WO2014082080A3 - Procédés de couplage de peptides de ciblage sur des enzymes lysosomales recombinantes pour des traitements améliorés de maladies lysosomales - Google Patents
Procédés de couplage de peptides de ciblage sur des enzymes lysosomales recombinantes pour des traitements améliorés de maladies lysosomales Download PDFInfo
- Publication number
- WO2014082080A3 WO2014082080A3 PCT/US2013/071994 US2013071994W WO2014082080A3 WO 2014082080 A3 WO2014082080 A3 WO 2014082080A3 US 2013071994 W US2013071994 W US 2013071994W WO 2014082080 A3 WO2014082080 A3 WO 2014082080A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- crosslinking agent
- methods
- lysosomal enzymes
- targeting peptides
- storage diseases
- Prior art date
Links
- 102000004190 Enzymes Human genes 0.000 title abstract 5
- 108090000790 Enzymes Proteins 0.000 title abstract 5
- 230000002132 lysosomal effect Effects 0.000 title abstract 5
- 238000000034 method Methods 0.000 title abstract 4
- 102000004196 processed proteins & peptides Human genes 0.000 title abstract 2
- 108090000765 processed proteins & peptides Proteins 0.000 title abstract 2
- 230000008685 targeting Effects 0.000 title abstract 2
- 238000011282 treatment Methods 0.000 title abstract 2
- 208000015439 Lysosomal storage disease Diseases 0.000 title 1
- 230000008878 coupling Effects 0.000 title 1
- 238000010168 coupling process Methods 0.000 title 1
- 238000005859 coupling reaction Methods 0.000 title 1
- 239000003431 cross linking reagent Substances 0.000 abstract 6
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 abstract 3
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 abstract 2
- 102100037182 Cation-independent mannose-6-phosphate receptor Human genes 0.000 abstract 1
- 101710145225 Cation-independent mannose-6-phosphate receptor Proteins 0.000 abstract 1
- 101001076292 Homo sapiens Insulin-like growth factor II Proteins 0.000 abstract 1
- 102100025947 Insulin-like growth factor II Human genes 0.000 abstract 1
- 150000001413 amino acids Chemical class 0.000 abstract 1
- 230000004700 cellular uptake Effects 0.000 abstract 1
- 230000001268 conjugating effect Effects 0.000 abstract 1
- 125000003588 lysine group Chemical group [H]N([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 abstract 1
Classifications
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- 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
- 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
-
- 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
- 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)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
-
- 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
- C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
-
- 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)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Diabetes (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Endocrinology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
Abstract
La présente invention concerne des procédés de fabrication de peptides de ciblage, conjugués à des enzymes lysosomales recombinantes, par la modification de l'extrémité (N)-terminale aminée et d'un ou de plusieurs résidus lysine sur des enzymes lysosomales humaines, recombinantes, à l'aide d'un premier agent de réticulation pour donner naissance à des enzymes lysosomales humaines, recombinantes, modifiées par un premier agent de réticulation, modifiant le premier acide aminé dans un lieur court au niveau de l'extrémité (N)-terminale aminée sur un variant de peptide IGF-2 à l'aide d'un second agent de réticulation pour donner naissance à un variant de peptide IGF-2 modifié par un second agent de réticulation, puis par la conjugaison de l'enzyme lysosomale humaine, recombinante, modifiée par un premier agent de réticulation, et du variant de peptide IGF-2 modifié par un second agent de réticulation contenant un lieur court. La présente invention concerne également des conjugués synthétisés, caractérisés comme ayant des affinités et des avidités supérieures pour le récepteur IGF-2/CI-MPR et une capture cellulaire supérieur à l'aide des procédés de l'invention. La présente invention concerne également des méthodes de traitement à l'aide des conjugués décrits.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261729857P | 2012-11-26 | 2012-11-26 | |
| US61/729,857 | 2012-11-26 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| WO2014082080A2 WO2014082080A2 (fr) | 2014-05-30 |
| WO2014082080A3 true WO2014082080A3 (fr) | 2014-07-24 |
Family
ID=50776681
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PCT/US2013/071994 WO2014082080A2 (fr) | 2012-11-26 | 2013-11-26 | Procédés de couplage de peptides de ciblage sur des enzymes lysosomales recombinantes pour des traitements améliorés de maladies lysosomales |
Country Status (1)
| Country | Link |
|---|---|
| WO (1) | WO2014082080A2 (fr) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| SG11202106209RA (en) | 2018-12-19 | 2021-07-29 | Univ Leland Stanford Junior | Bifunctional molecules for lysosomal targeting and related compositions and methods |
| CN110373405B (zh) * | 2019-07-24 | 2023-12-15 | 杭州恩和生物科技有限公司 | 一种接枝聚合物的生物酶及其制备方法和固定方法 |
| AU2020361703A1 (en) * | 2019-10-10 | 2022-04-28 | Amicus Therapeutics, Inc. | Variant IGF2 constructs |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7811786B1 (en) * | 2004-05-06 | 2010-10-12 | Daewoong Co., Ltd. | Preparation method for the production of active and soluble proteins in prokaryotes and polycistronic vectors therefor |
| US20110003969A1 (en) * | 2009-06-03 | 2011-01-06 | Immunogen Inc. | Conjugation methods |
| US20110223147A1 (en) * | 2008-05-07 | 2011-09-15 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| WO2012121973A1 (fr) * | 2011-03-04 | 2012-09-13 | Life Technologies Corporation | Composés et procédés de conjugaison de biomolécules |
| EP1877099B1 (fr) * | 2005-04-06 | 2012-09-19 | Genzyme Corporation | Conjugués thérapeutiques comprenant une enzyme lysosomale, l'acide polysialique et un agent de ciblage |
-
2013
- 2013-11-26 WO PCT/US2013/071994 patent/WO2014082080A2/fr active Application Filing
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7811786B1 (en) * | 2004-05-06 | 2010-10-12 | Daewoong Co., Ltd. | Preparation method for the production of active and soluble proteins in prokaryotes and polycistronic vectors therefor |
| EP1877099B1 (fr) * | 2005-04-06 | 2012-09-19 | Genzyme Corporation | Conjugués thérapeutiques comprenant une enzyme lysosomale, l'acide polysialique et un agent de ciblage |
| US20110223147A1 (en) * | 2008-05-07 | 2011-09-15 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| US20110003969A1 (en) * | 2009-06-03 | 2011-01-06 | Immunogen Inc. | Conjugation methods |
| WO2012121973A1 (fr) * | 2011-03-04 | 2012-09-13 | Life Technologies Corporation | Composés et procédés de conjugaison de biomolécules |
Non-Patent Citations (2)
| Title |
|---|
| LIAO ET AL.: "Cloning, expression, purification, and characterization of the human broad specificity lysosomal acid alpha-mannosidase.", J BIOL CHEM, vol. 271, no. 45, 8 November 1996 (1996-11-08), pages 28348 - 28358 * |
| SOLULINK.: "All-Purpose Crosslinking Kit-Technical Manual", 30 August 2012 (2012-08-30), Retrieved from the Internet <URL:http:/twww.solulink.com/library> [retrieved on 20140221] * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2014082080A2 (fr) | 2014-05-30 |
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