PT2925776T - Proteínas de fusão de enzima lisossomal terapêuticas alvo e suas utilizações - Google Patents

Proteínas de fusão de enzima lisossomal terapêuticas alvo e suas utilizações

Info

Publication number
PT2925776T
PT2925776T PT138028345T PT13802834T PT2925776T PT 2925776 T PT2925776 T PT 2925776T PT 138028345 T PT138028345 T PT 138028345T PT 13802834 T PT13802834 T PT 13802834T PT 2925776 T PT2925776 T PT 2925776T
Authority
PT
Portugal
Prior art keywords
fusion proteins
lysosomal enzyme
targeted therapeutic
enzyme fusion
therapeutic lysosomal
Prior art date
Application number
PT138028345T
Other languages
English (en)
Portuguese (pt)
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 PT2925776T publication Critical patent/PT2925776T/pt

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, i.e. 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (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)
PT138028345T 2012-11-27 2013-11-27 Proteínas de fusão de enzima lisossomal terapêuticas alvo e suas utilizações PT2925776T (pt)

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
PT2925776T true PT2925776T (pt) 2018-07-30

Family

ID=49753532

Family Applications (2)

Application Number Title Priority Date Filing Date
PT138028345T PT2925776T (pt) 2012-11-27 2013-11-27 Proteínas de fusão de enzima lisossomal terapêuticas alvo e suas utilizações
PT16182420T PT3115372T (pt) 2012-11-27 2013-11-27 Proteínas de fusão de enzima lisossomal terapêuticas alvo e suas utilizações

Family Applications After (1)

Application Number Title Priority Date Filing Date
PT16182420T PT3115372T (pt) 2012-11-27 2013-11-27 Proteínas de fusão de enzima lisossomal terapêuticas alvo e suas utilizações

Country Status (26)

Country Link
US (9) US9376480B2 (enExample)
EP (2) EP2925776B1 (enExample)
JP (2) JP6831176B2 (enExample)
KR (4) KR102262882B1 (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) DK2925776T3 (enExample)
ES (2) ES2679374T3 (enExample)
HR (2) HRP20181351T1 (enExample)
HU (2) HUE039334T2 (enExample)
IL (3) IL238824B (enExample)
LT (1) LT3115372T (enExample)
MX (2) MX367024B (enExample)
PL (2) PL3115372T3 (enExample)
PT (2) PT2925776T (enExample)
RS (1) RS58916B1 (enExample)
RU (1) RU2680581C2 (enExample)
SI (1) SI3115372T1 (enExample)
TW (2) TWI626250B (enExample)
WO (1) WO2014085621A1 (enExample)
ZA (1) ZA201503509B (enExample)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009137721A2 (en) 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
ES2679374T3 (es) 2012-11-27 2018-08-24 Biomarin Pharmaceutical Inc. Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas
JP2016523835A (ja) 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
WO2016025519A1 (en) * 2014-08-11 2016-02-18 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
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
JP6993958B2 (ja) 2015-07-06 2022-02-04 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 多重特異性抗原結合分子およびその使用
HK1253529A1 (zh) 2015-11-06 2019-06-21 Biomarin Pharmaceutical Inc. 用於检测中和溶酶体酶的摄取的抗体或其它因子的基於细胞的测定
EP3419651B1 (en) 2016-02-24 2023-10-25 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
US11253612B2 (en) 2016-04-15 2022-02-22 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
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
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
EP3762505A4 (en) * 2018-03-09 2021-12-01 Avrobio, Inc. COMPOSITIONS AND METHODS OF TREATMENT FOR PARKINSON'S DISEASE
TW202344691A (zh) * 2018-04-30 2023-11-16 美商阿米庫斯醫療股份有限公司 基因治療構築體及使用方法
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
JP2021526026A (ja) 2018-06-08 2021-09-30 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 翻訳後修飾が低減したペプチドリンカー
JP7577650B2 (ja) 2018-10-10 2024-11-05 アミカス セラピューティックス インコーポレイテッド ジスルフィド結合により安定したポリペプチド組成物及びその使用方法
CA3120087A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence
JP2022552254A (ja) * 2019-10-10 2022-12-15 アミカス セラピューティックス インコーポレイテッド バリアントigf2構築体
EP4081241A4 (en) 2019-12-24 2024-05-01 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
EP4359425B1 (en) 2021-06-21 2026-08-05 Juvena Therapeutics, Inc. Igf2 fusion polypeptide and use in the treatment of muscular dystrophy, sarcopenia, cachexia, or muscle injury
EP4359430A4 (en) * 2021-06-23 2025-07-16 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
EP4654982A1 (en) * 2023-01-27 2025-12-03 Regeneron Pharmaceuticals, Inc. Modified rhabdovirus glycoproteins and uses thereof
CN117122690A (zh) * 2023-08-28 2023-11-28 河北工业大学 一种溶酶体靶向嵌合体及其制备方法和应用

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
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
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
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
EP1436316B1 (en) * 2001-04-30 2008-01-23 ZyStor Therapeutics , Inc. Subcellular targeting of therapeutic proteins
WO2003032913A2 (en) 2001-10-16 2003-04-24 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
ES2371072T3 (es) 2003-06-12 2011-12-27 Eli Lilly And Company Proteínas de fusión análogas de 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
WO2006096653A2 (en) 2005-03-04 2006-09-14 Biogen Idec Ma Inc. Methods of humanizing immunoglobulin variable regions through rational modification of complementarity determining residues
WO2009137721A2 (en) 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
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
US8785168B2 (en) * 2009-06-17 2014-07-22 Biomarin Pharmaceutical Inc. Formulations for lysosomal enzymes
CN103269709A (zh) * 2010-06-25 2013-08-28 夏尔人类遗传性治疗公司 Sanfilippo综合症类型B的治疗
MX335966B (es) 2010-06-25 2016-01-06 Amcor Ltd Sistema de depuración de oxígeno para un recipiente.
US9409950B2 (en) * 2010-12-23 2016-08-09 Biogen Ma Inc. Linker peptides and polypeptides comprising same
BR112013022557A2 (pt) * 2011-03-04 2017-08-01 Shire Human Genetic Therapies ligantes de peptídeo composições de polipeptídeo e métodos para uso dos mesmos
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
ES2679374T3 (es) 2012-11-27 2018-08-24 Biomarin Pharmaceutical Inc. Proteínas de fusión terapéuticas dirigidas de enzima lisosómica y usos de las mismas

Also Published As

Publication number Publication date
MX367024B (es) 2019-08-02
TWI626250B (zh) 2018-06-11
US9771408B2 (en) 2017-09-26
CN104822701A (zh) 2015-08-05
IL262976A (en) 2018-12-31
DK2925776T3 (en) 2018-09-03
JP6831176B2 (ja) 2021-02-17
HRP20190918T1 (hr) 2019-09-20
TW201827468A (zh) 2018-08-01
KR20220047892A (ko) 2022-04-19
IL272854A (en) 2020-04-30
EP3115372A1 (en) 2017-01-11
HUE043679T2 (hu) 2019-09-30
EP3115372B1 (en) 2019-03-06
IL262976B (en) 2020-02-27
KR20150088317A (ko) 2015-07-31
PL3115372T3 (pl) 2019-09-30
KR20230054482A (ko) 2023-04-24
US9834588B2 (en) 2017-12-05
JP2019206556A (ja) 2019-12-05
LT3115372T (lt) 2019-06-25
US20160031964A1 (en) 2016-02-04
US20160031965A1 (en) 2016-02-04
JP2016505539A (ja) 2016-02-25
IL238824A0 (en) 2015-06-30
AU2013352184B2 (en) 2018-05-31
HK1216026A1 (en) 2016-10-07
PT3115372T (pt) 2019-06-12
US9834587B2 (en) 2017-12-05
US11254725B2 (en) 2022-02-22
EP2925776B1 (en) 2018-05-30
HRP20181351T1 (hr) 2018-11-02
BR112015012152A2 (pt) 2017-08-15
DK3115372T3 (da) 2019-06-11
PL2925776T3 (pl) 2018-11-30
US20160039900A1 (en) 2016-02-11
KR102521039B1 (ko) 2023-04-12
BR112015012152B1 (pt) 2023-04-25
TWI711632B (zh) 2020-12-01
US20170355744A1 (en) 2017-12-14
AR093626A1 (es) 2015-06-17
US20190225666A1 (en) 2019-07-25
US20250388644A1 (en) 2025-12-25
KR102385392B1 (ko) 2022-04-11
ES2679374T3 (es) 2018-08-24
IL272854B (en) 2021-07-29
US9376480B2 (en) 2016-06-28
IL238824B (en) 2018-11-29
RS58916B1 (sr) 2019-08-30
JP6913719B2 (ja) 2021-08-04
RU2680581C2 (ru) 2019-02-22
ZA201503509B (en) 2016-11-30
SI3115372T1 (sl) 2019-08-30
MX2015006644A (es) 2015-08-10
CL2015001371A1 (es) 2015-10-09
KR20210070389A (ko) 2021-06-14
ES2729997T3 (es) 2019-11-07
RU2015125491A (ru) 2017-01-10
US9845346B2 (en) 2017-12-19
TW201431884A (zh) 2014-08-16
WO2014085621A1 (en) 2014-06-05
CN104822701B (zh) 2018-09-21
US10301369B2 (en) 2019-05-28
HUE039334T2 (hu) 2018-12-28
US20140161788A1 (en) 2014-06-12
MX377150B (es) 2025-03-07
KR102262882B1 (ko) 2021-06-10
EP2925776A1 (en) 2015-10-07
CY1122555T1 (el) 2021-01-27
AU2013352184A1 (en) 2015-06-04
CA2892146A1 (en) 2014-06-05
MX2019009191A (es) 2019-10-09
US20220127326A1 (en) 2022-04-28
US20160031963A1 (en) 2016-02-04

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