MX2019009191A - Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas. - Google Patents

Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas.

Info

Publication number
MX2019009191A
MX2019009191A MX2019009191A MX2019009191A MX2019009191A MX 2019009191 A MX2019009191 A MX 2019009191A MX 2019009191 A MX2019009191 A MX 2019009191A MX 2019009191 A MX2019009191 A MX 2019009191A MX 2019009191 A MX2019009191 A MX 2019009191A
Authority
MX
Mexico
Prior art keywords
fusion proteins
peptide
targeted therapeutic
lysosomal enzyme
lysosomal
Prior art date
Application number
MX2019009191A
Other languages
English (en)
Inventor
Aoyagi-Scharber Mika
Margaret Christianson Teresa
Dvorak-Ewell Melita
J Wendt Daniel
Long Shinong
Lebowitz Jonathan
Solomon Gold Daniel
Original Assignee
Biomarin Pharm 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 Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of MX2019009191A publication Critical patent/MX2019009191A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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)
    • 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)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal

Abstract

La presente invención se refiere en general a proteínas de fusión terapéutica útiles para tratar enfermedades de almacenamiento lisosomal y métodos para tratar tales enfermedades. Proteínas de fusión terapéutica ejemplares comprenden una enzima lisosomal, una porción de objetivo lisosomal, por ejemplo, un péptido IGF-II, y un péptido espaciador. También se proporcionan composiciones y métodos para tratar Mucopolisacaridosis Tipo IIIB (Síndrome de Sanfilippo B), que comprenden una proteína de fusión terapéutica objetivo que comprende alfa-N-acetilglucosaminidasa (Naglu), una porción de objetivo lisosomal, por ejemplo, un péptido IGF-II, y un péptido espaciador.
MX2019009191A 2012-11-27 2015-05-26 Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas. MX2019009191A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261730378P 2012-11-27 2012-11-27
US201361788968P 2013-03-15 2013-03-15

Publications (1)

Publication Number Publication Date
MX2019009191A true MX2019009191A (es) 2019-10-09

Family

ID=49753532

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2015006644A MX367024B (es) 2012-11-27 2013-11-27 Proteinas de fusion de enzima lisosomal terapeutica objetivo y usos de las mismas.
MX2019009191A MX2019009191A (es) 2012-11-27 2015-05-26 Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2015006644A MX367024B (es) 2012-11-27 2013-11-27 Proteinas de fusion de enzima lisosomal terapeutica objetivo y usos de las mismas.

Country Status (27)

Country Link
US (8) US9376480B2 (es)
EP (2) EP3115372B1 (es)
JP (2) JP6831176B2 (es)
KR (4) KR102262882B1 (es)
CN (1) CN104822701B (es)
AR (1) AR093626A1 (es)
AU (1) AU2013352184B2 (es)
BR (1) BR112015012152B1 (es)
CA (1) CA2892146A1 (es)
CL (1) CL2015001371A1 (es)
CY (1) CY1122555T1 (es)
DK (2) DK2925776T3 (es)
ES (2) ES2679374T3 (es)
HK (1) HK1216026A1 (es)
HR (2) HRP20181351T1 (es)
HU (2) HUE043679T2 (es)
IL (3) IL238824B (es)
LT (1) LT3115372T (es)
MX (2) MX367024B (es)
PL (2) PL2925776T3 (es)
PT (2) PT3115372T (es)
RS (1) RS58916B1 (es)
RU (1) RU2680581C2 (es)
SI (1) SI3115372T1 (es)
TW (2) TWI626250B (es)
WO (1) WO2014085621A1 (es)
ZA (1) ZA201503509B (es)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2279210T3 (pl) * 2008-05-07 2017-10-31 Biomarin Pharm Inc Lizosomalne peptydy kierujące i ich zastosowania
KR102262882B1 (ko) 2012-11-27 2021-06-10 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들
CN115120746A (zh) * 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
EP3185889B1 (en) * 2014-08-11 2020-07-15 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
WO2016049510A2 (en) * 2014-09-25 2016-03-31 The General Hospital Corporation Cell-based targeted delivery of pseudonomas exotoxin
MA40791A (fr) 2014-10-24 2017-09-27 Shire Human Genetic Therapies Ciblage lysosomal d'enzymes et utilisations associées
TWI752907B (zh) 2015-05-08 2022-01-21 美商拜奧馬林製藥公司 用於治療cln2疾病之tpp1調配物及方法
CA3003750A1 (en) 2015-11-06 2017-05-11 Biomarin Pharmaceutical Inc. Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes
AU2017222620B2 (en) * 2016-02-24 2022-06-16 Biomarin Pharmaceutical Inc. Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
JP7171439B2 (ja) 2016-04-15 2022-11-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉ型を処置するための遺伝子療法
CL2016003282A1 (es) 2016-12-21 2017-08-18 Univ Chile Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington
US11819539B2 (en) 2017-09-22 2023-11-21 The Trustees Of The University Of Pennsylvania Gene therapy for treating Mucopolysaccharidosis type II
MA50746A (fr) 2017-10-02 2020-08-12 Denali Therapeutics Inc Protéines de fusion comprenant des enzymes d'enzymothérapie substitutive
AU2019231889A1 (en) * 2018-03-09 2020-10-01 Avrobio, Inc. Compositions and methods for treating Parkinson's disease
JP2021521851A (ja) * 2018-04-30 2021-08-30 アミカス セラピューティックス インコーポレイテッド 遺伝子治療構築物及び使用方法
CN112243444A (zh) 2018-06-08 2021-01-19 豪夫迈·罗氏有限公司 具有减少的翻译后修饰的肽接头
US11097015B2 (en) 2018-10-10 2021-08-24 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
AU2019381803A1 (en) * 2018-11-16 2021-06-10 Asklepios Biopharmaceutical, Inc. Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence
CN115087458A (zh) * 2019-10-10 2022-09-20 阿米库斯治疗学公司 变体igf2构建体
WO2021183895A1 (en) 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
CN115975039A (zh) * 2021-10-15 2023-04-18 中山大学 重组融合抗体和抗体-药物偶联物及其用途

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5206161A (en) 1991-02-01 1993-04-27 Genentech, Inc. Human plasma carboxypeptidase B
AUPN674895A0 (en) 1995-11-23 1995-12-14 Women's And Children's Hospital Synthetic mammalian alpha-N-acetylglucosaminidase and genetic sequences encoding same
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
DE10035433C2 (de) 2000-07-20 2002-07-18 Tuma Wolfgang Schonende Hochanreicherung von fetalen Zellen aus pripherem Blut und Verwendung derselben
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
JP4641705B2 (ja) 2001-04-30 2011-03-02 ザイストール セラピューティクス， インコーポレイテッド 治療的タンパク質の亜細胞標的化
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
WO2003102583A1 (en) 2002-05-29 2003-12-11 Symbiontics, Inc. Targeted therapeutic proteins
AU2002347910A1 (en) 2001-10-16 2003-04-28 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US7485314B2 (en) 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
SI1641823T1 (sl) 2003-06-12 2011-12-30 Lilly Co Eli Fuzijski proteini analogni glp-1
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
DE602005020745D1 (de) * 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
CA2600929A1 (en) 2005-03-04 2006-09-14 Biogen Idec Ma Inc. Methods of humanizing immunoglobulin variable regions through rational modification of complementarity determining residues
PL2279210T3 (pl) * 2008-05-07 2017-10-31 Biomarin Pharm Inc Lizosomalne peptydy kierujące i ich zastosowania
US8697654B2 (en) * 2008-12-18 2014-04-15 E I Du Pont De Nemours And Company Peptide linkers for effective multivalent peptide binding
US9029530B2 (en) 2009-01-02 2015-05-12 Biomarin Pharmaceutical Inc. Detection of oligosaccharides
EP3075386B1 (en) 2009-06-17 2019-10-16 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
ES2773071T3 (es) 2010-06-25 2020-07-09 Amcor Rigid Plastics Usa Llc The Corp Trust Company Sistema de extracción de oxígeno para un recipiente
JP6063380B2 (ja) * 2010-06-25 2017-01-18 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド サンフィリポ症候群ｂ型の処置
US9409950B2 (en) * 2010-12-23 2016-08-09 Biogen Ma Inc. Linker peptides and polypeptides comprising same
EP2680879B1 (en) * 2011-03-04 2021-01-06 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
WO2012122477A1 (en) * 2011-03-10 2012-09-13 Novozymes A/S Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same
KR102262882B1 (ko) 2012-11-27 2021-06-10 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들

Also Published As

Publication number Publication date
KR20150088317A (ko) 2015-07-31
PL3115372T3 (pl) 2019-09-30
US20160039900A1 (en) 2016-02-11
HRP20181351T1 (hr) 2018-11-02
SI3115372T1 (sl) 2019-08-30
HUE039334T2 (hu) 2018-12-28
AU2013352184B2 (en) 2018-05-31
ZA201503509B (en) 2016-11-30
IL238824B (en) 2018-11-29
US20190225666A1 (en) 2019-07-25
MX2015006644A (es) 2015-08-10
KR102262882B1 (ko) 2021-06-10
JP6831176B2 (ja) 2021-02-17
HUE043679T2 (hu) 2019-09-30
CY1122555T1 (el) 2021-01-27
RU2680581C2 (ru) 2019-02-22
KR102385392B1 (ko) 2022-04-11
US10301369B2 (en) 2019-05-28
TW201431884A (zh) 2014-08-16
EP2925776A1 (en) 2015-10-07
IL272854A (en) 2020-04-30
ES2679374T3 (es) 2018-08-24
IL238824A0 (en) 2015-06-30
US9834588B2 (en) 2017-12-05
CL2015001371A1 (es) 2015-10-09
EP3115372B1 (en) 2019-03-06
TW201827468A (zh) 2018-08-01
US9834587B2 (en) 2017-12-05
AU2013352184A1 (en) 2015-06-04
JP6913719B2 (ja) 2021-08-04
CN104822701A (zh) 2015-08-05
TWI711632B (zh) 2020-12-01
JP2016505539A (ja) 2016-02-25
TWI626250B (zh) 2018-06-11
DK3115372T3 (da) 2019-06-11
HRP20190918T1 (hr) 2019-09-20
DK2925776T3 (en) 2018-09-03
KR102521039B1 (ko) 2023-04-12
ES2729997T3 (es) 2019-11-07
IL262976A (en) 2018-12-31
AR093626A1 (es) 2015-06-17
HK1216026A1 (zh) 2016-10-07
KR20210070389A (ko) 2021-06-14
US11254725B2 (en) 2022-02-22
US9376480B2 (en) 2016-06-28
LT3115372T (lt) 2019-06-25
JP2019206556A (ja) 2019-12-05
KR20230054482A (ko) 2023-04-24
US9845346B2 (en) 2017-12-19
RU2015125491A (ru) 2017-01-10
US20220127326A1 (en) 2022-04-28
US20160031963A1 (en) 2016-02-04
WO2014085621A1 (en) 2014-06-05
PT3115372T (pt) 2019-06-12
CA2892146A1 (en) 2014-06-05
PT2925776T (pt) 2018-07-30
US20160031965A1 (en) 2016-02-04
EP3115372A1 (en) 2017-01-11
US20160031964A1 (en) 2016-02-04
CN104822701B (zh) 2018-09-21
PL2925776T3 (pl) 2018-11-30
KR20220047892A (ko) 2022-04-19
EP2925776B1 (en) 2018-05-30
US20170355744A1 (en) 2017-12-14
US20140161788A1 (en) 2014-06-12
RS58916B1 (sr) 2019-08-30
US9771408B2 (en) 2017-09-26
MX367024B (es) 2019-08-02
IL262976B (en) 2020-02-27
IL272854B (en) 2021-07-29
BR112015012152B1 (pt) 2023-04-25
BR112015012152A2 (pt) 2017-08-15

Similar Documents

Publication Publication Date Title
MX2019009191A (es) Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas.
PH12019550261A1 (en) Compositions and methods for internalizing enzymes
CY1123676T1 (el) Συνθεσεις και μεθοδοι για την εσωτερικοποιηση ενζυμων
WO2011163652A3 (en) Treatment of sanfilippo syndrome type b
CA2818969A1 (en) Improved n-terminal capping modules for designed ankyrin repeat proteins
WO2014062697A3 (en) Drug delivery conjugates containing unnatural amino acids and methods for using
MX2012007684A (es) Modificacion covalente ligando dirigida de proteina.
MX348071B (es) Variantes de fc.
WO2014039585A3 (en) Chimeric polypeptides having targeted binding specificity
WO2012135805A3 (en) Delivery and formulation of engineered nucleic acids
WO2013040093A3 (en) Glucagon-like peptide-2 compositions and methods of making and using same
MX2014004359A (es) Proteina naglu humana recombinante y usos de la misma.
WO2014055836A3 (en) Serine protease molecules and therapies
MX2019004487A (es) Metodos y composiciones para el tratamiento de la enfermedad de fabry.
MX2014015551A (es) Compuestos iduronidasa apuntados.
MX2022014532A (es) Metodo para seleccionar proteinas recombinantes ricas en m6p.
MX2017001898A (es) Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales.
MX2018010196A (es) Proteinas de fusion de enzimas lisosomales terapeuticas dirigidas, formulaciones asociadas y usos de las mismas.
WO2012122042A3 (en) Peptide linkers for polypeptide compositions and methods for using same

Legal Events

Date Code Title Description
FG Grant or registration