CA2940401C - Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof - Google Patents

Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof Download PDF

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Publication number
CA2940401C
CA2940401C CA2940401A CA2940401A CA2940401C CA 2940401 C CA2940401 C CA 2940401C CA 2940401 A CA2940401 A CA 2940401A CA 2940401 A CA2940401 A CA 2940401A CA 2940401 C CA2940401 C CA 2940401C
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CA
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Prior art keywords
cdkl5
fusion protein
protein
nucleic acid
tatk
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Active
Application number
CA2940401A
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English (en)
French (fr)
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CA2940401A1 (en
Inventor
Elisabetta CIANI
Franco Laccone
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Universita di Bologna
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Universita di Bologna
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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
    • 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)
    • 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

Landscapes

  • 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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Toxicology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (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)
CA2940401A 2014-02-28 2015-02-27 Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof Active CA2940401C (en)

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
CA2940401A1 CA2940401A1 (en) 2015-09-03
CA2940401C true CA2940401C (en) 2022-11-29

Family

ID=53539745

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2940401A Active CA2940401C (en) 2014-02-28 2015-02-27 Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof

Country Status (33)

Country Link
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

Family Cites Families (2)

* 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

Also Published As

Publication number Publication date
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
BR112016019868B1 (pt) 2023-11-28
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
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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