BR112018077225A2 - proteínas de fusão tatk-cdkl5, composições, formulações e uso das mesmas - Google Patents

proteínas de fusão tatk-cdkl5, composições, formulações e uso das mesmas

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Publication number
BR112018077225A2
BR112018077225A2 BR112018077225-9A BR112018077225A BR112018077225A2 BR 112018077225 A2 BR112018077225 A2 BR 112018077225A2 BR 112018077225 A BR112018077225 A BR 112018077225A BR 112018077225 A2 BR112018077225 A2 BR 112018077225A2
Authority
BR
Brazil
Prior art keywords
tatk
fusion proteins
compositions
formulations
cdk1
Prior art date
Application number
BR112018077225-9A
Other languages
English (en)
Inventor
Ciani Elisabetta
Laccone Franco
clark Sean
Original Assignee
Alma Mater Studiorum - Università di Bologna
Amicus Therapeutics, Inc.
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 Alma Mater Studiorum - Università di Bologna, Amicus Therapeutics, Inc. filed Critical Alma Mater Studiorum - Università di Bologna
Publication of BR112018077225A2 publication Critical patent/BR112018077225A2/pt

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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
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45Transferases (2)
    • 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
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/20Hypnotics; Sedatives
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11022Cyclin-dependent kinase (2.7.11.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/20Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11023[RNA-polymerase-subunit] kinase (2.7.11.23)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Anesthesiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

são aqui divulgadas composições e formulações contendo uma proteína de fusão tat?-cdkl5. também divulgados métodos de produção de uma proteína de fusão tatk-cdkl5 a partir de vetores contendo um cdna de tatk-cdkl5 e métodos de transdução de células com os vetores contendo um cdna de tatk-cdkl5 e a proteína de fusão tatk-cdkl5. também são divulgados usos de proteínas de fusão tatk-cdkl5 para tratar deficiências de cdkl5 por administração sistêmica ou intravenosa das proteínas de fusão.
BR112018077225-9A 2016-06-28 2017-06-28 proteínas de fusão tatk-cdkl5, composições, formulações e uso das mesmas BR112018077225A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662355579P 2016-06-28 2016-06-28
US62/355,579 2016-06-28
US201662381886P 2016-08-31 2016-08-31
US62/381,886 2016-08-31
PCT/US2017/039692 WO2018005617A2 (en) 2016-06-28 2017-06-28 TATĸ-CDKL5 FUSION PROTEINS, COMPOSITIONS, FORMULATIONS, AND USE THEREOF

Publications (1)

Publication Number Publication Date
BR112018077225A2 true BR112018077225A2 (pt) 2019-04-09

Family

ID=60787683

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018077225-9A BR112018077225A2 (pt) 2016-06-28 2017-06-28 proteínas de fusão tatk-cdkl5, composições, formulações e uso das mesmas

Country Status (12)

Country Link
US (1) US20210268072A1 (pt)
EP (1) EP3475418A4 (pt)
JP (1) JP2019522989A (pt)
KR (1) KR20190034546A (pt)
CN (1) CN109844109A (pt)
AU (1) AU2017290047A1 (pt)
BR (1) BR112018077225A2 (pt)
CA (1) CA3029473A1 (pt)
IL (1) IL263842A (pt)
MX (1) MX2018016417A (pt)
TW (1) TW201803893A (pt)
WO (1) WO2018005617A2 (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220332780A1 (en) 2019-09-10 2022-10-20 Obsidian Therapeutics, Inc. Ca2-il15 fusion proteins for tunable regulation
KR20220106981A (ko) * 2019-10-30 2022-08-01 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 cdkl5 단백질, 유전자 치료 및 생산 방법
JP2023551911A (ja) 2020-12-01 2023-12-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アンジェルマン症候群の治療のための組成物及びその使用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN100489101C (zh) * 2006-12-19 2009-05-20 浙江大学 G-eGFP蛋白及其制备方法与应用
CN101704893A (zh) * 2009-11-02 2010-05-12 江苏大学 带穿膜序列的人Scurfin蛋白全长及片段以及制备方法
SI3110837T1 (sl) * 2014-02-28 2019-12-31 Alma Mater Studiorum - Universita Di Bologna Fuzijski proteini TATK-CDKL5, sestavki, formulacije in njihova uporaba
JP2019511495A (ja) * 2016-03-10 2019-04-25 アルマ マータ ステューディオラム — ユニバーシタ ディ ボローニャ GSK3β阻害薬チデグルシブによるCDKL5障害の治療

Also Published As

Publication number Publication date
WO2018005617A3 (en) 2018-02-08
EP3475418A4 (en) 2020-03-04
KR20190034546A (ko) 2019-04-02
IL263842A (en) 2019-01-31
US20210268072A1 (en) 2021-09-02
JP2019522989A (ja) 2019-08-22
CN109844109A (zh) 2019-06-04
WO2018005617A2 (en) 2018-01-04
EP3475418A2 (en) 2019-05-01
CA3029473A1 (en) 2018-01-04
TW201803893A (zh) 2018-02-01
MX2018016417A (es) 2019-09-09
AU2017290047A1 (en) 2019-01-17

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Legal Events

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B11A Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing
B11Y Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette]
B350 Update of information on the portal [chapter 15.35 patent gazette]