WO2012016162A3 - Compositions, procédés et kits pour modéliser, diagnostiquer et traiter des troubles du complément - Google Patents

Compositions, procédés et kits pour modéliser, diagnostiquer et traiter des troubles du complément Download PDF

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Publication number
WO2012016162A3
WO2012016162A3 PCT/US2011/045933 US2011045933W WO2012016162A3 WO 2012016162 A3 WO2012016162 A3 WO 2012016162A3 US 2011045933 W US2011045933 W US 2011045933W WO 2012016162 A3 WO2012016162 A3 WO 2012016162A3
Authority
WO
WIPO (PCT)
Prior art keywords
protein
amino acid
methods
kits
complement
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.)
Ceased
Application number
PCT/US2011/045933
Other languages
English (en)
Other versions
WO2012016162A2 (fr
Inventor
Rajendra Kumar-Singh
Siobhan M. Cashman
John Harry Sweigard
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.)
Tufts University
Original Assignee
Tufts University
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 Tufts University filed Critical Tufts University
Publication of WO2012016162A2 publication Critical patent/WO2012016162A2/fr
Publication of WO2012016162A3 publication Critical patent/WO2012016162A3/fr
Priority to US13/692,734 priority Critical patent/US8877896B2/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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Classifications

    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
    • G01N2333/46—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
    • G01N2333/47—Assays involving proteins of known structure or function as defined in the subgroups
    • G01N2333/4701—Details
    • G01N2333/4716—Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/16—Ophthalmology

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pathology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Food Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

L'invention concerne des systèmes, des compositions, des procédés et des kits pour identifier des agents thérapeutiques potentiels pour le traitement de maladies oculaires basées sur le complément. Les procédés et kits comprennent une protéine ou un dérivé de composant du complément 3 (C3) qui est en contact avec les cellules ou le tissu oculaires. Un autre mode de réalisation de la présente invention concerne le diagnostic et/ou le pronostic d'une maladie oculaire associée au complément. L'invention concerne des compositions, des procédés et des kits pour réguler ou traiter une affection associée au complément au moyen d'au moins un élément parmi la protéine CD46, la protéine CD55, et une protéine de terminateur du complément activé (STAC) soluble chimérique recombiné ou une source de protéine de STAC. La protéine de STAC comprend une séquence d'acides aminés incluant au moins deux éléments parmi une séquence d'acides aminés d'une protéine CD59, une séquence d'acides aminés dérivée d'une protéine CD46 et une séquence d'acides aminés dérivée d'une protéine CD55, comprenant éventuellement en outre un lieur pour relier les séquences d'acides aminés.
PCT/US2011/045933 2008-02-15 2011-07-29 Compositions, procédés et kits pour modéliser, diagnostiquer et traiter des troubles du complément Ceased WO2012016162A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
US13/692,734 US8877896B2 (en) 2008-02-15 2012-12-03 Compositions, methods and kits for modeling, diagnosing, and treating complement disorders

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US36893110P 2010-07-29 2010-07-29
US61/368,931 2010-07-29
US201161441501P 2011-02-10 2011-02-10
US61/441,501 2011-02-10
US201161445430P 2011-02-22 2011-02-22
US61/445,430 2011-02-22
US201161480082P 2011-04-28 2011-04-28
US61/480,082 2011-04-28

Related Child Applications (3)

Application Number Title Priority Date Filing Date
PCT/US2009/000947 Continuation WO2009102488A2 (fr) 2008-02-15 2009-02-13 Modèle humanisé de la formation du complexe d’attaque membranaire (mac) sur la rétine murine et compositions, trousses et procédés de traitement de la dégénérescence maculaire
US86756610A Continuation 2008-02-15 2010-08-13
US13/692,734 Continuation US8877896B2 (en) 2008-02-15 2012-12-03 Compositions, methods and kits for modeling, diagnosing, and treating complement disorders

Publications (2)

Publication Number Publication Date
WO2012016162A2 WO2012016162A2 (fr) 2012-02-02
WO2012016162A3 true WO2012016162A3 (fr) 2012-05-24

Family

ID=45530747

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/045933 Ceased WO2012016162A2 (fr) 2008-02-15 2011-07-29 Compositions, procédés et kits pour modéliser, diagnostiquer et traiter des troubles du complément

Country Status (1)

Country Link
WO (1) WO2012016162A2 (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3892291A1 (fr) * 2014-08-28 2021-10-13 Tufts University Compositions, procédés et kits pour le traitement des troubles liés au complément
WO2020204149A1 (fr) * 2019-03-29 2020-10-08 公立大学法人横浜市立大学 Procédé de criblage et procédé d'évaluation de toxicité
US20220195012A1 (en) * 2019-04-13 2022-06-23 National Centre For Cell Science Daf-mcp chimeric proteins, process to manufacture the same and use of the chimeric protein for treating pathological conditions involving the complement system
WO2025034741A1 (fr) * 2023-08-07 2025-02-13 Avirmax Biopharma Inc. Compositions et procédés d'expression d'agents thérapeutiques

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009102488A2 (fr) * 2008-02-15 2009-08-20 Tufts University Modèle humanisé de la formation du complexe d’attaque membranaire (mac) sur la rétine murine et compositions, trousses et procédés de traitement de la dégénérescence maculaire
US20100120665A1 (en) * 2007-03-01 2010-05-13 Advanced Vision Therapies, Inc. Treatment of diseases characterized by inflammation
US20100130413A1 (en) * 2006-10-20 2010-05-27 Marsh Henry C Treatment for age-related macular degeneration and other diseases of the eye

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100130413A1 (en) * 2006-10-20 2010-05-27 Marsh Henry C Treatment for age-related macular degeneration and other diseases of the eye
US20100120665A1 (en) * 2007-03-01 2010-05-13 Advanced Vision Therapies, Inc. Treatment of diseases characterized by inflammation
WO2009102488A2 (fr) * 2008-02-15 2009-08-20 Tufts University Modèle humanisé de la formation du complexe d’attaque membranaire (mac) sur la rétine murine et compositions, trousses et procédés de traitement de la dégénérescence maculaire

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
RAMO, K. ET AL.: "Evaluation of Adenovirus-Delivered Human CD59 as a Potential Therapy for AMD in a Model of Human Membrane Attack Complex Formation on Murine RPE", INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE., vol. 49, no. 9, September 2008 (2008-09-01), pages 4126 - 4136, XP055013364, DOI: doi:10.1167/iovs.08-2025 *

Also Published As

Publication number Publication date
WO2012016162A2 (fr) 2012-02-02

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