LT3115372T - Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas - Google Patents

Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas

Info

Publication number
LT3115372T
LT3115372T LTEP16182420.6T LT16182420T LT3115372T LT 3115372 T LT3115372 T LT 3115372T LT 16182420 T LT16182420 T LT 16182420T LT 3115372 T LT3115372 T LT 3115372T
Authority
LT
Lithuania
Prior art keywords
fusion proteins
lysosomal enzyme
targeted therapeutic
enzyme fusion
therapeutic lysosomal
Prior art date
Application number
LTEP16182420.6T
Other languages
English (en)
Lithuanian (lt)
Inventor
Mika Aoyagi-Scharber
Teresa Margaret Christianson
Melita Dvorak-Ewell
Daniel Wendt
Shinong Long
Jonathan Lebowitz
Daniel Solomon Gold
Original Assignee
Biomarin Pharmaceutical 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 Pharmaceutical Inc. filed Critical Biomarin Pharmaceutical Inc.
Publication of LT3115372T publication Critical patent/LT3115372T/lt

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)
LTEP16182420.6T 2012-11-27 2013-11-27 Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas LT3115372T (lt)

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
LT3115372T true LT3115372T (lt) 2019-06-25

Family

ID=49753532

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP16182420.6T LT3115372T (lt) 2012-11-27 2013-11-27 Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas

Country Status (26)

Country Link
US (9) US9376480B2 (enExample)
EP (2) EP2925776B1 (enExample)
JP (2) JP6831176B2 (enExample)
KR (4) KR20230054482A (enExample)
CN (1) CN104822701B (enExample)
AR (1) AR093626A1 (enExample)
AU (1) AU2013352184B2 (enExample)
BR (1) BR112015012152B1 (enExample)
CA (1) CA2892146A1 (enExample)
CL (1) CL2015001371A1 (enExample)
CY (1) CY1122555T1 (enExample)
DK (2) DK3115372T3 (enExample)
ES (2) ES2679374T3 (enExample)
HR (2) HRP20181351T1 (enExample)
HU (2) HUE043679T2 (enExample)
IL (3) IL238824B (enExample)
LT (1) LT3115372T (enExample)
MX (2) MX377150B (enExample)
PL (2) PL3115372T3 (enExample)
PT (2) PT2925776T (enExample)
RS (1) RS58916B1 (enExample)
RU (1) RU2680581C2 (enExample)
SI (1) SI3115372T1 (enExample)
TW (2) TWI711632B (enExample)
WO (1) WO2014085621A1 (enExample)
ZA (1) ZA201503509B (enExample)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2629853T3 (es) 2008-05-07 2017-08-16 Biomarin Pharmaceutical Inc. Péptidos de dirección lisosómica y usos de los mismos
LT3115372T (lt) 2012-11-27 2019-06-25 Biomarin Pharmaceutical Inc. Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas
KR20160010526A (ko) * 2013-05-15 2016-01-27 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
MX2017001898A (es) * 2014-08-11 2017-04-11 Shire Human Genetic Therapies Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales.
US20180303877A1 (en) * 2014-09-25 2018-10-25 The General Hospital Corporation Cell-based targeted delivery of pseudonomas exotoxin
EP3220958A1 (en) * 2014-10-24 2017-09-27 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
MY189159A (en) 2015-07-06 2022-01-29 Regeneron Pharma Multispecific antigen-binding molecules and uses thereof
MX2018005258A (es) 2015-11-06 2019-09-04 Biomarin Pharm Inc Ensayos basados en células para la detección de anticuerpos u otros factores que neutralizan la absorción de enzimas lisosómicas.
IL261246B2 (en) * 2016-02-24 2023-03-01 Biomarin Pharm Inc Therapeutic fusion proteins targeting lysosomal enzymes, their formulations and their uses
AU2017250298B2 (en) 2016-04-15 2024-10-03 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type 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
ES3065816T3 (en) 2017-06-07 2026-05-08 Regeneron Pharma Compositions and methods for internalizing enzymes
JP7449223B2 (ja) 2017-09-22 2024-03-13 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型を治療するための遺伝子療法
MX2020002918A (es) 2017-10-02 2020-07-22 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas.
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
CA3092961A1 (en) * 2018-03-09 2019-09-12 Avrobio, Inc. Compositions and methods for treating parkinson's disease
JP7749322B2 (ja) 2018-04-30 2025-10-06 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
BR112020023145A2 (pt) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto
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
EA202190999A1 (ru) 2018-10-10 2021-10-15 Амикус Терапьютикс, Инк. Композиции на основе полипептидов, стабилизированных с помощью дисульфидной связи, и способы применения
EP3880830A4 (en) * 2018-11-16 2022-08-24 Asklepios Biopharmaceutical, Inc. VECTORS WITH A NUCLEIC ACID ENCODING LYSOSOMAL ENZYMES CONDENSED TO A LYSOSOMAL TARGETING SEQUENCE
AU2020361703A1 (en) * 2019-10-10 2022-04-28 Amicus Therapeutics, Inc. Variant IGF2 constructs
US12358963B2 (en) 2019-12-24 2025-07-15 Juvena Therapeutics, Inc. Regenerative polypeptides and uses thereof
WO2021183895A1 (en) 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
CA3223707A1 (en) 2021-06-21 2022-12-29 Hanadie Yousef Regenerative polypeptides and uses thereof
US20240279360A1 (en) * 2021-06-23 2024-08-22 Lycia Therapeutics, Inc. Bifunctional compounds containing igf-2 polypeptides
CN117343197A (zh) * 2021-10-15 2024-01-05 中山大学 重组融合抗体
WO2024159071A1 (en) * 2023-01-27 2024-08-02 Regeneron Pharmaceuticals, Inc. Modified rhabdovirus glycoproteins and uses thereof

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
WO2003102583A1 (en) 2002-05-29 2003-12-11 Symbiontics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
EP1974752B1 (en) * 2001-04-30 2012-09-26 BioMarin Pharmaceutical Inc. Subcellular targeting of therapeutic proteins
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
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
IL161352A0 (en) 2001-10-16 2004-09-27 Symbiontics Inc Methods and compositions for targeting underglycosylated 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
JP4629047B2 (ja) 2003-06-12 2011-02-09 イーライ リリー アンド カンパニー Glp−1アナログ複合タンパク質
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
CA2553955C (en) * 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
JP2008531060A (ja) 2005-03-04 2008-08-14 バイオジェン・アイデック・エムエイ・インコーポレイテッド 相補性決定残基の合理的改変を介して免疫グロブリン可変領域をヒト化する方法
ES2629853T3 (es) 2008-05-07 2017-08-16 Biomarin Pharmaceutical Inc. Péptidos de dirección lisosómica y usos de los mismos
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
EP2475376B1 (en) * 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
NZ702801A (en) * 2010-06-25 2016-08-26 Shire Human Genetic Therapies Treatment of sanfilippo syndrome type b
EP2585385B1 (en) 2010-06-25 2019-12-18 Amcor Rigid Plastics USA, LLC Oxygen scavenging system for a container
EP2655624B1 (en) * 2010-12-23 2017-11-29 Biogen MA Inc. Linker peptides and polypeptides comprising same
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
CA3080181A1 (en) * 2011-03-04 2012-09-13 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
LT3115372T (lt) 2012-11-27 2019-06-25 Biomarin Pharmaceutical Inc. Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas

Also Published As

Publication number Publication date
DK2925776T3 (en) 2018-09-03
PL2925776T3 (pl) 2018-11-30
IL238824B (en) 2018-11-29
JP6913719B2 (ja) 2021-08-04
RS58916B1 (sr) 2019-08-30
IL272854B (en) 2021-07-29
ES2729997T3 (es) 2019-11-07
US20160031965A1 (en) 2016-02-04
US9834588B2 (en) 2017-12-05
CN104822701A (zh) 2015-08-05
MX367024B (es) 2019-08-02
CY1122555T1 (el) 2021-01-27
MX2015006644A (es) 2015-08-10
JP6831176B2 (ja) 2021-02-17
EP3115372B1 (en) 2019-03-06
HRP20190918T1 (hr) 2019-09-20
CL2015001371A1 (es) 2015-10-09
SI3115372T1 (sl) 2019-08-30
KR20220047892A (ko) 2022-04-19
CN104822701B (zh) 2018-09-21
AR093626A1 (es) 2015-06-17
US20160031964A1 (en) 2016-02-04
KR102262882B1 (ko) 2021-06-10
ZA201503509B (en) 2016-11-30
TW201827468A (zh) 2018-08-01
HUE039334T2 (hu) 2018-12-28
AU2013352184B2 (en) 2018-05-31
KR20210070389A (ko) 2021-06-14
US20140161788A1 (en) 2014-06-12
WO2014085621A1 (en) 2014-06-05
KR102385392B1 (ko) 2022-04-11
TW201431884A (zh) 2014-08-16
RU2680581C2 (ru) 2019-02-22
KR102521039B1 (ko) 2023-04-12
US10301369B2 (en) 2019-05-28
AU2013352184A1 (en) 2015-06-04
RU2015125491A (ru) 2017-01-10
JP2019206556A (ja) 2019-12-05
US9845346B2 (en) 2017-12-19
BR112015012152A2 (pt) 2017-08-15
IL262976B (en) 2020-02-27
KR20150088317A (ko) 2015-07-31
PL3115372T3 (pl) 2019-09-30
PT2925776T (pt) 2018-07-30
EP2925776A1 (en) 2015-10-07
US9834587B2 (en) 2017-12-05
US11254725B2 (en) 2022-02-22
EP3115372A1 (en) 2017-01-11
DK3115372T3 (da) 2019-06-11
HRP20181351T1 (hr) 2018-11-02
HUE043679T2 (hu) 2019-09-30
US20160039900A1 (en) 2016-02-11
ES2679374T3 (es) 2018-08-24
JP2016505539A (ja) 2016-02-25
KR20230054482A (ko) 2023-04-24
US20160031963A1 (en) 2016-02-04
HK1216026A1 (en) 2016-10-07
MX2019009191A (es) 2019-10-09
IL272854A (en) 2020-04-30
EP2925776B1 (en) 2018-05-30
US20170355744A1 (en) 2017-12-14
BR112015012152B1 (pt) 2023-04-25
IL262976A (en) 2018-12-31
TWI711632B (zh) 2020-12-01
IL238824A0 (en) 2015-06-30
MX377150B (es) 2025-03-07
PT3115372T (pt) 2019-06-12
US20190225666A1 (en) 2019-07-25
US9771408B2 (en) 2017-09-26
US20220127326A1 (en) 2022-04-28
TWI626250B (zh) 2018-06-11
US9376480B2 (en) 2016-06-28
CA2892146A1 (en) 2014-06-05
US20250388644A1 (en) 2025-12-25

Similar Documents

Publication Publication Date Title
IL272854A (en) Chimeric proteins Enzyme medical lysosomes are designated and their uses
IL281148A (en) New and used KIF5B-RET fusion compounds
PL2844667T3 (pl) Ukierunkowane/immunomodulujące białka fuzyjne i sposoby ich otrzymywania
ZA201409304B (en) Interleukin-10 fusion proteins and uses thereof
PT3744835T (pt) Proteínas de fusão modificadoras do adn e métodos de utilização das mesmas
IL279676A (en) Recombinant proteins and therapeutic uses
IL236933A0 (en) Interleukin-2 fusion proteins and their use
PT3434775T (pt) Proteína que tem atividade de nuclease, proteínas de fusão e utilizações das mesmas
EP2785837A4 (en) TARGETED ENZYMATIC COMPOUNDS AND ITS USES
IL229753A0 (en) Proteins that release relaxin and their use
SG11201502455TA (en) Fgfr3 fusion gene and pharmaceutical drug targeting same
SG2014010482A (en) Modified proteins and peptides
IL222382A0 (en) Robo1-fc fusion protein and use thereof for treating tumours
EP2880050A4 (en) NOVEL ENZYMES, ENZYME COMPONENTS AND USES THEREOF
PL2809669T3 (pl) Skondensowane pirolodikarboksamidy i ich zastosowanie jako środki farmaceutyczne
EP2718432A4 (en) NUCLEASE FUSION PROTEIN AND USES THEREOF
GB201413180D0 (en) MCT protein inhibitor-related prognostic and therapeutic methods
EP2536741A4 (en) THERAPY TARGETING FUSIONS OF GENES
AU2012901619A0 (en) Fusion Proteins and Use Thereof
AU2011902431A0 (en) Fusion Proteins and Use Thereof
AU2011905077A0 (en) Radiotracers and Therapeutic Agents