CO2024016724A2 - Métodos para tratar la enfermedad de pompe - Google Patents

Métodos para tratar la enfermedad de pompe

Info

Publication number
CO2024016724A2
CO2024016724A2 CONC2024/0016724A CO2024016724A CO2024016724A2 CO 2024016724 A2 CO2024016724 A2 CO 2024016724A2 CO 2024016724 A CO2024016724 A CO 2024016724A CO 2024016724 A2 CO2024016724 A2 CO 2024016724A2
Authority
CO
Colombia
Prior art keywords
methods
pompe disease
treating pompe
treating
population
Prior art date
Application number
CONC2024/0016724A
Other languages
English (en)
Spanish (es)
Inventor
Jay Barth
Sheela Sitaraman Das
Jeff Castelli
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 CO2024016724A2 publication Critical patent/CO2024016724A2/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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • A61K47/38—Cellulose; Derivatives thereof
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Inorganic Chemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CONC2024/0016724A 2022-05-05 2024-12-05 Métodos para tratar la enfermedad de pompe CO2024016724A2 (es)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US202263338601P 2022-05-05 2022-05-05
US202263399498P 2022-08-19 2022-08-19
US202263399491P 2022-08-19 2022-08-19
US202263404475P 2022-09-07 2022-09-07
US202263414897P 2022-10-10 2022-10-10
US202263428970P 2022-11-30 2022-11-30
US202263431920P 2022-12-12 2022-12-12
US202263434791P 2022-12-22 2022-12-22
US202363447222P 2023-02-21 2023-02-21
PCT/US2023/066657 WO2023215865A1 (en) 2022-05-05 2023-05-05 Methods for treating pompe disease

Publications (1)

Publication Number Publication Date
CO2024016724A2 true CO2024016724A2 (es) 2025-04-28

Family

ID=86692686

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2024/0016724A CO2024016724A2 (es) 2022-05-05 2024-12-05 Métodos para tratar la enfermedad de pompe

Country Status (13)

Country Link
US (1) US20260041744A1 (enExample)
EP (1) EP4518885A1 (enExample)
JP (1) JP2025515159A (enExample)
KR (1) KR20250007602A (enExample)
CN (1) CN119343147A (enExample)
AU (1) AU2023265155A1 (enExample)
CA (1) CA3252291A1 (enExample)
CL (1) CL2024003377A1 (enExample)
CO (1) CO2024016724A2 (enExample)
IL (1) IL316776A (enExample)
MX (1) MX2024013673A (enExample)
TW (1) TW202400212A (enExample)
WO (1) WO2023215865A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026006625A1 (en) 2024-06-26 2026-01-02 Amicus Therapeutics, Inc. Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
US11382980B2 (en) 2013-10-08 2022-07-12 Taiyo Kagaku Co., Ltd. Oil/fat composition containing polyunsaturated fatty acid
JP6851964B2 (ja) * 2014-09-30 2021-03-31 アミカス セラピューティックス インコーポレイテッド 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ
IL299470B2 (en) * 2015-12-30 2026-05-01 Amicus Therapeutics Inc Improved acid alpha-glucosidase for the treatment of Pompe disease
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
KR102618519B1 (ko) * 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
CN119113091A (zh) * 2017-05-15 2024-12-13 阿米库斯治疗学公司 重组人类酸性α-葡萄糖苷酶
WO2020163480A1 (en) 2019-02-05 2020-08-13 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase and uses thereof
JP2024506346A (ja) * 2021-02-11 2024-02-13 アミカス セラピューティックス インコーポレイテッド 組換えヒト酸性アルファグルコシダーゼ及びその使用

Also Published As

Publication number Publication date
MX2024013673A (es) 2024-12-06
IL316776A (en) 2025-01-01
CN119343147A (zh) 2025-01-21
CA3252291A1 (en) 2023-11-09
AU2023265155A1 (en) 2024-12-12
KR20250007602A (ko) 2025-01-14
WO2023215865A1 (en) 2023-11-09
CL2024003377A1 (es) 2025-03-28
US20260041744A1 (en) 2026-02-12
JP2025515159A (ja) 2025-05-13
EP4518885A1 (en) 2025-03-12
TW202400212A (zh) 2024-01-01

Similar Documents

Publication Publication Date Title
BR112023016212A2 (pt) Alfa-glucosidase ácida humana recombinante e usos da mesma
BR112022011760A2 (pt) Fragmento de alfa glicosidase ácida recombinante e/ou de alfa glicosidase ácida recombinante biologicamente ativa, alfa glicosidase ácida recombinante, composição, sequência polinucleotídica recombinante, vetor de expressão, vetor de expressão pdh, célula hospedeira, método para produzir uma variante de alfa glicosidase ácida recombinante, variante de alfa glicosidase ácida recombinante, composição farmacêutica para o tratamento da doença de pompe, composição farmacêutica, método para tratar e/ou prevenir os sintomas da doença de pompe em um indivíduo, e, uso das composições
CL2024003377A1 (es) Métodos para tratar la enfermedad de pompe
CO2025008934A2 (es) Fexamétodos para tratar la enfermedad de pompe de inicio infantil en pacientes pediátricos
MX349992B (es) Composiciones de alta concentracion de alfa-glucosidasa para el tratamiento de la enfermedad de pompe.
CL2018002768A1 (es) Formulaciones que comprenden alfa-glucosidasa ácida recombinante
US20140187755A1 (en) Clostridial neurotoxins with altered persistency
BR112023001121A2 (pt) Cepa c de prevotella histicola como uma terapia oral para doenças inflamatórias
Jeong et al. Sphingosine kinase 1 activation enhances epidermal innate immunity through sphingosine-1-phosphate stimulation of cathelicidin production
BR112022019510A2 (pt) Variantes de beta-glucocerebrosidase para uso no tratamento de doença de gaucher
CO2025008932A2 (es) Métodos para tratar la enfermedad de pompe de inicio tardío en pacientes pediátricos
EA202091205A1 (ru) Композиции для терапии гипергликемии и сопутствующих состояний
Wei et al. Mechanical ventilation induces lung and brain injury through ATP production, P2Y1 receptor activation and dopamine release
WO2023150493A3 (en) Cell therapy for diabetes
AR125351A1 (es) Composiciones y métodos para modular la expresión de pnpla3
ES2537579T3 (es) Preparación que contiene toxina botulínica de tipo A muy purificada derivada de patógeno de botulismo del lactante
BR112022014119A2 (pt) Administração oral de peptídeos
Song et al. A glycolytic shunt via the pentose phosphate pathway is a metabolic checkpoint for nervous system sensory homeostasis and axonal regeneration
BRPI0416434A (pt) 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
Yu et al. Exosome-Mediated Rewiring of Oxidative Stress–Inflammation–ECM Remodeling Axis Mitigates UVB-Triggered Skin Photoaging
Lu et al. Successful treatment of kerion with itraconazole and ALA-PDT: a case report
BR112021019139A2 (pt) Célula de mamífero manipulada, composição, molécula isolada de dna de fita dupla, dispositivo implantável, cápsula de hidrogel, e, método de tratamento de um paciente com doença de fabry
EP4282423A4 (en) Pharmaceutical composition for prevention or treatment of inflammatory disease or pain, comprising mesenchymal stem cells expressing ptx-3, timp1 and bdnf as active ingredient
BR112022006125A2 (pt) Composições compreendendo hialuronidase e/ou colagenase e/ou 4-metilumbeliferona (4-mu) e métodos de tratamento usando as mesmas
Zhang et al. Crotoxin potentiates L-type calcium currents and modulates the action potential of neonatal rat cardiomyocytes