CL2015001371A1 - Proteínas de fusión de enzima lisosomal terapéutica objetivo y usos de las mismas - Google Patents

Proteínas de fusión de enzima lisosomal terapéutica objetivo y usos de las mismas

Info

Publication number
CL2015001371A1
CL2015001371A1 CL2015001371A CL2015001371A CL2015001371A1 CL 2015001371 A1 CL2015001371 A1 CL 2015001371A1 CL 2015001371 A CL2015001371 A CL 2015001371A CL 2015001371 A CL2015001371 A CL 2015001371A CL 2015001371 A1 CL2015001371 A1 CL 2015001371A1
Authority
CL
Chile
Prior art keywords
fusion proteins
lysosomal enzyme
target therapeutic
enzyme fusion
therapeutic lysosomal
Prior art date
Application number
CL2015001371A
Other languages
English (en)
Inventor
Mika Aoyagi-Scharber
Teresa Margaret Christianson
Melita Dvorak-Ewell
Daniel J Wendt
Shinong Long
Jonathan Lebowits
Daniel Solomon Gold
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 CL2015001371A1 publication Critical patent/CL2015001371A1/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575Hormones
    • C07K14/65Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105Alpha-N-acetylglucosaminidase (3.2.1.50)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
CL2015001371A 2012-11-27 2015-05-20 Proteínas de fusión de enzima lisosomal terapéutica objetivo y usos de las mismas CL2015001371A1 (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
CL2015001371A1 true CL2015001371A1 (es) 2015-10-09

Family

ID=49753532

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2015001371A CL2015001371A1 (es) 2012-11-27 2015-05-20 Proteínas de fusión de enzima lisosomal terapéutica objetivo y usos de las mismas

Country Status (27)

Country Link
US (8) US9376480B2 (es)
EP (2) EP2925776B1 (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) ES2729997T3 (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) PT2925776T (es)
RS (1) RS58916B1 (es)
RU (1) RU2680581C2 (es)
SI (1) SI3115372T1 (es)
TW (2) TWI711632B (es)
WO (1) WO2014085621A1 (es)
ZA (1) ZA201503509B (es)

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KR102262882B1 (ko) 2012-11-27 2021-06-10 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들
KR20220119187A (ko) * 2013-05-15 2022-08-26 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
BR112017002741A2 (pt) * 2014-08-11 2018-02-27 Shire Human Genetic Therapies, Inc peptídeos contendo manose-6-fosfato fundidos a enzimas lisossômicas
WO2016049510A2 (en) * 2014-09-25 2016-03-31 The General Hospital Corporation Cell-based targeted delivery of pseudonomas exotoxin
WO2016065319A1 (en) * 2014-10-24 2016-04-28 Shire Human Genetic Therapies, Inc. Lysosomal targeting of enzymes, and uses thereof
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
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BR112018071156A2 (pt) 2016-04-15 2019-03-12 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
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
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
WO2019070577A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. FUSION PROTEINS COMPRISING SUBSTITUTE ENZYM THERAPY ENZYMES
EP3762505A4 (en) * 2018-03-09 2021-12-01 Avrobio, Inc. COMPOSITIONS AND METHODS OF TREATMENT FOR 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
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SG11202103640VA (en) 2018-10-10 2021-05-28 Amicus Therapeutics Inc Disulfide bond stabilized polypeptide compositions and methods of use
WO2020102667A2 (en) * 2018-11-16 2020-05-22 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

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Also Published As

Publication number Publication date
US10301369B2 (en) 2019-05-28
AR093626A1 (es) 2015-06-17
CY1122555T1 (el) 2021-01-27
BR112015012152B1 (pt) 2023-04-25
HK1216026A1 (zh) 2016-10-07
PT2925776T (pt) 2018-07-30
LT3115372T (lt) 2019-06-25
TW201827468A (zh) 2018-08-01
CN104822701A (zh) 2015-08-05
IL238824B (en) 2018-11-29
TWI711632B (zh) 2020-12-01
EP3115372B1 (en) 2019-03-06
HRP20181351T1 (hr) 2018-11-02
US11254725B2 (en) 2022-02-22
KR20220047892A (ko) 2022-04-19
TW201431884A (zh) 2014-08-16
MX367024B (es) 2019-08-02
US20160039900A1 (en) 2016-02-11
RU2680581C2 (ru) 2019-02-22
IL272854B (en) 2021-07-29
IL272854A (en) 2020-04-30
US20190225666A1 (en) 2019-07-25
US9376480B2 (en) 2016-06-28
DK3115372T3 (da) 2019-06-11
JP2016505539A (ja) 2016-02-25
KR20210070389A (ko) 2021-06-14
KR102521039B1 (ko) 2023-04-12
MX2015006644A (es) 2015-08-10
HRP20190918T1 (hr) 2019-09-20
US9845346B2 (en) 2017-12-19
CN104822701B (zh) 2018-09-21
US20170355744A1 (en) 2017-12-14
JP6913719B2 (ja) 2021-08-04
AU2013352184B2 (en) 2018-05-31
TWI626250B (zh) 2018-06-11
DK2925776T3 (en) 2018-09-03
PT3115372T (pt) 2019-06-12
HUE039334T2 (hu) 2018-12-28
US20160031965A1 (en) 2016-02-04
US20160031964A1 (en) 2016-02-04
WO2014085621A1 (en) 2014-06-05
ES2729997T3 (es) 2019-11-07
PL2925776T3 (pl) 2018-11-30
ES2679374T3 (es) 2018-08-24
IL262976B (en) 2020-02-27
US20160031963A1 (en) 2016-02-04
US20220127326A1 (en) 2022-04-28
US20140161788A1 (en) 2014-06-12
HUE043679T2 (hu) 2019-09-30
SI3115372T1 (sl) 2019-08-30
KR20150088317A (ko) 2015-07-31
PL3115372T3 (pl) 2019-09-30
US9834588B2 (en) 2017-12-05
JP6831176B2 (ja) 2021-02-17
US9771408B2 (en) 2017-09-26
JP2019206556A (ja) 2019-12-05
KR20230054482A (ko) 2023-04-24
US9834587B2 (en) 2017-12-05
BR112015012152A2 (pt) 2017-08-15
IL262976A (en) 2018-12-31
RS58916B1 (sr) 2019-08-30
EP2925776B1 (en) 2018-05-30
KR102385392B1 (ko) 2022-04-11
AU2013352184A1 (en) 2015-06-04
EP3115372A1 (en) 2017-01-11
MX2019009191A (es) 2019-10-09
KR102262882B1 (ko) 2021-06-10
EP2925776A1 (en) 2015-10-07
RU2015125491A (ru) 2017-01-10
IL238824A0 (en) 2015-06-30
ZA201503509B (en) 2016-11-30
CA2892146A1 (en) 2014-06-05

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