AU2022256043A1 - A composition for use in treating mhc-1-opathies - Google Patents

A composition for use in treating mhc-1-opathies Download PDF

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Publication number
AU2022256043A1
AU2022256043A1 AU2022256043A AU2022256043A AU2022256043A1 AU 2022256043 A1 AU2022256043 A1 AU 2022256043A1 AU 2022256043 A AU2022256043 A AU 2022256043A AU 2022256043 A AU2022256043 A AU 2022256043A AU 2022256043 A1 AU2022256043 A1 AU 2022256043A1
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AU
Australia
Prior art keywords
hla
nucleic acid
acid molecule
inhibitor
subject
Prior art date
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Pending
Application number
AU2022256043A
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English (en)
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AU2022256043A9 (en
Inventor
Aris BARAS
Giovanni Coppola
Manuel Allen Revez FERREIRA
Sahar GELFMAN
Jack A. KOSMICKI
Ann LIGOCKI
Arden MOSCATI
Carmelo Romano
Eli A. STAHL
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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Publication date
Application filed by Regeneron Pharmaceuticals Inc filed Critical Regeneron Pharmaceuticals Inc
Publication of AU2022256043A1 publication Critical patent/AU2022256043A1/en
Publication of AU2022256043A9 publication Critical patent/AU2022256043A9/en
Pending legal-status Critical Current

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    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485Exopeptidases (3.4.11-3.4.19)
    • 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
    • A61K39/3955Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803Immunoglobulins [IG], 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
    • C07K16/2833Immunoglobulins [IG], 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 against MHC-molecules, e.g. HLA-molecules
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6881Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for tissue or cell typing, e.g. human leukocyte antigen [HLA] probes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • 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
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic or mutational markers

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Analytical Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Endocrinology (AREA)
  • Biomedical Technology (AREA)
  • Cell Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Compositions Of Macromolecular Compounds (AREA)
AU2022256043A 2021-04-07 2022-04-06 A composition for use in treating mhc-1-opathies Pending AU2022256043A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163171672P 2021-04-07 2021-04-07
US63/171,672 2021-04-07
PCT/US2022/023685 WO2022216832A1 (en) 2021-04-07 2022-04-06 A composition for use in treating mhc-1-opathies

Publications (2)

Publication Number Publication Date
AU2022256043A1 true AU2022256043A1 (en) 2023-10-12
AU2022256043A9 AU2022256043A9 (en) 2023-10-26

Family

ID=81580496

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2022256043A Pending AU2022256043A1 (en) 2021-04-07 2022-04-06 A composition for use in treating mhc-1-opathies

Country Status (10)

Country Link
US (2) US12370257B2 (https=)
EP (1) EP4320166A1 (https=)
JP (1) JP2024515240A (https=)
KR (1) KR20230167355A (https=)
CN (1) CN117321084A (https=)
AU (1) AU2022256043A1 (https=)
CA (1) CA3215902A1 (https=)
IL (1) IL306046A (https=)
MX (1) MX2023011720A (https=)
WO (1) WO2022216832A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL248777B1 (pl) * 2024-03-05 2026-01-26 Inst Immunologii I Terapii Doswiadczalnej Im Ludwika Hirszfelda Polskiej Akademii Nauk Sposób określania ciężkości reumatoidalnego zapalenia stawów na podstawie pomiaru stężenia białka ERAP2
CN118047824B (zh) * 2024-04-01 2024-06-28 北京瑞博奥医药科技有限公司 二烷基膦酸在寡核苷酸合成中的应用

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CA2804763C (en) 2010-07-14 2023-01-03 The Regents Of The University Of California Biomarkers for diagnosis of transient ischemic attacks
US20130315933A1 (en) 2010-10-06 2013-11-28 Christoph Renner Antibodies Directed Against HLA-B27 Homodimers and Methods and Uses Thereof in Diagnosis and Therapy
US8969254B2 (en) 2010-12-16 2015-03-03 Dana-Farber Cancer Institute, Inc. Oligonucleotide array for tissue typing
TWI619729B (zh) 2012-04-02 2018-04-01 再生元醫藥公司 抗-hla-b*27抗體及其用途
WO2013162368A1 (en) 2012-04-26 2013-10-31 Stichting Vu-Vumc Biomarkers
HK1213830A1 (zh) 2012-10-10 2016-07-15 Teva Pharmaceutical Industries Ltd. 用於预测醋酸格拉替雷临床反应的生物标志物
KR101748867B1 (ko) 2013-04-19 2017-06-20 주식회사 젠큐릭스 조기 유방암 예후 예측 진단용 자동화 시스템
US20160145687A1 (en) 2013-06-25 2016-05-26 Turun Yliopisto Method of Predicting Risk for Type 1 Diabetes
US9907833B2 (en) 2013-07-25 2018-03-06 University Of Florida Research Foundation, Incorporated Use of relaxin to treat placental syndromes
US10927412B2 (en) 2013-10-01 2021-02-23 The Regents Of The University Of California Endometriosis classifier
US10166210B2 (en) 2014-06-12 2019-01-01 Nsabp Foundation, Inc. Methods of subtyping CRC and their association with treatment of colon cancer patients with oxaliplatin
EP3730188B1 (en) 2015-08-21 2024-07-31 The Children's Hospital of Philadelphia Compositions and methods for use in combination for the treatment and diagnosis of autoimmune diseases
WO2017191274A2 (en) 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
GB201609712D0 (en) 2016-06-03 2016-07-20 Babraham Inst Disease targets and biomarkers
US20190375842A1 (en) 2018-01-04 2019-12-12 Vanderbilt University Check point inhibition in organ fibrosis
WO2019140380A1 (en) 2018-01-12 2019-07-18 Kymera Therapeutics, Inc. Protein degraders and uses thereof
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Publication number Publication date
MX2023011720A (es) 2023-10-12
US20220323581A1 (en) 2022-10-13
US12370257B2 (en) 2025-07-29
KR20230167355A (ko) 2023-12-08
CN117321084A (zh) 2023-12-29
EP4320166A1 (en) 2024-02-14
AU2022256043A9 (en) 2023-10-26
WO2022216832A1 (en) 2022-10-13
IL306046A (en) 2023-11-01
JP2024515240A (ja) 2024-04-08
US20250375519A1 (en) 2025-12-11
CA3215902A1 (en) 2022-10-13

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