CA3145291A1 - Methods for treating ran protein-associated neurological diseases - Google Patents

Methods for treating ran protein-associated neurological diseases Download PDF

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Publication number
CA3145291A1
CA3145291A1 CA3145291A CA3145291A CA3145291A1 CA 3145291 A1 CA3145291 A1 CA 3145291A1 CA 3145291 A CA3145291 A CA 3145291A CA 3145291 A CA3145291 A CA 3145291A CA 3145291 A1 CA3145291 A1 CA 3145291A1
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Prior art keywords
poly
seq
ran
protein
antibody
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CA3145291A
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English (en)
French (fr)
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Laura Ranum
Lien Nguyen
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University of Florida Research Foundation Inc
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University of Florida Research Foundation Inc
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • 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
    • A61K31/13—Amines
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/27—Esters, e.g. nitroglycerine, selenocyanates of carbamic or thiocarbamic acids, meprobamate, carbachol, neostigmine
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • 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
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
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    • C12N2310/50—Physical structure
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    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
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  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Analytical Chemistry (AREA)
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  • Hospice & Palliative Care (AREA)
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CA3145291A 2019-07-05 2020-07-02 Methods for treating ran protein-associated neurological diseases Pending CA3145291A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962871031P 2019-07-05 2019-07-05
US62/871,031 2019-07-05
US202063025096P 2020-05-14 2020-05-14
US63/025,096 2020-05-14
PCT/US2020/040725 WO2021007110A1 (en) 2019-07-05 2020-07-02 Methods for treating ran protein-associated neurological diseases

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CA3145291A1 true CA3145291A1 (en) 2021-01-14

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CA3145291A Pending CA3145291A1 (en) 2019-07-05 2020-07-02 Methods for treating ran protein-associated neurological diseases

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US (1) US20220267776A1 (enExample)
EP (1) EP3994159A4 (enExample)
JP (2) JP7579589B2 (enExample)
AU (1) AU2020310843A1 (enExample)
CA (1) CA3145291A1 (enExample)
WO (1) WO2021007110A1 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
CA3019847A1 (en) 2016-04-04 2017-10-12 University Of Florida Research Foundation, Incorporated Manipulation of eif3 to modulate repeat associated non-atg (ran) translation
AU2018255293B2 (en) 2017-04-17 2024-03-07 University Of Florida Research Foundation, Incorporated Regulation of RAN translation by PKR and eIF2a-P pathways
JP7368856B2 (ja) 2017-07-25 2023-10-25 トゥルーバインディング，インコーポレイテッド Ｔｉｍ－３とそのリガンドとの相互作用の遮断によるがん治療
AU2018338451A1 (en) 2017-09-25 2020-04-02 University Of Florida Research Foundation, Incorporated Immunoassays for detection of RAN proteins
JP7350337B2 (ja) 2017-09-26 2023-09-26 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 神経障害の処置においてｒａｎタンパク質レベルを低下させるためのメトホルミンおよびそのアナログの使用
CN116063505B (zh) 2019-01-30 2024-05-03 真和制药有限公司 抗gal3抗体及其用途
CA3154741A1 (en) 2019-09-20 2021-03-25 University Of Florida Research Foundation, Incorporated Detection of antibodies against ran proteins from serum and tissue lysates
US12281166B2 (en) 2020-05-26 2025-04-22 Truebinding, Inc. Methods of treating inflammatory diseases by blocking Galectin-3
EP4427049A4 (en) * 2021-11-01 2025-08-20 Univ Florida POLY-GA PROTEINS IN ALZHEIMER'S DISEASE
WO2023102111A1 (en) 2021-12-01 2023-06-08 University Of Florida Research Foundation, Incorporated Small molecule inhibitors of repeat associated non-aug (ran) translation and combination therapies
AU2023373378A1 (en) * 2022-11-01 2025-04-24 University Of Florida Research Foundation, Incorporated Interrupted ran proteins in disease
WO2024173816A1 (en) * 2023-02-17 2024-08-22 The University Of North Carolina At Chapel Hill Methods of detecting telomere-encoded dipeptide repeat proteins and therapeutic applications
CN116693621B (zh) * 2023-03-02 2023-11-14 东北农业大学 抑制革兰氏阴性菌的窄谱抗菌肽pc及其制备方法和应用
WO2025019730A1 (en) * 2023-07-20 2025-01-23 University Of Florida Research Foundation, Incorporated Engineered ga antibodies for therapy development
WO2025128551A1 (en) * 2023-12-11 2025-06-19 University Of Florida Research Foundation, Incorporated Aav-based anti-ga therapy

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RS59283B1 (sr) 2013-01-22 2019-10-31 Deutsches Zentrum Fuer Neurodegenerative Erkrankungen E V Proteini sa dipeptidnim ponovkom kao terapeutsko ciljno mesto kod neurodegenerativnih bolesti sa ekspanzijom heksanukleotidnog ponovka
CA2904960A1 (en) * 2013-03-14 2014-10-02 University Of Florida Research Foundation, Inc. Di-amino acid repeat-containing proteins associated with als
CA2955086A1 (en) * 2014-08-08 2016-02-11 Alector Llc Anti-trem2 antibodies and methods of use thereof
JP6432023B2 (ja) 2014-12-02 2018-12-05 国立大学法人 東京医科歯科大学 脊髄小脳失調症３１型（ｓｃａ３１）治療剤
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
CA3019847A1 (en) * 2016-04-04 2017-10-12 University Of Florida Research Foundation, Incorporated Manipulation of eif3 to modulate repeat associated non-atg (ran) translation
JP2020534253A (ja) * 2017-08-08 2020-11-26 ウェーブ ライフ サイエンシーズ リミテッド オリゴヌクレオチド組成物及びその方法
AU2018338451A1 (en) 2017-09-25 2020-04-02 University Of Florida Research Foundation, Incorporated Immunoassays for detection of RAN proteins
JP7350337B2 (ja) * 2017-09-26 2023-09-26 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 神経障害の処置においてｒａｎタンパク質レベルを低下させるためのメトホルミンおよびそのアナログの使用

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AU2020310843A1 (en) 2022-01-20
EP3994159A4 (en) 2023-08-09
JP7579589B2 (ja) 2024-11-08
JP2022538926A (ja) 2022-09-06
JP2025026843A (ja) 2025-02-26
WO2021007110A1 (en) 2021-01-14
EP3994159A1 (en) 2022-05-11
US20220267776A1 (en) 2022-08-25

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