BR112016018721A2 - proteínas de fusão p97-ids - Google Patents
proteínas de fusão p97-idsInfo
- Publication number
- BR112016018721A2 BR112016018721A2 BR112016018721A BR112016018721A BR112016018721A2 BR 112016018721 A2 BR112016018721 A2 BR 112016018721A2 BR 112016018721 A BR112016018721 A BR 112016018721A BR 112016018721 A BR112016018721 A BR 112016018721A BR 112016018721 A2 BR112016018721 A2 BR 112016018721A2
- Authority
- BR
- Brazil
- Prior art keywords
- fusion proteins
- ids
- cns
- ids fusion
- melanotransferrin
- Prior art date
Links
- 102000037865 fusion proteins Human genes 0.000 title abstract 2
- 108020001507 fusion proteins Proteins 0.000 title abstract 2
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 abstract 3
- 201000002273 mucopolysaccharidosis II Diseases 0.000 abstract 2
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 abstract 1
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 abstract 1
- 208000015439 Lysosomal storage disease Diseases 0.000 abstract 1
- 102000051089 Melanotransferrin Human genes 0.000 abstract 1
- 108700038051 Melanotransferrin Proteins 0.000 abstract 1
- 230000008499 blood brain barrier function Effects 0.000 abstract 1
- 210000001218 blood-brain barrier Anatomy 0.000 abstract 1
- 210000003169 central nervous system Anatomy 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 230000035515 penetration Effects 0.000 abstract 1
- 230000002093 peripheral effect Effects 0.000 abstract 1
Classifications
-
- 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)
-
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/40—Transferrins, e.g. lactoferrins, ovotransferrins
-
- 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)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/04—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; involved in cellular and subcellular movement (3.6.4)
- C12Y306/04006—Vesicle-fusing ATPase (3.6.4.6)
-
- 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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
- C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
- C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/50—Fusion polypeptide containing protease site
Abstract
trata-se de proteínas de fusão entre p97 (melanotransferrina) e iduronato-2-sulfatase (ids) e composições e métodos relacionados de uso dos mesmos, por exemplo, para facilitar a entrega de ids através da barreira hematoencefálica (bbb) e/ou aprimorar sua penetração de tecido em cns e/ou tecidos periféricos, e, assim, tratar e/ou diagnosticar síndrome de hunter (mucopolissacaridose tipo ii; mps ii) e distúrbios de armazenamento lisossômico relacionados, incluindo aqueles que têm um componente do sistema nervoso central (cns).
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201461941896P | 2014-02-19 | 2014-02-19 | |
PCT/US2015/015662 WO2015126729A1 (en) | 2014-02-19 | 2015-02-12 | P97-ids fusion proteins |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112016018721A2 true BR112016018721A2 (pt) | 2017-10-10 |
Family
ID=52629674
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112016018721A BR112016018721A2 (pt) | 2014-02-19 | 2015-02-12 | proteínas de fusão p97-ids |
Country Status (11)
Country | Link |
---|---|
US (2) | US10392605B2 (pt) |
EP (1) | EP3107562B1 (pt) |
JP (2) | JP6605482B2 (pt) |
AU (1) | AU2015219339B2 (pt) |
BR (1) | BR112016018721A2 (pt) |
CA (1) | CA2935805C (pt) |
DK (1) | DK3107562T3 (pt) |
ES (1) | ES2762672T3 (pt) |
HK (1) | HK1232136A1 (pt) |
IL (1) | IL246355B (pt) |
WO (1) | WO2015126729A1 (pt) |
Families Citing this family (15)
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JP2006506317A (ja) | 2002-01-11 | 2006-02-23 | バイオマリン ファーマシューティカル インコーポレイテッド | 治療用リソソーム酵素の送達のための酵素送達系としてのp97の使用 |
AU2012278944B2 (en) | 2011-07-05 | 2015-09-17 | Bioasis Technologies Inc. | p97-antibody conjugates and methods of use |
JP6433424B2 (ja) | 2012-07-31 | 2018-12-05 | バイオアシス テクノロジーズ インコーポレイテッド | 脱リン酸化されたリソソーム蓄積症タンパク質およびその使用方法 |
WO2014160438A1 (en) | 2013-03-13 | 2014-10-02 | Bioasis Technologies Inc. | Fragments of p97 and uses thereof |
AU2015210612B2 (en) | 2014-02-03 | 2020-04-09 | Bioasis Technologies Inc. | P97 fusion proteins |
JP6605482B2 (ja) | 2014-02-19 | 2019-11-13 | バイオアシス テクノロジーズ インコーポレイテッド | P97−ids融合タンパク質 |
WO2015168521A2 (en) | 2014-05-01 | 2015-11-05 | Bioasis Technologies, Inc. | P97-polynucleotide conjugates |
EP3101125A1 (en) * | 2015-06-05 | 2016-12-07 | Laboratorios Del Dr. Esteve, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
SG11201901494UA (en) * | 2016-09-12 | 2019-03-28 | Genethon | Acid-alpha glucosidase variants and uses thereof |
EP3537961A1 (en) | 2016-11-10 | 2019-09-18 | The Research Foundation for The State University of New York | System, method and biomarkers for airway obstruction |
KR20190139951A (ko) * | 2017-04-14 | 2019-12-18 | 리젠엑스바이오 인크. | 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료 |
MX2020002918A (es) | 2017-10-02 | 2020-07-22 | Denali Therapeutics Inc | Proteinas de fusion que comprenden enzimas de terapia de reemplazo de enzimas. |
EP3924475A1 (en) | 2019-02-15 | 2021-12-22 | Sigma-Aldrich Co. LLC | Crispr/cas fusion proteins and systems |
CN114181318B (zh) * | 2021-11-08 | 2023-08-01 | 四川大学 | 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用 |
CN114940712B (zh) * | 2022-06-01 | 2023-12-26 | 山西锦波生物医药股份有限公司 | 一种生物合成人体结构性材料的制备方法 |
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JP6433424B2 (ja) | 2012-07-31 | 2018-12-05 | バイオアシス テクノロジーズ インコーポレイテッド | 脱リン酸化されたリソソーム蓄積症タンパク質およびその使用方法 |
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US20150093399A1 (en) | 2013-08-28 | 2015-04-02 | Bioasis Technologies, Inc. | Cns-targeted conjugates having modified fc regions and methods of use thereof |
AU2015210612B2 (en) | 2014-02-03 | 2020-04-09 | Bioasis Technologies Inc. | P97 fusion proteins |
JP6605482B2 (ja) | 2014-02-19 | 2019-11-13 | バイオアシス テクノロジーズ インコーポレイテッド | P97−ids融合タンパク質 |
WO2015168521A2 (en) | 2014-05-01 | 2015-11-05 | Bioasis Technologies, Inc. | P97-polynucleotide conjugates |
US20190022244A1 (en) | 2016-01-13 | 2019-01-24 | Bioasis Technologies, Inc. | Blood-brain barrier vector compounds and conjugates thereof |
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JP6605482B2 (ja) | 2019-11-13 |
JP2020059710A (ja) | 2020-04-16 |
DK3107562T3 (da) | 2019-12-16 |
JP6930756B6 (ja) | 2021-10-20 |
AU2015219339B2 (en) | 2020-03-05 |
IL246355B (en) | 2020-06-30 |
CA2935805A1 (en) | 2015-08-27 |
JP2017506886A (ja) | 2017-03-16 |
US11124781B2 (en) | 2021-09-21 |
AU2015219339A1 (en) | 2016-07-07 |
JP6930756B2 (ja) | 2021-09-01 |
EP3107562B1 (en) | 2019-09-18 |
US20170204386A1 (en) | 2017-07-20 |
CA2935805C (en) | 2023-07-11 |
ES2762672T3 (es) | 2020-05-25 |
EP3107562A1 (en) | 2016-12-28 |
IL246355A0 (en) | 2016-08-31 |
US20200270590A1 (en) | 2020-08-27 |
HK1232136A1 (zh) | 2018-01-05 |
WO2015126729A1 (en) | 2015-08-27 |
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