CA2921856A1 - Compositions and methods for treatment of hsct-associated thrombotic microangiopathy - Google Patents

Compositions and methods for treatment of hsct-associated thrombotic microangiopathy Download PDF

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Publication number
CA2921856A1
CA2921856A1 CA2921856A CA2921856A CA2921856A1 CA 2921856 A1 CA2921856 A1 CA 2921856A1 CA 2921856 A CA2921856 A CA 2921856A CA 2921856 A CA2921856 A CA 2921856A CA 2921856 A1 CA2921856 A1 CA 2921856A1
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Prior art keywords
hsct
eculizumab
complement
tma
subject
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CA2921856A
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English (en)
French (fr)
Inventor
Sonata JODELE
Benjamin L. Laskin
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Cincinnati Children's Hospital Medical Center
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Cincinnati Children's Hospital Medical Center
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Publication of CA2921856A1 publication Critical patent/CA2921856A1/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • 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
    • G01N33/94—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving narcotics or drugs or pharmaceuticals, neurotransmitters or associated receptors
    • 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
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76—Antagonist effect on antigen, e.g. neutralization or inhibition of binding
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/94—Stability, e.g. half-life, pH, temperature or enzyme-resistance
    • 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
    • G01N2800/22—Haematology
    • G01N2800/226—Thrombotic disorders, i.e. thrombo-embolism irrespective of location/organ involved, e.g. renal vein thrombosis, venous thrombosis
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Urology & Nephrology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Hematology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Food Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Transplantation (AREA)
  • Diabetes (AREA)
  • Vascular Medicine (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CA2921856A 2013-09-16 2014-09-16 Compositions and methods for treatment of hsct-associated thrombotic microangiopathy Pending CA2921856A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361878119P 2013-09-16 2013-09-16
US61/878,119 2013-09-16
PCT/US2014/055922 WO2015039126A1 (en) 2013-09-16 2014-09-16 Compositions and methods for treatment of hsct-associated thrombotic microangiopathy

Publications (1)

Publication Number Publication Date
CA2921856A1 true CA2921856A1 (en) 2015-03-19

Family

ID=52666441

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2921856A Pending CA2921856A1 (en) 2013-09-16 2014-09-16 Compositions and methods for treatment of hsct-associated thrombotic microangiopathy

Country Status (6)

Country Link
US (1) US10815296B2 (cg-RX-API-DMAC10.html)
EP (2) EP3046581B1 (cg-RX-API-DMAC10.html)
JP (4) JP6654137B2 (cg-RX-API-DMAC10.html)
CA (1) CA2921856A1 (cg-RX-API-DMAC10.html)
ES (1) ES2796078T3 (cg-RX-API-DMAC10.html)
WO (1) WO2015039126A1 (cg-RX-API-DMAC10.html)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2796078T3 (es) * 2013-09-16 2020-11-25 Childrens Hospital Med Ct Composiciones y métodos para el tratamiento de la microangiopatía trombótica asociada al hsct
RS61351B1 (sr) 2013-10-17 2021-02-26 Omeros Corp Postupci za lečenje stanja udruženih sa aktivacijom komplementa zavisnom od masp-2
CN106999559A (zh) * 2014-12-19 2017-08-01 儿童医院医疗中心 与移植相关血栓性微血管病相关的方法和组合物
WO2016200627A1 (en) * 2015-06-09 2016-12-15 Children's Hospital Medical Center Dosing algorithm for complement inhibitor
TW201718014A (zh) * 2015-10-12 2017-06-01 諾華公司 C5抑制劑於移植相關微血管病之用途
CN117398458A (zh) * 2015-11-09 2024-01-16 奥默罗斯公司 用于治疗与masp-2依赖性补体活化相关的病况的方法
IL263481B2 (en) 2016-06-14 2024-06-01 Regeneron Pharma Anti-c5 antibodies and uses thereof
WO2019108456A1 (en) * 2017-12-01 2019-06-06 Children's Hospital Medical Center Compositions for interferon blockade and methods of using same
AU2018383751B2 (en) 2017-12-13 2025-05-08 Regeneron Pharmaceuticals, Inc. Anti-C5 antibody combinations and uses thereof
JP2023503776A (ja) * 2019-09-27 2023-02-01 ヴォリューション イミュノ ファーマシューティカルズ エスエイ 造血幹細胞移植後の血栓性微小血管症（ｈｓｃｔ－ｔｍａ）の治療方法
JP7821160B2 (ja) * 2020-08-13 2026-02-26 アレクシオン ファーマシューティカルズ， インコーポレイテッド 造血幹細胞移植関連血栓性微小血管症（ｈｓｃｔ－ｔｍａ）を処置するための抗ｃ５抗体の用量及び投与
US20250066461A1 (en) * 2021-12-21 2025-02-27 Children's Hospital Medical Center Compositions and methods for treating transplant associated thrombotic microangiopathy in bleeding patients
TW202426048A (zh) * 2022-09-06 2024-07-01 美商阿雷希昂製藥公司 用於治療造血幹細胞移植相關血栓性（ｈｓｃｔ－ｔｍａ）的抗ｃ５抗體的補充劑量和投與
CA3266977A1 (en) 2022-09-06 2024-03-14 Alexion Pharmaceuticals, Inc. DIAGNOSTIC AND PROGNOSIS BIOMARKER PROFILES IN PATIENTS WITH HEMATOPOIETIC STEM CELL TRANSPLANT-ASSOCIATED THROMBOTIC MICROANGIOPATHY (HSCT-TMA)
WO2024137904A1 (en) * 2022-12-21 2024-06-27 Children's Hospital Medical Center Improved eculizumab precision dosing

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US20040013664A1 (en) 1997-01-14 2004-01-22 Gentz Reiner L. Tumor necrosis factor receptors 6 alpha & 6 beta
WO2004034988A2 (en) 2002-10-16 2004-04-29 Amgen Inc. Human anti-ifn-ϝ neutralizing antibodies as selective ifn-ϝ pathway inhibitors
US20070116710A1 (en) 2004-02-03 2007-05-24 Leonard Bell Methods of treating hemolytic anemia
DK1991275T3 (en) * 2006-03-08 2014-12-08 Archemix Llc Complement aptamers AND ANTI-C5 AGENTS FOR USE IN THE TREATMENT OF EYE DISEASES
JP6223659B2 (ja) 2008-11-10 2017-11-01 アレクシオン ファーマシューティカルズ， インコーポレイテッド 補体関連障害を処置するための方法および組成物
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Also Published As

Publication number Publication date
JP2016531158A (ja) 2016-10-06
JP7572777B2 (ja) 2024-10-24
EP3046581B1 (en) 2020-04-01
JP6654137B2 (ja) 2020-02-26
EP3046581A1 (en) 2016-07-27
EP3046581A4 (en) 2017-04-19
ES2796078T3 (es) 2020-11-25
EP3750914A1 (en) 2020-12-16
JP2020073531A (ja) 2020-05-14
WO2015039126A1 (en) 2015-03-19
JP2024133399A (ja) 2024-10-01
JP2022062161A (ja) 2022-04-19
US20160194386A1 (en) 2016-07-07
US10815296B2 (en) 2020-10-27

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