO-OPTOGENETIC LIGAND
AU2020341454A1 (en) 2019-09-03 2022-03-10 Sana Biotechnology, Inc. CD24-associated particles and related methods and uses thereof
US20220348925A1 (en) * 2019-09-09 2022-11-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
CA3153700A1 (en) * 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy
CN114616336B (zh) * 2019-09-20 2025-11-14 博德研究所 用于将货物递送至靶细胞的组合物和方法
WO2021069593A1 (en) 2019-10-09 2021-04-15 INSERM (Institut National de la Santé et de la Recherche Médicale) T cells modified to express mutated cxcr4 or partially deleted and uses thereof
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3159316A1 (en) 2019-12-06 2021-06-10 Benjamin OAKES Compositions and methods for the targeting of rhodopsin
AU2020398658A1 (en) * 2019-12-06 2022-07-07 Scribe Therapeutics Inc. Particle delivery systems
EP4069846A1 (en) * 2019-12-07 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of htt
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
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
US12565647B2 (en) 2021-05-28 2026-03-03 The Regents Of The University Of California Compositions and methods for targeted delivery of CRISPR-Cas effector polypeptides and transgenes
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
WO2022261150A2 (en) * 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
JP2024534523A (ja) 2021-09-21 2024-09-20 スクライブ・セラピューティクス・インコーポレイテッド 操作されたｃａｓｘリプレッサー系
US20250230511A1 (en) 2021-10-29 2025-07-17 Sana Biotechnology, Inc. Methods and reagents for amplifying viral vector nucleic acid products
EP4441073A2 (en) * 2021-12-03 2024-10-09 The Broad Institute, Inc. Self-assembling virus-like particles for delivery of nucleic acid programmable fusion proteins and methods of making and using same
US20250223584A2 (en) * 2021-12-03 2025-07-10 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
WO2023102538A1 (en) * 2021-12-03 2023-06-08 The Broad Institute, Inc. Self-assembling virus-like particles for delivery of prime editors and methods of making and using same
US20250059560A1 (en) 2021-12-16 2025-02-20 Sana Biotechnology, Inc. Methods and systems of particle production
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
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
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
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
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
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
WO2024197008A2 (en) * 2023-03-20 2024-09-26 The Broad Institute, Inc. Nuclease-guided non-ltr retrotransposons and uses thereof
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
WO2025137322A1 (en) * 2023-12-20 2025-06-26 The Board Of Trustees Of The Leland Stanford Junior University Secreted particle information transfer (spit) system for genetic engineering
WO2025151838A1 (en) 2024-01-12 2025-07-17 Sana Biotechnology, Inc. Safety switches to control in vitro and in vivo proliferation of cell therapy products
WO2025184529A1 (en) 2024-03-01 2025-09-04 Sana Biotechnology, Inc. Viral particles with fusogen display and related compositions and methods
CN118547011A (zh) * 2024-06-05 2024-08-27 广州达博生物制品有限公司 一种稳定高表达人源悬浮细胞系的构建方法
WO2025262311A1 (en) 2024-06-21 2025-12-26 Institut National De La Sante Et De La Recherche Medicale Methods and products for prime editing genetic engineering
WO2025262314A1 (en) 2024-06-21 2025-12-26 Institut National De La Sante Et De La Recherche Medicale Methods and products for prime editing genetic engineering

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5989886A (en) * 1991-01-02 1999-11-23 The Johns Hopkins University Method for the inhibition and prevention of viral replication using fusions of a virus protein and a destructive enzyme
GB9227068D0 (en) 1992-12-29 1993-02-24 British Bio Technology Novel proteinaceous particles
FR2716459B1 (fr) 1994-02-22 1996-05-10 Univ Paris Curie Système hôte-vecteur utilisable en thérapie génique.
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
WO2000039302A2 (en) * 1998-12-31 2000-07-06 Chiron Corporation Improved expression of hiv polypeptides and production of virus-like particles
US9296790B2 (en) * 2008-10-03 2016-03-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Methods and compositions for protein delivery
AU2009319979B2 (en) * 2008-11-03 2016-10-20 Takeda Vaccines, Inc. Improved methods for isolating enveloped virus-based VLPs free of infectious agents
DE102010025907A1 (de) 2010-07-02 2012-01-05 Robert Bosch Gmbh Wellenenergiewandler zur Umwandlung von kinetischer Energie in elektrische Energie
ES2874233T3 (es) 2011-11-11 2021-11-04 Variation Biotechnologies Inc Composiciones y métodos para el tratamiento de citomegalovirus
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
AU2013359262C1 (en) 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
WO2014131833A1 (en) 2013-02-27 2014-09-04 Helmholtz Zentrum München Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Gene editing in the oocyte by cas9 nucleases
US9593356B2 (en) 2013-06-11 2017-03-14 Takara Bio Usa, Inc. Protein enriched microvesicles and methods of making and using the same
SMT202100691T1 (it) * 2013-07-11 2022-01-10 Modernatx Inc Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso
EP3079473B1 (en) 2013-12-12 2020-11-11 Dow AgroSciences LLC Selective weed control with halauxifen
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
AU2014361781B2 (en) * 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
EP3149171A1 (en) * 2014-05-30 2017-04-05 The Board of Trustees of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
AU2015311704B2 (en) * 2014-09-07 2021-12-09 Selecta Biosciences, Inc. Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
CN107406838A (zh) * 2014-11-06 2017-11-28 纳幕尔杜邦公司 Rna引导的内切核酸酶向细胞中的肽介导的递送
GB2560250A (en) 2015-06-30 2018-09-05 Ethris Gmbh ATP-Binding cassette family coding polyribonucleotides and formulations thereof
WO2017068077A1 (en) 2015-10-20 2017-04-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
US10316295B2 (en) * 2015-12-17 2019-06-11 The Penn State Research Foundation Paramyxovirus virus-like particles as protein delivery vehicles

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