JP2020531047A - 神経疾患及び神経損傷における使用のためのbpifb4タンパク質のvtftアイソフォーム - Google Patents

神経疾患及び神経損傷における使用のためのbpifb4タンパク質のvtftアイソフォーム Download PDF

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JP2020531047A
JP2020531047A JP2020530730A JP2020530730A JP2020531047A JP 2020531047 A JP2020531047 A JP 2020531047A JP 2020530730 A JP2020530730 A JP 2020530730A JP 2020530730 A JP2020530730 A JP 2020530730A JP 2020531047 A JP2020531047 A JP 2020531047A
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protein
disease
vtft
vector
seq
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プカ,アンニバレ・アレッサンドロ
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エッレジヴ1・ソチエタ・ア・レスポンサビリタ・リミタータ
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4742Bactericidal/Permeability-increasing protein [BPI]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1751Bactericidal/permeability-increasing protein [BPI]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/20Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C07K2319/21Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag

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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Molecular Biology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Toxicology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020530730A 2017-08-16 2018-08-16 神経疾患及び神経損傷における使用のためのbpifb4タンパク質のvtftアイソフォーム Pending JP2020531047A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP17186528 2017-08-16
EP17186528.0 2017-08-16
PCT/EP2018/072184 WO2019034723A1 (en) 2017-08-16 2018-08-16 VTFT ISOFORM OF BPIFB4 PROTEIN INTENDED FOR USE IN NEURONAL DISEASES AND INJURIES

Publications (2)

Publication Number Publication Date
JP2020531047A true JP2020531047A (ja) 2020-11-05
JP2020531047A5 JP2020531047A5 (https=) 2021-09-24

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JP2020530730A Pending JP2020531047A (ja) 2017-08-16 2018-08-16 神経疾患及び神経損傷における使用のためのbpifb4タンパク質のvtftアイソフォーム

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Country Link
US (1) US12311009B2 (https=)
EP (1) EP3668891B1 (https=)
JP (1) JP2020531047A (https=)
CN (1) CN111094327A (https=)
AU (1) AU2018317807A1 (https=)
CA (1) CA3109159A1 (https=)
ES (1) ES2970198T3 (https=)
WO (1) WO2019034723A1 (https=)

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EP4434534A1 (en) 2023-03-22 2024-09-25 ADvantage Therapeutics, Inc. Klotho mrna

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004500829A (ja) * 2000-04-18 2004-01-15 メルク パテント ゲゼルシャフト ミット ベシュレンクテル ハフトング 新規な脂質結合タンパク質4
JP2016508036A (ja) * 2012-12-28 2016-03-17 アンニバレ・アレッサンドロ・プカ Bpifb4タンパク質の変異体

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9506197D0 (en) * 1995-03-27 1995-05-17 Hoffmann La Roche Inhibition of tau-tau association.
AU2144999A (en) * 1998-01-13 1999-08-02 Claude Gravel Viral vectors encoding neurofilament heavy proteins and their use
WO1999036560A2 (en) * 1998-01-13 1999-07-22 Julien Jean Pierre Viral vectors encoding neurofilament light proteins and their use
US6472378B2 (en) * 1998-08-31 2002-10-29 Pro-Neuron, Inc. Compositions and methods for treatment of mitochondrial diseases
AU2002313992B2 (en) * 1998-08-31 2006-09-21 Pro-Neuron, Inc. Compositions and Methods for Treatment of Mitochondrial Diseases
AU6234700A (en) * 1999-07-27 2001-02-13 Abgenix, Inc. Methods and compositions for inhibiting polypeptide accumulation associated with neurological disorders
US6515197B1 (en) * 2000-08-24 2003-02-04 Cedars-Sinai Medical Center Transgenic mouse expressing a polynucleotide encoding a human ataxin-2 polypeptide
GB0100119D0 (en) * 2001-01-03 2001-02-14 Univ Aberdeen Materials and methods relating to protein aggregation in neurodegenerative disease
JP2004532006A (ja) * 2001-02-08 2004-10-21 インサイト・ゲノミックス・インコーポレイテッド 細胞内シグナル伝達分子
WO2002090504A2 (en) * 2001-05-03 2002-11-14 Curagen Corporation Novel antibodies that bind to antigenic polypeptides, nucleic acids encoding the antigens, and methods of use
WO2003004660A1 (en) * 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
NZ533833A (en) * 2001-12-21 2008-01-31 Salk Inst For Biological Studi Targeted retrograde gene delivery to motor neurons
US20040092715A1 (en) * 2002-02-07 2004-05-13 Li Ding Intracellular signaling molecules
EP1489423A1 (en) * 2003-06-20 2004-12-22 Universite Louis Pasteur Use of mitochondrial uncoupling proteins for diagnostic, prevention and treatment of diseases involving neuromuscular affection
WO2005095610A1 (en) * 2004-03-31 2005-10-13 Agt Biosciences Limited Nucleic acid expressed in the hypothalamus or muscle tissue and uses thereof in diagnoses
WO2006022619A2 (en) * 2004-07-22 2006-03-02 Sequenom, Inc. Methods for identifying risk of type ii diabetes and treatments thereof
CA2584986A1 (en) * 2004-10-22 2006-05-04 Neurologix, Inc. Use of apoptosis inhibiting compounds in degenerative neurological disorders
PL1879623T3 (pl) * 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
KR20080080109A (ko) * 2005-11-04 2008-09-02 바이오겐 아이덱 엠에이 인코포레이티드 도파민성 뉴런의 신경돌기 성장 및 생존의 촉진 방법
PL2066791T3 (pl) * 2006-10-03 2013-02-28 Genzyme Corp Terapia Genowa do leczenia stwardnienia zanikowego bocznego i innych zaburzeń rdzenia kręgowego
EP2150117A4 (en) * 2007-05-02 2011-10-05 Mclean Hospital Corp METHODS AND COMPOSITIONS FOR MITOCHONDRIA REPLACEMENT THERAPY
WO2009020624A1 (en) * 2007-08-08 2009-02-12 The Board Of Trustees Of The University Of Alabama For And On Behalf Of The University Of Alabama Regulators of protein misfolding and neuroprotection and methods of use
MX2010010495A (es) * 2008-04-03 2010-10-15 Hoffmann La Roche Uso de variantes del factor de crecimiento similar a la insulina (igf-i) pegiladas para el tratamiento de trastornos neuromusculares.
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
WO2010088729A1 (en) * 2009-02-04 2010-08-12 University Of Tasmania Through The Menzies Research Institute Compositions and uses therefor
JP5879256B2 (ja) * 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
CA2800375C (en) * 2010-05-27 2021-03-09 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glutaminyl-trna synthetases
US20140147434A1 (en) * 2011-05-06 2014-05-29 Ecole Polytechnique Federale De Lausanne (Epfl) NCoR1 is a Physiological Modulator of Muscle Mass and Oxidative Function
US10815285B2 (en) * 2011-07-01 2020-10-27 University Of South Florida Recombinant adeno-associated virus-mediated expression of fractalkine for treatment of neuroinflammatory and neurodegenerative diseases
JP6253596B2 (ja) * 2012-02-16 2017-12-27 ザ ペン ステイト リサーチ ファンデーション アシル補酵素a:リゾカルジオリピン・アシル基転移酵素1(alcat1)の発現、機能または活性の阻害物質を同定する方法
EP2895621B1 (en) * 2012-09-14 2020-10-21 Population Bio, Inc. Methods and compositions for diagnosing, prognosing, and treating neurological conditions
WO2014161095A1 (en) * 2013-04-04 2014-10-09 Crp40 Inc. Crp40 fragments for the treatment of neurological disorders
AU2014329606B2 (en) * 2013-10-01 2020-02-06 Dana-Farber Cancer Institute, Inc. Methods for the identification, assessment, prevention, and treatment of neurological disorders and diseases using Fndc5
US20170129930A1 (en) * 2014-06-19 2017-05-11 Board Of Trustees Of The University Of Arkansas Compositions and methods for modulating neuronal degeneration
US11027024B2 (en) * 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
CA2973891A1 (en) * 2016-07-21 2018-01-21 Stealth Biotherapeutics Corp Therapeutic compositions including modified creatine compounds and uses thereof to treat and prevent mitochondrial diseases and conditions

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004500829A (ja) * 2000-04-18 2004-01-15 メルク パテント ゲゼルシャフト ミット ベシュレンクテル ハフトング 新規な脂質結合タンパク質4
JP2016508036A (ja) * 2012-12-28 2016-03-17 アンニバレ・アレッサンドロ・プカ Bpifb4タンパク質の変異体

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
AM. J. PATHOL. (2016) VOL.186, ISSUE 10, SUPPLEMENT, P.S1(A1), JPN6022033984, ISSN: 0004848734 *
CIRC. RES. (2015) VOL.117, ISSUE 4, P.333-345, JPN6022033983, ISSN: 0004848735 *

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Publication number Publication date
CN111094327A (zh) 2020-05-01
EP3668891A1 (en) 2020-06-24
US20210128681A1 (en) 2021-05-06
AU2018317807A1 (en) 2020-02-06
EP3668891B1 (en) 2023-07-26
ES2970198T3 (es) 2024-05-27
US12311009B2 (en) 2025-05-27
WO2019034723A1 (en) 2019-02-21
EP3668891C0 (en) 2023-07-26
CA3109159A1 (en) 2019-02-21

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