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AR133829A1 - CELL PENETRATING AGENTS AND THEIR USES - Google Patents

CELL PENETRATING AGENTS AND THEIR USES

Info

Publication number
AR133829A1
AR133829A1 ARP240102462A ARP240102462A AR133829A1 AR 133829 A1 AR133829 A1 AR 133829A1 AR P240102462 A ARP240102462 A AR P240102462A AR P240102462 A ARP240102462 A AR P240102462A AR 133829 A1 AR133829 A1 AR 133829A1
Authority
AR
Argentina
Prior art keywords
cell
amyloid
antibody
nucleic acid
internalization
Prior art date
Application number
ARP240102462A
Other languages
Spanish (es)
Inventor
Tarlochan S Nijjar
Stephen Jed Tam
Original Assignee
Othair Prothena Ltd
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 Othair Prothena Ltd filed Critical Othair Prothena Ltd
Publication of AR133829A1 publication Critical patent/AR133829A1/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896Neurological disorders, e.g. Alzheimer's disease
    • 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
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/435Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
    • G01N2333/46Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
    • G01N2333/47Assays involving proteins of known structure or function as defined in the subgroups
    • G01N2333/4701Details
    • G01N2333/4709Amyloid plaque core protein

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Biochemistry (AREA)
  • Analytical Chemistry (AREA)
  • Psychiatry (AREA)
  • Food Science & Technology (AREA)
  • Microbiology (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Cell Biology (AREA)
  • Biotechnology (AREA)
  • Hospice & Palliative Care (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente divulgación proporciona agentes penetrantes de células que comprenden un módulo de internalización celular y un anticuerpo o fragmento de anticuerpo que se une a antígeno del mismo que se une específicamente al amiloide b humano y método de uso de estos agentes penetrantes de células para tratar pacientes con enfermedades relacionadas con beta amiloide, incluyendo la Miositis por cuerpos de inclusión (MCI). Reivindicación 1: Un agente penetrante de células (APC) anti-péptido Amiloide b (Ab) que comprende: (i) un módulo de internalización celular (MIC) y (ii) un anticuerpo que se une específicamente al péptido Amiloide b (Ab). Reivindicación 72: Una composición farmacéutica que comprende el APC anti-Ab de una cualquiera de las reivindicaciones 1 - 71 y un portador o diluyente farmacéuticamente aceptable. Reivindicación 73: Un ácido nucleico que codifica al menos una porción del APC anti-Ab de una cualquiera de las reivindicaciones 1 - 71. Reivindicación 78: Un vector que comprende el ácido nucleico de una cualquiera de las reivindicaciones 73 - 77 unido de manera operativa a una o más secuencias reguladoras para efectuar la expresión en una célula de mamífero del agente penetrante de células de una cualquiera de las reivindicaciones 1 a 71. Reivindicación 81: Una célula huésped transformada con el vector de una cualquiera de las reivindicaciones 78 - 80. Reivindicación 82: Una célula huésped que comprende el ácido nucleico de una cualquiera de las reivindicaciones 73 - 77. Reivindicación 85: Un método de unión una proteína Ab intracelular en una célula, en donde el método comprende: poner en contacto el APC anti-Ab de una cualquiera de las reivindicaciones 1 a 71 con la célula, dando como resultado de ese modo la internalización de, y transferencia al citosol de, como mínimo, un fragmento de unión a antígeno del anticuerpo.This disclosure provides cell-penetrating agents comprising a cell internalization module and an antibody or antibody fragment binding to an antigen thereof that specifically binds to human amyloid b, and a method of using these cell-penetrating agents to treat patients with beta-amyloid-related diseases, including inclusion body myositis (IBM). Claim 1: An anti-amyloid b peptide (Ab) cell-penetrating agent (CPA) comprising: (i) a cell internalization module (CPM) and (ii) an antibody that specifically binds to the amyloid b peptide (Ab). Claim 72: A pharmaceutical composition comprising the anti-Ab CPA of any one of claims 1-71 and a pharmaceutically acceptable carrier or diluent. Claim 73: A nucleic acid encoding at least a portion of the anti-Ab APC of any one of claims 1-71. Claim 78: A vector comprising the nucleic acid of any one of claims 73-77 operatively linked to one or more regulatory sequences to effect expression in a mammalian cell of the cell-penetrating agent of any one of claims 1-71. Claim 81: A host cell transformed with the vector of any one of claims 78-80. Claim 82: A host cell comprising the nucleic acid of any one of claims 73-77. Claim 85: A method of attaching an intracellular Ab protein to a cell, wherein the method comprises: contacting the anti-Ab APC of any one of claims 1-71 with the cell, thereby resulting in the internalization of, and transfer to the cytosol of, at least an antigen-binding fragment of the antibody.

ARP240102462A 2023-09-15 2024-09-13 CELL PENETRATING AGENTS AND THEIR USES AR133829A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202363538600P 2023-09-15 2023-09-15

Publications (1)

Publication Number Publication Date
AR133829A1 true AR133829A1 (en) 2025-11-05

Family

ID=93010600

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP240102462A AR133829A1 (en) 2023-09-15 2024-09-13 CELL PENETRATING AGENTS AND THEIR USES

Country Status (3)

Country Link
AR (1) AR133829A1 (en)
TW (1) TW202530246A (en)
WO (1) WO2025059487A2 (en)

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US5786464C1 (en) 1994-09-19 2012-04-24 Gen Hospital Corp Overexpression of mammalian and viral proteins
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Also Published As

Publication number Publication date
WO2025059487A2 (en) 2025-03-20
TW202530246A (en) 2025-08-01
WO2025059487A3 (en) 2025-06-12

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