CY1120485T1 - Μεθοδοι για τη συζευξη πεπτιδιων-στοχων με ανασυνδυασμενα λυσοσωμικα ενζυμα για βελτιωμενες θεραπευτικες αγωγες διαταραχων λυσοσωμικης αποθηκευσης - Google Patents

Μεθοδοι για τη συζευξη πεπτιδιων-στοχων με ανασυνδυασμενα λυσοσωμικα ενζυμα για βελτιωμενες θεραπευτικες αγωγες διαταραχων λυσοσωμικης αποθηκευσης

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Publication number
CY1120485T1
CY1120485T1 CY20181100212T CY181100212T CY1120485T1 CY 1120485 T1 CY1120485 T1 CY 1120485T1 CY 20181100212 T CY20181100212 T CY 20181100212T CY 181100212 T CY181100212 T CY 181100212T CY 1120485 T1 CY1120485 T1 CY 1120485T1
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Cyprus
Prior art keywords
methods
peptide
lysosomal enzymes
targets
connection
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CY20181100212T
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English (en)
Inventor
Hung Do
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Amicus Therapeutics, Inc.
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Publication date
Application filed by Amicus Therapeutics, Inc. filed Critical Amicus Therapeutics, Inc.
Publication of CY1120485T1 publication Critical patent/CY1120485T1/el

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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/107—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length by chemical modification of precursor peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/68—Medicinal 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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—Medicinal 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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6847—Medicinal 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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a hormone or a hormone-releasing or -inhibiting factor
    • 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
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/62—Medicinal 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
    • A61K47/64—Drug-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
    • A61K47/642—Drug-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 the peptide or protein in the drug conjugate being a cytokine, e.g. IL2, chemokine, growth factors or interferons being the inactive part of the conjugate
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
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    • 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
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/16—Extraction; Separation; Purification by chromatography
    • C07K1/18—Ion-exchange chromatography
    • C—CHEMISTRY; METALLURGY
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    • C07K1/14—Extraction; Separation; Purification
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    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
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    • C07—ORGANIC CHEMISTRY
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    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/30—Extraction; Separation; Purification by precipitation
    • C07K1/306—Extraction; Separation; Purification by precipitation by crystallization
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    • C07—ORGANIC CHEMISTRY
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    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/36—Extraction; Separation; Purification by a combination of two or more processes of different types
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    • 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
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    • C07—ORGANIC CHEMISTRY
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    • 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
    • C07K14/575—Hormones
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    • 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
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site

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Abstract

To παρόν έγγραφο περιγράφει μεθόδους σύζευξης των πεπτιδίων-στόχων με ανασυνδυασμένα λυσοσωμικά ένζυμα μέσω της τροποποίησης του αμινο (Ν)-τερματικού και ενός ή περισσοτέρων υπολειμμάτων λυσίνης με ανασυνδυασμένα ανθρώπινα λυσοσωμικά ένζυμα, χρησιμοποιώντας έναν πρώτο παράγοντα διασταύρωσης, ώστε να δημιουργηθούν ανασυνδυασμένα ανθρώπινα λυσοσωμικά ένζυμα τροποποιημένα με έναν πρώτο παράγοντα διασταύρωσης, τροποποιώντας το πρώτο αμινοξύ εντός ενός βραχέως συνδέτη στο αμινο (Ν)-τερματικό σε μια παραλλαγή του πεπτιδίου IGF-2 χρησιμοποιώντας έναν δεύτερο παράγοντα διασταύρωσης, ώστε να δημιουργηθεί μια δεύτερη παραλλαγή του πεπτιδίου IGF-2 τροποποιημένου με έναν δεύτερο παράγοντα διασταύρωσης, και κατόπιν με τη σύζευξη του πρώτου ανασυνδυασμένου ανθρώπινου λυσοσωμικού ενζύμου που έχει τροποποιηθεί με έναν πρώτο παράγοντα διασταύρωσης με τη δεύτερη παραλλαγή του πεπτιδίου IGF-2 που έχει τροποποιηθεί με έναν δεύτερο παράγοντα διασταύρωσης που περιέχει έναν βραχύ συνδέτη. Το παρόν έγγραφο περιγράφει, επίσης, συνθετικά συζεύγματα που χαρακτηρίζονται από μεγαλύτερη συγγένεια με τον υποδοχέα IGF2/CI-MPR και κυτταρική πρόσληψη με χρήση των μεθόδων που αποκαλύπτονται στο παρόν έγγραφο. Επίσης, στο παρόν έγγραφο περιγράφονται μέθοδοι θεραπευτικής αντιμετώπισης οι οποίες χρησιμοποιούν τα αποκαλυφθέντα συζεύγματα.
CY20181100212T 2011-05-27 2018-02-22 Μεθοδοι για τη συζευξη πεπτιδιων-στοχων με ανασυνδυασμενα λυσοσωμικα ενζυμα για βελτιωμενες θεραπευτικες αγωγες διαταραχων λυσοσωμικης αποθηκευσης CY1120485T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161490957P 2011-05-27 2011-05-27
PCT/US2012/039705 WO2012166653A2 (en) 2011-05-27 2012-05-25 Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases

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CY1120485T1 true CY1120485T1 (el) 2019-07-10

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CY20181100212T CY1120485T1 (el) 2011-05-27 2018-02-22 Μεθοδοι για τη συζευξη πεπτιδιων-στοχων με ανασυνδυασμενα λυσοσωμικα ενζυμα για βελτιωμενες θεραπευτικες αγωγες διαταραχων λυσοσωμικης αποθηκευσης

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CA2836318C (en) 2018-11-27
US9545450B2 (en) 2017-01-17
US20170319710A1 (en) 2017-11-09
US20140302001A1 (en) 2014-10-09
SI2714752T1 (en) 2018-03-30
CN104160033B (zh) 2018-06-01
LT2714752T (lt) 2018-03-12
EP2714752A2 (en) 2014-04-09
CN108586621A (zh) 2018-09-28
JP2014518063A (ja) 2014-07-28
HRP20180291T1 (hr) 2018-03-23
BR112013030432A2 (pt) 2016-12-13
PL2714752T3 (pl) 2018-04-30
DK2714752T3 (en) 2018-02-26
PT2714752T (pt) 2018-02-26
KR20140033155A (ko) 2014-03-17
EP2714752B1 (en) 2017-11-22
WO2012166653A2 (en) 2012-12-06
ES2660185T3 (es) 2018-03-21
HUE038172T2 (hu) 2018-09-28
WO2012166653A3 (en) 2014-05-01
EP2714752A4 (en) 2015-08-26
JP2017225444A (ja) 2017-12-28
CN104160033A (zh) 2014-11-19
KR101955054B1 (ko) 2019-03-07
JP6300720B2 (ja) 2018-03-28
NO2820016T3 (el) 2017-12-30
US10660972B2 (en) 2020-05-26
TR201802230T4 (tr) 2018-03-21
KR20190022943A (ko) 2019-03-06
RS56913B1 (sr) 2018-05-31
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