[go: up one dir, main page]

MX2023001109A - Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas. - Google Patents

Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.

Info

Publication number
MX2023001109A
MX2023001109A MX2023001109A MX2023001109A MX2023001109A MX 2023001109 A MX2023001109 A MX 2023001109A MX 2023001109 A MX2023001109 A MX 2023001109A MX 2023001109 A MX2023001109 A MX 2023001109A MX 2023001109 A MX2023001109 A MX 2023001109A
Authority
MX
Mexico
Prior art keywords
methods
dna molecules
compositions
therefor
making therefor
Prior art date
Application number
MX2023001109A
Other languages
English (en)
Inventor
Beer Joel De
Monique Maurer
Nicolas Meier
Lavaniya Kunalingam
Marcello Clerici
Original Assignee
Anjarium Biosciences Ag
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 Anjarium Biosciences Ag filed Critical Anjarium Biosciences Ag
Publication of MX2023001109A publication Critical patent/MX2023001109A/es

Links

Classifications

    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal 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
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00Preparation of compounds containing saccharide radicals
    • C12P19/26Preparation of nitrogen-containing carbohydrates
    • C12P19/28N-glycosides
    • C12P19/30Nucleotides
    • C12P19/34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6844Nucleic acid amplification reactions
    • C12Q1/6853Nucleic acid amplification reactions using modified primers or templates
    • 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
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/533Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • 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
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • 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
    • C12N2750/14171Demonstrated in vivo effect
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • 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
    • C12N2820/00Vectors comprising a special origin of replication system
    • C12N2820/60Vectors comprising a special origin of replication system from viruses

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Analytical Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

La presente invención se refiere a moléculas de ADN de doble cadena que comprenden repeticiones invertidas, casete de expresión y uno o más sitios de restricción para endonucleasas de recorte, los métodos de uso de las mismas y los métodos para manufacturarlas.
MX2023001109A 2020-07-27 2021-07-26 Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas. MX2023001109A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063057179P 2020-07-27 2020-07-27
US202163139486P 2021-01-20 2021-01-20
PCT/EP2021/070884 WO2022023284A1 (en) 2020-07-27 2021-07-26 Compositions of dna molecules, methods of making therefor, and methods of use thereof

Publications (1)

Publication Number Publication Date
MX2023001109A true MX2023001109A (es) 2023-04-24

Family

ID=77431273

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023001109A MX2023001109A (es) 2020-07-27 2021-07-26 Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.

Country Status (11)

Country Link
US (3) US20230272432A1 (es)
EP (1) EP4189098A1 (es)
JP (1) JP2023535632A (es)
KR (1) KR20230056669A (es)
CN (1) CN116234917A (es)
AU (1) AU2021314809A1 (es)
BR (1) BR112023001648A2 (es)
CA (1) CA3189740A1 (es)
IL (1) IL299925A (es)
MX (1) MX2023001109A (es)
WO (1) WO2022023284A1 (es)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015110957A2 (en) 2014-01-21 2015-07-30 De Beer Joel Hybridosomes, compositions comprising the same, processes for their production and uses thereof
CA3108799A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
US20230272432A1 (en) 2020-07-27 2023-08-31 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
AU2022334714A1 (en) * 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
EP4392443A1 (en) * 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Optimized factor viii genes
US20250207206A1 (en) * 2022-03-31 2025-06-26 The Board Of Trustees Of The Leland Stanford Junior University Modification-dependent enrichment of dna by genome of origin
AU2023253718A1 (en) * 2022-04-15 2024-11-28 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
CN119866375A (zh) 2022-07-19 2025-04-22 朗帕特生物科学公司 非免疫原性环状非病毒dna载体
EP4665865A1 (en) 2023-02-17 2025-12-24 Anjarium Biosciences AG Methods of making dna molecules and compositions and uses thereof

Family Cites Families (156)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0018120D0 (en) 2000-07-24 2000-09-13 Fermentas Ab Nuclease
US6660475B2 (en) * 2000-12-15 2003-12-09 New England Biolabs, Inc. Use of site-specific nicking endonucleases to create single-stranded regions and applications thereof
WO2003055975A2 (en) 2001-08-23 2003-07-10 New England Biolabs, Inc. A method for engineering strand-specific, sequence-specific, dna-nicking enzymes
US7011966B2 (en) 2003-04-16 2006-03-14 New England Biolabs, Inc. Method for cloning and expression of AcuI restriction endonuclease and AcuI methylase in E. coli
US7943303B2 (en) 2003-12-18 2011-05-17 New England Biolabs, Inc. Method for engineering strand-specific nicking endonucleases from restriction endonucleases
EP1766035B1 (en) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
WO2005120152A2 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
US7820424B2 (en) 2004-07-22 2010-10-26 New England Biolabs, Inc. Nicking endonuclease methods and compositions
PL1830888T3 (pl) 2004-12-27 2015-12-31 Silence Therapeutics Gmbh Kompleksy lipidowe pokryte PEG i ich wykorzystanie
EP1885863B1 (en) 2005-05-31 2014-11-19 The Regents of the University of Colorado, a body corporate Methods for delivering genes
AU2007303205A1 (en) 2006-10-03 2008-04-10 Tekmira Pharmaceuticals Corporation Lipid containing formulations
EP3100718B1 (en) 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
US20090305272A1 (en) * 2008-01-04 2009-12-10 Zhengne Wang Method of characterizing endogenous polynucleotide-polypeptide interactions
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
WO2009132131A1 (en) 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
US8703489B2 (en) 2008-08-22 2014-04-22 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
EP2350043B9 (en) 2008-10-09 2014-08-20 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
US8709778B2 (en) 2008-10-28 2014-04-29 Xavier Danthinne Method of adenoviral vector synthesis
CN111909020A (zh) 2008-11-10 2020-11-10 阿布特斯生物制药公司 用于递送治疗剂的脂质和组合物
WO2010054384A1 (en) 2008-11-10 2010-05-14 Alnylam Pharmaceuticals, Inc. Lipids and compositions for the delivery of therapeutics
US20120101148A1 (en) 2009-01-29 2012-04-26 Alnylam Pharmaceuticals, Inc. lipid formulation
KR20240015723A (ko) 2009-05-05 2024-02-05 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
CA2764609C (en) 2009-06-10 2018-10-02 Alnylam Pharmaceuticals, Inc. Improved cationic lipid of formula i
WO2011000106A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Improved cationic lipids and methods for the delivery of therapeutic agents
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
EP2467357B1 (en) 2009-08-20 2016-03-30 Sirna Therapeutics, Inc. Novel cationic lipids with various head groups for oligonucleotide delivery
EP2480668A2 (en) 2009-09-23 2012-08-01 Protiva Biotherapeutics Inc. Compositions and methods for silencing genes expressed in cancer
EP2506879A4 (en) 2009-12-01 2014-03-19 Protiva Biotherapeutics Inc PREPARATIONS OF SNALP CONTAINING ANTIOXIDANTS
AU2010328336B2 (en) 2009-12-07 2017-03-02 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
US9670487B2 (en) 2010-01-22 2017-06-06 Sirna Therapeutics, Inc. Cationic lipids for oligonucleotide delivery
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
EP2575767B1 (en) 2010-06-04 2017-01-04 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
CN103237889B (zh) 2010-08-16 2017-04-05 萨克生物研究学院 腺病毒组装方法
EP4066857B1 (en) 2010-08-31 2022-12-21 GlaxoSmithKline Biologicals SA Pegylated liposomes for delivery of immunogen-encoding rna
WO2012028681A1 (en) 2010-09-02 2012-03-08 Molmed Spa Stable production of lentiviral vectors
JP5897576B2 (ja) 2010-09-02 2016-03-30 モルメド エスピーエー レンチウイルスベクターの半−安定産生
AU2011305617A1 (en) 2010-09-20 2013-02-21 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
CN103260611A (zh) 2010-09-30 2013-08-21 默沙东公司 用于寡核苷酸递送的低分子量阳离子脂质
US9029590B2 (en) 2010-10-21 2015-05-12 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
EP3202760B1 (en) 2011-01-11 2019-08-21 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
EP2673286B1 (en) 2011-02-12 2019-07-03 University of Iowa Research Foundation Therapeutic compounds
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
WO2012162210A1 (en) 2011-05-26 2012-11-29 Merck Sharp & Dohme Corp. Ring constrained cationic lipids for oligonucleotide delivery
RU2014104090A (ru) 2011-07-06 2015-08-20 Новартис Аг Липосомы с эффективным соотношением n:p для доставки молекул phk
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
SI2750707T1 (sl) 2011-08-31 2019-02-28 Glaxosmithkline Biologicals Sa Pegilirani liposomi za dostavo imunogen-kodirajoče RNA
JP6250543B2 (ja) 2011-09-27 2017-12-20 アルニラム・ファーマシューティカルズ・インコーポレーテッド ジ脂肪族置換peg化脂質
ES2921724T1 (es) 2011-12-07 2022-08-31 Alnylam Pharmaceuticals Inc Lípidos biodegradables para la administración de agentes activos
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
JP6305343B2 (ja) 2011-12-07 2018-04-04 アルニラム・ファーマシューティカルズ・インコーポレーテッド 活性薬剤の送達のための分岐アルキルおよびシクロアルキルを末端とする生分解性脂質
TWI594767B (zh) 2011-12-12 2017-08-11 協和醱酵麒麟有限公司 含有陽離子性脂質之藥物傳遞系統用脂質奈米粒子
WO2013116126A1 (en) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Novel low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
RS58077B1 (sr) 2012-02-24 2019-02-28 Arbutus Biopharma Corp Trialkil katjonski lipidi i postupci njihove primene
EP2830594B1 (en) 2012-03-27 2018-05-09 Sirna Therapeutics, Inc. DIETHER BASED BIODEGRADABLE CATIONIC LIPIDS FOR siRNA DELIVERY
JP6449175B2 (ja) 2013-02-15 2019-01-09 ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド バイ ザ セクレタリー, デパートメント オブ ヘルス アンド ヒューマン サービシーズ 眼の遺伝子関連疾患の治療のための方法及び組成物
EP3517612A1 (en) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
WO2014197624A1 (en) 2013-06-07 2014-12-11 The Regents Of The University Of California Cycle adenosine monophosphate-incompetent adenylyl cyclase and compositions and methods for treating heart failure and increasing cardiac function
AU2014340155B2 (en) 2013-10-22 2018-11-01 Massachusetts Institute Of Technology Lipid formulations for delivery of messenger RNA
CN105873902B (zh) 2013-11-18 2019-03-08 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
PL3083556T3 (pl) 2013-12-19 2020-06-29 Novartis Ag Lipidy i kompozycje lipidowe dla dostarczania środków czynnych
WO2015095346A1 (en) 2013-12-19 2015-06-25 Novartis Ag Lipids and lipid compositions for the delivery of active agents
WO2015110957A2 (en) 2014-01-21 2015-07-30 De Beer Joel Hybridosomes, compositions comprising the same, processes for their production and uses thereof
EP3160938B1 (en) 2014-06-25 2020-09-16 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
CN104095665B (zh) 2014-07-15 2016-06-08 中国人民解放军第二军医大学 膜式输尿管结石阻挡取出器
WO2016033338A1 (en) 2014-08-27 2016-03-03 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Methods and compositions for treating leber congenital amaurosis
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
LT3221293T (lt) 2014-11-18 2023-03-27 Arcturus Therapeutics, Inc. Jonizuojamas katijoninis lipidas, skirtas rnr pristatymui
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EP3632923A1 (en) 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
GB201502645D0 (en) 2015-02-17 2015-04-01 Touchlight Genetics Ltd Method
AU2016285852B2 (en) 2015-06-29 2020-12-17 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
ES2969082T3 (es) 2015-09-17 2024-05-16 Modernatx Inc Compuestos y composiciones para la administración intracelular de agentes terapéuticos
US11230720B2 (en) 2015-10-14 2022-01-25 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
FI3368507T3 (fi) 2015-10-28 2023-03-21 Acuitas Therapeutics Inc Uusia lipidejä ja lipidinanopartikkeliformulaatioita nukleiinihappojen annostelemiseksi
LT3386484T (lt) 2015-12-10 2022-06-10 Modernatx, Inc. Gydomųjų medžiagų sudėtis ir pristatymo metodai
WO2017117528A1 (en) 2015-12-30 2017-07-06 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
PL3405215T3 (pl) 2016-01-19 2022-10-31 The Regents Of The University Of California Sposoby leczenia choroby danona i innych zaburzeń autofagii
MX2018010633A (es) 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
WO2018004514A1 (en) 2016-06-27 2018-01-04 Nokia Solutions And Networks Oy Duplex distance modification and blank nb-iot subcarriers
US20180020547A1 (en) 2016-07-13 2018-01-18 Alcatel-Lucent Canada Inc. Underlying recessed component placement
CN110139933B (zh) 2016-11-09 2024-03-08 英特瑞克斯顿股份有限公司 共济蛋白表达构建体
WO2018187552A1 (en) 2017-04-05 2018-10-11 University Of Massachusetts Minigene therapy
WO2018191450A2 (en) 2017-04-14 2018-10-18 National Taiwan University Hospital Gene therapy for aadc deficiency
TWI897844B (zh) 2017-05-05 2025-09-21 美商航海家醫療公司 調節多核苷酸
CA3063907A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type iii
CN109252337B (zh) * 2017-07-14 2021-06-04 重庆海尔滚筒洗衣机有限公司 一种滚筒洗衣机
KR102850887B1 (ko) 2017-08-03 2025-08-28 보이저 테라퓨틱스, 인크. Aav의 전달을 위한 조성물 및 방법
TWI904068B (zh) 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
EP3665290A1 (en) 2017-08-09 2020-06-17 The U.S.A. As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus dna, and uses thereof
MX2020002500A (es) 2017-09-08 2020-09-17 Generation Bio Co Adn de extremo cerrado modificado (adnec).
SG11202000765PA (en) 2017-09-08 2020-03-30 Generation Bio Co Lipid nanoparticle formulations of non-viral, capsid-free dna vectors
KR20200060456A (ko) 2017-09-27 2020-05-29 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항-VEGF Fab를 이용한 안구 질환의 치료
GB2568255A (en) 2017-11-08 2019-05-15 Evox Therapeutics Ltd Exosomes comprising RNA therapeutics
GB201718681D0 (en) 2017-11-13 2017-12-27 Evox Therapeutics Ltd Protein engineered extracellular vesicles
CA3084185A1 (en) * 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
WO2019140353A1 (en) 2018-01-12 2019-07-18 Camena Bioscience Limited Compositions and methods for template-free geometric enzymatic nucleic acid synthesis
MX2020005790A (es) * 2018-01-19 2020-10-28 Generation Bio Co Vectores de adn de extremo cerrado que pueden obtenerse de síntesis libre de células y un proceso para obtener vectores de ceadn.
GB201800921D0 (en) 2018-01-19 2018-03-07 Evox Therapeutics Ltd Intracellular delivery of target silencing technology
US20220042035A1 (en) 2018-02-14 2022-02-10 Generation Bio Co. Non-viral dna vectors and uses thereof for antibody and fusion protein production
EP3755803A4 (en) 2018-02-22 2022-01-19 Generation Bio Co. REGULATED EXPRESSION OF TRANSGENES USING CLOSED-ENDED DNA VECTOR (CEDNA)
MA52116A (fr) * 2018-03-02 2021-01-06 Generation Bio Co Vecteurs d'adn à extrémité fermée (cedna) pour l'insertion de transgènes au niveau de havres génomiques sécuritaires (gsh) dans des génomes humains et murins
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
CA3098807A1 (en) 2018-05-08 2019-11-14 Neuracle Science Co., Ltd. Adeno-associated virus (aav) delivery of anti-fam19a5 antibodies
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3796893A1 (en) 2018-05-23 2021-03-31 Modernatx, Inc. Delivery of dna
EP3810782A2 (en) 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
AU2019307911B2 (en) 2018-07-20 2022-06-09 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Intraocular delivery of gene therapy expression vectors
CN112654698A (zh) 2018-08-08 2021-04-13 吉尼松公司 用于治疗糖原贮积病iii的小型gde
CA3108799A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
CA3117738A1 (en) 2018-10-24 2020-04-30 Bristol-Myers Squibb Company Miniaturized dystrophins and uses thereof
CN113316640A (zh) 2018-11-09 2021-08-27 世代生物公司 包含对称的被修饰的反向末端重复序列的被修饰的封闭端dna(cedna)
EP3911757A2 (en) 2019-01-14 2021-11-24 Camena Bioscience Limited Compositions and methods for template-free geometric enzymatic nucleic acid synthesis
CA3127799A1 (en) 2019-01-24 2020-07-30 Generation Bio Co. Close-ended dna (cedna) and use in methods of reducing gene or nucleic acid therapy related immune response
EP3924477A4 (en) 2019-02-14 2023-03-29 Metagenomi, Inc. ENZYMES WITH RUVC DOMAINS
CN113454232B (zh) 2019-02-15 2024-10-29 世代生物公司 封闭端dna(cedna)产生中对rep蛋白活性的调节
MA55219A (fr) * 2019-03-06 2022-01-12 Generation Bio Co Nanoparticules lipidiques non actives avec adn dépourvu de capside, non viral
MA55223A (fr) 2019-03-06 2022-01-12 Generation Bio Co Adn à extrémité fermée (cedna) et composés de modulation immunitaire
BR112021017853A2 (pt) 2019-03-13 2021-11-30 Generation Bio Co Vetores de dna não virais e usos dos mesmos para expressar terapêuticas de fviii
EP3938515A4 (en) 2019-03-13 2022-12-28 Generation Bio Co. NON-VIRAL DNA VECTORS AND THEIR USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) THERAPEUTIC AGENTS
WO2020198233A1 (en) 2019-03-25 2020-10-01 The Regents Of The University Of California Methods of treating tnni3-mediated cardiomyopathy
CA3136720A1 (en) 2019-04-17 2020-10-22 Codiak Biosciences, Inc. Compositions of exosomes and aav
CA3137135A1 (en) 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for stargardt disease (abca4)
EP3956452A4 (en) 2019-04-19 2023-03-15 University of Massachusetts GENE THERAPIES FOR USHER SYNDROME (USH2A)
EP3956453A4 (en) 2019-04-19 2023-02-22 University of Massachusetts GENE THERAPIES FOR USHER SYNDROME (USH1B)
WO2020219941A1 (en) 2019-04-26 2020-10-29 Genevant Sciences Gmbh Lipid nanoparticles
EP3959324B1 (en) 2019-04-26 2025-09-17 President and Fellows of Harvard College Aav vectors encoding mini-pcdh15 and uses thereof
EP3972610B1 (en) 2019-05-20 2025-09-03 University of Massachusetts Minigene therapy
WO2020257590A1 (en) 2019-06-21 2020-12-24 Asklepios Biopharmaceutical, Inc. Production of vectors using phage origin of replication
AU2020300519A1 (en) 2019-06-30 2022-02-24 John Fraser Wright Recombinant AAV vectors with altered immunogencity and methods of making the same
US20220228171A1 (en) 2019-07-17 2022-07-21 Generation Bio Co. Compositions and production of nicked closed-ended dna vectors
WO2021011842A1 (en) 2019-07-17 2021-01-21 Generation Bio Co. Synthetic production of single-stranded adeno associated viral dna vectors
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
EP4013879A4 (en) 2019-08-12 2023-09-13 Generation Bio Co. METHODS AND COMPOSITIONS FOR REDUCING IMMUNE RESPONSES ASSOCIATED WITH GENE OR NUCLEIC ACID THERAPY
GEP20257820B (en) 2019-08-14 2025-11-10 Acuitas Therapeutics Inc Improved lipid nanoparticles for delivery of nucleic acids
US20220280603A1 (en) 2019-08-15 2022-09-08 University Of Massachusetts Targeting of the cytoskeleton as a therapeutic approach for neurodegenerative disease
US20220280427A1 (en) 2019-09-06 2022-09-08 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
EP3792367A1 (en) 2019-09-11 2021-03-17 Universität Bielefeld Method for the production of raav and method for the in vitro generation of genetically engineered, linear, single-stranded nucleic acid fragments containing itr sequences flanking a gene of interest
JP7713130B2 (ja) 2019-09-30 2025-07-25 イーライ リリー アンド カンパニー 遺伝性難聴の処置のためのアデノ随伴ウイルス(aav)システム
WO2021072201A1 (en) 2019-10-11 2021-04-15 University Of Massachusetts Raav-mediated in vivo delivery of suppressor trnas
WO2021076634A1 (en) 2019-10-15 2021-04-22 University Of Massachusetts Aav production strategy using a cell line expressing an inducible rep gene
WO2021076566A1 (en) 2019-10-16 2021-04-22 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Compositions and methods for treating liver disease
US20230040275A1 (en) 2019-11-19 2023-02-09 Spark Therapeutics, Inc. Secretable protein induced immune tolerization and treatment of autoimmune, allergic and other diseases and disorders
WO2021102390A1 (en) 2019-11-22 2021-05-27 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
CN114787127B (zh) 2019-11-22 2024-04-26 世代生物公司 可电离脂质及其纳米颗粒组合物
US20230057380A1 (en) 2019-11-26 2023-02-23 University Of Massachusetts Recombinant adeno-associated virus for delivery of kh902 (conbercept) and uses thereof
US20230272432A1 (en) 2020-07-27 2023-08-31 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof

Also Published As

Publication number Publication date
US11634742B2 (en) 2023-04-25
BR112023001648A2 (pt) 2023-04-04
CN116234917A (zh) 2023-06-06
US20230323418A1 (en) 2023-10-12
KR20230056669A (ko) 2023-04-27
US20220098633A1 (en) 2022-03-31
US20230272432A1 (en) 2023-08-31
EP4189098A1 (en) 2023-06-07
AU2021314809A1 (en) 2023-02-23
IL299925A (en) 2023-03-01
WO2022023284A1 (en) 2022-02-03
JP2023535632A (ja) 2023-08-18
CA3189740A1 (en) 2022-02-03

Similar Documents

Publication Publication Date Title
MX2023001109A (es) Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
CA3197311A1 (en) Oligonucleotide compositions and methods thereof
PH12021500030A1 (en) Il-2 conjugates and methods of use thereof
WO2021127283A3 (en) Irak degraders and uses thereof
PH12022551524A1 (en) Irak degraders and uses thereof
WO2020243415A3 (en) Tead inhibitors and uses thereof
EP4349413A3 (en) Combination therapies for treating myelodysplastic syndromes and acute myeloid leukemia
MX2021014441A (es) Inhibidores del dominio asociado mejorador de la transcripcion (tead) y usos de los mismos.
MX2023012245A (es) Compuestos, composiciones y metodos para el tratamiento del cancer.
WO2020010250A3 (en) Anti-tcr antibody molecules and uses thereof
MY203849A (en) Compositions and methods for ttr gene editing and treating attr amyloidosis
EP4389889A3 (en) Compositions and methods for editing rna
PH12022551522A1 (en) Smarca degraders and uses thereof
MX2021015411A (es) Composiciones acopladoras especificas de macrofagos y metodos de uso de las mismas.
WO2021055577A3 (en) Anti-klk7 antibodies, anti-klk5 antibodies, multispecific anti-klk5/klk7 antibodies, and methods of use
MY209474A (en) Il-2 conjugates and methods of use to treat autoimmune diseases
MX2021003187A (es) Inhibición de la peptidasa específica de ubiquitina 9x.
PH12021500009A1 (en) Rna molecules comprising non-canonical base pairs
PH12023500023A1 (en) Cdk2 degraders and uses thereof
MX363846B (es) Moduladores de oligonucleotido de linfoma linfocitico cronico/linfoma de celulas b 11a (bcl11a) y sus usos.
AR119366A1 (es) Composiciones y métodos para la estimulación con espuma
WO2020051375A3 (en) Papd5 inhibitors and methods of use thereof
WO2020092557A3 (en) Methods for altering gene expression for genetic disorders
ZA202204072B (en) Co-delivery of tgf-β sirna and pdl1 sirna to treat cancer
PH12022550130A1 (en) Enzyme inhibitors