EA038000B1 - Слитый белок для лечения одного или нескольких симптомов дефицита cdkl5 и его применение - Google Patents

Слитый белок для лечения одного или нескольких симптомов дефицита cdkl5 и его применение Download PDF

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Publication number
EA038000B1
EA038000B1 EA201691691A EA201691691A EA038000B1 EA 038000 B1 EA038000 B1 EA 038000B1 EA 201691691 A EA201691691 A EA 201691691A EA 201691691 A EA201691691 A EA 201691691A EA 038000 B1 EA038000 B1 EA 038000B1
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EA
Eurasian Patent Office
Prior art keywords
cdkl5
fusion protein
tatk
cells
protein
Prior art date
Application number
EA201691691A
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English (en)
Russian (ru)
Other versions
EA201691691A1 (ru
Inventor
Элизабетта Чани
Франко Лакконе
Original Assignee
Альма Матер Студиорум-Юниверсита Ди Болонья
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Application filed by Альма Матер Студиорум-Юниверсита Ди Болонья filed Critical Альма Матер Студиорум-Юниверсита Ди Болонья
Publication of EA201691691A1 publication Critical patent/EA201691691A1/ru
Publication of EA038000B1 publication Critical patent/EA038000B1/ru

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • C07K14/4737—C-reactive protein
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • C07K14/4738—Cell cycle regulated proteins, e.g. cyclin, CDC, INK-CCR
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/60—Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/40—Regulators of development
    • C12N2501/405—Cell cycle regulated proteins, e.g. cyclins, cyclin-dependant kinases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11022—Cyclin-dependent kinase (2.7.11.22)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
EA201691691A 2014-02-28 2015-02-27 Слитый белок для лечения одного или нескольких симптомов дефицита cdkl5 и его применение EA038000B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461946280P 2014-02-28 2014-02-28
PCT/IB2015/000999 WO2015128746A2 (en) 2014-02-28 2015-02-27 Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof

Publications (2)

Publication Number Publication Date
EA201691691A1 EA201691691A1 (ru) 2017-05-31
EA038000B1 true EA038000B1 (ru) 2021-06-22

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Application Number Title Priority Date Filing Date
EA201691691A EA038000B1 (ru) 2014-02-28 2015-02-27 Слитый белок для лечения одного или нескольких симптомов дефицита cdkl5 и его применение
EA202190727A EA202190727A3 (ru) 2014-02-28 2015-02-27 Рекомбинантная нуклеиновая кислота, вектор, содержащий ее, и способ, предусматривающий трансфекцию клеток

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EA202190727A EA202190727A3 (ru) 2014-02-28 2015-02-27 Рекомбинантная нуклеиновая кислота, вектор, содержащий ее, и способ, предусматривающий трансфекцию клеток

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HUE055282T2 (hu) * 2014-02-28 2021-11-29 Univ Bologna Alma Mater Studiorum TATK-CDKL5 fúziós fehérjék, készítmények, formulák és azok alkalmazása
US20210268072A1 (en) * 2016-06-28 2021-09-02 Alma Mater Studiorum - Universitá Di Bologna TATk-CDKL5 Fusion Proteins, Compositions, Formulations, and Use Thereof
CN111936624A (zh) * 2017-11-30 2020-11-13 阿米库斯治疗学公司 Cdkl5表达变体和cdkl5融合蛋白
CN114651003A (zh) 2019-09-10 2022-06-21 黑曜石疗法公司 用于可调调节的ca2-il15融合蛋白
WO2021087282A1 (en) * 2019-10-30 2021-05-06 Amicus Therapeutics, Inc. Recombinant cdkl5 proteins, gene therapy and production methods
AU2021218703A1 (en) 2020-02-14 2022-07-28 Ultragenyx Pharmaceutical Inc. Gene therapy for treating CDKL5 deficiency disorder
IT202000025423A1 (it) * 2020-10-27 2022-04-27 Ulisse Biomed S P A Uso di proteine per il trattamento di patologie associate a tossine formanti pori o a proteine virali
WO2022119890A1 (en) 2020-12-01 2022-06-09 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
CA3229127A1 (en) * 2021-08-18 2023-02-23 Julie Yu Wei Recombinant aav formulations
CN116874571A (zh) * 2021-12-17 2023-10-13 博格隆(上海)生物技术有限公司 突变的蛋白a结构域c及其应用
WO2025050032A1 (en) * 2023-08-31 2025-03-06 Ultragenyx Pharmaceutical Inc. Methods of producing cdkl5 proteins and uses of the same

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NZ609640A (en) * 2010-10-25 2015-06-26 Univ D’Aix Marseille Treatment of mecp2-associated disorders
HUE055282T2 (hu) * 2014-02-28 2021-11-29 Univ Bologna Alma Mater Studiorum TATK-CDKL5 fúziós fehérjék, készítmények, formulák és azok alkalmazása

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Anonymous: "Gene therapy reverses Rett syndrome symptoms in mice -", 30 September 2013 (2013-09-30), XP55208894, retrieved from the Internet: URL:http://sfari.org/news-and-opinion/news/2013/gene-therapy-reverses-rett-syndrome-symptoms-in-mice [retrieved on 2015-08-21] the whole document *
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MAE, M. ; LANGEL, U.: "Cell-penetrating peptides as vectors for peptide, protein and oligonucleotide delivery", CURRENT OPINION IN PHARMACOLOGY, ELSEVIER SCIENCE PUBLISHERS,, NL, vol. 6, no. 5, 1 October 2006 (2006-10-01), NL, pages 509 - 514, XP028058465, ISSN: 1471-4892, DOI: 10.1016/j.coph.2006.04.004 *
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IL247481A0 (en) 2016-11-30
ZA201906304B (en) 2022-03-30
DK3110837T3 (da) 2019-09-16
IL275435A (en) 2020-08-31
EP3608334A1 (en) 2020-02-12
LT3110837T (lt) 2019-10-10
RS62244B1 (sr) 2021-09-30
ZA201606655B (en) 2020-05-27
US20160194617A1 (en) 2016-07-07
HUE045393T2 (hu) 2019-12-30
US9290746B2 (en) 2016-03-22
DK3608334T3 (da) 2021-08-23
PH12016501689B1 (en) 2016-10-03
AU2015221860A1 (en) 2016-09-08
LT3608334T (lt) 2021-11-25
MY181566A (en) 2020-12-29
MX2016011167A (es) 2017-04-06
CR20160392A (es) 2017-06-12
US20180327725A1 (en) 2018-11-15
CA2940401C (en) 2022-11-29
CY1122644T1 (el) 2021-03-12
IL275435B (en) 2021-07-29
MX392778B (es) 2025-03-24
JP2017507655A (ja) 2017-03-23
AU2015221860B2 (en) 2019-09-12
IL247481B (en) 2020-07-30
PL3110837T3 (pl) 2020-04-30
US10584318B2 (en) 2020-03-10
CY1124727T1 (el) 2022-07-22
CL2016002142A1 (es) 2017-09-08
JP6629747B2 (ja) 2020-01-15
KR102307276B1 (ko) 2021-09-30
SMT202100478T1 (it) 2021-09-14
US10907138B2 (en) 2021-02-02
HUE055282T2 (hu) 2021-11-29
EP3608334B1 (en) 2021-05-19
ES2745335T3 (es) 2020-02-28
DOP2016000220A (es) 2016-12-15
WO2015128746A3 (en) 2015-11-26
KR20170002372A (ko) 2017-01-06
WO2015128746A2 (en) 2015-09-03
AU2019219743B2 (en) 2020-10-29
SI3608334T1 (sl) 2021-11-30
CN106255706A (zh) 2016-12-21
EA202190727A3 (ru) 2021-11-30
EP3110837B1 (en) 2019-06-12
SG11201606863YA (en) 2016-09-29
US20200199549A1 (en) 2020-06-25
JP6896050B2 (ja) 2021-06-30
PT3110837T (pt) 2019-09-12
SG10202000537RA (en) 2020-03-30
SI3110837T1 (sl) 2019-12-31
NI201600127A (es) 2017-03-13
EP3110837A2 (en) 2017-01-04
NZ723371A (en) 2023-11-24
EA201691691A1 (ru) 2017-05-31
SV2016005264A (es) 2017-02-15
SMT201900504T1 (it) 2019-11-13
PT3608334T (pt) 2021-08-25
BR112016019868A2 (pt) 2017-10-17
JP2020062022A (ja) 2020-04-23
PL3608334T3 (pl) 2021-11-22
HRP20191511T1 (hr) 2019-11-29
PH12016501689A1 (en) 2016-10-03
PE20161406A1 (es) 2017-01-14
ES2885245T3 (es) 2021-12-13
BR112016019868B1 (pt) 2023-11-28
HRP20211324T1 (hr) 2021-11-26
MX2020001223A (es) 2022-05-31
CA2940401A1 (en) 2015-09-03
RS59344B1 (sr) 2019-10-31
US9944910B2 (en) 2018-04-17
EA202190727A2 (ru) 2021-07-30
AU2019219743A1 (en) 2019-09-19
US20150247134A1 (en) 2015-09-03

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