MX2022002342A - Composiciones y metodos para suministro de acidos nucleicos a celulas. - Google Patents

Composiciones y metodos para suministro de acidos nucleicos a celulas.

Info

Publication number
MX2022002342A
MX2022002342A MX2022002342A MX2022002342A MX2022002342A MX 2022002342 A MX2022002342 A MX 2022002342A MX 2022002342 A MX2022002342 A MX 2022002342A MX 2022002342 A MX2022002342 A MX 2022002342A MX 2022002342 A MX2022002342 A MX 2022002342A
Authority
MX
Mexico
Prior art keywords
nucleic acid
compositions
cells
methods
delivery
Prior art date
Application number
MX2022002342A
Other languages
English (en)
Inventor
Elias Quijano
Peter Glazer
Original Assignee
Univ Yale
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
Priority claimed from PCT/US2019/048962 external-priority patent/WO2020047353A1/en
Priority claimed from PCT/US2019/048953 external-priority patent/WO2020047344A1/en
Application filed by Univ Yale filed Critical Univ Yale
Publication of MX2022002342A publication Critical patent/MX2022002342A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/44—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material not provided for elsewhere, e.g. haptens, metals, DNA, RNA, amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6851—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a determinant of a tumour cell
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4747—Apoptosis related proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • 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
    • 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
    • C12N15/11—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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/31—Immunoglobulins specific features characterized by aspects of specificity or valency multispecific
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
    • C07K2317/565—Complementarity determining region [CDR]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77—Internalization into the cell
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
  • Developmental Biology & Embryology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se proporciona composiciones y métodos de uso de las mismas para suministrar carga de ácido nucleico en las células. Las composiciones típicamente incluyen (a) un anticuerpo monoclonal 3E10 o un fragmento de penetración en célula, de enlace de antígeno del mismo; un fragmento variable de una sola cadena (scFv) monovalente, divalente o multivalente; o un diacuerpo; o forma humanizada o variante del mismo y (b) una carga de ácido nucleico que incluye, por ejemplo, un ácido nucleico que codifica un polipéptido, un ácido nucleico funcional, un ácido nucleico que codifica un ácido nucleico funcional, o una combinación de los mismos. Los elementos (a) y (b) típicamente se enlazan no covalentemente para formar un complejo.
MX2022002342A 2019-08-30 2020-08-31 Composiciones y metodos para suministro de acidos nucleicos a celulas. MX2022002342A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
PCT/US2019/048962 WO2020047353A1 (en) 2018-08-31 2019-08-30 Compositions and methods for enhancing triplex and nuclease-based gene editing
PCT/US2019/048953 WO2020047344A1 (en) 2018-08-31 2019-08-30 Compositions and methods for enhancing donor oligonucleotide-based gene editing
US201962944281P 2019-12-05 2019-12-05
PCT/US2020/048823 WO2021042060A1 (en) 2019-08-30 2020-08-31 Compositions and methods for delivery of nucleic acids to cells

Publications (1)

Publication Number Publication Date
MX2022002342A true MX2022002342A (es) 2022-06-14

Family

ID=74684750

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022002342A MX2022002342A (es) 2019-08-30 2020-08-31 Composiciones y metodos para suministro de acidos nucleicos a celulas.

Country Status (10)

Country Link
US (5) US20230227583A1 (es)
EP (1) EP4021496A1 (es)
JP (1) JP2022546699A (es)
KR (1) KR20220101073A (es)
CN (1) CN115151275A (es)
AU (1) AU2020336992A1 (es)
CA (1) CA3149421A1 (es)
IL (1) IL290862A (es)
MX (1) MX2022002342A (es)
WO (1) WO2021042060A1 (es)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115151275A (zh) 2019-08-30 2022-10-04 耶鲁大学 用于向细胞递送核酸的组合物和方法
EP4204002A1 (en) * 2020-08-31 2023-07-05 Yale University Compositions and methods for delivery of nucleic acids to cells
CN117295753A (zh) * 2020-12-04 2023-12-26 基那奥生物公司 用于将核酸递送到细胞的组合物和方法
JP2024508309A (ja) * 2021-03-03 2024-02-26 イエール ユニバーシティ 骨格筋疾患を治療するための組成物及び方法
EP4346907A1 (en) * 2021-05-25 2024-04-10 Transmab Pty Ltd Improved immunoglobulin i
KR20240043747A (ko) * 2021-07-02 2024-04-03 예일 유니버시티 암을 치료하기 위한 조성물 및 방법
CA3226284A1 (en) * 2021-07-08 2023-01-12 Temple University - Of The Commonwealth System Of Higher Education Oligonucleotides and viral untranslated region (utr) for increasing expression of target genes and proteins
AU2022340804A1 (en) * 2021-08-31 2024-03-21 Gennao Bio, Inc. Compositions and methods for treating cancers
WO2023168401A1 (en) * 2022-03-03 2023-09-07 Yale University Compositions and methods for treating disease

Family Cites Families (76)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4965204A (en) 1984-02-06 1990-10-23 The Johns Hopkins University Human stem cells and monoclonal antibodies
US4714680B1 (en) 1984-02-06 1995-06-27 Univ Johns Hopkins Human stem cells
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
ATE171185T1 (de) 1985-03-15 1998-10-15 Antivirals Inc Immunotestmittel für polynukleotid und verfahren
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US4812397A (en) 1987-02-10 1989-03-14 The Regents Of The University Of California MAB-anti-DNA related to nephritis
US5061620A (en) 1990-03-30 1991-10-29 Systemix, Inc. Human hematopoietic stem cell
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5527675A (en) 1993-08-20 1996-06-18 Millipore Corporation Method for degradation and sequencing of polymers which sequentially eliminate terminal residues
DE4331012A1 (de) 1993-09-13 1995-03-16 Bayer Ag Nukleinsäuren-bindende Oligomere mit N-Verzweigung für Therapie und Diagnostik
US5409813A (en) 1993-09-30 1995-04-25 Systemix, Inc. Method for mammalian cell separation from a mixture of cell populations
US5677136A (en) 1994-11-14 1997-10-14 Systemix, Inc. Methods of obtaining compositions enriched for hematopoietic stem cells, compositions derived therefrom and methods of use thereof
WO1997032602A1 (en) 1996-03-08 1997-09-12 The Regents Of The University Of California DELIVERY SYSTEM USING mAb 3E10 AND MUTANTS AND/OR FUNCTIONAL FRAGMENTS THEREOF
US5945337A (en) 1996-10-18 1999-08-31 Quality Biological, Inc. Method for culturing CD34+ cells in a serum-free medium
US5786571A (en) 1997-05-09 1998-07-28 Lexmark International, Inc. Wrapped temperature sensing assembly
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
AU5886000A (en) 1999-06-25 2001-01-31 Northwestern University Compositions, kits, and methods for modulating survival and differentiation of multi-potential hematopoietic progenitor cells
US6919208B2 (en) 2000-05-22 2005-07-19 The Children's Hospital Of Philadelphia Methods and compositions for enhancing the delivery of a nucleic acid to a cell
EP1873259B1 (en) 2000-12-01 2012-01-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. RNA interference mediated by 21 and 22nt RNAs
US7067617B2 (en) 2001-02-21 2006-06-27 The Scripps Research Institute Zinc finger binding domains for nucleotide sequence ANN
US20040197892A1 (en) 2001-04-04 2004-10-07 Michael Moore Composition binding polypeptides
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
DE102004042546A1 (de) * 2004-09-02 2006-03-09 Curevac Gmbh Kombinationstherapie zur Immunstimulation
JP2009520463A (ja) 2005-11-28 2009-05-28 ザ スクリプス リサーチ インスティテュート Ｔｎｎのための亜鉛フィンガー結合ドメイン
US20070154989A1 (en) 2006-01-03 2007-07-05 The Scripps Research Institute Zinc finger domains specifically binding agc
CN1986792A (zh) * 2006-12-14 2007-06-27 方炳良 致病细胞的外来抗原标记方法
WO2008091911A2 (en) 2007-01-22 2008-07-31 The Regents Of The University Of California Use of antibody conjugates
EP2443155B1 (en) * 2009-06-15 2017-03-29 Valerion Therapeutics, LLC Methods and compositions for treatment of myotubular myopathy using chimeric polypeptides comprising myotubularin 1 (mtm1) polypeptides
PT2816112T (pt) 2009-12-10 2018-11-20 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
WO2012135831A1 (en) * 2011-04-01 2012-10-04 Yale University Cell-penetrating anti-dna antibodies and uses thereof inhibit dna repair
AU2012240135B2 (en) 2011-04-08 2016-09-08 Baylor College Of Medicine Reversing the effects of the tumor microenvironment using chimeric cytokine receptors
US20130071414A1 (en) 2011-04-27 2013-03-21 Gianpietro Dotti Engineered cd19-specific t lymphocytes that coexpress il-15 and an inducible caspase-9 based suicide gene for the treatment of b-cell malignancies
US9272043B2 (en) 2011-12-02 2016-03-01 Yale University Enzymatic synthesis of poly(amine-co-esters) and methods of use thereof for gene delivery
WO2013082529A1 (en) 2011-12-02 2013-06-06 Yale University Enzymatic synthesis of poly(amine-co-esters) and methods of use thereof for gene delivery
WO2013138662A1 (en) 2012-03-16 2013-09-19 4S3 Bioscience, Inc. Antisense conjugates for decreasing expression of dmpk
US9732146B2 (en) 2012-03-30 2017-08-15 The United States Of America As Represented By The Department Of Veterans Affairs Antibody-mediated transduction of heat shock proteins into living cells
US9283272B2 (en) 2012-03-30 2016-03-15 The United States Of America As Represented By The Department Of Veterans Affairs Targeting intracellular target-binding determinants with intracellular antibodies
ES2960803T3 (es) 2012-05-25 2024-03-06 Univ California Métodos y composiciones para la modificación de ADN diana dirigida por RNA y para la modulación de la transcripción dirigida por RNA
CA2876734A1 (en) 2012-07-13 2014-01-16 The Trustees Of The University Of Pennsylvania Use of cart19 to deplete normal b cells to induce tolerance
CN116622704A (zh) 2012-07-25 2023-08-22 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
CN105189542A (zh) 2013-02-20 2015-12-23 瓦莱里昂治疗有限责任公司 用于治疗庞贝氏症的方法和组合物
US20150017120A1 (en) 2013-06-13 2015-01-15 Massachusetts Institute Of Technology Synergistic tumor treatment with extended-pk il-2 and adoptive cell therapy
JP6709733B2 (ja) 2014-01-13 2020-06-17 バレリオン セラピューティクス， エルエルシー 内在化部分
US10040867B2 (en) 2014-03-04 2018-08-07 Yale University Cell penetrating anti-guanosine antibody based therapy for cancers with Ras mutations
SG10202109752XA (en) 2014-04-07 2021-10-28 Novartis Ag Treatment of cancer using anti-cd19 chimeric antigen receptor
CA2989294A1 (en) * 2014-06-13 2015-12-17 Valerion Therapeutics, Llc Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
WO2016033324A1 (en) 2014-08-27 2016-03-03 Valerion Therapeutics, Llc Internalizing moieties for treatment of cancer
WO2016033321A1 (en) 2014-08-28 2016-03-03 Yale University Multivalent fragments of antibody 3e10 and methods of use thereof
WO2016081621A1 (en) 2014-11-18 2016-05-26 Yale University Formulations for targeted release of agents under low ph conditions and methods of use thereof
WO2017015101A1 (en) 2015-07-17 2017-01-26 University Of Washington Methods for maximizing the efficiency of targeted gene correction
WO2017015630A2 (en) 2015-07-23 2017-01-26 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
JP2019508037A (ja) 2016-02-16 2019-03-28 イェール ユニバーシティーＹａｌｅ Ｕｎｉｖｅｒｓｉｔ 標的化遺伝子編集を増強するための組成物およびその使用方法
MA45328A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
CA3027960C (en) 2016-06-15 2022-06-14 Yale University Antibody-mediated autocatalytic, targeted delivery of nanocarriers to tumors
WO2018187493A1 (en) 2017-04-04 2018-10-11 Yale University Compositions and methods for in utero delivery
US20200216568A1 (en) 2017-07-17 2020-07-09 Nucleus Therapeutics Pty. Ltd. Binding proteins 2
WO2019079215A1 (en) * 2017-10-16 2019-04-25 Aadigen, Llc PEPTIDES AND NANOPARTICLES FOR INTRACELLULAR MRRNA DELIVERY
US20210054102A1 (en) 2018-02-01 2021-02-25 Yale University Compositions and methods for enhancing nuclear translocation
US20210137960A1 (en) 2018-02-01 2021-05-13 Yale University Compositions and methods for inhibition of nuclear-penetrating antibodies
EP3844287B1 (en) 2018-08-31 2024-05-22 Yale University Compositions and methods for enhancing donor oligonucleotide-based gene editing
KR20210054547A (ko) 2018-08-31 2021-05-13 예일 유니버시티 삼중체 및 뉴클레아제 기반 유전자 편집을 향상시키기 위한 조성물 및 방법
FI20186112A1 (en) 2018-12-19 2020-06-20 Actim Oy SYSTEM AND METHOD FOR ANALYZING THE RESULT OF THE CARE SITE TEST
CN115151275A (zh) 2019-08-30 2022-10-04 耶鲁大学 用于向细胞递送核酸的组合物和方法
CN111218458B (zh) 2020-02-27 2020-11-20 珠海丽凡达生物技术有限公司 编码SARS-CoV-2病毒抗原的mRNA和疫苗及疫苗的制备方法

Also Published As

Publication number Publication date
US20230227583A1 (en) 2023-07-20
JP2022546699A (ja) 2022-11-07
US11872286B2 (en) 2024-01-16
KR20220101073A (ko) 2022-07-19
CN115151275A (zh) 2022-10-04
EP4021496A1 (en) 2022-07-06
US11850284B2 (en) 2023-12-26
US20230093460A1 (en) 2023-03-23
WO2021042060A1 (en) 2021-03-04
CA3149421A1 (en) 2021-03-04
US20230085308A1 (en) 2023-03-16
AU2020336992A1 (en) 2022-04-14
US20230032060A1 (en) 2023-02-02
IL290862A (en) 2022-04-01
US20230093888A1 (en) 2023-03-30

Similar Documents

Publication Publication Date Title
MX2022002342A (es) Composiciones y metodos para suministro de acidos nucleicos a celulas.
MX2023006403A (es) Composiciones y métodos para el suministro de ácidos nucleicos a las células.
JOP20180042A1 (ar) نطاقات ربط مولد ضد متوافقة مع البشر وطرق الاستخدام
AR106184A1 (es) Proteínas de unión a pd-1 y sus métodos de uso
CY1118709T1 (el) Αντιγονικα πεπτιδια του παραγοντα διεγερσης αποικιων των κοκκινοκυτταρων (gm-csf) και αντισωματα για ton gm-csf
AU2018256487A1 (en) Antibodies that bind to TL1A and their uses
WO2016207304A8 (en) Monoclonal anti-il-1racp antibodies
EP4257611A3 (en) Anti-muc1 antibody
MX2023002480A (es) "composiciones y métodos para el suministro de ácidos nucleicos a las células.
AR084141A1 (es) Anticuerpos neutralizadores anti-ccl20
AR096617A1 (es) Anticuerpos anti-tweakr y sus usos
PE20120475A1 (es) Anticuerpos especificos para dkk-1
NZ603226A (en) Anti-c5a antibodies and methods for using the antibodies
NZ630610A (en) Tau peptides, anti-tau antibodies, and methods of use thereof
MY196874A (en) Humanized anti-basigin antibodies and the use thereof
UA120048C2 (uk) Антигензв'язуючий фрагмент антитіла, який специфічно зв'язується з рецептором інсуліноподібного фактора росту 1 (igf1r)
EA201691782A1 (ru) Антитела, специфические к рецептору инсулиноподобного фактора роста 1, и их применения
MX2022001942A (es) Moléculas de union a antígeno multiespecíficas para el direcciónamiento célular y usos de las mismas.
MX2022011951A (es) Anticuerpos de la lectina 15 similar a la inmunoglobulina que se une a acido sialico (siglec15) y usos de los mismos.
AR114281A1 (es) Anticuerpos anti-klk5 y métodos de uso
JOP20210286A1 (ar) جسم مضاد أحادي النسيلة يرتبط نوعياً مع gitr
JP2017511152A5 (es)
MX2022003074A (es) Anticuerpos anti-cd371, y usos de los mismos.
WO2024081736A3 (en) Compositions and methods of using cell-penetrating antibodies
PE20220763A1 (es) Materiales y metodos para modular la inmunidad mediada por celulas t