TW202242112A - 靶向hbv之類轉錄活化子效應物核酸酶(talen) - Google Patents

靶向hbv之類轉錄活化子效應物核酸酶(talen) Download PDF

Info

Publication number
TW202242112A
TW202242112A TW110149158A TW110149158A TW202242112A TW 202242112 A TW202242112 A TW 202242112A TW 110149158 A TW110149158 A TW 110149158A TW 110149158 A TW110149158 A TW 110149158A TW 202242112 A TW202242112 A TW 202242112A
Authority
TW
Taiwan
Prior art keywords
nucleic acid
hbv
sequence
acid sequence
seq
Prior art date
Application number
TW110149158A
Other languages
English (en)
Chinese (zh)
Inventor
雷蒙 迪亞茲 崔李斯
敏儀 林
立川潔
普里亞 普雷卡許 卡爾瑪里
帕德瑪納 奇福庫拉
Original Assignee
美商詹森藥物公司
美商亞克圖羅斯醫療公司
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 美商詹森藥物公司, 美商亞克圖羅斯醫療公司 filed Critical 美商詹森藥物公司
Publication of TW202242112A publication Critical patent/TW202242112A/zh

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0337Animal models for infectious diseases
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2730/00Reverse transcribing DNA viruses
    • C12N2730/00011Details
    • C12N2730/10011Hepadnaviridae
    • C12N2730/10111Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
TW110149158A 2020-12-28 2021-12-28 靶向hbv之類轉錄活化子效應物核酸酶(talen) TW202242112A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063131145P 2020-12-28 2020-12-28
US63/131,145 2020-12-28

Publications (1)

Publication Number Publication Date
TW202242112A true TW202242112A (zh) 2022-11-01

Family

ID=80034902

Family Applications (1)

Application Number Title Priority Date Filing Date
TW110149158A TW202242112A (zh) 2020-12-28 2021-12-28 靶向hbv之類轉錄活化子效應物核酸酶(talen)

Country Status (8)

Country Link
US (1) US20240316090A1 (enExample)
EP (1) EP4267740A1 (enExample)
JP (1) JP2024501022A (enExample)
AU (1) AU2021412833A1 (enExample)
CA (1) CA3203442A1 (enExample)
TW (1) TW202242112A (enExample)
UY (1) UY39592A (enExample)
WO (1) WO2022146654A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024211625A1 (en) * 2023-04-07 2024-10-10 Arcturus Therapeutics, Inc. Hbv gene editing
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0173552B1 (en) 1984-08-24 1991-10-09 The Upjohn Company Recombinant dna compounds and the expression of polypeptides such as tpa
US5168062A (en) 1985-01-30 1992-12-01 University Of Iowa Research Foundation Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence
US5273525A (en) 1992-08-13 1993-12-28 Btx Inc. Injection and electroporation apparatus for drug and gene delivery
US6041252A (en) 1995-06-07 2000-03-21 Ichor Medical Systems Inc. Drug delivery system and method
US6261281B1 (en) 1997-04-03 2001-07-17 Electrofect As Method for genetic immunization and introduction of molecules into skeletal muscle and immune cells
CA2285056C (en) 1997-04-03 2004-12-14 Iacob Mathiesen Method for introducing pharmaceutical drugs and nucleic acids into skeletal muscle
US5873849A (en) 1997-04-24 1999-02-23 Ichor Medical Systems, Inc. Electrodes and electrode arrays for generating electroporation inducing electrical fields
US6117660A (en) 1997-06-10 2000-09-12 Cytopulse Sciences, Inc. Method and apparatus for treating materials with electrical fields having varying orientations
AU8444798A (en) 1997-06-30 1999-01-25 Centre National De La Recherche Scientifique Improved method for transferring nucleic acid into the striped muscle and combination therefor
US6697669B2 (en) 1998-07-13 2004-02-24 Genetronics, Inc. Skin and muscle-targeted gene therapy by pulsed electrical field
US6319901B1 (en) 1998-10-15 2001-11-20 Ichor Medical Systems, Inc. Methods for prolonging cell membrane permeability
US8209006B2 (en) 2002-03-07 2012-06-26 Vgx Pharmaceuticals, Inc. Constant current electroporation device and methods of use
US7245963B2 (en) 2002-03-07 2007-07-17 Advisys, Inc. Electrode assembly for constant-current electroporation and use
US6912417B1 (en) 2002-04-05 2005-06-28 Ichor Medical Systmes, Inc. Method and apparatus for delivery of therapeutic agents
US7328064B2 (en) 2002-07-04 2008-02-05 Inovio As Electroporation device and injection apparatus
CA2532228C (en) 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
HUE025052T2 (en) 2004-03-08 2016-01-28 Ichor Medical Systems Inc A sharpened device for electrically mediated delivery of medical substances
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
US8304529B2 (en) 2006-07-28 2012-11-06 Life Technologies Corporation Dinucleotide MRNA cap analogs
CA2666501C (en) 2006-10-17 2017-03-21 Vgx Pharmaceuticals, Inc. Electroporation devices and methods of using same for electroporation of cells in mammals
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
WO2010048536A2 (en) 2008-10-23 2010-04-29 Alnylam Pharmaceuticals, Inc. Processes for preparing lipids
CN111808084A (zh) 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
SG10201911942UA (en) 2009-05-05 2020-02-27 Muthiah Manoharan Lipid compositions
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2011072246A2 (en) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Tal effector-mediated dna modification
KR20190039347A (ko) 2010-06-03 2019-04-10 알닐람 파마슈티칼스 인코포레이티드 활성제의 전달을 위한 생분해성 지질
MX342608B (es) 2010-07-06 2016-10-06 Novartis Ag * Particulas de suministro similares a viriones para moleculas de arn de autorreplicacion.
KR101556359B1 (ko) * 2011-01-03 2015-10-01 주식회사 툴젠 디자인된 tal 이펙터 뉴클레아제를 통한 게놈 엔지니어링
MX366055B (es) 2011-08-31 2019-06-26 Novartis Ag Liposomas pegilados para admistracion de acido ribonucleico (arn) que codifica para inmunogeno.
EP2817287B1 (en) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
JP6506749B2 (ja) 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのmRNA療法
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
CN120842296A (zh) 2015-09-21 2025-10-28 垂林克生物技术有限公司 用于合成5’-加帽rna的组合物和方法
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
WO2017117528A1 (en) 2015-12-30 2017-07-06 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
US9834510B2 (en) 2015-12-30 2017-12-05 Arcturus Therapeutics, Inc. Aromatic ionizable cationic lipid
AU2017241925B2 (en) 2016-03-28 2022-01-13 Ichor Medical Systems, Inc. Method and apparatus for delivery of therapeutic agents
US10487105B2 (en) 2016-10-19 2019-11-26 Arcturus Therapeutics, Inc. Trinucleotide MRNA cap analogs
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018119163A1 (en) 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
WO2018191657A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
CN110799492B (zh) 2017-04-28 2023-06-27 爱康泰生治疗公司 用于递送核酸的新型羰基脂质和脂质纳米颗粒制剂
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036030A1 (en) 2017-08-17 2019-02-21 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
EP4501322A3 (en) 2017-08-17 2025-04-16 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations

Also Published As

Publication number Publication date
AU2021412833A1 (en) 2023-07-06
EP4267740A1 (en) 2023-11-01
JP2024501022A (ja) 2024-01-10
CA3203442A1 (en) 2022-07-07
WO2022146654A1 (en) 2022-07-07
US20240316090A1 (en) 2024-09-26
UY39592A (es) 2022-06-30

Similar Documents

Publication Publication Date Title
US20220249654A1 (en) Nucleic acid based combination vaccines
JP2024502210A (ja) SARS-CoV-2バリアントに対するRNAワクチン
KR102305215B1 (ko) B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제
JP2023519173A (ja) 免疫応答を誘導するための組成物及び方法
WO2022043551A2 (en) Multivalent nucleic acid based coronavirus vaccines
US20230219996A1 (en) Rna vaccines
JP2023533528A (ja) Hbvに対するrnaレプリコンワクチン
US12186389B2 (en) Nucleic acid base vaccine against emerging SARS-CoV-2 variants
TW202242112A (zh) 靶向hbv之類轉錄活化子效應物核酸酶(talen)
WO2024211625A1 (en) Hbv gene editing
TW202438672A (zh) 四價流感疫苗之方法及組成物
WO2023218420A1 (en) Mrna compositions for inducing latent hiv-1 reversal
HK40110708A (zh) Rna疫苗
CN117440824A (zh) 抗SARS-CoV-2变体的RNA疫苗
HK40091626A (zh) 针对hbv的rna复制子疫苗