JP2022534809A5 - - Google Patents

Info

Publication number
JP2022534809A5
JP2022534809A5 JP2021572556A JP2021572556A JP2022534809A5 JP 2022534809 A5 JP2022534809 A5 JP 2022534809A5 JP 2021572556 A JP2021572556 A JP 2021572556A JP 2021572556 A JP2021572556 A JP 2021572556A JP 2022534809 A5 JP2022534809 A5 JP 2022534809A5
Authority
JP
Japan
Prior art keywords
sequence
improved
grna
protein
casx
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.)
Granted
Application number
JP2021572556A
Other languages
English (en)
Japanese (ja)
Other versions
JP7744011B2 (ja
JP2022534809A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/036505 external-priority patent/WO2020247882A1/en
Publication of JP2022534809A publication Critical patent/JP2022534809A/ja
Publication of JP2022534809A5 publication Critical patent/JP2022534809A5/ja
Priority to JP2025147107A priority Critical patent/JP2025183286A/ja
Application granted granted Critical
Publication of JP7744011B2 publication Critical patent/JP7744011B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021572556A 2019-06-07 2020-06-05 操作されたcasxシステム Active JP7744011B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025147107A JP2025183286A (ja) 2019-06-07 2025-09-04 操作されたcasxシステム

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962858750P 2019-06-07 2019-06-07
US62/858,750 2019-06-07
US201962944892P 2019-12-06 2019-12-06
US62/944,892 2019-12-06
US202063030838P 2020-05-27 2020-05-27
US63/030,838 2020-05-27
PCT/US2020/036505 WO2020247882A1 (en) 2019-06-07 2020-06-05 Engineered casx systems

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025147107A Division JP2025183286A (ja) 2019-06-07 2025-09-04 操作されたcasxシステム

Publications (3)

Publication Number Publication Date
JP2022534809A JP2022534809A (ja) 2022-08-03
JP2022534809A5 true JP2022534809A5 (enExample) 2023-06-12
JP7744011B2 JP7744011B2 (ja) 2025-09-25

Family

ID=71899867

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021572556A Active JP7744011B2 (ja) 2019-06-07 2020-06-05 操作されたcasxシステム
JP2025147107A Pending JP2025183286A (ja) 2019-06-07 2025-09-04 操作されたcasxシステム

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025147107A Pending JP2025183286A (ja) 2019-06-07 2025-09-04 操作されたcasxシステム

Country Status (17)

Country Link
US (5) US11560555B2 (enExample)
EP (1) EP3980533A1 (enExample)
JP (2) JP7744011B2 (enExample)
KR (1) KR20220032050A (enExample)
CN (1) CN114375334B (enExample)
AU (1) AU2020289591B2 (enExample)
BR (1) BR112021024288A2 (enExample)
CA (1) CA3142883A1 (enExample)
CL (1) CL2021003233A1 (enExample)
GB (1) GB2600274B (enExample)
IL (1) IL288738A (enExample)
MX (1) MX2021015058A (enExample)
PE (1) PE20220256A1 (enExample)
PH (1) PH12021553076A1 (enExample)
SG (1) SG11202113253SA (enExample)
TW (1) TW202113074A (enExample)
WO (1) WO2020247882A1 (enExample)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3841205A4 (en) * 2018-08-22 2022-08-17 The Regents of The University of California VARIANT TYPE V CRISPR/CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF
SG11202113253SA (en) 2019-06-07 2021-12-30 Scribe Therapeutics Inc Engineered casx systems
US12551560B2 (en) 2019-09-09 2026-02-17 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy
EP4028522A1 (en) 2019-09-09 2022-07-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
WO2021113763A1 (en) 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
JP2023510352A (ja) * 2020-01-10 2023-03-13 スクライブ・セラピューティクス・インコーポレイテッド Pcsk9の標的化のための組成物および方法
MX2022011460A (es) 2020-03-18 2022-12-15 Scribe Therapeutics Inc Composiciones y métodos para el direccionamiento de c9orf72.
GB202010075D0 (en) * 2020-07-01 2020-08-12 Imp College Innovations Ltd Therapeutic nucleic acids, peptides and uses
WO2022119957A1 (en) 2020-12-02 2022-06-09 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
AU2021392719B2 (en) 2020-12-03 2025-08-14 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
US20240100185A1 (en) 2020-12-03 2024-03-28 Scribe Therapeutics Inc. Compositions and methods for the targeting of ptbp1
KR20230129395A (ko) 2020-12-09 2023-09-08 스크라이브 테라퓨틱스 인크. 유전자 편집을 위한 aav 벡터
AU2022234760A1 (en) 2021-03-09 2023-09-14 Arbor Biotechnologies, Inc. Compositions comprising a variant polypeptide and uses thereof
AU2022284804A1 (en) 2021-06-01 2023-12-07 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof
WO2022261150A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
IL311610A (en) * 2021-09-21 2024-05-01 Scribe Therapeutics Inc Engineered CASX suppressor systems
EP4405490A2 (en) 2021-09-23 2024-07-31 Scribe Therapeutics Inc. Self-inactivating vectors for gene editing
CN116497002A (zh) * 2022-01-19 2023-07-28 中国科学院动物研究所 工程化的CasX核酸酶、效应蛋白及其用途
CA3257824A1 (en) 2022-06-02 2023-12-07 Scribe Therapeutics Inc. MODIFIED TYPE V, CLASS 2 CRISPR SYSTEMS
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
EP4536819A1 (en) 2022-06-07 2025-04-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
CN119998446A (zh) * 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
EP4536286A1 (en) 2022-06-08 2025-04-16 Scribe Therapeutics Inc. Aav vectors for gene editing
WO2023240157A2 (en) 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
US20260139236A1 (en) * 2022-10-11 2026-05-21 The Broad Institute, Inc. Reprogramable tnpb polypeptides and use thereof
US20260232844A1 (en) 2023-02-15 2026-08-13 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript
WO2024192291A1 (en) 2023-03-15 2024-09-19 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2024206620A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Messenger rna encoding casx
WO2024206676A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa
WO2024206555A1 (en) * 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
TW202444914A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 抑制子融合蛋白系統
CN116926043B (zh) * 2023-04-28 2024-09-06 新乡医学院第三附属医院 一种dCasX蛋白核酸酶、重组载体及其应用
WO2024228975A2 (en) * 2023-05-02 2024-11-07 Inari Agriculture Technology, Inc. Ribozyme sequences for processing rna
JP2026523341A (ja) 2023-05-15 2026-07-14 エヌクロマ・バイオ,インコーポレーテッド Hbv遺伝子発現のエピジェネティック調節のための組成物および方法
EP4729547A1 (en) * 2023-06-16 2026-04-22 VCGT Inc. Zinc finger protein, zinc finger nuclease, vector, and genome editing method using said substances
EP4771154A2 (en) 2023-09-01 2026-07-08 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025072293A1 (en) 2023-09-27 2025-04-03 Scribe Therapeutics Inc. Optimized mrnas encoding casx proteins
CN117296799B (zh) * 2023-11-28 2024-02-02 四川省医学科学院·四川省人民医院 一种视网膜色素变性疾病模型的构建方法及其应用
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025240940A1 (en) 2024-05-17 2025-11-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of apolipoprotein c3
WO2026015832A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Reverse transcriptases and gene editing systems comprising such
WO2026015829A2 (en) 2024-07-12 2026-01-15 Arbor Biotechnologies, Inc. Small reverse transcriptases and gene editing systems comprising such
CN120424908B (zh) * 2025-07-10 2025-09-12 南京农业大学 一种基于LAMP与CRISPR/Cas的检测方法和应用

Family Cites Families (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5208036A (en) 1985-01-07 1993-05-04 Syntex (U.S.A.) Inc. N-(ω, (ω-1)-dialkyloxy)- and N-(ω, (ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
ATE219660T1 (de) 1994-09-30 2002-07-15 Inex Pharmaceuticals Corp Mittel zum einbringen polyanionischer materialien in zellen
US5695937A (en) 1995-09-12 1997-12-09 The Johns Hopkins University School Of Medicine Method for serial analysis of gene expression
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
CA2597724A1 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
EP3100718B1 (en) 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
EP2279254B1 (en) 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. Novel lipid formulations for nucleic acid delivery
US9221902B2 (en) 2008-11-07 2015-12-29 Fabrus, Inc. Combinatorial antibody libraries and uses thereof
WO2010075303A1 (en) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
US20110071208A1 (en) 2009-06-05 2011-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated dicer-substrate interfering rna
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
US9580714B2 (en) 2010-11-24 2017-02-28 The University Of Western Australia Peptides for the specific binding of RNA targets
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
CA2953341C (en) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
EP3247328B1 (en) 2015-01-21 2026-01-07 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
HUE067372T2 (hu) 2015-06-29 2024-10-28 Acuitas Therapeutics Inc Lipidek és nanoszemcsés lipid formulázások nukleinsavak beadására
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
CN108368028B (zh) 2015-10-28 2021-09-03 爱康泰生治疗公司 用于递送核酸的新型脂质和脂质纳米颗粒制剂
EP3374494A4 (en) 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE THEREOF FOR GENE THERAPY
EP3390624A4 (en) 2015-12-18 2019-07-10 The Regents of The University of California Modified site-directed modifying polypeptides and methods of use thereof
US9896696B2 (en) 2016-02-15 2018-02-20 Benson Hill Biosystems, Inc. Compositions and methods for modifying genomes
WO2017212007A1 (en) 2016-06-09 2017-12-14 Curevac Ag Cationic carriers for nucleic acid delivery
EP3468612A1 (en) 2016-06-09 2019-04-17 CureVac AG Hybrid carriers for nucleic acid cargo
CN109688820B (zh) 2016-06-24 2023-01-10 科罗拉多州立大学董事会(法人团体) 用于生成条形码化组合文库的方法
WO2018035250A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
JP2019532644A (ja) 2016-09-30 2019-11-14 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Rna誘導型核酸修飾酵素及びその使用方法
US9982267B2 (en) 2016-10-12 2018-05-29 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
EP3568470B1 (en) 2017-01-10 2022-07-06 Christiana Care Health Services, Inc. Methods for in vitro site-directed mutagenesis using gene editing technologies
US20190367924A1 (en) * 2017-02-17 2019-12-05 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
MX2019011215A (es) 2017-03-24 2020-02-12 Curevac Ag Ácidos nucleicos que codifican proteínas asociadas a crispr y usos de los mismos.
US11773409B2 (en) 2017-04-21 2023-10-03 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas 9-mediated integration of polynucleotides by sequential homologous recombination of AAV donor vectors
WO2019084148A1 (en) 2017-10-25 2019-05-02 Monsanto Technology Llc TARGETED RNA GUIDED ENDONUCLEASE ENDONUCLEASE ACTIVITY IN EUKARYOTES
US10253365B1 (en) 2017-11-22 2019-04-09 The Regents Of The University Of California Type V CRISPR/Cas effector proteins for cleaving ssDNAs and detecting target DNAs
EP3758714A4 (en) 2018-02-27 2021-12-01 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR TREATMENT OF ANGELMAN'S SYNDROME
US20200224160A1 (en) 2018-02-27 2020-07-16 Sorrento Therapeutics, Inc. Process for dna integration using rna-guided endonucleases
US12522811B2 (en) 2018-05-01 2026-01-13 The Children's Medical Center Corporation Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9
US20210139892A1 (en) 2018-07-05 2021-05-13 The Regents Of The University Of California Compositions and methods for delivery of rna to a cell
US20210284981A1 (en) 2018-07-24 2021-09-16 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
EP3841205A4 (en) 2018-08-22 2022-08-17 The Regents of The University of California VARIANT TYPE V CRISPR/CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF
SG11202113253SA (en) 2019-06-07 2021-12-30 Scribe Therapeutics Inc Engineered casx systems
WO2020247883A2 (en) 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Deep mutational evolution of biomolecules
WO2021007177A1 (en) 2019-07-08 2021-01-14 The Regents Of The University Of California Variant type v crispr/cas effector polypeptides and methods of use thereof
AU2020324386B2 (en) 2019-08-02 2026-04-23 Monsanto Technology Llc Methods and compositions to promote targeted genome modifications using HUH endonucleases
US12551560B2 (en) 2019-09-09 2026-02-17 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy
EP4028522A1 (en) 2019-09-09 2022-07-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
IL292571A (en) 2019-10-28 2022-06-01 Targetgene Biotechnologies Ltd Pam-reduced and pam-abolished cas derivatives compositions and uses thereof in genetic modulation
WO2021113763A1 (en) 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
WO2021113772A1 (en) 2019-12-06 2021-06-10 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
JP2023510352A (ja) 2020-01-10 2023-03-13 スクライブ・セラピューティクス・インコーポレイテッド Pcsk9の標的化のための組成物および方法
MX2022011460A (es) 2020-03-18 2022-12-15 Scribe Therapeutics Inc Composiciones y métodos para el direccionamiento de c9orf72.
AU2021392719B2 (en) 2020-12-03 2025-08-14 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2022261150A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
IL311610A (en) 2021-09-21 2024-05-01 Scribe Therapeutics Inc Engineered CASX suppressor systems
EP4405490A2 (en) 2021-09-23 2024-07-31 Scribe Therapeutics Inc. Self-inactivating vectors for gene editing
CA3257824A1 (en) 2022-06-02 2023-12-07 Scribe Therapeutics Inc. MODIFIED TYPE V, CLASS 2 CRISPR SYSTEMS

Similar Documents

Publication Publication Date Title
JP7374119B2 (ja) 減少した肝臓向性を有するAAV9とAAVrh74とのハイブリッド組換えアデノ随伴ウイルス血清型
US12472239B2 (en) Guide RNA for repairing a mutant human titin gene using CRISPR technology
JP7651190B2 (ja) 遺伝子操作rnaを用いた内因性adarによる標的化rna編集
GB2600274A (en) Engineered CasX systems
JP7381494B2 (ja) 転写調節要素及びその使用
JP2023552374A5 (enExample)
JP7832262B2 (ja) 肝特異的ウイルスプロモーター及びその使用方法
JP2023518541A5 (enExample)
EP3931331A1 (en) Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
JP2022505381A5 (enExample)
JP2023510352A5 (enExample)
JP2017529854A (ja) 高効率ゲノム編集のためのアデノ随伴ウイルスベクターバリアント及びその方法
JP2022541070A (ja) 調節可能な発現系
JP2023537625A5 (enExample)
JP7481755B2 (ja) 組換えパルボウイルスベクターならびにその作製および使用方法
CN114072497B (zh) 用于调控酶表达和使酶表达自失活的组合物以及用于调节酶的脱靶活性的方法
JP2022553573A5 (enExample)
CN119403823A (zh) 具有增强的肌肉转导效率的肽修饰的aav衣壳
WO2025061173A1 (zh) 一种融合蛋白及其应用
CN120818522B (zh) 一种基于itr-增强子驱动核酸表达的调控元件组合及其应用
EP4441232A2 (en) Functional aav capsids for intravitreal administration
JP2023507174A (ja) Dmd変異の修正のための方法及び組成物
TWI838812B (zh) 用於治療法布瑞氏症之組合物及方法
Fakhiri et al. 254. New Chimeric Gene Therapy Vectors Based on Four Different Mammalian Bocaviruses
WO2025006957A2 (en) Non-neutralizing aav constructs and methods of generating and using same