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AR126718A1 - Composiciones y métodos para la degradación selectiva de proteínas diseñadas por ingeniería - Google Patents

Composiciones y métodos para la degradación selectiva de proteínas diseñadas por ingeniería

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Publication number
AR126718A1
AR126718A1 ARP220102109A ARP220102109A AR126718A1 AR 126718 A1 AR126718 A1 AR 126718A1 AR P220102109 A ARP220102109 A AR P220102109A AR P220102109 A ARP220102109 A AR P220102109A AR 126718 A1 AR126718 A1 AR 126718A1
Authority
AR
Argentina
Prior art keywords
tautomers
pharmaceutically acceptable
acceptable salts
degradation
compounds
Prior art date
Application number
ARP220102109A
Other languages
English (en)
Inventor
Christopher Walton Carroll
Laura Akullian Dagostino
Haibo Liu
Veerabahu Shanmugasundaram
Brook Barajas
Original Assignee
Celgene Corp
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.)
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Publication date
Application filed by Celgene Corp filed Critical Celgene Corp
Publication of AR126718A1 publication Critical patent/AR126718A1/es

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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/4523Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/496Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
    • 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
    • A61K40/421Immunoglobulin superfamily
    • A61K40/4211CD19 or B4
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
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    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D401/00Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/14Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
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    • C07D417/00Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
    • C07D417/14Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing three or more hetero rings
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    • C07D471/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04Ortho-condensed systems
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    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • 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
    • C12N5/0636T lymphocytes
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K2239/46Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the cancer treated
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K2319/00Fusion polypeptide
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    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/95Fusion polypeptide containing a motif/fusion for degradation (ubiquitin fusions, PEST sequence)

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Immunology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Cell Biology (AREA)
  • Wood Science & Technology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente descripción se refiere a polipéptidos diseñados por ingeniería que comprenden dominios de degradación, compuestos, composiciones y métodos para su preparación y uso en la degradación de proteínas diseñadas por ingeniería en células. Reivindicación 1: Un polipéptido diseñado por ingeniería que comprende un dominio de degradación, en donde el dominio de degradación comprende la secuencia de aminoácidos FCX¹X²CGX³X⁴ (SEC ID Nº 1), en donde: X¹ se selecciona de asparagina, aspartato, glicina, glutamina, metionina, histidina, triptófano, isoleucina, arginina, leucina, valina, treonina y fenilalanina, X² se selecciona de glutamina, arginina, histidina, leucina, fenilalanina, tirosina, triptófano, isoleucina, valina y metionina, X³ se selecciona de alanina, serina, cisteína, arginina, leucina, isoleucina, metionina y glicina, y X⁴ se selecciona de serina, metionina, lisina, isoleucina, valina, histidina, glutamina, arginina, fenilalanina y triptófano. Reivindicación 72: Un método para reducir el nivel del polipéptido diseñado por ingeniería de acuerdo con cualquiera de las reivindicaciones 1 a 61, que comprende poner en contacto el polipéptido diseñado por ingeniería con un agente de degradación seleccionado de: los compuestos del grupo de fórmula (1), y tautómeros de los mismos, y sales farmacéuticamente aceptables de los mismos, y sales farmacéuticamente aceptables de tautómeros de los mismos. Reivindicación 73: Un método para reducir el nivel de un polipéptido diseñado por ingeniería en una célula, que comprende poner en contacto la célula de acuerdo con cualquiera de las reivindicaciones 64 a 70 con un agente de degradación seleccionado de: los compuestos del grupo de fórmula (1), y tautómeros de los mismos, y sales farmacéuticamente aceptables de los mismos, y sales farmacéuticamente aceptables de tautómeros de los mismos. Reivindicación 94: El método de cualquiera de las reivindicaciones 91 a 93, en donde el agente de degradación es un compuesto seleccionado a partir de: los compuestos del grupo de fórmula (1), y tautómeros de los mismos, sales farmacéuticamente aceptables de los mismos y sales farmacéuticamente aceptables de tautómeros de los mismos. Reivindicación 95: Un compuesto seleccionado a partir de: los compuestos del grupo de fórmula (1), y tautómeros de los mismos, sales farmacéuticamente aceptables de los mismos y sales farmacéuticamente aceptables de tautómeros de los mismos.
ARP220102109A 2021-08-06 2022-08-05 Composiciones y métodos para la degradación selectiva de proteínas diseñadas por ingeniería AR126718A1 (es)

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US (1) US20230095912A1 (es)
EP (1) EP4380582A1 (es)
JP (1) JP2024528988A (es)
KR (1) KR20240043780A (es)
CN (1) CN117794554A (es)
AR (1) AR126718A1 (es)
AU (1) AU2022324621A1 (es)
CA (1) CA3223636A1 (es)
CL (1) CL2024000305A1 (es)
CO (1) CO2024001133A2 (es)
IL (1) IL309653A (es)
MX (1) MX2024000901A (es)
TW (1) TW202321278A (es)
WO (1) WO2023015283A1 (es)

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IL309653A (en) 2024-02-01
JP2024528988A (ja) 2024-08-01
US20230095912A1 (en) 2023-03-30
CO2024001133A2 (es) 2024-03-07
EP4380582A1 (en) 2024-06-12
CA3223636A1 (en) 2023-02-09
CN117794554A (zh) 2024-03-29
TW202321278A (zh) 2023-06-01
KR20240043780A (ko) 2024-04-03
AU2022324621A1 (en) 2024-01-18
WO2023015283A1 (en) 2023-02-09
MX2024000901A (es) 2024-02-06
CL2024000305A1 (es) 2024-06-14

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