AR068144A1 - Administracion subcutanea de alfa-galactosidasa a - Google Patents

Administracion subcutanea de alfa-galactosidasa a

Info

Publication number
AR068144A1
AR068144A1 ARP080103780A ARP080103780A AR068144A1 AR 068144 A1 AR068144 A1 AR 068144A1 AR P080103780 A ARP080103780 A AR P080103780A AR P080103780 A ARP080103780 A AR P080103780A AR 068144 A1 AR068144 A1 AR 068144A1
Authority
AR
Argentina
Prior art keywords
alpha
galactosidase
gal
cell
composition
Prior art date
Application number
ARP080103780A
Other languages
English (en)
Original Assignee
Shire Human Genetic Therapies
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of AR068144A1 publication Critical patent/AR068144A1/es

Links

Classifications

    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/12—Carboxylic acids; Salts or anhydrides thereof
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Dermatology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Métodos para administrar alfa-galactosidase A para el tratamiento de las deficiencias de alfa-galactosidasa A que incluye la enfermedad de Fabri. Reivindicacion 1: Una composicion que comprende de aproximadamente 1 mg/ml a aproximadamente 60 mg/ml de alfa-Gal A, de aproximadamente 2% a aproximadamente 10% (p/v) de carbohidrato, de aproximadamente 5 mM a aproximadamente 10 mM de citrato, hasta 3% (v/v) de excipiente, de aproximadamente 0,05% a aproximadamente 0,5% (v/v) de tensioactivo, y que tiene un pH de 6,0. Reivindicacion 2: La composicion de la reivindicacion 1, en donde el carbohidrato es sacarosa. Reivindicacion 3: La composicion de la reivindicacion 1, en donde el excipiente es glicerol. Reivindicacion 16: El método de cualquiera de las reivindicaciones 11 - 14, en donde alfa-Gal A es alfa-Gal A aislada manipulada genéticamente. Reivindicacion 17: El método de la reivindicacion 16, en donde la alfa-Gal A manipulada genéticamente se produce en una célula humana, una célula de levadura, una célula bacteriana, una célula de insecto o una célula vegetal.
ARP080103780A 2007-08-29 2008-08-29 Administracion subcutanea de alfa-galactosidasa a AR068144A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US96672207P 2007-08-29 2007-08-29

Publications (1)

Publication Number Publication Date
AR068144A1 true AR068144A1 (es) 2009-11-04

Family

ID=40230079

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP080103780A AR068144A1 (es) 2007-08-29 2008-08-29 Administracion subcutanea de alfa-galactosidasa a

Country Status (4)

Country Link
US (3) US20100291060A1 (es)
EP (1) EP2185187A1 (es)
AR (1) AR068144A1 (es)
WO (1) WO2009032171A1 (es)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE429927T1 (de) 2000-02-04 2009-05-15 Children S Hospital Res Founda Verwendung von lysosomal acid lipase zur behandlung von atherosklerose und ähnlichen krankheiten
SI2561069T1 (sl) 2010-04-23 2017-07-31 Alexion Pharmaceuticals, Inc. Encim za bolezen lizosomskega shranjevanja
SG10201507199UA (en) 2010-09-09 2015-10-29 Synageva Biopharma Corp Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
KR20140006037A (ko) 2011-02-15 2014-01-15 시나게바 바이오파르마, 코포레이션 리소좀의 산 리파제 결핍을 치료하는 방법들
US20140219986A1 (en) 2011-03-11 2014-08-07 Amicus Therapeutics ,Inc. Dosing regimens for the treatment of fabry disease
WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease
US9907856B2 (en) 2013-07-08 2018-03-06 The Regents Of The University Of California Carboxymethylcellulose-peptide conjugates and methods for using the same
US9981021B1 (en) 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5179023A (en) * 1989-03-24 1993-01-12 Research Corporation Technologies, Inc. Recombinant α-galactosidase a therapy for Fabry disease
US6458574B1 (en) * 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US6083725A (en) * 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
EP2361631A1 (en) * 2002-04-25 2011-08-31 Shire Human Genetic Therapies, Inc. Treatment of alpha-galactosidase a deficiency

Also Published As

Publication number Publication date
WO2009032171A1 (en) 2009-03-12
EP2185187A1 (en) 2010-05-19
US20140037612A1 (en) 2014-02-06
US20150306187A1 (en) 2015-10-29
US20100291060A1 (en) 2010-11-18

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