CA3054808A1 - Compositions and methods for inhibition lineage specific proteins - Google Patents

Compositions and methods for inhibition lineage specific proteins Download PDF

Info

Publication number
CA3054808A1
CA3054808A1 CA3054808A CA3054808A CA3054808A1 CA 3054808 A1 CA3054808 A1 CA 3054808A1 CA 3054808 A CA3054808 A CA 3054808A CA 3054808 A CA3054808 A CA 3054808A CA 3054808 A1 CA3054808 A1 CA 3054808A1
Authority
CA
Canada
Prior art keywords
cells
lineage
cell
epitope
surface protein
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3054808A
Other languages
English (en)
French (fr)
Inventor
Joseph BOLEN
Aleksandar Filip Radovic-Moreno
John LYDEARD
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Vor Biopharma Inc
Original Assignee
Vor Biopharma Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Vor Biopharma Inc filed Critical Vor Biopharma Inc
Publication of CA3054808A1 publication Critical patent/CA3054808A1/en
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596Molecules with a "CD"-designation not provided for elsewhere
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14Blood; Artificial blood
    • A61K35/15Cells of the myeloid line, e.g. granulocytes, basophils, eosinophils, neutrophils, leucocytes, monocytes, macrophages or mast cells; Myeloid precursor cells; Antigen-presenting cells, e.g. dendritic cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14Blood; Artificial blood
    • A61K35/17Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/0005Vertebrate antigens
    • A61K39/0011Cancer antigens
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/395Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/39558Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against tumor tissues, cells, antigens
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • A61K40/11T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31Chimeric antigen receptors [CAR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/421Immunoglobulin superfamily
    • A61K40/4211CD19 or B4
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/4224Molecules with a "CD" designation not provided for elsewhere
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/68Medicinal 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
    • A61K47/6801Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6811Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
    • A61K47/6817Toxins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/68Medicinal 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
    • A61K47/6835Medicinal 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
    • A61K47/6851Medicinal 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
    • A61K47/6867Medicinal 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 the tumour determinant being from a cell of a blood cancer
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
    • C07K14/7051T-cell receptor (TcR)-CD3 complex
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
    • C07K14/70517CD8
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
    • C07K14/70521CD28, CD152
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/30Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
    • C07K16/3061Blood 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/505Medicinal preparations containing antigens or antibodies comprising antibodies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/515Animal cells
    • A61K2039/5156Animal cells expressing foreign proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/515Animal cells
    • A61K2039/5158Antigen-pulsed cells, e.g. T-cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00Preparations for use in therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/38Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/46Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the cancer treated
    • A61K2239/48Blood cells, e.g. leukemia or lymphoma
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/30Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/34Identification of a linear epitope shorter than 20 amino acid residues or of a conformational epitope defined by amino acid residues
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/60Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/73Inducing cell death, e.g. apoptosis, necrosis or inhibition of cell proliferation
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/33Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2510/00Genetically modified cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • General Engineering & Computer Science (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3054808A 2017-02-28 2018-02-28 Compositions and methods for inhibition lineage specific proteins Pending CA3054808A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762464975P 2017-02-28 2017-02-28
US62/464,975 2017-02-28
PCT/US2018/020327 WO2018160768A1 (en) 2017-02-28 2018-02-28 Compositions and methods for inhibition lineage specific proteins

Publications (1)

Publication Number Publication Date
CA3054808A1 true CA3054808A1 (en) 2018-09-07

Family

ID=63370206

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3054808A Pending CA3054808A1 (en) 2017-02-28 2018-02-28 Compositions and methods for inhibition lineage specific proteins

Country Status (13)

Country Link
US (2) US11975029B2 (enExample)
EP (1) EP3589291A4 (enExample)
JP (2) JP7386082B2 (enExample)
KR (1) KR102709603B1 (enExample)
CN (1) CN110662554A (enExample)
AU (2) AU2018229351B2 (enExample)
BR (1) BR112019017751A2 (enExample)
CA (1) CA3054808A1 (enExample)
IL (1) IL268895B2 (enExample)
MX (1) MX2019010196A (enExample)
RU (1) RU2019130504A (enExample)
SG (1) SG11201907870VA (enExample)
WO (1) WO2018160768A1 (enExample)

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4265633A3 (en) 2015-10-16 2024-01-24 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
BR112019017751A2 (pt) 2017-02-28 2020-04-07 Vor Biopharma, Inc. composições e métodos de inibição de proteínas de linhagem específica
WO2019046285A1 (en) * 2017-08-28 2019-03-07 The Trustees Of Columbia University In The City Of New York CD33 EXON 2 DONOR STEM CELLS FOR USE WITH CD33 TARGETING AGENTS
US12133884B2 (en) 2018-05-11 2024-11-05 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
SG11202101994XA (en) 2018-08-28 2021-03-30 Vor Biopharma Inc Genetically engineered hematopoietic stem cells and uses thereof
US11866494B2 (en) * 2018-08-31 2024-01-09 Innovative Cellular Therapeutics Holdings, Ltd. CAR T therapy through uses of co-stimulation
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
CN114026237B (zh) * 2019-02-13 2025-03-25 比姆医疗股份有限公司 用于治疗1a型糖原贮积病的组成物和方法
KR20210138603A (ko) * 2019-02-13 2021-11-19 빔 테라퓨틱스, 인크. 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기를 갖는 변형된 면역 세포
US20220202900A1 (en) * 2019-02-22 2022-06-30 The Trustees Of The University Of Pennsylvania Compositions and methods for crispr/cas9 knock-out of cd33 in human hematopoietic stem / progenitor cells for allogenic transplantation in patients with relapsed - refractory acute myeloid leukemia
AU2020280103A1 (en) 2019-05-23 2021-12-23 Vor Biopharma Inc. Compositions and methods for CD33 modification
US20220290160A1 (en) 2019-08-28 2022-09-15 Vor Biopharma Inc. Compositions and methods for cll1 modification
CA3151669A1 (en) 2019-08-28 2021-03-04 Vor Biopharma Inc. Compositions and methods for cd123 modification
WO2021058795A2 (en) * 2019-09-27 2021-04-01 Stark Labs Senescent cell-associated antigen-binding domains, antibodies and chimeric antigen receptors comprising the same, and uses thereof
US11723926B2 (en) 2019-09-27 2023-08-15 Starkage Therapeutics Senescent cell-associated antigen-binding domains, antibodies and chimeric antigen receptors comprising the same, and uses thereof
EP4048790A4 (en) * 2019-10-22 2024-03-27 Fred Hutchinson Cancer Center Base editor-mediated cd33 reduction to selectively protect therapeutic cells
EP4126248A4 (en) * 2020-03-31 2024-07-10 Fred Hutchinson Cancer Center HUMAN ANTI-CD33 ANTIBODIES AND THEIR USES
CN115348973A (zh) * 2020-03-31 2022-11-15 弗莱德哈钦森癌症中心 靶向cd33的嵌合抗原受体
US20240110189A1 (en) 2020-08-28 2024-04-04 Vor Biopharma Inc. Compositions and methods for cll1 modification
EP4204564A1 (en) 2020-08-28 2023-07-05 Vor Biopharma Inc. Compositions and methods for cd123 modification
JP2023541457A (ja) 2020-09-14 2023-10-02 ブイオーアール バイオファーマ インコーポレーテッド Cd38修飾のための化合物および方法
WO2022056459A1 (en) 2020-09-14 2022-03-17 Vor Biopharma, Inc. Compositions and methods for cd5 modification
EP4214318A1 (en) 2020-09-18 2023-07-26 Vor Biopharma Inc. Compositions and methods for cd7 modification
US20230364233A1 (en) 2020-09-28 2023-11-16 Vor Biopharma Inc. Compositions and methods for cd6 modification
US20230364146A1 (en) 2020-09-30 2023-11-16 Vor Biopharma Inc. Compositions and methods for cd30 gene modification
KR20230097089A (ko) * 2020-10-27 2023-06-30 보르 바이오파마 인크. 조혈 악성종양을 치료하기 위한 조성물 및 방법
WO2022094245A1 (en) 2020-10-30 2022-05-05 Vor Biopharma, Inc. Compositions and methods for bcma modification
US20230414755A1 (en) * 2020-11-13 2023-12-28 Vor Biopharma Inc. Methods and compositions relating to genetically engineered cells expressing chimeric antigen receptors
CA3202219A1 (en) 2020-12-31 2022-07-07 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
CN120249215A (zh) * 2021-04-27 2025-07-04 上海驯鹿生物技术有限公司 一种基因编辑的造血干细胞及其与car-t细胞的联合应用
CA3224408A1 (en) 2021-08-06 2023-02-09 Rosalba LEPORE Discernible cell surface protein variants for use in cell therapy
AU2022387087A1 (en) 2021-11-09 2024-05-02 Vor Biopharma Inc. Compositions and methods for erm2 modification
WO2023111311A1 (en) 2021-12-16 2023-06-22 Universität Basel Discernible cell surface protein variants of cd117 for use in cell therapy
KR20240119146A (ko) 2021-12-23 2024-08-06 유니버시타트 바셀 세포 치료에 이용하기 위한 cd45의 식별 가능한 세포 표면 단백질 변이체
EP4555091A2 (en) 2022-07-13 2025-05-21 Vor Biopharma Inc. Compositions and methods for artificial protospacer adjacent motif (pam) generation
CN118684759A (zh) * 2023-01-17 2024-09-24 武汉大学 用于治疗急性髓系白血病的成分和方法
WO2024208836A1 (en) 2023-04-03 2024-10-10 Universität Basel Discernible cell surface protein variants of cd33 for use in cell therapy
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof

Family Cites Families (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101903403B (zh) 2007-10-19 2016-03-16 西雅图基因公司 Cd19结合剂及其应用
PH12012500714A1 (en) 2009-10-19 2012-10-29 Tristem Trading Cyprus Ltd Treatment using reprogrammed mature adult cells
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
US20130309223A1 (en) 2012-05-18 2013-11-21 Seattle Genetics, Inc. CD33 Antibodies And Use Of Same To Treat Cancer
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
DK3004337T3 (da) 2013-05-29 2017-11-13 Cellectis Fremgangsmåde til konstruktion af T-celler til immunoterapi ved brug af RNA-guidet Cas nuklease-system
EP2833591A1 (en) 2013-07-31 2015-02-04 Siemens Aktiengesellschaft Subsea data communication interface unit
EP3087101B1 (en) 2013-12-20 2024-06-05 Novartis AG Regulatable chimeric antigen receptor
JP6673838B2 (ja) 2014-02-14 2020-04-01 セレクティスCellectis 免疫細胞と病的細胞の両方に存在する抗原を標的とするように操作された、免疫療法のための細胞
ES2740903T3 (es) 2014-03-19 2020-02-07 Cellectis Receptores antigénicos quiméricos específicos de CD123 para inmunoterapia del cáncer
ES2765710T3 (es) 2014-04-03 2020-06-10 Cellectis Receptores de antígeno quimérico específicos de CD33 para la inmunoterapia del cáncer
EP3556858A3 (en) 2014-04-09 2020-01-22 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
CA2945393C (en) 2014-04-24 2021-03-23 Board Of Regents, The University Of Texas System Application of induced pluripotent stem cells to generate adoptive cell therapy products
WO2015195547A1 (en) 2014-06-16 2015-12-23 University Of Washington Methods for controlling stem cell potential and for gene editing in stem cells
US9212225B1 (en) 2014-07-01 2015-12-15 Amphivena Therapeutics, Inc. Bispecific CD33 and CD3 binding proteins
CN107109420A (zh) 2014-07-21 2017-08-29 诺华股份有限公司 使用cll-1嵌合抗原受体的癌症治疗
BR112017001242A2 (pt) 2014-07-21 2017-12-05 Novartis Ag tratamento de câncer usando um receptor antigênico quimérico a cd33
CN106795488B (zh) 2014-09-16 2021-03-30 桑格摩治疗股份有限公司 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物
KR101799975B1 (ko) 2014-10-31 2017-11-21 에스케이씨하이테크앤마케팅(유) 액정표시장치용 색순도 향상 및 휘도 보상 필름 및 이를 포함하는 액정표시장치
ES2692206T3 (es) 2014-11-26 2018-11-30 Miltenyi Biotec Gmbh Inmunoterapia combinada de receptores de reconocimiento de antígenos y células hematopoyéticas para el tratamiento de enfermedades
CN107250148B (zh) 2014-12-03 2021-04-16 安捷伦科技有限公司 具有化学修饰的指导rna
US11459390B2 (en) 2015-01-16 2022-10-04 Novartis Ag Phosphoglycerate kinase 1 (PGK) promoters and methods of use for expressing chimeric antigen receptor
CA2973529A1 (en) 2015-01-26 2016-08-04 Cellectis Cll1-specific multi-chain chimeric antigen receptor
ES2884838T3 (es) 2015-04-06 2021-12-13 Univ Leland Stanford Junior ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS
IL254817B2 (en) * 2015-04-08 2023-12-01 Novartis Ag Cd20 therapies, cd22 therapies, and combination therapies with a cd19 chimeric antigen receptor (car) - expressing cell
AU2016251687C1 (en) 2015-04-22 2023-07-27 CureVac SE RNA containing composition for treatment of tumor diseases
GB201507119D0 (en) 2015-04-27 2015-06-10 Ucl Business Plc Nucleic Acid Construct
JP6985934B2 (ja) 2015-04-29 2021-12-22 フレッド ハッチンソン キャンサー リサーチ センター 操作された造血幹細胞/前駆細胞及び非tエフェクター細胞、ならびにその使用
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
JP6996983B2 (ja) 2015-06-16 2022-02-21 ジェネンテック, インコーポレイテッド 抗cll-1抗体及び使用方法
EP4265633A3 (en) 2015-10-16 2024-01-24 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
EP3370741B1 (en) 2015-11-04 2025-10-08 The Trustees of the University of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells
HK1258375A1 (zh) 2015-11-24 2019-11-08 Onk Therapeutics Limited 人源化抗-cll-1抗体
AU2017244108B2 (en) 2016-03-29 2021-03-18 University Of Southern California Chimeric antigen receptors targeting cancer
US11499168B2 (en) 2016-04-25 2022-11-15 Universitat Basel Allele editing and applications thereof
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
US11787848B2 (en) 2016-06-08 2023-10-17 Precigen, Inc. CD33 specific chimeric antigen receptors
EP4353750A3 (en) 2016-06-24 2024-07-24 iCell Gene Therapeutics LLC Chimeric antigen receptors (cars), compositions and methods thereof
AU2017291851B2 (en) 2016-07-06 2022-10-13 Cellectis Sequential gene editing in primary immune cells
ES2978486T3 (es) 2016-10-21 2024-09-13 Ose Immunotherapeutics Métodos para promover la respuesta de linfocitos T
US20190365806A1 (en) * 2016-11-02 2019-12-05 Universität Basel Immunologically discernible cell surface variants for use in cell therapy
KR102758180B1 (ko) 2016-12-30 2025-01-23 에디타스 메디신, 인코포레이티드 합성 가이드 분자, 그와 관련된 조성물 및 방법
BR112019017751A2 (pt) 2017-02-28 2020-04-07 Vor Biopharma, Inc. composições e métodos de inibição de proteínas de linhagem específica
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
CA3058790A1 (en) 2017-04-24 2018-11-01 Memorial Sloan Kettering Cancer Center Anti-cd33 antibody agents
WO2019046285A1 (en) 2017-08-28 2019-03-07 The Trustees Of Columbia University In The City Of New York CD33 EXON 2 DONOR STEM CELLS FOR USE WITH CD33 TARGETING AGENTS
US10704938B2 (en) 2018-01-31 2020-07-07 Hydroacoustics, Inc. Pumpjack production well including fluid sensor having 2-dimensional venturi and capacitive flow sensor
WO2019157326A1 (en) 2018-02-08 2019-08-15 Zymergen Inc. Genome editing using crispr in corynebacterium
TWI669677B (zh) 2018-03-07 2019-08-21 光陽工業股份有限公司 充電座管理方法、用於充電座管理方法的伺服器及充電座
SG11202101994XA (en) 2018-08-28 2021-03-30 Vor Biopharma Inc Genetically engineered hematopoietic stem cells and uses thereof
WO2020095107A1 (en) 2018-11-07 2020-05-14 Crispr Therapeutics Ag Anti-cd33 immune cell cancer therapy
AU2020209218A1 (en) 2019-01-16 2021-07-29 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
AU2020208616A1 (en) 2019-01-16 2021-08-12 Beam Therapeutics Inc. Modified immune cells having enhanced anti-neoplasia activity and immunosuppression resistance
KR20210138603A (ko) 2019-02-13 2021-11-19 빔 테라퓨틱스, 인크. 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기를 갖는 변형된 면역 세포
AU2020280103A1 (en) 2019-05-23 2021-12-23 Vor Biopharma Inc. Compositions and methods for CD33 modification
EA202191979A1 (ru) 2019-05-24 2021-11-01 Зе Трастис Оф Коламбия Юниверсити Ин Зе Сити Оф Нью-Йорк Композиции и способы ингибирования линиеспецифических антигенов
US20220290160A1 (en) 2019-08-28 2022-09-15 Vor Biopharma Inc. Compositions and methods for cll1 modification
CA3151669A1 (en) 2019-08-28 2021-03-04 Vor Biopharma Inc. Compositions and methods for cd123 modification

Also Published As

Publication number Publication date
EP3589291A1 (en) 2020-01-08
JP2020509050A (ja) 2020-03-26
MX2019010196A (es) 2019-12-19
BR112019017751A2 (pt) 2020-04-07
IL268895B1 (en) 2025-01-01
CN110662554A (zh) 2020-01-07
AU2018229351A1 (en) 2019-09-26
IL268895A (en) 2019-10-31
US20200030381A1 (en) 2020-01-30
RU2019130504A (ru) 2021-03-30
SG11201907870VA (en) 2019-09-27
AU2018229351B2 (en) 2024-01-04
AU2024202117A1 (en) 2024-05-02
EP3589291A4 (en) 2020-11-25
WO2018160768A1 (en) 2018-09-07
JP7386082B2 (ja) 2023-11-24
US11975029B2 (en) 2024-05-07
WO2018160768A8 (en) 2019-04-18
IL268895B2 (en) 2025-05-01
JP2024019176A (ja) 2024-02-08
KR102709603B1 (ko) 2024-09-27
RU2019130504A3 (enExample) 2021-11-29
US20240350550A1 (en) 2024-10-24
KR20190141657A (ko) 2019-12-24

Similar Documents

Publication Publication Date Title
US20240350550A1 (en) Compositions and methods for inhibition of lineage specific proteins
US11464807B2 (en) Genetically engineered hematopoietic stem cells and uses thereof
HK40020660A (en) Compositions and methods for inhibition of lineage specific proteins
WO2025030010A1 (en) Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220930

EEER Examination request

Effective date: 20220930

EEER Examination request

Effective date: 20220930

EEER Examination request

Effective date: 20220930

EEER Examination request

Effective date: 20220930