CN106255706A - TATk‑CDKL5融合蛋白、其组合物、配制品以及用途 - Google Patents

TATk‑CDKL5融合蛋白、其组合物、配制品以及用途 Download PDF

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Publication number
CN106255706A
CN106255706A CN201580010858.9A CN201580010858A CN106255706A CN 106255706 A CN106255706 A CN 106255706A CN 201580010858 A CN201580010858 A CN 201580010858A CN 106255706 A CN106255706 A CN 106255706A
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China
Prior art keywords
cdkl5
fusion protein
polypeptide
tatk
seq
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Pending
Application number
CN201580010858.9A
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English (en)
Chinese (zh)
Inventor
伊丽莎白·恰妮
佛朗科·拉科内
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Mother Of Bologna University - University Of China
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Mother Of Bologna University - University Of China
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Publication of CN106255706A publication Critical patent/CN106255706A/zh
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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
    • 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)
    • 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)
    • C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Immunology (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)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
CN201580010858.9A 2014-02-28 2015-02-27 TATk‑CDKL5融合蛋白、其组合物、配制品以及用途 Pending CN106255706A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461946280P 2014-02-28 2014-02-28
US61/946,280 2014-02-28
PCT/IB2015/000999 WO2015128746A2 (en) 2014-02-28 2015-02-27 Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof

Publications (1)

Publication Number Publication Date
CN106255706A true CN106255706A (zh) 2016-12-21

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CN201580010858.9A Pending CN106255706A (zh) 2014-02-28 2015-02-27 TATk‑CDKL5融合蛋白、其组合物、配制品以及用途

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Country Link
US (4) US9290746B2 (https=)
EP (2) EP3608334B1 (https=)
JP (2) JP6629747B2 (https=)
KR (1) KR102307276B1 (https=)
CN (1) CN106255706A (https=)
AU (2) AU2015221860B2 (https=)
BR (1) BR112016019868B1 (https=)
CA (1) CA2940401C (https=)
CL (1) CL2016002142A1 (https=)
CR (1) CR20160392A (https=)
CY (2) CY1122644T1 (https=)
DK (2) DK3110837T3 (https=)
DO (1) DOP2016000220A (https=)
EA (2) EA202190727A3 (https=)
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HR (2) HRP20191511T1 (https=)
HU (2) HUE055282T2 (https=)
IL (2) IL247481B (https=)
LT (2) LT3608334T (https=)
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PE (1) PE20161406A1 (https=)
PH (1) PH12016501689B1 (https=)
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Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10202000537RA (en) * 2014-02-28 2020-03-30 Univ Bologna Alma Mater Studiorum Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof
EP3475418A4 (en) * 2016-06-28 2020-03-04 Alma Mater Studiorum -Universita' di Bologna TATK-CDKL5 FUSION PROTEINS, COMPOSITIONS, FORMULATIONS AND THEIR USE
JP2021505135A (ja) * 2017-11-30 2021-02-18 アミカス セラピューティックス インコーポレイテッド Ｃｄｋｌ５発現変異体及びｃｄｋｌ５融合タンパク質
WO2021050789A1 (en) 2019-09-10 2021-03-18 Obsidian Therapeutics, Inc. Ca2-il15 fusion proteins for tunable regulation
US20230043046A1 (en) * 2019-10-30 2023-02-09 Amicus Therapeutics, Inc. Recombinant CDKL5 Proteins, Gene Therapy and Production Methods
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
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
EP4387641A4 (en) * 2021-08-18 2025-07-09 Ultragenyx Pharmaceutical Inc RECOMBINANT AAV FORMULATIONS
WO2025050032A1 (en) * 2023-08-31 2025-03-06 Ultragenyx Pharmaceutical Inc. Methods of producing cdkl5 proteins and uses of the same

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103260612A (zh) * 2010-10-25 2013-08-21 艾克斯-马赛大学 MeCP2相关性病症的治疗

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10202000537RA (en) * 2014-02-28 2020-03-30 Univ Bologna Alma Mater Studiorum Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103260612A (zh) * 2010-10-25 2013-08-21 艾克斯-马赛大学 MeCP2相关性病症的治疗

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
ANONYMOUS: "Bandi per Assegni di ricerca", 《HTTP://BANDI.MIUR.IT/BANDI.PHP/PUBLIC/FELLOWSHIP/ID_FELLOW/67724》 *
FLINTERMAN M ET AL.: "Delivery of therapeutic proteins as secretable TAT fusion products", 《MOL THER》 *
L WEAVING ET AL.: "Rett syndrome:clinical review and genetic update", 《J MED GENET》 *
L WEAVING ET AL.: "Rett syndrome:clinical review and genetic update.", 《J MED GENET》 *
SCALA E ET AL.: "CDKL5/STK9 is mutated in Rett syndrome variant with infantile spasms", 《J MED GENET》 *
孟洪弟等: "Rett综合征患儿线粒体结构及功能缺陷的研究", 《中华儿科杂志》 *

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PT3608334T (pt) 2021-08-25
CY1122644T1 (el) 2021-03-12
EA202190727A2 (ru) 2021-07-30
NI201600127A (es) 2017-03-13
JP2020062022A (ja) 2020-04-23
US20180327725A1 (en) 2018-11-15
PL3110837T3 (pl) 2020-04-30
SMT201900504T1 (it) 2019-11-13
ES2885245T3 (es) 2021-12-13
DK3110837T3 (da) 2019-09-16
US9944910B2 (en) 2018-04-17
EP3110837A2 (en) 2017-01-04
SMT202100478T1 (it) 2021-09-14
PH12016501689A1 (en) 2016-10-03
IL247481A0 (en) 2016-11-30
MX2016011167A (es) 2017-04-06
KR102307276B1 (ko) 2021-09-30
AU2015221860A1 (en) 2016-09-08
HRP20211324T1 (hr) 2021-11-26
HUE045393T2 (hu) 2019-12-30
EA202190727A3 (ru) 2021-11-30
JP6629747B2 (ja) 2020-01-15
SV2016005264A (es) 2017-02-15
KR20170002372A (ko) 2017-01-06
PH12016501689B1 (en) 2016-10-03
EP3110837B1 (en) 2019-06-12
SI3110837T1 (sl) 2019-12-31
ZA201906304B (en) 2022-03-30
IL275435A (en) 2020-08-31
WO2015128746A3 (en) 2015-11-26
BR112016019868A2 (pt) 2017-10-17
MX2020001223A (es) 2022-05-31
HUE055282T2 (hu) 2021-11-29
ES2745335T3 (es) 2020-02-28
PL3608334T3 (pl) 2021-11-22
SG11201606863YA (en) 2016-09-29
RS59344B1 (sr) 2019-10-31
AU2019219743B2 (en) 2020-10-29
DOP2016000220A (es) 2016-12-15
EA201691691A1 (ru) 2017-05-31
DK3608334T3 (da) 2021-08-23
SG10202000537RA (en) 2020-03-30
JP6896050B2 (ja) 2021-06-30
SI3608334T1 (sl) 2021-11-30
AU2019219743A1 (en) 2019-09-19
WO2015128746A2 (en) 2015-09-03
US20150247134A1 (en) 2015-09-03
LT3608334T (lt) 2021-11-25
MY181566A (en) 2020-12-29
US20160194617A1 (en) 2016-07-07
US10907138B2 (en) 2021-02-02
LT3110837T (lt) 2019-10-10
JP2017507655A (ja) 2017-03-23
HRP20191511T1 (hr) 2019-11-29
EA038000B1 (ru) 2021-06-22
RS62244B1 (sr) 2021-09-30
US20200199549A1 (en) 2020-06-25
CL2016002142A1 (es) 2017-09-08
PT3110837T (pt) 2019-09-12
AU2015221860B2 (en) 2019-09-12
IL247481B (en) 2020-07-30
CY1124727T1 (el) 2022-07-22
EP3608334B1 (en) 2021-05-19
EP3608334A1 (en) 2020-02-12
ZA201606655B (en) 2020-05-27
BR112016019868B1 (pt) 2023-11-28
PE20161406A1 (es) 2017-01-14
CA2940401A1 (en) 2015-09-03
US9290746B2 (en) 2016-03-22
MX392778B (es) 2025-03-24
CR20160392A (es) 2017-06-12
NZ723371A (en) 2023-11-24
CA2940401C (en) 2022-11-29
US10584318B2 (en) 2020-03-10
IL275435B (en) 2021-07-29

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