JP2022512726A5 - - Google Patents

Info

Publication number
JP2022512726A5
JP2022512726A5 JP2021521097A JP2021521097A JP2022512726A5 JP 2022512726 A5 JP2022512726 A5 JP 2022512726A5 JP 2021521097 A JP2021521097 A JP 2021521097A JP 2021521097 A JP2021521097 A JP 2021521097A JP 2022512726 A5 JP2022512726 A5 JP 2022512726A5
Authority
JP
Japan
Application number
JP2021521097A
Other languages
Japanese (ja)
Other versions
JP2022512726A (ja
JPWO2020082041A5 (https=
JP7593920B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/057084 external-priority patent/WO2020082041A1/en
Publication of JP2022512726A publication Critical patent/JP2022512726A/ja
Publication of JP2022512726A5 publication Critical patent/JP2022512726A5/ja
Publication of JPWO2020082041A5 publication Critical patent/JPWO2020082041A5/ja
Priority to JP2024203045A priority Critical patent/JP2025028917A/ja
Application granted granted Critical
Publication of JP7593920B2 publication Critical patent/JP7593920B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021521097A 2018-10-18 2019-10-18 核酸構築物及び使用方法 Active JP7593920B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024203045A JP2025028917A (ja) 2018-10-18 2024-11-21 核酸構築物及び使用方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862747393P 2018-10-18 2018-10-18
US62/747,393 2018-10-18
US201962840343P 2019-04-29 2019-04-29
US62/840,343 2019-04-29
PCT/US2019/057084 WO2020082041A1 (en) 2018-10-18 2019-10-18 Nucleic acid constructs and methods of use

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024203045A Division JP2025028917A (ja) 2018-10-18 2024-11-21 核酸構築物及び使用方法

Publications (4)

Publication Number Publication Date
JP2022512726A JP2022512726A (ja) 2022-02-07
JP2022512726A5 true JP2022512726A5 (https=) 2022-10-26
JPWO2020082041A5 JPWO2020082041A5 (https=) 2022-10-26
JP7593920B2 JP7593920B2 (ja) 2024-12-03

Family

ID=68503214

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021521097A Active JP7593920B2 (ja) 2018-10-18 2019-10-18 核酸構築物及び使用方法
JP2024203045A Pending JP2025028917A (ja) 2018-10-18 2024-11-21 核酸構築物及び使用方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024203045A Pending JP2025028917A (ja) 2018-10-18 2024-11-21 核酸構築物及び使用方法

Country Status (16)

Country Link
US (2) US20200268906A1 (https=)
EP (2) EP3867376A1 (https=)
JP (2) JP7593920B2 (https=)
CN (1) CN113272428A (https=)
AU (2) AU2019360269A1 (https=)
BR (1) BR112021007323A2 (https=)
CA (1) CA3116580A1 (https=)
CO (1) CO2021006362A2 (https=)
IL (2) IL322336A (https=)
MA (1) MA53919A (https=)
MX (1) MX2021004282A (https=)
MY (1) MY208708A (https=)
PH (1) PH12021550843A1 (https=)
SG (1) SG11202103732RA (https=)
TW (1) TW202028461A (https=)
WO (1) WO2020082041A1 (https=)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7460643B2 (ja) 2018-10-16 2024-04-02 ブルーアレル, エルエルシー 遺伝子へのdnaの標的化挿入のための方法
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス,インコーポレーテッド 第ix因子を発現するための組成物及び方法
US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
KR20240099164A (ko) * 2021-09-08 2024-06-28 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Pah-조절 조성물 및 방법
US20230149563A1 (en) 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
EP4423271A2 (en) 2021-10-28 2024-09-04 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
EP4473103A2 (en) 2022-02-02 2024-12-11 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
WO2023212677A2 (en) 2022-04-29 2023-11-02 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
US20250302998A1 (en) 2022-05-09 2025-10-02 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
CN119421951A (zh) 2022-05-31 2025-02-11 瑞泽恩制药公司 用于c9orf72重复序列扩增疾病的crispr干扰疗法
IL318625A (en) 2022-07-29 2025-03-01 Regeneron Pharma Compositions and methods for transferrin receptor (TFR)-mediated delivery to brain and muscle
CN120265314A (zh) 2022-09-28 2025-07-04 瑞泽恩制药公司 抗体抗性修饰受体以增强基于细胞的疗法
IL320016A (en) 2022-11-04 2025-06-01 Regeneron Pharma Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
AU2024309884A1 (en) 2023-06-30 2025-12-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for increasing homology-directed repair
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
AU2024315073A1 (en) 2023-07-28 2026-01-22 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
AU2024317483A1 (en) 2023-07-28 2026-01-29 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
AU2024349204A1 (en) * 2023-09-29 2026-04-09 The Board Of Regents Of The University Of Texas System Endogenous nucleus targeting signal peptides and uses thereof
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025160340A2 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Combination immunosuppression for inhibiting an immune response and enabling immunogen administration and re-administration
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
WO2025265017A1 (en) 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i
WO2026072529A1 (en) 2024-09-24 2026-04-02 University Of Florida Research Foundation, Incorporated Mettl7a for improved embryo competence

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4946787A (en) 1985-01-07 1990-08-07 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
US4897355A (en) 1985-01-07 1990-01-30 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
US5049386A (en) 1985-01-07 1991-09-17 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
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
KR940703846A (ko) 1991-12-24 1994-12-12 비. 린네 파샬 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides)
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
JP4285766B2 (ja) 1994-03-23 2009-06-24 オハイオ ユニバーシティ 緻密にした核酸および細胞へのデリバリ
US6169169B1 (en) 1994-05-19 2001-01-02 Dako A/S PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
WO2008133938A2 (en) 2007-04-26 2008-11-06 Sangamo Biosciences, Inc. Targeted integration into the ppp1r12c locus
EP2281050B1 (en) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US8771985B2 (en) 2010-04-26 2014-07-08 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using zinc-finger nucleases
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
AU2012312260B2 (en) 2011-09-21 2017-08-31 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
JP6188703B2 (ja) 2011-10-27 2017-08-30 サンガモ セラピューティクス, インコーポレイテッド Hprt遺伝子座を修飾するための方法および組成物
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
DK3444342T3 (da) * 2012-07-11 2020-08-24 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme
EP4357457B1 (en) 2012-10-23 2024-10-16 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
EP3608308B1 (en) 2013-03-08 2021-07-21 Novartis AG Lipids and lipid compositions for the delivery of active agents
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
EP3169309B1 (en) 2014-07-16 2023-05-10 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
CA2969151A1 (en) 2014-12-23 2016-06-30 Syngenta Participations Ag Methods and compositions for identifying and enriching for cells comprising site specific genomic modifications
EP3265559B1 (en) 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
CA3006618A1 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
TWI773666B (zh) 2016-03-30 2022-08-11 美商英特利亞醫療公司 Crispr/cas 組分之脂質奈米粒子調配物
AR113154A1 (es) 2017-09-29 2020-01-29 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para edición del genoma
JP7460643B2 (ja) * 2018-10-16 2024-04-02 ブルーアレル, エルエルシー 遺伝子へのdnaの標的化挿入のための方法

Similar Documents

Publication Publication Date Title
JP2022512726A5 (https=)
JP2022512731A5 (https=)
BR112021017339A2 (https=)
BR112021018450A2 (https=)
BR112021017637A2 (https=)
BR112021017892A2 (https=)
BR112021017782A2 (https=)
BR112021017939A2 (https=)
BR112021017738A2 (https=)
BR112021017728A2 (https=)
BR112021018452A2 (https=)
BR112021017703A2 (https=)
BR112021018102A2 (https=)
BR112021017732A2 (https=)
BR112021017234A2 (https=)
BR112021018168A2 (https=)
BR112021018093A2 (https=)
BR112021017173A2 (https=)
BR112021017083A2 (https=)
BR112021018250A2 (https=)
BR112021018584A2 (https=)
BR112021017310A2 (https=)
BR112021013128A2 (https=)
BR112021018484A2 (https=)
BR112021021629A2 (https=)