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

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 (1) EP3746115A1 (es)
JP (2) JP2021512853A (es)
KR (1) KR20200118011A (es)
CN (1) CN112703010A (es)
AU (1) AU2019214519A1 (es)
BR (1) BR112020015003A2 (es)
CA (1) CA3088225A1 (es)
CL (1) CL2020001856A1 (es)
IL (1) IL276284A (es)
MX (1) 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
WO2018132667A1 (en) * 2017-01-13 2018-07-19 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 (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2012272815B2 (en) * 2011-06-22 2017-09-07 The General Hospital Corporation Treatment of proteinopathies
BR112015015948B1 (pt) * 2013-01-09 2022-05-17 Takeda Pharmaceutical Company Limited Método de purificação da proteína arilsulfatase a (asa) recombinante
MX2015012114A (es) * 2013-03-08 2016-01-12 Genzyme Corp Fabricacion continua integrada de principios activos proteinicos terapeuticos.
EP2968479B1 (en) 2013-03-14 2019-07-17 Icahn School of Medicine at Mount Sinai Therapeutic acid ceramidase compositions and methods of making and using them
WO2018132667A1 (en) 2017-01-13 2018-07-19 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
PH12020551146A1 (en) 2021-06-28
EP3746115A1 (en) 2020-12-09
MX2020007501A (es) 2020-10-16
CA3088225A1 (en) 2019-08-08
JP2021512853A (ja) 2021-05-20
RU2020118032A (ru) 2022-03-02
US20230123505A1 (en) 2023-04-20
AU2019214519A1 (en) 2020-06-18
US20190240298A1 (en) 2019-08-08
JP2023113809A (ja) 2023-08-16
WO2019150192A1 (en) 2019-08-08
SG11202006506TA (en) 2020-08-28
IL276284A (en) 2020-09-30
KR20200118011A (ko) 2020-10-14
BR112020015003A2 (pt) 2020-12-29

Similar Documents

Publication Publication Date Title
ECSP20003147A (es) Carboxamidas como moduladores de los canales de sodio
CL2020002043A1 (es) Métodos y composiciones para la entrega de proteínas terapeuticas.
CO2020016619A2 (es) Anticuerpos biespecificos anti-pvrig/anti-tigit y métodos de uso
CO2017005784A2 (es) Compuestos bicíclicos fusionados para el tratamiento de enfermedades
CL2020003257A1 (es) Compuestos antagonistas de pcsk9.
CL2020001856A1 (es) Métodos para tratar la enfermedad de farber.
SV2018005610A (es) Derivados de oxopiridina sustituidos
CR20190376A (es) Formulaciones de anticuerpos de her2 subcutáneas
ECSP20046463A (es) Compuestos, composiciones y métodos para tratar enfermedades que involucren tejidos con enfermedades ácidas o hipóxicas
CL2016002667A1 (es) Anticuerpos anti-rnf43 novedosos y métodos para su uso
CL2019001993A1 (es) Inhibidores selectivos de jak1.
BR112018075597A2 (pt) derivados de carbamato de 2-amino-4-(amino substituído)fenila fluorado
EA202191632A1 (ru) Ахромосомные динамические активные системы
BR112017025781A2 (pt) derivados de 2-(pirazolopiridin-3-il)pirimidina como inibidores de jak
CL2015002835A1 (es) Nuevos derivados de piridina
BR112018070497A2 (pt) métodos e formulações farmacêuticas para tratamento de condições oculares
CO2022005759A2 (es) Inhibición de integrina α4β7 humana
BR112021014944A2 (pt) Toxinas apxia, apxiia e apxiiia inativas
CO2017010621A2 (es) Compuestos bicíclicos fusionados para el tratamiento de enfermedades
UY36117A (es) Composiciones farmacéuticas para el tratamiento de enfermedades infecciosas.
BR112018009745A8 (pt) compostos heterocíclicos para o tratamento de doença
BR112017005666A2 (pt) micropartículas injetáveis para liberação hiper-localizada de agentes terapêuticos
UY38352A (es) Inhibidores de integrina alfavbeta6
BR112018068960A2 (pt) formulações de enalapril
BR112018008415A2 (pt) formulações de liberação sustentada de anestésicos locais