IL255912B2 - Preparations and methods for reducing unfolded proteins - Google Patents

Preparations and methods for reducing unfolded proteins

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Publication number
IL255912B2
IL255912B2 IL255912A IL25591217A IL255912B2 IL 255912 B2 IL255912 B2 IL 255912B2 IL 255912 A IL255912 A IL 255912A IL 25591217 A IL25591217 A IL 25591217A IL 255912 B2 IL255912 B2 IL 255912B2
Authority
IL
Israel
Prior art keywords
pml
atxnl
proteins
protein
amyloidosis
Prior art date
Application number
IL255912A
Other languages
English (en)
Hebrew (he)
Other versions
IL255912B1 (en
IL255912A (en
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of IL255912A publication Critical patent/IL255912A/en
Publication of IL255912B1 publication Critical patent/IL255912B1/en
Publication of IL255912B2 publication Critical patent/IL255912B2/en

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    • 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
    • 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
    • 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
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/21—Interferons [IFN]
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/12—Mucolytics
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • G01N33/5058—Neurological cells
    • 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
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/566—Immunoassay; Biospecific binding assay; Materials therefor using specific carrier or receptor proteins as ligand binding reagents where possible specific carrier or receptor proteins are classified with their target compounds
    • 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
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2814—Dementia; Cognitive disorders

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Analytical Chemistry (AREA)
  • Food Science & Technology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
IL255912A 2015-05-29 2016-05-27 Preparations and methods for reducing unfolded proteins IL255912B2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562168309P 2015-05-29 2015-05-29
PCT/US2016/034751 WO2016196328A1 (en) 2015-05-29 2016-05-27 Compositions and methods for degradation of misfolded proteins

Publications (3)

Publication Number Publication Date
IL255912A IL255912A (en) 2018-01-31
IL255912B1 IL255912B1 (en) 2023-06-01
IL255912B2 true IL255912B2 (en) 2023-10-01

Family

ID=57441753

Family Applications (3)

Application Number Title Priority Date Filing Date
IL255912A IL255912B2 (en) 2015-05-29 2016-05-27 Preparations and methods for reducing unfolded proteins
IL322085A IL322085A (en) 2015-05-29 2016-05-27 Compositions and methods for reducing unfolded proteins
IL302965A IL302965B2 (en) 2015-05-29 2016-05-27 Compositions and methods for degradation of misfolded proteins

Family Applications After (2)

Application Number Title Priority Date Filing Date
IL322085A IL322085A (en) 2015-05-29 2016-05-27 Compositions and methods for reducing unfolded proteins
IL302965A IL302965B2 (en) 2015-05-29 2016-05-27 Compositions and methods for degradation of misfolded proteins

Country Status (15)

Country Link
US (2) US11542500B2 (https=)
EP (1) EP3302706A4 (https=)
JP (3) JP7011467B2 (https=)
KR (2) KR20240108531A (https=)
CN (1) CN108271364A (https=)
AU (3) AU2016271635B2 (https=)
BR (1) BR112017025574A2 (https=)
CA (1) CA2987057A1 (https=)
HK (1) HK1252757A1 (https=)
IL (3) IL255912B2 (https=)
MX (3) MX2017015373A (https=)
RU (2) RU2021134920A (https=)
SG (1) SG10202011833TA (https=)
WO (1) WO2016196328A1 (https=)
ZA (1) ZA201708067B (https=)

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CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CN110753557B (zh) * 2017-03-22 2024-03-22 神济昌华(北京)生物科技有限公司 Trim72通过泛素化fus蛋白突变体作为als的潜在治疗性靶标
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN109609614B (zh) * 2017-09-30 2022-07-15 首都医科大学附属北京胸科医院 检测trim2、trim4、trim32和/或trim46基因或蛋白产品的用途
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
CN108018303A (zh) * 2017-12-20 2018-05-11 江苏浩欧博生物医药股份有限公司 一种人Ro52重组蛋白的制备方法
WO2019175881A1 (en) 2018-03-14 2019-09-19 Technion Research & Development Foundation Limited A prognostic biomarker in cancer
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
US11147815B2 (en) 2018-08-21 2021-10-19 Synkrino Biotherapeutics, Inc. Compositions and methods for treating diseases associated with uncontrolled inflammatory responses
EP3863642A4 (en) * 2018-10-09 2022-06-29 The Regents of the University of California Covalent targeting of e3 ligases
WO2020227515A1 (en) * 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
CN110302383A (zh) * 2019-07-29 2019-10-08 深圳先进技术研究院 Trim11基因和蛋白靶点以及抑制剂在乳腺癌中的用途
CN110669791B (zh) * 2019-10-29 2020-08-04 成都益安博生物技术有限公司 一种提高细胞抗体表达量的方法
KR102114880B1 (ko) * 2020-01-15 2020-05-26 서울대학교병원 코돈 최적화된 TIF1γ 폴리뉴클레오티드를 포함하는 재조합 벡터 및 이의 용도
GB202005876D0 (en) * 2020-04-22 2020-06-03 Univ Dundee Protein degradation
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CN113292658B (zh) * 2021-04-17 2023-02-28 复旦大学 一种融合蛋白及其在靶向降解细胞内蛋白中的应用
CN113651879A (zh) * 2021-08-18 2021-11-16 武汉华美生物工程有限公司 一种trim21全长蛋白的制备方法及应用
CN117159747B (zh) * 2023-08-30 2024-12-20 暨南大学 一种亨廷顿舞蹈症的药物及其制备方法
CN118272444A (zh) * 2024-02-29 2024-07-02 宜昌市中心人民医院(三峡大学第一临床医学院、三峡大学附属中心人民医院) 一种慢病毒载体LV-Trim69及其在制备改善心脏衰老药物上的应用
CN120209082B (zh) * 2025-05-28 2025-08-05 中南大学 一种靶向阻断trim25与brd7蛋白结合的多肽及其应用

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JP2024161501A (ja) 2024-11-19
RU2017144862A (ru) 2019-07-02
RU2761564C2 (ru) 2021-12-10
US20180163202A1 (en) 2018-06-14
US11542500B2 (en) 2023-01-03
RU2017144862A3 (https=) 2019-11-20
IL302965B1 (en) 2025-08-01
MX2024001616A (es) 2024-02-28
RU2761564C9 (ru) 2022-03-17
KR20240108531A (ko) 2024-07-09
IL322085A (en) 2025-09-01
RU2021134920A (ru) 2022-04-15
BR112017025574A2 (en) 2018-08-14
AU2022200673A1 (en) 2022-02-24
CN108271364A (zh) 2018-07-10
JP2018521014A (ja) 2018-08-02
IL302965B2 (en) 2025-12-01
IL302965A (en) 2023-07-01
ZA201708067B (en) 2018-11-28
IL255912B1 (en) 2023-06-01
KR20180011276A (ko) 2018-01-31
AU2025230665A1 (en) 2025-10-02
MX2021014954A (es) 2022-01-31
CA2987057A1 (en) 2016-12-08
JP2022000459A (ja) 2022-01-04
JP7011467B2 (ja) 2022-02-10
EP3302706A1 (en) 2018-04-11
US20230313190A1 (en) 2023-10-05
WO2016196328A1 (en) 2016-12-08
EP3302706A4 (en) 2019-05-08
AU2016271635A1 (en) 2018-01-25
MX2017015373A (es) 2018-07-06
SG10202011833TA (en) 2021-01-28
AU2022200673B2 (en) 2025-06-05
HK1252757A1 (zh) 2019-05-31
IL255912A (en) 2018-01-31
AU2016271635B2 (en) 2021-11-04

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