RU2014117291A - Рекомбинантный человеческий белок naglu и его применение - Google Patents
Рекомбинантный человеческий белок naglu и его применение Download PDFInfo
- Publication number
- RU2014117291A RU2014117291A RU2014117291/10A RU2014117291A RU2014117291A RU 2014117291 A RU2014117291 A RU 2014117291A RU 2014117291/10 A RU2014117291/10 A RU 2014117291/10A RU 2014117291 A RU2014117291 A RU 2014117291A RU 2014117291 A RU2014117291 A RU 2014117291A
- Authority
- RU
- Russia
- Prior art keywords
- rhnaglu
- composition according
- naglu
- recombinant human
- glucosaminidase
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title claims 6
- 102000004169 proteins and genes Human genes 0.000 title claims 6
- 102100034561 Alpha-N-acetylglucosaminidase Human genes 0.000 title 1
- 108010009380 alpha-N-acetyl-D-glucosaminidase Proteins 0.000 title 1
- 239000000203 mixture Substances 0.000 claims abstract 50
- 208000005340 mucopolysaccharidosis III Diseases 0.000 claims abstract 14
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims abstract 14
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 claims abstract 9
- 210000004027 cell Anatomy 0.000 claims abstract 5
- OVRNDRQMDRJTHS-UHFFFAOYSA-N N-acelyl-D-glucosamine Natural products CC(=O)NC1C(O)OC(CO)C(O)C1O OVRNDRQMDRJTHS-UHFFFAOYSA-N 0.000 claims abstract 4
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims abstract 4
- MBLBDJOUHNCFQT-LXGUWJNJSA-N N-acetylglucosamine Natural products CC(=O)N[C@@H](C=O)[C@@H](O)[C@H](O)[C@H](O)CO MBLBDJOUHNCFQT-LXGUWJNJSA-N 0.000 claims abstract 4
- 125000003275 alpha amino acid group Chemical group 0.000 claims abstract 4
- 230000000694 effects Effects 0.000 claims abstract 4
- 210000004962 mammalian cell Anatomy 0.000 claims abstract 4
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 claims abstract 3
- 239000008103 glucose Substances 0.000 claims abstract 3
- 150000004676 glycans Chemical group 0.000 claims abstract 3
- WQZGKKKJIJFFOK-QTVWNMPRSA-N D-mannopyranose Chemical compound OC[C@H]1OC(O)[C@@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-QTVWNMPRSA-N 0.000 claims abstract 2
- 229930091371 Fructose Natural products 0.000 claims abstract 2
- RFSUNEUAIZKAJO-ARQDHWQXSA-N Fructose Chemical compound OC[C@H]1O[C@](O)(CO)[C@@H](O)[C@@H]1O RFSUNEUAIZKAJO-ARQDHWQXSA-N 0.000 claims abstract 2
- 239000005715 Fructose Substances 0.000 claims abstract 2
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical compound CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 claims abstract 2
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims abstract 2
- OVRNDRQMDRJTHS-RTRLPJTCSA-N N-acetyl-D-glucosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@@H](O)[C@@H]1O OVRNDRQMDRJTHS-RTRLPJTCSA-N 0.000 claims abstract 2
- OVRNDRQMDRJTHS-FMDGEEDCSA-N N-acetyl-beta-D-glucosamine Chemical compound CC(=O)N[C@H]1[C@H](O)O[C@H](CO)[C@@H](O)[C@@H]1O OVRNDRQMDRJTHS-FMDGEEDCSA-N 0.000 claims abstract 2
- WQZGKKKJIJFFOK-VFUOTHLCSA-N beta-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 claims abstract 2
- 229930182830 galactose Natural products 0.000 claims abstract 2
- 229950006780 n-acetylglucosamine Drugs 0.000 claims abstract 2
- 230000010837 receptor-mediated endocytosis Effects 0.000 claims abstract 2
- 241000894007 species Species 0.000 claims abstract 2
- 210000004556 brain Anatomy 0.000 claims 7
- 241000124008 Mammalia Species 0.000 claims 6
- 230000009261 transgenic effect Effects 0.000 claims 4
- 241000287828 Gallus gallus Species 0.000 claims 3
- 210000005260 human cell Anatomy 0.000 claims 3
- 125000000539 amino acid group Chemical group 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 238000007913 intrathecal administration Methods 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 238000007910 systemic administration Methods 0.000 claims 2
- 229940124597 therapeutic agent Drugs 0.000 claims 2
- 241000272525 Anas platyrhynchos Species 0.000 claims 1
- 241000271566 Aves Species 0.000 claims 1
- 229920002971 Heparan sulfate Polymers 0.000 claims 1
- 108010058846 Ovalbumin Proteins 0.000 claims 1
- 241000286209 Phasianidae Species 0.000 claims 1
- 230000008499 blood brain barrier function Effects 0.000 claims 1
- 210000001218 blood-brain barrier Anatomy 0.000 claims 1
- 230000037396 body weight Effects 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000012202 endocytosis Effects 0.000 claims 1
- 230000002255 enzymatic effect Effects 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 229960003444 immunosuppressant agent Drugs 0.000 claims 1
- 230000001861 immunosuppressant effect Effects 0.000 claims 1
- 239000003018 immunosuppressive agent Substances 0.000 claims 1
- 238000001727 in vivo Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 210000002540 macrophage Anatomy 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 229940092253 ovalbumin Drugs 0.000 claims 1
- 210000003101 oviduct Anatomy 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 102000005962 receptors Human genes 0.000 claims 1
- 108020003175 receptors Proteins 0.000 claims 1
- 210000001626 skin fibroblast Anatomy 0.000 claims 1
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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7024—Esters of saccharides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/02—Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/12—Antidiarrhoeals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/20—Hypnotics; Sedatives
-
- 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
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- 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
-
- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
- C07K7/04—Linear peptides containing only normal peptide links
- C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
- C07K7/04—Linear peptides containing only normal peptide links
- C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
-
- 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
- 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)
- C12N9/2474—Hyaluronoglucosaminidase (3.2.1.35), i.e. hyaluronidase
-
- 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)
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biophysics (AREA)
- Dermatology (AREA)
- Neurology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Neurosurgery (AREA)
- Transplantation (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Rheumatology (AREA)
- Physical Education & Sports Medicine (AREA)
- Anesthesiology (AREA)
- Obesity (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161546248P | 2011-10-12 | 2011-10-12 | |
| US61/546,248 | 2011-10-12 | ||
| PCT/US2012/059708 WO2013055888A2 (en) | 2011-10-12 | 2012-10-11 | Recombinant human naglu protein and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2014117291A true RU2014117291A (ru) | 2015-11-20 |
Family
ID=48082738
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2014117291/10A RU2014117291A (ru) | 2011-10-12 | 2012-10-11 | Рекомбинантный человеческий белок naglu и его применение |
Country Status (18)
| Country | Link |
|---|---|
| US (3) | US9579366B2 (enExample) |
| EP (1) | EP2766477B1 (enExample) |
| JP (1) | JP6329483B2 (enExample) |
| KR (1) | KR20140108519A (enExample) |
| CN (1) | CN103958676A (enExample) |
| AU (1) | AU2012322811A1 (enExample) |
| BR (1) | BR112014008873A2 (enExample) |
| CA (1) | CA2852027A1 (enExample) |
| CL (1) | CL2014000927A1 (enExample) |
| CO (1) | CO7010783A2 (enExample) |
| HK (1) | HK1200489A1 (enExample) |
| IL (1) | IL232073A0 (enExample) |
| IN (1) | IN2014MN00740A (enExample) |
| MX (1) | MX2014004359A (enExample) |
| RU (1) | RU2014117291A (enExample) |
| SG (2) | SG11201401457SA (enExample) |
| TW (1) | TW201321515A (enExample) |
| WO (1) | WO2013055888A2 (enExample) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2007285763B2 (en) | 2006-08-18 | 2011-12-15 | Armagen Technologies, Inc. | Agents for blood-brain barrier delivery |
| CA3184105A1 (en) | 2007-07-27 | 2009-02-05 | Armagen Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
| JP2013507131A (ja) | 2009-10-09 | 2013-03-04 | アーメイゲン・テクノロジーズ・インコーポレイテッド | Ｃｎｓにおけるイズロン酸２−スルファターゼ活性を増加させるための方法および組成物 |
| BR122019021757B1 (pt) | 2010-09-09 | 2020-12-08 | Alexion Pharmaceuticals, Inc | composição farmacêutica para tratamento de um paciente humano sofrendo de uma deficiência de lipase ácida lisossomal (lal) |
| EP2675472A4 (en) | 2011-02-15 | 2014-09-17 | Synageva Biopharma Corp | METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE |
| WO2013055888A2 (en) * | 2011-10-12 | 2013-04-18 | Synageva Biopharma Corp. | Recombinant human naglu protein and uses thereof |
| US8486399B2 (en) | 2011-12-02 | 2013-07-16 | Armagen Technologies, Inc. | Methods and compositions for increasing arylsulfatase A activity in the CNS |
| 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 |
| TWI793159B (zh) * | 2013-10-23 | 2023-02-21 | 美商健臻公司 | 重組醣蛋白及其用途 |
| SG11201609314XA (en) * | 2014-05-14 | 2016-12-29 | Esteve Labor Dr | Adenoassociated virus vectors for the treatment of lysosomal storage disorders |
| EP3160494B1 (en) * | 2014-06-25 | 2019-11-20 | Shire Human Genetic Therapies, Inc. | Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase |
| WO2016025519A1 (en) * | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| AU2015323966A1 (en) | 2014-09-29 | 2017-03-16 | Alexion Pharmaceuticals, Inc. | Methods of treating mucopolysaccharidosis IIIB (MPSIIIB) |
| CN104524662B (zh) * | 2014-12-31 | 2017-02-22 | 迈得医疗工业设备股份有限公司 | 一种医用针座组装的上料机构 |
| US10538589B2 (en) * | 2015-01-14 | 2020-01-21 | Armagen Inc. | Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU |
| US20190083583A1 (en) * | 2016-01-29 | 2019-03-21 | Alexion Pharmaceuticals, Inc. | Enzyme replacement therapy in mucopolysaccharidosis iiib patients |
| US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| US10633705B2 (en) | 2016-10-14 | 2020-04-28 | Alexion Pharmaceuticals, Inc. | N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods |
| 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 |
| EP4100532B1 (en) | 2020-03-23 | 2026-08-12 | The University of North Carolina at Chapel Hill | Aav-naglu vectors for treatment of mucopolysaccharidosis iiib |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
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| US6825396B2 (en) | 1996-06-12 | 2004-11-30 | Board Of Trustees Operating Michigan State University | Methods for tissue specific synthesis of protein in eggs of transgenic hens |
| US5897998A (en) | 1997-08-04 | 1999-04-27 | The University Of Georgia Research Foundation, Inc. | Method for manipulating avian eggs |
| AU752946B2 (en) | 1997-10-16 | 2002-10-03 | Avigenics, Inc. | Vectors comprising a magnum-specific promoter for avian transgenesis |
| US7511120B2 (en) | 1997-10-16 | 2009-03-31 | Synageva Biopharma Corp. | Glycosylated G-CSF obtained from a transgenic chicken |
| US20040019923A1 (en) | 1997-10-16 | 2004-01-29 | Ivarie Robert D. | Exogenous proteins expressed in avians and their eggs |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| US6534300B1 (en) * | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| US7138262B1 (en) | 2000-08-18 | 2006-11-21 | Shire Human Genetic Therapies, Inc. | High mannose proteins and methods of making high mannose proteins |
| US7001994B2 (en) * | 2001-01-18 | 2006-02-21 | Genzyme Corporation | Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins |
| US7294507B2 (en) | 2001-11-30 | 2007-11-13 | Avigenics, Inc. | Ovomucoid promoters and methods of use |
| US6875588B2 (en) | 2001-11-30 | 2005-04-05 | Avigenics, Inc. | Ovomucoid promoter and methods of use |
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2012
- 2012-10-11 WO PCT/US2012/059708 patent/WO2013055888A2/en not_active Ceased
- 2012-10-11 US US13/649,595 patent/US9579366B2/en not_active Expired - Fee Related
- 2012-10-11 SG SG11201401457SA patent/SG11201401457SA/en unknown
- 2012-10-11 US US14/350,644 patent/US20140255383A1/en not_active Abandoned
- 2012-10-11 IN IN740MUN2014 patent/IN2014MN00740A/en unknown
- 2012-10-11 CN CN201280049876.4A patent/CN103958676A/zh active Pending
- 2012-10-11 AU AU2012322811A patent/AU2012322811A1/en not_active Abandoned
- 2012-10-11 MX MX2014004359A patent/MX2014004359A/es unknown
- 2012-10-11 RU RU2014117291/10A patent/RU2014117291A/ru not_active Application Discontinuation
- 2012-10-11 HK HK15100907.5A patent/HK1200489A1/xx unknown
- 2012-10-11 KR KR1020147012617A patent/KR20140108519A/ko not_active Ceased
- 2012-10-11 EP EP12840612.1A patent/EP2766477B1/en not_active Not-in-force
- 2012-10-11 SG SG10201510106PA patent/SG10201510106PA/en unknown
- 2012-10-11 BR BR112014008873-0A patent/BR112014008873A2/pt not_active Application Discontinuation
- 2012-10-11 JP JP2014535865A patent/JP6329483B2/ja not_active Expired - Fee Related
- 2012-10-11 CA CA2852027A patent/CA2852027A1/en not_active Abandoned
- 2012-10-12 TW TW101137865A patent/TW201321515A/zh unknown
-
2014
- 2014-04-10 IL IL232073A patent/IL232073A0/en unknown
- 2014-04-11 CL CL2014000927A patent/CL2014000927A1/es unknown
- 2014-05-08 CO CO14098853A patent/CO7010783A2/es unknown
-
2017
- 2017-02-10 US US15/429,309 patent/US20170216412A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| SG10201510106PA (en) | 2016-01-28 |
| WO2013055888A2 (en) | 2013-04-18 |
| US20170216412A1 (en) | 2017-08-03 |
| SG11201401457SA (en) | 2014-05-29 |
| EP2766477B1 (en) | 2018-09-26 |
| WO2013055888A3 (en) | 2013-06-13 |
| CA2852027A1 (en) | 2013-04-18 |
| IN2014MN00740A (enExample) | 2015-05-22 |
| US20140255383A1 (en) | 2014-09-11 |
| CO7010783A2 (es) | 2014-07-31 |
| CN103958676A (zh) | 2014-07-30 |
| EP2766477A4 (en) | 2015-07-15 |
| HK1200489A1 (en) | 2015-08-07 |
| NZ623690A (en) | 2016-08-26 |
| TW201321515A (zh) | 2013-06-01 |
| MX2014004359A (es) | 2014-05-28 |
| EP2766477A2 (en) | 2014-08-20 |
| US20130095092A1 (en) | 2013-04-18 |
| US9579366B2 (en) | 2017-02-28 |
| IL232073A0 (en) | 2014-05-28 |
| BR112014008873A2 (pt) | 2020-09-15 |
| AU2012322811A1 (en) | 2014-05-01 |
| JP6329483B2 (ja) | 2018-05-23 |
| JP2014534962A (ja) | 2014-12-25 |
| KR20140108519A (ko) | 2014-09-11 |
| CL2014000927A1 (es) | 2014-11-14 |
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| FA94 | Acknowledgement of application withdrawn (non-payment of fees) |
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