BR112016019868B1 - Uso da proteína de fusão tatk-cdkl5, formulação farmacêutica e seus usos - Google Patents

Uso da proteína de fusão tatk-cdkl5, formulação farmacêutica e seus usos Download PDF

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Publication number
BR112016019868B1
BR112016019868B1 BR112016019868-9A BR112016019868A BR112016019868B1 BR 112016019868 B1 BR112016019868 B1 BR 112016019868B1 BR 112016019868 A BR112016019868 A BR 112016019868A BR 112016019868 B1 BR112016019868 B1 BR 112016019868B1
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Prior art keywords
cdkl5
fusion protein
tatk
cells
gfp
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BR112016019868-9A
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English (en)
Portuguese (pt)
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BR112016019868A2 (pt
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Elisabetta Ciani
Franco Laccone
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Alma Mater Studiorum-Universita Di Bologna
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Publication of BR112016019868B1 publication Critical patent/BR112016019868B1/pt

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    • 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)
    • 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
    • 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
    • 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)
    • 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
    • A61P25/28Drugs 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
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • 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
    • C07K14/4737C-reactive protein
    • 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
    • C07K14/4738Cell cycle regulated proteins, e.g. cyclin, CDC, INK-CCR
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    • 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)
    • C12N9/1205Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
    • CCHEMISTRY; METALLURGY
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    • 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
    • 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
    • C07K2319/21Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/40Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/60Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/40Regulators of development
    • C12N2501/405Cell cycle regulated proteins, e.g. cyclins, cyclin-dependant kinases

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  • Health & Medical Sciences (AREA)
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  • 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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • 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)
BR112016019868-9A 2014-02-28 2015-02-27 Uso da proteína de fusão tatk-cdkl5, formulação farmacêutica e seus usos BR112016019868B1 (pt)

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 (2)

Publication Number Publication Date
BR112016019868A2 BR112016019868A2 (pt) 2017-10-17
BR112016019868B1 true BR112016019868B1 (pt) 2023-11-28

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BR112016019868-9A BR112016019868B1 (pt) 2014-02-28 2015-02-27 Uso da proteína de fusão tatk-cdkl5, formulação farmacêutica e seus usos

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US (4) US9290746B2 (https=)
EP (2) EP3110837B1 (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) DK3608334T3 (https=)
DO (1) DOP2016000220A (https=)
EA (2) EA038000B1 (https=)
ES (2) ES2745335T3 (https=)
HR (2) HRP20191511T1 (https=)
HU (2) HUE055282T2 (https=)
IL (2) IL247481B (https=)
LT (2) LT3608334T (https=)
MX (2) MX2016011167A (https=)
MY (1) MY181566A (https=)
NI (1) NI201600127A (https=)
PE (1) PE20161406A1 (https=)
PH (1) PH12016501689A1 (https=)
PL (2) PL3608334T3 (https=)
PT (2) PT3110837T (https=)
RS (2) RS59344B1 (https=)
SG (2) SG10202000537RA (https=)
SI (2) SI3110837T1 (https=)
SM (2) SMT202100478T1 (https=)
SV (1) SV2016005264A (https=)
WO (1) WO2015128746A2 (https=)
ZA (2) ZA201606655B (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106255706A (zh) * 2014-02-28 2016-12-21 万校之母-博洛尼亚大学 TATk‑CDKL5融合蛋白、其组合物、配制品以及用途
US20210268072A1 (en) * 2016-06-28 2021-09-02 Alma Mater Studiorum - Universitá Di Bologna TATk-CDKL5 Fusion Proteins, Compositions, Formulations, and Use Thereof
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
CA3150224A1 (en) 2019-09-10 2021-03-18 Obsidian Therapeutics, Inc. Ca2 compositions and methods for tunable regulation
CA3156506A1 (en) * 2019-10-30 2021-05-06 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
KR20230128001A (ko) 2020-12-01 2023-09-01 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도
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

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2011322715A1 (en) * 2010-10-25 2013-05-23 Centre National De La Recherche Scientifique Treatment of MeCP2-associated disorders
CN106255706A (zh) * 2014-02-28 2016-12-21 万校之母-博洛尼亚大学 TATk‑CDKL5融合蛋白、其组合物、配制品以及用途

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KR102307276B1 (ko) 2021-09-30
CN106255706A (zh) 2016-12-21
NZ723371A (en) 2023-11-24
HUE045393T2 (hu) 2019-12-30
CA2940401A1 (en) 2015-09-03
US9944910B2 (en) 2018-04-17
SMT201900504T1 (it) 2019-11-13
ES2885245T3 (es) 2021-12-13
EA202190727A3 (ru) 2021-11-30
KR20170002372A (ko) 2017-01-06
IL247481A0 (en) 2016-11-30
EP3608334B1 (en) 2021-05-19
HRP20211324T1 (hr) 2021-11-26
US9290746B2 (en) 2016-03-22
US20180327725A1 (en) 2018-11-15
PH12016501689B1 (en) 2016-10-03
PL3608334T3 (pl) 2021-11-22
EA202190727A2 (ru) 2021-07-30
JP2020062022A (ja) 2020-04-23
AU2015221860A1 (en) 2016-09-08
DK3608334T3 (da) 2021-08-23
PT3110837T (pt) 2019-09-12
NI201600127A (es) 2017-03-13
PH12016501689A1 (en) 2016-10-03
PL3110837T3 (pl) 2020-04-30
MX2020001223A (es) 2022-05-31
LT3608334T (lt) 2021-11-25
MY181566A (en) 2020-12-29
SV2016005264A (es) 2017-02-15
JP6629747B2 (ja) 2020-01-15
US10907138B2 (en) 2021-02-02
MX392778B (es) 2025-03-24
IL275435B (en) 2021-07-29
IL275435A (en) 2020-08-31
SG10202000537RA (en) 2020-03-30
CR20160392A (es) 2017-06-12
AU2015221860B2 (en) 2019-09-12
US20200199549A1 (en) 2020-06-25
CY1124727T1 (el) 2022-07-22
HUE055282T2 (hu) 2021-11-29
JP6896050B2 (ja) 2021-06-30
SI3110837T1 (sl) 2019-12-31
EA201691691A1 (ru) 2017-05-31
ZA201906304B (en) 2022-03-30
RS59344B1 (sr) 2019-10-31
AU2019219743B2 (en) 2020-10-29
ES2745335T3 (es) 2020-02-28
EP3110837B1 (en) 2019-06-12
JP2017507655A (ja) 2017-03-23
CL2016002142A1 (es) 2017-09-08
SG11201606863YA (en) 2016-09-29
SI3608334T1 (sl) 2021-11-30
RS62244B1 (sr) 2021-09-30
LT3110837T (lt) 2019-10-10
EP3608334A1 (en) 2020-02-12
US20150247134A1 (en) 2015-09-03
DOP2016000220A (es) 2016-12-15
CA2940401C (en) 2022-11-29
US20160194617A1 (en) 2016-07-07
DK3110837T3 (da) 2019-09-16
BR112016019868A2 (pt) 2017-10-17
SMT202100478T1 (it) 2021-09-14
WO2015128746A2 (en) 2015-09-03
US10584318B2 (en) 2020-03-10
PT3608334T (pt) 2021-08-25
ZA201606655B (en) 2020-05-27
PE20161406A1 (es) 2017-01-14
AU2019219743A1 (en) 2019-09-19
IL247481B (en) 2020-07-30
MX2016011167A (es) 2017-04-06
EA038000B1 (ru) 2021-06-22
HRP20191511T1 (hr) 2019-11-29
WO2015128746A3 (en) 2015-11-26
CY1122644T1 (el) 2021-03-12
EP3110837A2 (en) 2017-01-04

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