CA3215902A1 - 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
CA3215902A1
CA3215902A1 CA3215902A CA3215902A CA3215902A1 CA 3215902 A1 CA3215902 A1 CA 3215902A1 CA 3215902 A CA3215902 A CA 3215902A CA 3215902 A CA3215902 A CA 3215902A CA 3215902 A1 CA3215902 A1 CA 3215902A1
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CA
Canada
Prior art keywords
hla
nucleic acid
acid molecule
inhibitor
subject
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.)
Pending
Application number
CA3215902A
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English (en)
French (fr)
Inventor
Sahar GELFMAN
Ann LIGOCKI
Giovanni Coppola
Aris BARAS
Arden MOSCATI
Eli A. STAHL
Jack A. KOSMICKI
Manuel Allen Revez FERREIRA
Carmelo Romano
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.)
Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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Filing date
Publication date
Application filed by Regeneron Pharmaceuticals Inc filed Critical Regeneron Pharmaceuticals Inc
Publication of CA3215902A1 publication Critical patent/CA3215902A1/en
Pending legal-status Critical Current

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    • 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)
CA3215902A 2021-04-07 2022-04-06 A composition for use in treating mhc-1-opathies Pending CA3215902A1 (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 (1)

Publication Number Publication Date
CA3215902A1 true CA3215902A1 (en) 2022-10-13

Family

ID=81580496

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3215902A Pending CA3215902A1 (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

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US11632728B2 (en) 2019-07-18 2023-04-18 Qualcomm Incorporated Directional measurements for sidelink synchronization signal transmission
US12195723B2 (en) 2019-11-08 2025-01-14 The Broad Institute, Inc. Engineered antigen presenting cells and uses thereof
WO2021113805A1 (en) 2019-12-05 2021-06-10 The General Hospital Corporation Multi-biomarker prediction models for multiple infection episodes following blunt trauma

Also Published As

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
AU2022256043A1 (en) 2023-10-12
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

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