DK3436077T3 - Lipidnanopartikelformuleringer til crispr/cas-komponenter - Google Patents

Lipidnanopartikelformuleringer til crispr/cas-komponenter

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Publication number
DK3436077T3
DK3436077T3 DK17719387.7T DK17719387T DK3436077T3 DK 3436077 T3 DK3436077 T3 DK 3436077T3 DK 17719387 T DK17719387 T DK 17719387T DK 3436077 T3 DK3436077 T3 DK 3436077T3
Authority
DK
Denmark
Prior art keywords
crispr
lipid nanoparticle
nanoparticle formulations
cas components
cas
Prior art date
Application number
DK17719387.7T
Other languages
English (en)
Inventor
David V Morrissey
Mihir Chandrakant Patel
Jonathan D Finn
Amy Madison Rhoden Smith
Lucinda J Shaw
Christian Dombrowski
Ruchi Rudraprasad Shah
Original Assignee
Intellia Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Application granted granted Critical
Publication of DK3436077T3 publication Critical patent/DK3436077T3/da

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
DK17719387.7T 2016-03-30 2017-03-30 Lipidnanopartikelformuleringer til crispr/cas-komponenter DK3436077T3 (da)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662315602P 2016-03-30 2016-03-30
US201662375776P 2016-08-16 2016-08-16
US201662433228P 2016-12-12 2016-12-12
US201762468300P 2017-03-07 2017-03-07
PCT/US2017/024973 WO2017173054A1 (en) 2016-03-30 2017-03-30 Lipid nanoparticle formulations for crispr/cas components

Publications (1)

Publication Number Publication Date
DK3436077T3 true DK3436077T3 (da) 2025-06-30

Family

ID=58632585

Family Applications (1)

Application Number Title Priority Date Filing Date
DK17719387.7T DK3436077T3 (da) 2016-03-30 2017-03-30 Lipidnanopartikelformuleringer til crispr/cas-komponenter

Country Status (23)

Country Link
US (2) US20190136231A1 (da)
EP (2) EP4585272A3 (da)
JP (3) JP7245651B2 (da)
KR (1) KR102617874B1 (da)
CN (2) CN117731805A (da)
AU (2) AU2017244143B2 (da)
BR (1) BR112018069795A2 (da)
CA (1) CA3018978A1 (da)
CO (1) CO2018011554A2 (da)
DK (1) DK3436077T3 (da)
ES (1) ES3030658T3 (da)
FI (1) FI3436077T3 (da)
HR (1) HRP20250670T1 (da)
HU (1) HUE071550T2 (da)
LT (1) LT3436077T (da)
MX (1) MX2024008101A (da)
PL (1) PL3436077T3 (da)
PT (1) PT3436077T (da)
RS (1) RS66865B1 (da)
SI (1) SI3436077T1 (da)
SM (1) SMT202500211T1 (da)
TW (1) TWI773666B (da)
WO (1) WO2017173054A1 (da)

Families Citing this family (361)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
PT3766916T (pt) 2014-06-25 2022-11-28 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3289083A4 (en) 2015-04-27 2018-12-19 The Trustees Of The University Of Pennsylvania Nucleoside-modified rna for inducing an adaptive immune response
HRP20240865T1 (hr) 2015-06-29 2024-10-11 Acuitas Therapeutics Inc. Lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
WO2017075531A1 (en) 2015-10-28 2017-05-04 Acuitas Therapeutics, Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
KR20190019168A (ko) * 2016-06-17 2019-02-26 더 브로드 인스티튜트, 인코퍼레이티드 제vi형 crispr 오솔로그 및 시스템
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
SG11201900907YA (en) 2016-08-03 2019-02-27 Harvard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
SG10202106058WA (en) * 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
KR102551664B1 (ko) * 2016-12-22 2023-07-05 인텔리아 테라퓨틱스, 인크. 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
CA3049991A1 (en) 2017-01-11 2018-07-19 The Trustees Of The University Of Pennsylvania Nucleoside-modified rna for inducing an immune response against zika virus
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
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
IL269458B2 (en) 2017-03-23 2024-02-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
EP3615054A4 (en) 2017-04-27 2021-03-24 The Trustees Of The University Of Pennsylvania NUCLEOSIDE MODIFIED MRNA-LIPID NANOPARTICLE LINE VACCINE FOR THE HEPATITIS C VIRUS
WO2018200943A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
US11173190B2 (en) 2017-05-16 2021-11-16 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mRNA encoding CFTR
EP3641834B1 (en) 2017-06-19 2023-10-04 Translate Bio, Inc. Messenger rna therapy for the treatment of friedreich's ataxia
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
SG11201912236YA (en) 2017-07-31 2020-01-30 Regeneron Pharma Crispr reporter non-human animals and uses thereof
WO2019028029A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. EVALUATION OF CRISPR / CAS INDUCED RECOMBINATION WITH IN VIVO EXOGENIC DONOR NUCLEIC ACID
CA3067872A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. Cas-transgenic mouse embryonic stem cells and mice and uses thereof
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
ES2997124T3 (en) 2017-08-17 2025-02-14 Acuitas Therapeutics Inc Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
SG11202000771UA (en) 2017-09-29 2020-04-29 Regeneron Pharma Non-human animals comprising a humanized ttr locus and methods of use
KR102832035B1 (ko) 2017-09-29 2025-07-09 인텔리아 테라퓨틱스, 인크. 지질 나노파티클을 이용한 mRNA 전달의 체외 방법
RS65493B1 (sr) * 2017-09-29 2024-05-31 Intellia Therapeutics Inc Formulacije
CN118530993A (zh) * 2017-09-29 2024-08-23 因特利亚治疗公司 用于ttr基因编辑及治疗attr淀粉样变性的组合物及方法
IL311278B1 (en) 2017-09-29 2026-01-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
EP3701065A4 (en) 2017-10-23 2021-07-28 Montrose Biosystems LLC METAL AND MIXED METAL NANOPARTICLES, CONJUGATES OF NANOPARTICLES, DEVICES FOR MANUFACTURING NANOPARTICLES, AND RELATED METHODS OF USE
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND PROCEDURES FOR PREDICTING REPAIR RESULTS IN GENE ENGINEERING
MA51306A (fr) 2017-12-20 2020-10-28 Translate Bio Inc Compositions et procédés améliorés pour le traitement du déficit en ornithine transcarbamylase
CN111885915B (zh) 2018-03-19 2023-04-28 瑞泽恩制药公司 使用crispr/cas系统对动物进行转录调制
CA3092497A1 (en) 2018-03-19 2019-09-26 Crispr Therapeutics Ag Novel rna-programmable endonuclease systems and uses thereof
JP7642531B2 (ja) 2018-05-11 2025-03-10 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法
CA3097912A1 (en) 2018-05-15 2019-11-21 Translate Bio, Inc. Subcutaneous delivery of messenger rna
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
CA3100254A1 (en) 2018-05-24 2019-11-28 Translate Bio, Inc. Thioester cationic lipids
JP2021525725A (ja) 2018-05-30 2021-09-27 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａワクチンおよびその使用
EP3802507A1 (en) 2018-05-30 2021-04-14 Translate Bio, Inc. Vitamin cationic lipids
AU2019277355A1 (en) 2018-05-30 2020-12-17 Translate Bio, Inc. Phosphoester cationic lipids
ES2989886T3 (es) 2018-05-30 2024-11-28 Translate Bio Inc Lípidos catiónicos que comprenden una fracción esteroidal
CA3102950A1 (en) * 2018-06-08 2019-12-12 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
US12357580B2 (en) 2018-06-19 2025-07-15 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
US20210269828A1 (en) 2018-06-22 2021-09-02 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
SG11202100602SA (en) 2018-07-23 2021-02-25 Translate Bio Inc Dry power formulations for messenger rna
WO2020028327A1 (en) 2018-07-31 2020-02-06 Intellia Therapeutics, Inc. Compositions and methods for hydroxyacid oxidase 1 ( hao1) gene editing for treating primary hyperoxaluria type 1 (ph1)
KR20210049859A (ko) * 2018-08-28 2021-05-06 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하는 방법 및 조성물
CN112839639A (zh) 2018-08-29 2021-05-25 川斯勒佰尔公司 制备负载mRNA的脂质纳米颗粒的改进方法
GB2592504B (en) * 2018-09-04 2023-02-15 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
JP7425066B2 (ja) * 2018-09-04 2024-01-30 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム 核酸を臓器特異的送達するための組成物および方法
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
WO2020056294A1 (en) 2018-09-14 2020-03-19 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
CA3113449A1 (en) 2018-09-21 2020-03-26 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
MX2021003457A (es) 2018-09-28 2021-07-16 Intellia Therapeutics Inc Composiciones y métodos para la edición génica de lactato deshidrogenasa (ldha).
EP3867379A1 (en) 2018-10-16 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for immunotherapy
AU2019361204A1 (en) 2018-10-18 2021-05-27 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey
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
CA3116918A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
AU2019361129A1 (en) 2018-10-18 2021-05-20 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
CA3116580A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Nucleic acid constructs and methods of use
US20210378962A1 (en) 2018-10-19 2021-12-09 Translate Bio, Inc. Pumpless encapsulation of messenger rna
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
EP3876994A2 (en) 2018-11-09 2021-09-15 Translate Bio, Inc. Peg lipidoid compounds
WO2020097384A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. 2,5-dioxopiperazine lipids with intercalated ester, thioester, disulfide and anhydride moieities
WO2020097376A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Multi-peg lipid compounds
WO2020097511A2 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Messenger rna therapy for treatment of ocular diseases
AU2019378763B2 (en) 2018-11-12 2025-06-26 Translate Bio, Inc. Methods for inducing immune tolerance
WO2020106903A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. Cationic lipid compounds and compositions thereof for use in the delivery of messenger rna
JP7564102B2 (ja) 2018-11-28 2024-10-08 クリスパー セラピューティクス アクチェンゲゼルシャフト ＬＮＰでの使用に最適化された、ＣＡＳ９をコードするｍＲＮＡ
TWI841639B (zh) * 2018-12-05 2024-05-11 美商英特利亞醫療公司 經修飾之胺脂質
CN116064550A (zh) 2018-12-20 2023-05-05 瑞泽恩制药公司 核酸酶介导的重复扩增
AU2020206109B2 (en) 2019-01-07 2024-12-19 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
JP7523449B2 (ja) 2019-01-11 2024-07-26 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020154774A1 (en) * 2019-01-31 2020-08-06 Newsouth Innovations Pty Limited Liposomal nanoparticle
CN120138047A (zh) * 2019-02-13 2025-06-13 比姆医疗股份有限公司 用于治疗1a型糖原贮积病的组成物和方法
EP3937963B1 (en) 2019-03-12 2025-05-07 CRISPR Therapeutics AG Novel high fidelity rna-programmable endonuclease systems and uses thereof
US11845931B2 (en) 2019-03-18 2023-12-19 Regeneron Pharmaceuticals, Inc. CRISPR/Cas screening platform to identify genetic modifiers of tau seeding or aggregation
CN113711046B (zh) 2019-03-18 2023-08-29 瑞泽恩制药公司 用于揭示与Tau聚集相关的基因脆弱性的CRISPR/Cas脱落筛选平台
GB2601618B (en) 2019-03-19 2024-11-06 Broad Inst Inc Methods and compositions for editing nucleotide sequences
KR20220004649A (ko) 2019-03-28 2022-01-11 인텔리아 테라퓨틱스, 인크. 폴리펩티드 발현을 위한 폴리뉴클레오티드, 조성물 및 방법
WO2020198706A1 (en) 2019-03-28 2020-10-01 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating attr amyloidosis comprising a corticosteroid or use thereof
CN113874076A (zh) * 2019-03-28 2021-12-31 因特利亚治疗公司 包括ttr向导rna和对rna引导的dna结合剂进行编码的多核苷酸的组合物和方法
BR112021019512A2 (pt) 2019-04-03 2022-02-15 Regeneron Pharma Métodos para inserir uma sequência de codificação de proteína de ligação ao antígeno e para tratar ou efetuar a profilaxia de uma doença em um animal, animal, célula, genoma, ácido nucleico doador exógeno, gene de porto seguro, e, agente de nuclease ou um ou mais ácidos nucleicos
ES2966625T3 (es) 2019-04-04 2024-04-23 Regeneron Pharma Roedores que comprenden un locus del factor de coagulación 12 humanizado
WO2020206134A1 (en) 2019-04-04 2020-10-08 Regeneron Pharmaceuticals, Inc. Methods for scarless introduction of targeted modifications into targeting vectors
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220177423A1 (en) 2019-04-18 2022-06-09 Translate Bio, Inc. Cystine cationic lipids
US20220233444A1 (en) 2019-04-22 2022-07-28 Translate Bio, Inc. Thioester cationic lipids
AU2020261419B2 (en) * 2019-04-25 2026-01-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles
WO2020227085A1 (en) 2019-05-03 2020-11-12 Translate Bio, Inc. Di-thioester cationic lipids
CN110157778A (zh) * 2019-05-30 2019-08-23 浙江大学 一种用于核酸扩增产物防污染检测的crispr试剂体系的储存方法及检测管
EP3976593A1 (en) 2019-05-31 2022-04-06 Translate Bio, Inc. Macrocyclic lipids
AU2020286382B2 (en) 2019-06-04 2026-03-26 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized TTR locus with a beta-slip mutation and methods of use
JP7698587B2 (ja) 2019-06-07 2025-06-25 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヒト化アルブミン遺伝子座を含む非ヒト動物
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
WO2020252340A1 (en) 2019-06-14 2020-12-17 Regeneron Pharmaceuticals, Inc. Models of tauopathy
WO2020257611A1 (en) 2019-06-21 2020-12-24 Translate Bio, Inc. Cationic lipids comprising an hydroxy moiety
CN114401942B (zh) 2019-06-21 2024-01-26 川斯勒佰尔公司 三(羟甲基)甲基甘氨酸和柠檬酸脂质
KR20220078557A (ko) 2019-07-08 2022-06-10 트랜슬레이트 바이오 인코포레이티드 개선된 mrna 로딩된 지질 나노입자 및 이의 제조 방법
JP7759311B2 (ja) 2019-07-19 2025-10-23 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー リコンビナーゼ組成物及び使用方法
MX2022000934A (es) 2019-07-23 2022-02-14 Translate Bio Inc Composiciones estables de nanoparticulas lipidicas cargadas de arnm y procesos de fabricacion.
CN114502542A (zh) * 2019-07-29 2022-05-13 佐治亚技术研究公司 包含限制性脂质的纳米材料及其用途
JP2022548031A (ja) * 2019-09-13 2022-11-16 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 脂質ナノ粒子によって送達されるｃｒｉｓｐｒ／ｃａｓシステムを使用する動物における転写調節
US20240197825A1 (en) 2019-09-20 2024-06-20 Translate Bio, Inc. mRNA Encoding Engineered CFTR
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
WO2021081058A1 (en) 2019-10-21 2021-04-29 Translate Bio, Inc. Compositions, methods and uses of messenger rna
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
WO2021127641A1 (en) 2019-12-20 2021-06-24 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
EP4076393A2 (en) 2019-12-20 2022-10-26 Translate Bio, Inc. Rectal delivery of messenger rna
KR20220123536A (ko) 2020-01-09 2022-09-07 가이드 테라퓨틱스 엘엘씨 나노물질
WO2021142245A1 (en) 2020-01-10 2021-07-15 Translate Bio, Inc. Compounds, pharmaceutical compositions and methods for modulating expression of muc5b in lung cells and tissues
KR20220133248A (ko) 2020-01-28 2022-10-04 리제너론 파마슈티칼스 인코포레이티드 인간화 pnpla3 좌위를 포함하는 비-인간 동물 및 사용 방법
AU2021216451A1 (en) 2020-02-07 2022-09-29 Intellia Therapeutics, Inc. Compositions and methods for kallikrein ( KLKB1) gene editing
EP4099821A1 (en) 2020-02-07 2022-12-14 Regeneron Pharmaceuticals, Inc. <smallcaps/>? ? ?klkb1? ? ? ? ?non-human animals comprising a humanizedlocus and methods of use
CN115427021A (zh) 2020-02-25 2022-12-02 翻译生物公司 制备负载mrna的脂质纳米颗粒的改进方法
CN115485385A (zh) 2020-03-04 2022-12-16 瑞泽恩制药公司 用于使肿瘤细胞对免疫疗法敏感的方法和组合物
GB2629329B (en) 2020-03-04 2025-02-26 Verve Therapeutics Inc Formulation comprising GalNAc-lipid nanoparticle
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
EP4125348A1 (en) 2020-03-23 2023-02-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
CN111388677B (zh) * 2020-03-24 2021-08-06 河南大学 药物载体、基于crispr基因编辑技术的脑靶向纳米药物及其制备方法和应用
BR112022020428A2 (pt) 2020-04-09 2023-03-07 Verve Therapeutics Inc Edição base do angptl3 e métodos de uso do mesmo para tratamento de doenças
TW202204612A (zh) 2020-04-28 2022-02-01 美商英特利亞醫療公司 活體外細胞遞送之方法
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy
US20230190954A1 (en) 2020-05-07 2023-06-22 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
AU2021269042A1 (en) 2020-05-07 2023-02-02 Translate Bio, Inc. Optimized nucleotide sequences encoding SARS-CoV-2 antigens
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
WO2021231697A1 (en) 2020-05-14 2021-11-18 Translate Bio, Inc. Peg lipidoid compounds
CN115916158A (zh) 2020-05-15 2023-04-04 翻译生物公司 用于mRNA递送的脂质纳米颗粒配制品
MX2022014653A (es) 2020-05-20 2023-02-16 Flagship Pioneering Innovations Vi Llc Composiciones de antigeno de coronavirus y sus usos.
CA3179444A1 (en) 2020-05-20 2021-11-25 Avak Kahvejian Immunogenic compositions and uses thereof
US20230203510A1 (en) 2020-05-29 2023-06-29 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods relating thereto
WO2021243301A2 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
US20240307556A1 (en) 2020-07-02 2024-09-19 Maritime Therapeutics, Inc. Compositions and methods for reverse gene therapy
BR112023000327A2 (pt) 2020-07-16 2023-01-31 Acuitas Therapeutics Inc Lipídeos catiônicos para o uso em nanopartículas lipídicas
AU2021310950A1 (en) 2020-07-24 2023-02-16 President And Fellows Of Harvard College Enhanced virus-like particles and methods of use thereof for delivery to cells
JP2023542492A (ja) 2020-09-03 2023-10-10 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 免疫原性組成物及びその使用
WO2022060871A1 (en) 2020-09-15 2022-03-24 Verve Therapeutics, Inc. Lipid formulations for gene editing
CN116685349A (zh) 2020-09-25 2023-09-01 比姆医疗股份有限公司 抗自相残杀的修饰的免疫细胞及其使用方法
AU2021358777A1 (en) 2020-10-06 2023-06-15 Translate Bio, Inc. Improved process and formulation of lipid nanoparticles
WO2022081548A1 (en) 2020-10-12 2022-04-21 Translate Bio, Inc. Improved process of preparing ice-based lipid nanoparticles
KR20230087536A (ko) 2020-10-12 2023-06-16 트랜슬레이트 바이오 인코포레이티드 Mrna-로딩된 지질 나노입자를 제조하는 개선된 프로세스
EP4240326A1 (en) 2020-11-09 2023-09-13 Translate Bio, Inc. Improved compositions for delivery of codon-optimized mrna
CN117098541A (zh) 2020-11-25 2023-11-21 阿卡格拉医药公司 用于递送核酸的脂质纳米粒及相关使用方法
CN116723829A (zh) * 2020-11-25 2023-09-08 翻译生物公司 稳定的液体脂质纳米颗粒配制品
WO2022120022A1 (en) 2020-12-02 2022-06-09 Regeneron Pharmaceuticals, Inc. Crispr sam biosensor cell lines and methods of use thereof
CR20230303A (es) 2020-12-11 2023-09-01 Intellia Therapeutics Inc Composiciones y métodos para reducir el mhc de clase ii en una célula
IL303506A (en) * 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
AU2021409740A1 (en) 2020-12-23 2023-07-06 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
CA3205042A1 (en) 2020-12-23 2022-06-30 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying ciita in a cell
JP2024500858A (ja) 2020-12-23 2024-01-10 インテリア セラピューティクス，インコーポレイテッド 細胞中のｈｌａ－ａを低減するための組成物及び方法
IL304069A (en) 2020-12-30 2023-08-01 Intellia Therapeutics Inc Engineered T cells
JP2024504614A (ja) 2021-01-14 2024-02-01 トランスレイト バイオ， インコーポレイテッド ｍＲＮＡがコードする抗体を送達する方法および組成物
CA3203628A1 (en) 2021-01-20 2022-07-28 Beam Therapeutics Inc. Nanomaterials comprising a biodegradable feature
WO2022170059A1 (en) 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
JP2024505672A (ja) 2021-02-08 2024-02-07 インテリア セラピューティクス，インコーポレイテッド 免疫療法のためのナチュラルキラー細胞受容体２ｂ４組成物及び方法
CN117042794A (zh) 2021-02-08 2023-11-10 因特利亚治疗公司 用于免疫疗法的t细胞免疫球蛋白和粘蛋白结构域3(tim3)组合物和方法
AU2021425941A1 (en) 2021-02-08 2023-08-17 The Board Of Regents Of The University Of Texas System Unsaturated dendrimers compositions,related formulations, and methods of use thereof
CN117042793A (zh) 2021-02-08 2023-11-10 因特利亚治疗公司 用于免疫疗法的淋巴细胞活化基因3(lag3)组合物和方法
EP4297729A4 (en) * 2021-02-26 2025-11-19 Univ Northwestern STRATEGIES FOR DEVELOPING GENOME-EDITIVE SPHERICAL NUCLEAR ACIDS (SNAs)
EP4312987A4 (en) 2021-03-22 2025-02-26 Recode Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TARGETED DELIVERY TO CELLS
US20250019409A1 (en) 2021-03-23 2025-01-16 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204549A1 (en) 2021-03-25 2022-09-29 Translate Bio, Inc. Optimized nucleotide sequences encoding the extracellular domain of human ace2 protein or a portion thereof
CA3214085A1 (en) 2021-03-31 2022-10-06 Darby Rye Schmidt Thanotransmission polypeptides and their use in treating cancer
MX2023012236A (es) 2021-04-17 2024-01-23 Intellia Therapeutics Inc Inhibidores de proteína cinasa dependente de acido desoxirribonucleico (and) y composiciones y usos de los mismos.
AU2022261865A1 (en) 2021-04-19 2023-11-30 Translate Bio, Inc. Improved compositions for delivery of mrna
WO2022240846A1 (en) 2021-05-10 2022-11-17 Sqz Biotechnologies Company Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof
KR20240032013A (ko) 2021-06-10 2024-03-08 인텔리아 테라퓨틱스, 인크. 유전자 편집을 위한 내부 링커를 포함하는 변형된 가이드 rna
US20240141312A1 (en) 2021-06-11 2024-05-02 Bayer Aktiengesellschaft Type v rna programmable endonuclease systems
EP4101928A1 (en) 2021-06-11 2022-12-14 Bayer AG Type v rna programmable endonuclease systems
KR20240038705A (ko) 2021-06-22 2024-03-25 인텔리아 테라퓨틱스, 인크. 간 유전자의 생체 내 편집 방법
CN113403313B (zh) * 2021-06-23 2022-08-19 北京理工大学 一种特异识别人PLK1位点的sgRNA、质粒、纳米复合物和应用
JP2024527541A (ja) 2021-07-01 2024-07-25 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａの送達のための組成物
WO2023288135A2 (en) * 2021-07-16 2023-01-19 Orthobio Therapeutics, Inc. Gene editing to improve joint function
WO2023009547A1 (en) 2021-07-26 2023-02-02 Flagship Pioneering Innovations Vi, Llc Trem compositions and uses thereof
JP2024530647A (ja) 2021-08-03 2024-08-23 ヴァーヴ・セラピューティクス，インコーポレーテッド 標的ｒｎａ送達のための組成物および方法
CN113637708B (zh) * 2021-08-09 2023-07-25 中国科学院过程工程研究所 一种CRISPR-cas9基因编辑系统递送载体及其制备方法和应用
CN117940153A (zh) 2021-08-24 2024-04-26 因特利亚治疗公司 用于基于细胞的疗法的程序性细胞死亡蛋白1(pd1)组合物和方法
EP4144841A1 (en) 2021-09-07 2023-03-08 Bayer AG Novel small rna programmable endonuclease systems with impoved pam specificity and uses thereof
IL311225A (en) 2021-09-08 2024-05-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for genome modulation
EP4464783A3 (en) 2021-09-17 2025-01-22 Flagship Pioneering Innovations VI, LLC Compositions and methods for producing circular polyribonucleotides
TW202322826A (zh) 2021-10-18 2023-06-16 美商旗艦先鋒創新有限責任公司 用於純化多核糖核苷酸之組成物及方法
WO2023069498A1 (en) 2021-10-22 2023-04-27 Senda Biosciences, Inc. Mrna vaccine composition
EP4422661A1 (en) 2021-10-26 2024-09-04 Regeneron Pharmaceuticals, Inc. Overexpression of lemd2, lemd3, or chmp7 as a therapeutic modality for tauopathy
CN118251491A (zh) 2021-10-28 2024-06-25 瑞泽恩制药公司 用于敲除C5的CRISPR/Cas相关方法及组合物
US20250302874A1 (en) 2021-11-03 2025-10-02 Intellia Therapeutics, Inc. Cd38 compositions and methods for immunotherapy
KR20240114296A (ko) 2021-11-03 2024-07-23 인텔리아 테라퓨틱스, 인크. 게놈 편집을 위한 폴리뉴클레오타이드, 조성물, 및 방법
WO2023081526A1 (en) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
JP2024545572A (ja) 2021-11-10 2024-12-10 トランスレイト バイオ， インコーポレイテッド 原発性線毛機能不全の治療のための組成物及び方法
KR20240107356A (ko) 2021-11-23 2024-07-09 세일 바이오메디슨스, 인크. 박테리아-유래 지질 조성물 및 이의 용도
WO2023096990A1 (en) 2021-11-24 2023-06-01 Flagship Pioneering Innovation Vi, Llc Coronavirus immunogen compositions and their uses
KR20240125931A (ko) 2021-11-24 2024-08-20 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 수두-대상포진 바이러스 면역원 조성물 및 이의 용도
US20250032604A1 (en) 2021-11-24 2025-01-30 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and their uses
US20250223584A2 (en) 2021-12-03 2025-07-10 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
CA3238939A1 (en) 2021-12-08 2023-06-15 Gaurang Patel Mutant myocilin disease model and uses thereof
IL313486A (en) 2021-12-16 2024-08-01 Acuitas Therapeutics Inc Lipids for use in lipid nanoparticle formulations
WO2023115013A1 (en) 2021-12-17 2023-06-22 Flagship Pioneering Innovations Vi, Llc Methods for enrichment of circular rna under denaturing conditions
JP2025500373A (ja) 2021-12-20 2025-01-09 セイル バイオメディシンズ インコーポレイテッド Ｍｒｎａ治療用組成物
WO2023122764A1 (en) * 2021-12-22 2023-06-29 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
CN118679254A (zh) 2021-12-22 2024-09-20 旗舰创业创新六公司 用于纯化多核糖核苷酸的组合物和方法
CA3243301A1 (en) 2021-12-23 2023-06-29 Bayer Aktiengesellschaft NEW SMALL PROGRAMMABLE TYPE V RNA ENDONUCLEASE SYSTEMS
MX2024007870A (es) 2021-12-23 2024-08-15 Flagship Pioneering Innovations Vi Llc Polirribonucleotidos circulares que codifican polipeptidos antifusogenicos.
US20250032642A1 (en) 2022-02-02 2025-01-30 Regeneron Pharmaceuticals, Inc. Crispr-mediated transgene insertion in neonatal cells
WO2023154451A1 (en) 2022-02-10 2023-08-17 Christiana Care Gene Editing Institute, Inc. Methods for lipid nanoparticle delivery of crispr/cas system
CA3242729A1 (en) 2022-02-11 2023-08-17 Regeneron Pharmaceuticals, Inc. Compositions and methods for screening 4r tau targeting agents
US20230293646A1 (en) * 2022-03-21 2023-09-21 Crispr Therapeutics Ag Methods and compositions for treating lipoprotein-related diseases
CA3245624A1 (en) 2022-03-25 2023-09-28 Sail Biomedicines, Inc. NEW IONIZABLE LIPIDS AND LIPID NANOPARTICLES AND THEIR METHODS OF USE
EP4499829A2 (en) 2022-03-29 2025-02-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
EP4504252A2 (en) 2022-04-08 2025-02-12 Flagship Pioneering Innovations VII, LLC Vaccines and related methods
IL316048A (en) 2022-04-13 2024-11-01 Caribou Biosciences Inc Therapeutic applications of V-type CRISPR systems
WO2023205606A1 (en) 2022-04-18 2023-10-26 Vertex Pharmaceuticals Incorporated Compositions and methods for enhancing aav therapy and decreasing tropism of aav to the liver
EP4511397A1 (en) 2022-04-19 2025-02-26 Intellia Therapeutics, Inc. Chimeric antigen receptor compositions and uses
CN119487200A (zh) 2022-04-29 2025-02-18 瑞泽恩制药公司 用于基因治疗方法的组织特异性基因外安全港的鉴定
CN119604610A (zh) 2022-05-09 2025-03-11 瑞泽恩制药公司 用于体内抗体产生的载体和方法
CA3257006A1 (en) 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc TREM COMPOSITIONS AND METHODS OF USE FOR TREATING PROLIFERATIVE DISORDERS
TW202409283A (zh) 2022-05-13 2024-03-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 雙股ｄｎａ組合物及相關方法
CN120303407A (zh) 2022-05-17 2025-07-11 恩维洛普治疗有限责任公司 用于有效体内递送的组合物和方法
AU2023275780B2 (en) 2022-05-25 2026-03-05 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
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
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
AU2023284048A1 (en) 2022-06-10 2024-11-14 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
EP4536293A1 (en) * 2022-06-10 2025-04-16 Renagade Therapeutics Management Inc. Nucleobase editing system and method of using same for modifying nucleic acid sequences
JP2025520367A (ja) 2022-06-16 2025-07-03 インテリア セラピューティクス，インコーポレイテッド 細胞を遺伝子改変するための方法及び組成物
WO2023250112A1 (en) 2022-06-22 2023-12-28 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
IL317790A (en) 2022-06-29 2025-02-01 Intellia Therapeutics Inc Engineered T cells
CA3260437A1 (en) 2022-07-20 2024-01-25 Beam Therapeutics Inc. NANOMATERIALS INCLUDING TRIOLS
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
JP2025525778A (ja) 2022-08-01 2025-08-07 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー 免疫調節タンパク質及び関連方法
JP2025525745A (ja) 2022-08-05 2025-08-07 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｔｄｐ－４３の凝集耐性バリアント
EP4569112A1 (en) 2022-08-12 2025-06-18 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
KR20250056256A (ko) 2022-08-31 2025-04-25 세일 바이오메디슨스, 인크. 신규 이온화성 지질 및 지질 나노입자, 그리고 이의 사용 방법
WO2024061296A2 (en) 2022-09-22 2024-03-28 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease
AU2023347320A1 (en) 2022-09-23 2025-04-10 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer
CN120513299A (zh) 2022-10-31 2025-08-19 旗舰创业创新第六有限责任公司 用于纯化多核糖核苷酸的组合物和方法
KR20250115463A (ko) 2022-11-04 2025-07-30 리제너론 파아마슈티컬스, 인크. 칼슘 전압 개폐 채널 보조 서브유닛 감마 1(cacng1) 결합 단백질 및 골격근으로의 cacng1-매개 전달
AU2023378958A1 (en) * 2022-11-07 2025-06-05 Ramot At Tel-Aviv University Ltd. Self-assembling lipid nanoparticles for targeted delivery of therapeutic agents
EP4615517A1 (en) 2022-11-08 2025-09-17 Orna Therapeutics, Inc. Circular rna compositions
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
AR131008A1 (es) 2022-11-08 2025-02-05 Flagship Pioneering Innovations Vi Llc Composiciones y métodos para producir polirribonucleótidos circulares
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102434A1 (en) 2022-11-10 2024-05-16 Senda Biosciences, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs
EP4619438A2 (en) 2022-11-14 2025-09-24 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024107670A1 (en) 2022-11-16 2024-05-23 Regeneron Pharmaceuticals, Inc. Chimeric proteins comprising membrane bound il-12 with protease cleavable linkers
EP4622630A1 (en) 2022-11-21 2025-10-01 Translate Bio, Inc. Compositions of dry powder formulations of messenger rna and methods of use thereof
EP4630057A1 (en) 2022-12-08 2025-10-15 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
AU2023398007A1 (en) 2022-12-13 2025-06-19 Bayer Aktiengesellschaft Engineered type v rna programmable endonucleases and their uses
TW202430215A (zh) 2022-12-14 2024-08-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於將治療劑遞送至骨之組成物和方法
EP4633673A1 (en) 2022-12-15 2025-10-22 Sanofi Pasteur Inc. Mrna encoding influenza virus-like particle
AU2023398825A1 (en) 2022-12-15 2025-06-26 Orna Therapeutics, Inc. Circular rna compositions and methods
AR131438A1 (es) 2022-12-20 2025-03-19 Sanofi Sa VACUNA DE ARNm DE RINOVIRUS
EP4638748A2 (en) 2022-12-21 2025-10-29 Intellia Therapeutics, Inc. Compositions and methods for proprotein convertase subtilisin kexin 9 (pcsk9) editing
EP4638783A2 (en) 2022-12-22 2025-10-29 Intellia Therapeutics, Inc. Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies
TW202428878A (zh) 2022-12-23 2024-07-16 美商英特利亞醫療公司 用於基因體編輯之系統及方法
WO2024151687A1 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
US20240252520A1 (en) 2023-01-09 2024-08-01 Beth Israel Deaconess Medical Center, Inc. Therapeutic agents and their use for treating chronic wounds
WO2024151673A2 (en) 2023-01-09 2024-07-18 President And Fellows Of Harvard College Recombinant nucleic acid molecules and their use in wound healing
EP4648794A2 (en) 2023-01-09 2025-11-19 Flagship Pioneering Innovations VII, LLC Vaccines and related methods
AU2024212425A1 (en) 2023-01-27 2025-08-07 Sail Biomedicines, Inc. A modified lipid composition and uses thereof
WO2024156291A1 (en) * 2023-01-29 2024-08-02 Geneditbio Limited Cationic lipids and lipid nanoparticles
TW202438515A (zh) 2023-02-06 2024-10-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 免疫調節組合物及相關方法
KR20250151427A (ko) 2023-02-13 2025-10-21 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 치료적 조성물을 위한 절단성 링커 함유 이온화 가능한 지질 및 지질 담체
TW202446956A (zh) 2023-02-17 2024-12-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 包含經修飾的胞嘧啶的ｄｎａ組成物
TW202438089A (zh) 2023-02-17 2024-10-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 包含經修飾的尿嘧啶的ｄｎａ組成物
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
WO2024186971A1 (en) 2023-03-07 2024-09-12 Intellia Therapeutics, Inc. Cish compositions and methods for immunotherapy
WO2024192422A1 (en) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
JP2026509462A (ja) 2023-03-15 2026-03-19 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー ポリリボヌクレオチドを含む組成物及びその使用
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
CN121219411A (zh) 2023-04-12 2025-12-26 旗舰先锋创新Vi有限责任公司 用于在错义突变的校正中使用的trem
EP4695393A1 (en) 2023-04-12 2026-02-18 Flagship Pioneering Innovations VI, LLC Modified trems, compositions, and related methods thereof
KR20250169625A (ko) 2023-04-17 2025-12-03 사노피 파스퇴르 인크 재구성 가능한 건조 분말 제형 및 이의 사용 방법
WO2024220625A1 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids
WO2024220712A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Vaccine compositions
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
EP4705481A2 (en) 2023-05-05 2026-03-11 Orna Therapeutics, Inc. Circular rna compositions and methods
EP4713453A1 (en) 2023-05-15 2026-03-25 nChroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024238700A1 (en) 2023-05-15 2024-11-21 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024254376A1 (en) 2023-06-08 2024-12-12 Regeneron Pharmaceuticals, Inc. Animal model with rapid onset of alzheimer's amyloid beta plaque pathology
WO2024258829A1 (en) 2023-06-12 2024-12-19 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2024259135A1 (en) 2023-06-13 2024-12-19 Intellia Therapeutics, Inc. Assays for analysis of ribonucleic acid (rna) molecules
AU2024305332A1 (en) 2023-06-15 2026-01-15 Regeneron Pharmaceuticals, Inc. Gene therapy for hearing disorders
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
AU2024309386A1 (en) 2023-06-30 2025-12-18 Christiana Care Gene Editing Institute, Inc. Nras gene knockout for treatment of cancer
AU2024309884A1 (en) 2023-06-30 2025-12-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for increasing homology-directed repair
WO2025024486A2 (en) 2023-07-25 2025-01-30 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
AR133384A1 (es) 2023-07-28 2025-09-24 Regeneron Pharma Anti-tfr:esfingomielinasa ácida para el tratamiento de la deficiencia de esfingomielinasa ácida
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
TW202515992A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對轉形生長因子β受體２型（ＴＧＦβＲ２）進行基因修飾之組合物及方法
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025049481A1 (en) 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods of editing an hla-a gene in vitro
WO2025054236A2 (en) 2023-09-06 2025-03-13 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
US20250268826A1 (en) 2023-09-18 2025-08-28 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025076291A1 (en) 2023-10-06 2025-04-10 Bluerock Therapeutics Lp Engineered type v rna programmable endonucleases and their uses
WO2025090926A1 (en) * 2023-10-26 2025-05-01 The Penn State Research Foundation Ultrasound-sensitive peptide particles with surface-bound gene editing tools for spatially resolved molecule delivery
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
US20250162981A1 (en) 2023-11-14 2025-05-22 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
WO2025106915A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 1 (glp-1) and uses thereof
WO2025106930A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 2 (glp-2) and uses thereof
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
WO2025117969A1 (en) * 2023-12-01 2025-06-05 Orna Therapeutics, Inc. Process for manufacturing lipid nanoparticles
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
US12553042B2 (en) 2023-12-01 2026-02-17 Recode Therapeutics, Inc. Method for quantifying an amount of capped messenger RNA
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
TW202542303A (zh) 2023-12-20 2025-11-01 美商英特利亞醫療公司 經工程改造之ｔ細胞
WO2025137301A1 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Methods for rapid engineering of cells
WO2025137646A1 (en) 2023-12-22 2025-06-26 Recode Therapeutics, Inc. Gene editing methods and compositions for treating cystic fibrosis
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
WO2025160334A1 (en) 2024-01-26 2025-07-31 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025166325A1 (en) * 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs
WO2025184520A1 (en) 2024-02-29 2025-09-04 Intellia Therapeutics, Inc. Compositions and methods for angiopoietin like 3 (angptl3) editing
US20250276092A1 (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
WO2025194019A1 (en) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Methods for treating liver fibrosis and non-alcoholic fatty liver disease
WO2025196065A1 (en) 2024-03-20 2025-09-25 Sanofi Novel homocysteine based lipids and their use for delivery of nucleic acids
US12460600B2 (en) 2024-03-26 2025-11-04 Ekstera Inc. Engine displacer with regenerator channels
US20250319206A1 (en) 2024-04-04 2025-10-16 Christina Care Gene Editing Institute, Inc. Crispr/cas gene editing of neh4 and/or neh5 domains in nrf2
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
WO2025226816A1 (en) 2024-04-23 2025-10-30 Christiana Care Gene Editing Institute, Inc. Methods of identifying and correlating crispr-induced exon skipping to phenotypic outcomes
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease
WO2025245111A1 (en) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Immunoreceptor targeting proteins and related methods
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy
US20250388890A1 (en) 2024-06-20 2025-12-25 Regeneron Pharmaceuticals, Inc. ASS1 Gene Insertion For The Treatment Of Citrullinemia Type I
WO2026006577A1 (en) 2024-06-26 2026-01-02 Flagship Pioneering Innovations Vii, Llc Therapeutic circular dna forms
WO2026008743A1 (en) 2024-07-02 2026-01-08 Sanofi Pasteur Inc. Water-soluble polyanionic polymer as adjuvant for carrier-formulated nucleic acid
WO2026033123A1 (en) 2024-08-08 2026-02-12 Sanofi Pasteur Inc. Lipid nanoparticle formulations for mrna delivery
WO2026055543A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Modified dna compositions and related methods
WO2026055547A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
EP2391343B1 (en) * 2009-01-29 2017-03-01 Arbutus Biopharma Corporation Improved lipid formulation for the delivery of nucleic acids
EP3504967A1 (en) * 2009-05-05 2019-07-03 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
US8691750B2 (en) * 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
CN110643600A (zh) * 2012-10-23 2020-01-03 基因工具股份有限公司 用于切割靶dna的系统及其用途
DK3327127T3 (da) * 2012-12-12 2021-06-28 Broad Inst Inc Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til sekvensmanipulation og terapeutiske anvendelser
CN105164102B (zh) 2013-03-08 2017-12-15 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
CA2914519A1 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
AU2014281028B2 (en) * 2013-06-17 2020-09-10 Massachusetts Institute Of Technology Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
CN106061466A (zh) * 2013-12-19 2016-10-26 诺华股份有限公司 瘦蛋白mRNA组合物和制剂
US10426737B2 (en) * 2013-12-19 2019-10-01 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
US10125092B2 (en) * 2014-09-05 2018-11-13 Novartis Ag Lipids and lipid compositions for the delivery of active agents
CN107002078A (zh) * 2014-10-09 2017-08-01 生命技术公司 Crispr寡核苷酸和基因剪辑

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