CL2020001856A1 - Métodos para tratar la enfermedad de farber. - Google Patents

Métodos para tratar la enfermedad de farber.

Info

Publication number
CL2020001856A1
CL2020001856A1 CL2020001856A CL2020001856A CL2020001856A1 CL 2020001856 A1 CL2020001856 A1 CL 2020001856A1 CL 2020001856 A CL2020001856 A CL 2020001856A CL 2020001856 A CL2020001856 A CL 2020001856A CL 2020001856 A1 CL2020001856 A1 CL 2020001856A1
Authority
CL
Chile
Prior art keywords
disease
methods
treating farber
farber
treating
Prior art date
Application number
CL2020001856A
Other languages
English (en)
Inventor
Eric Gaukel
Brante Sampey
Original Assignee
Enzyvant Therapeutics Gmbh
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 Enzyvant Therapeutics Gmbh filed Critical Enzyvant Therapeutics Gmbh
Publication of CL2020001856A1 publication Critical patent/CL2020001856A1/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
    • 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)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • 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)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • C12N9/2411—Amylases
    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)

Abstract

Se describen métodos para tratar la enfermedad de Farber mediante el uso de dosis y perfiles farmacocinéticos particulares.
CL2020001856A 2018-02-02 2020-07-13 Métodos para tratar la enfermedad de farber. CL2020001856A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201862625763P 2018-02-02 2018-02-02

Publications (1)

Publication Number Publication Date
CL2020001856A1 true CL2020001856A1 (es) 2020-12-04

Family

ID=65951809

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020001856A CL2020001856A1 (es) 2018-02-02 2020-07-13 Métodos para tratar la enfermedad de farber.

Country Status (15)

Country Link
US (2) US20190240298A1 (es)
EP (2) EP3746115A1 (es)
JP (3) JP2021512853A (es)
KR (2) KR20200118011A (es)
CN (1) CN112703010A (es)
AU (2) AU2019214519A1 (es)
BR (1) BR112020015003A2 (es)
CA (1) CA3088225A1 (es)
CL (1) CL2020001856A1 (es)
IL (2) IL312159A (es)
MX (2) MX2020007501A (es)
PH (1) PH12020551146A1 (es)
RU (1) RU2020118032A (es)
SG (1) SG11202006506TA (es)
WO (1) WO2019150192A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3568154B1 (en) * 2017-01-13 2023-07-12 Icahn School of Medicine at Mount Sinai Compositions and methods for treating farber disease
US20220088158A1 (en) * 2019-01-23 2022-03-24 Aceragen, Inc. Method of ameliorating a pro-inflammatory immunophenotype in farber disease subjects by repeated administration of a recombinant human acid ceramidase
US20220347276A1 (en) * 2019-09-11 2022-11-03 Icahn School Of Medicine At Mount Sinai Anc80 encoding sphingolipid-metabolizing proteins for mitigating disease-induced tissue damage

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2840224C (en) * 2011-06-22 2019-08-13 The General Hospital Corporation Treatment of proteinopathies
CN112126634A (zh) * 2013-01-09 2020-12-25 夏尔人类遗传性治疗公司 含用于纯化芳基硫酸酯酶a的方法
AU2014226126B2 (en) * 2013-03-08 2019-02-14 Genzyme Corporation Continuous purification of therapeutic proteins
PT2968479T (pt) 2013-03-14 2019-08-07 Icahn School Med Mount Sinai Composições terapêuticas de ceramidase ácida e métodos de fabrico e utilização
CA3036672C (en) 2016-09-16 2021-08-24 Vilter Manufacturing Llc High suction pressure single screw compressor with thrust balancing load using shaft seal pressure and related methods
EP3568154B1 (en) 2017-01-13 2023-07-12 Icahn School of Medicine at Mount Sinai Compositions and methods for treating farber disease
WO2019060837A1 (en) * 2017-09-25 2019-03-28 Enzyvant Farber Gmbh PROCESS FOR PRODUCING RECOMBINANT HUMAN ACID CERAMIDASE

Also Published As

Publication number Publication date
CN112703010A (zh) 2021-04-23
JP2023113809A (ja) 2023-08-16
AU2025202813A9 (en) 2026-03-05
KR20250016468A (ko) 2025-02-03
JP2025041867A (ja) 2025-03-26
WO2019150192A1 (en) 2019-08-08
US20230123505A1 (en) 2023-04-20
IL312159A (en) 2024-06-01
EP4556066A2 (en) 2025-05-21
EP4556066A3 (en) 2025-07-16
US20190240298A1 (en) 2019-08-08
IL276284A (en) 2020-09-30
BR112020015003A2 (pt) 2020-12-29
AU2025202813A1 (en) 2025-05-15
JP2021512853A (ja) 2021-05-20
RU2020118032A (ru) 2022-03-02
MX2025007132A (es) 2025-07-01
EP3746115A1 (en) 2020-12-09
PH12020551146A1 (en) 2021-06-28
AU2019214519A1 (en) 2020-06-18
KR20200118011A (ko) 2020-10-14
SG11202006506TA (en) 2020-08-28
CA3088225A1 (en) 2019-08-08
MX2020007501A (es) 2020-10-16

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