DK2279210T3 - Lysosomale targeting peptider og anvendelser deraf - Google Patents
Lysosomale targeting peptider og anvendelser deraf Download PDFInfo
- Publication number
- DK2279210T3 DK2279210T3 DK09743707.3T DK09743707T DK2279210T3 DK 2279210 T3 DK2279210 T3 DK 2279210T3 DK 09743707 T DK09743707 T DK 09743707T DK 2279210 T3 DK2279210 T3 DK 2279210T3
- Authority
- DK
- Denmark
- Prior art keywords
- igf
- fusion protein
- furin
- seq
- amino acid
- Prior art date
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Classifications
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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
- 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
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- 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
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
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- 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
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- 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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- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
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)
- Gastroenterology & Hepatology (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Diabetes (AREA)
- Biophysics (AREA)
- Toxicology (AREA)
- Endocrinology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Claims (15)
1. Målrettet terapeutisk fusionspro tein omfattende: en lysomal enzym; og en IGF-II mutein udvisende en aminosyresekvens, der er i det mindste 70% identisk med modent humant IGF-II (SEQ ID NO:l), og som omfatter en mutation inden for et område, der svarer til aminosyrer 30-40, der hører til SEQ ID NO:l, således at mutationen ophæver i det mindste et område med furin-protease hvor IGF-II-muteinet (a) binder til human kation-uafhængig mannose-6-phosphat-receptor på mannose-6-phosphat-uafhængig måde; (b) er modstandsdygtig mod furin-kløvning; og (c) har aftagende forløbende binding s-affinitet for insulin-receptoren i forhold til affiniteten for naturligt-optrædende human IGF-II for insulin-receptoren.
2. Målrettet terapeutisk fusionsprotein ifølge krav 1, hvor IGF-II muteinen omfatter en udeladelse af aminosyrer 30-40, der hører til SEQ ID NO:l.
3. Målrettet terapeutisk fusionsprotein ifølge krav 1, hvor mutationen er en aminosyre-substitution ved en position, der svarer til Arg37 hørende til SEQ ID NO:l, og som option, hvor aminosyre-substitutionen er en Lys- eller Ala-substitution.
4. Målrettet terapeutisk fusionsprotein ifølge et hvilket som helst af de foregående krav, hvor lysomal-enzymet er udvalgt blandt: al,4-glucosidasesyre; β-galactosidase; β-hexosaminidase A; β-hexosaminidase B; α-galactosidase A; glucocerebrosidase; arylsulfatase A; galactosylceramidase; sphingomyelinasesyre; ceramidasesyre; lipase-syre; α-L-iduronidase; iduronatsulfatase; heparan-N-sulfatase; a-N-acetylglucosamini-dase; acetyl-CoA-glucosaminidacetyltransferase; N-acetylglucosamin-6-sulfatase; galactosamin-6-sulfatase; β-galactosidase; arylsulfatase B; β-glucuronidase; a-manno-sidase; β-mannosidase; α-L-fucosidase; N-aspartyl^-glucosaminidase; a-neuramini-dase; lysosomalemprotektivprotein; α-N-acetyl-galactosaminidase; N-acetyl-glucosamin-1-phosphotransferase; og palmitoyl-protein-thioesterase.
5. Målrettet terapeutisk fusionsprotein ifølge et hvilket som helst af de foregående krav, hvor IGF-II mutein omfatter: (a) en udeladelse af eller en udskiftning af aminosyrer, der svarer til positioner 2-7 afSEQ ID NO:l; og/eller (b) en udeladelse af eller en udskiftning af aminosyrer, der svarer til positioner 1-7 afSEQ ID NO: 1; og/eller (c) en udeladelse af eller en udskiftning af aminosyrer svarende til positioner 62-67 afSEQ ID NO:l; og/eller (d) en amino syre-substitution ved en position, der svarer til Tyr27, Leu43 eller Ser26 af SEQ ID NO.l, som option udvalgt blandt gruppen omfattende Tyr27Leu, Leu43Val, Ser26Phe og kombinationer deraf; og/eller (e) aminosyrer, der svarer til positioner 48-55 af SEQ ID NO:l: og/eller (f) i det mindste tre aminosyrer udvalgt blandt gruppen bestående af amino-syrer, der svarer til positioner 8, 48, 49, 50, 54 og 56 af SEQ ID NO:l: og/eller (g) ved positioner, der svarer til positioner 54 og 56 af SEQ ID NO:l, aminosyrer, hvor hver af dem er uladet eller negativt ladet ved pH 7.4.
6. Målrettet terapeutisk fusionsprotein ifølge et hvilket som helst af de foregående krav, hvor IGF-II mutein udviser mindsket bindingsaffinitet over for IGF-I-receptor i forhold til affiniteten af naturligt optrædende humant IGF-II for IGF-I-receptoren.
7. Målrettet terapeutisk fusionsprotein ifølge et hvilket som helst af de foregående krav, hvor lysomal-enzymet er human alpha-glucosidasesyre (GAA), eller en funktionel variant deraf, som option hvor lysomal-enzymet omfatter aminosyrer 70-952 af human GAA.
8. Målrettet terapeutisk fusionsprotein ifølge et hvilket som helst af de foregående krav, hvor det målrettede terapeutiske fusionsprotein endvidere omfatter en mellemrumsgiver mellem lysomal-enzymet og IGF-II-mutein, som option hvor mellemrumsgiveren omfatter en aminosyresekvens Gly-Ala-Pro.
9. En nukleinsyre, der koder det målrettede fusionsprotein ifølge et hvilket som helst af de foregående krav.
10. En celle, der indeholder nukleinsyren ifølge krav 9.
11. Farmaceutisk sammensætning, som er egnet til behandling af lysomal storage lidelse omfattende et målrettet terapeutisk fusionsprotein ifølge et hvilket som helst af kravene 1 til 8.
12. Målrettet terapeutisk fusionsprotein ifølge et hvilket som helst af kravene 1 til 8 til anvendelse ved en fremgangsmåde til behandling af lysomal storage lidelse.
13. Målrettet terapeutisk fusionsprotein til anvendelse ifølge krav 12, hvor den lysomale storage lidelse er udvalgt blandt: Pompes sygdom, Wolmans sygdom og Sanfi-lippo B (MPS HIB).
14. Fremgangsmåde til fremstilling af et målrettet fusionsprotein omfattende et fremgangsmådetrin med dyrkning af pattedyrsceller i et cellekulturmedium, hvori pattedyrscellerne bærer kernesyren ifølge krav 9: og dyrkningen gennemføres under forhold, der tillader expression af det målrettede terapeutiske fusionsprotein.
15. Fremgangsmåde til fremstilling af et målrettet terapeutisk fusionsprotein ifølge et hvilket som helst af kravene 1 til 8 omfattende et fremgangsmådetrin med dyrkning af furin-deficiente celler i et cellekulturmedium, hvori de furin-deficiente celler hærer en kemesyre, der koder et fusionsprotein ifølge krav 1 til 8, og hvor dyrkningen gennemføres under forhold, som tillader expression af de målrettede terapeutiske fasionsproteiner.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US5133608P | 2008-05-07 | 2008-05-07 | |
| US14410609P | 2009-01-12 | 2009-01-12 | |
| PCT/US2009/043207 WO2009137721A2 (en) | 2008-05-07 | 2009-05-07 | Lysosomal targeting peptides and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2279210T3 true DK2279210T3 (da) | 2017-07-24 |
Family
ID=41265422
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK09743707.3T DK2279210T3 (da) | 2008-05-07 | 2009-05-07 | Lysosomale targeting peptider og anvendelser deraf |
Country Status (16)
| Country | Link |
|---|---|
| US (8) | US20110223147A1 (da) |
| EP (4) | EP3272773B8 (da) |
| JP (1) | JP5627571B2 (da) |
| CN (1) | CN102066422B (da) |
| AU (1) | AU2009244148B2 (da) |
| BR (2) | BRPI0912225A2 (da) |
| CA (1) | CA2723412A1 (da) |
| CY (1) | CY1119183T1 (da) |
| DK (1) | DK2279210T3 (da) |
| ES (2) | ES2830350T3 (da) |
| HR (1) | HRP20170698T1 (da) |
| HU (1) | HUE034850T2 (da) |
| IL (1) | IL209091A (da) |
| PL (1) | PL2279210T3 (da) |
| PT (1) | PT2279210T (da) |
| WO (1) | WO2009137721A2 (da) |
Families Citing this family (62)
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