HK1209625A1 - Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation - a 1- - Google Patents

Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation - a 1-

Info

Publication number
HK1209625A1
HK1209625A1 HK15110389.1A HK15110389A HK1209625A1 HK 1209625 A1 HK1209625 A1 HK 1209625A1 HK 15110389 A HK15110389 A HK 15110389A HK 1209625 A1 HK1209625 A1 HK 1209625A1
Authority
HK
Hong Kong
Prior art keywords
deoxygalactonojirimycin
galactosidase
alpha
formulation
Prior art date
Application number
HK15110389.1A
Other languages
English (en)
Chinese (zh)
Inventor
Richie Khanna
Kenneth Joseph Valenzano
Susan Elizabeth Fowles
Original Assignee
Amicus Therapeutics Inc
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 Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of HK1209625A1 publication Critical patent/HK1209625A1/xx

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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • 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
    • 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)
    • C12N9/24—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)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
HK15110389.1A 2012-07-17 2015-10-22 Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation - a 1- HK1209625A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261672566P 2012-07-17 2012-07-17
PCT/US2013/050721 WO2014014938A1 (en) 2012-07-17 2013-07-16 Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation

Publications (1)

Publication Number Publication Date
HK1209625A1 true HK1209625A1 (en) 2016-04-08

Family

ID=49949213

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15110389.1A HK1209625A1 (en) 2012-07-17 2015-10-22 Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation - a 1-

Country Status (6)

Country Link
US (2) US9694056B2 (xx)
EP (1) EP2874648A4 (xx)
CA (1) CA2917995C (xx)
HK (1) HK1209625A1 (xx)
TW (2) TWI594763B (xx)
WO (1) WO2014014938A1 (xx)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3457135A1 (en) 2006-05-16 2019-03-20 Amicus Therapeutics, Inc. Treatment options for fabry disease
PL3470077T3 (pl) 2008-02-12 2021-04-06 Amicus Therapeutics, Inc. Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi
CN106413401A (zh) * 2014-03-14 2017-02-15 索理思科技公司 有机酸抗微生物组合物
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP3397273B1 (en) 2015-12-30 2021-05-19 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
EA201891507A1 (ru) 2015-12-30 2018-12-28 Амикус Терапьютикс, Инк. Кислая альфа-глюкозидаза усиленного действия для лечения болезни помпе
CN114159436A (zh) 2016-03-22 2022-03-11 阿米库斯治疗学公司 治疗在gla基因中具有g9331a突变的患者的法布里病的方法
JP2020503900A (ja) * 2017-01-10 2020-02-06 アミカス セラピューティックス インコーポレイテッド ファブリー病の処置のための組換えアルファ−ガラクトシダーゼａ
KR20240017112A (ko) 2017-05-30 2024-02-06 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
ES2716305B2 (es) 2017-12-11 2019-11-27 Fund Biomedica Galicia Sur Uso de chaperonas farmacologicas para el tratamiento de enfermedades de deposito lisosomal
EP3749307A1 (en) 2018-02-06 2020-12-16 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
US11708569B2 (en) 2018-08-29 2023-07-25 University Of Copenhagen Modified recombinant lysosomal alpha-galactosidase A and aspartylglucoaminidase having low mannose-6-phosphate and high sialic acid
NL2021840B1 (en) 2018-10-19 2020-05-13 Univ Leiden Pharmacological Chaperones For Enzyme Treatment Therapy
WO2020046132A1 (en) 2018-08-31 2020-03-05 Leiden University Pharmacological chaperones for enzyme treatment therapy
CA3147055A1 (en) 2019-08-07 2021-02-11 Elfrida Benjamin Methods of treating fabry disease in patients having a mutation in the gla gene
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6458574B1 (en) * 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
GB9909066D0 (en) * 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
US20020095135A1 (en) * 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
EP1503788B1 (en) * 2002-04-25 2011-06-29 Shire Human Genetic Therapies, Inc. Treatment of alpha-galactosidase a deficiency
HUE033381T2 (en) 2003-01-31 2017-11-28 Mount Sinai School Of Medicine Of New York Univ Combination therapy for the treatment of protein deficiency disorders
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
BRPI0416434A (pt) * 2003-11-25 2007-02-21 Sinai School Medicine métodos para aumentar a atividade de uma proteìna de ácido esfingomielinase (asm), para estender a meia-vida e prolongar a atividade de uma proteìna asm purificada, para aumentar a produção de uma proteìna asm recombinante através de uma célula hospedeira, e usos de uma composição de asm tipo alelo-selvagem, purificada e um composto análogo de ceramida ou esfingomielina, e de um composto análogo de ceramida ou de esfingomielina
MX2009010557A (es) * 2007-03-30 2009-11-19 Amicus Therapeutics Inc Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas.
WO2008134628A2 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics

Also Published As

Publication number Publication date
TW201803589A (zh) 2018-02-01
EP2874648A1 (en) 2015-05-27
US9694056B2 (en) 2017-07-04
CA2917995C (en) 2021-01-26
US10357548B2 (en) 2019-07-23
US20170360901A1 (en) 2017-12-21
WO2014014938A1 (en) 2014-01-23
CA2917995A1 (en) 2014-01-23
TWI594763B (zh) 2017-08-11
US20150174214A1 (en) 2015-06-25
EP2874648A4 (en) 2015-12-30
TW201408319A (zh) 2014-03-01

Similar Documents

Publication Publication Date Title
HK1209625A1 (en) Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation - a 1-
HK1202545A1 (en) New diazaspirocycloalkane and azaspirocycloalkane
GB2500915B (en) Arrangement and method
EP2812777A4 (en) STRING COMPLETION
HK1208318A1 (en) A chair and supports
GB2508701B (en) A knee-walker
GB201216649D0 (en) Agents and methods
GB201223264D0 (en) A hydrogen-storage-material
EP2688588A4 (en) FAST AND LONG-TERM IMMUNOLOGICAL THERAPEUTIC
EP2697386A4 (en) DESIGN AND USE OF PEPDUCINE
SG11201500474PA (en) Nutriculture member and nutriculture method
AU349547S (en) Model
GB201219713D0 (en) A collapsableinfant chair
GB2510464B (en) A Footgauge
GB2508712B (en) A toaster
SG10201508411UA (en) A rotor-arm assembly and a multi-rotorcraft
GB201222541D0 (en) Kiddibidet and pottibidet
GB2506132B (en) A fuseboard
IL218974A0 (en) Conaction bitwen coutch and playels
GB201202021D0 (en) Co-sleeper and breastfeeder
GB201119443D0 (en) GoWinging Concept and patent
AU345605S (en) A daybed
HUP1200256A2 (en) Blebbistatin azido-derivatives
ZA201208126B (en) A biodigester
GB201223475D0 (en) A putting aid