CL2013003010A1 - “polipéptido que comprende una alfa glucosidasa acida humana o un fragmento cataliticamente activo de la misma que tiene una modificación en o cerca de un sitio de procesamiento de 70 kda n-terminal; ácido nucleico que lo codifica; célula húesped; composición farmacéutica; y su uso para reducir o prevenir la acumulación de glucógeno". - Google Patents
“polipéptido que comprende una alfa glucosidasa acida humana o un fragmento cataliticamente activo de la misma que tiene una modificación en o cerca de un sitio de procesamiento de 70 kda n-terminal; ácido nucleico que lo codifica; célula húesped; composición farmacéutica; y su uso para reducir o prevenir la acumulación de glucógeno".Info
- Publication number
- CL2013003010A1 CL2013003010A1 CL2013003010A CL2013003010A CL2013003010A1 CL 2013003010 A1 CL2013003010 A1 CL 2013003010A1 CL 2013003010 A CL2013003010 A CL 2013003010A CL 2013003010 A CL2013003010 A CL 2013003010A CL 2013003010 A1 CL2013003010 A1 CL 2013003010A1
- Authority
- CL
- Chile
- Prior art keywords
- polypeptide
- modification
- pharmaceutical composition
- terminal
- host cell
- Prior art date
Links
- 229920002527 Glycogen Polymers 0.000 title 1
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 title 1
- 238000009825 accumulation Methods 0.000 title 1
- 239000012634 fragment Substances 0.000 title 1
- 229940096919 glycogen Drugs 0.000 title 1
- 102000045921 human GAA Human genes 0.000 title 1
- 238000012986 modification Methods 0.000 title 1
- 230000004048 modification Effects 0.000 title 1
- 102000039446 nucleic acids Human genes 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 title 1
- 150000007523 nucleic acids Chemical class 0.000 title 1
- 239000008194 pharmaceutical composition Substances 0.000 title 1
- 229920001184 polypeptide Polymers 0.000 title 1
- 102000004196 processed proteins & peptides Human genes 0.000 title 1
- 108090000765 processed proteins & peptides Proteins 0.000 title 1
Classifications
-
- 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
-
- 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
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- 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)
-
- 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)
-
- 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
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
- C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Diabetes (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Endocrinology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Emergency Medicine (AREA)
- Epidemiology (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161478336P | 2011-04-22 | 2011-04-22 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2013003010A1 true CL2013003010A1 (es) | 2014-03-07 |
Family
ID=46000406
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2013003010A CL2013003010A1 (es) | 2011-04-22 | 2013-10-17 | “polipéptido que comprende una alfa glucosidasa acida humana o un fragmento cataliticamente activo de la misma que tiene una modificación en o cerca de un sitio de procesamiento de 70 kda n-terminal; ácido nucleico que lo codifica; célula húesped; composición farmacéutica; y su uso para reducir o prevenir la acumulación de glucógeno". |
Country Status (24)
| Country | Link |
|---|---|
| US (1) | US20140186326A1 (OSRAM) |
| EP (1) | EP2699676A1 (OSRAM) |
| JP (2) | JP2014513952A (OSRAM) |
| KR (1) | KR20140037082A (OSRAM) |
| CN (1) | CN103797115A (OSRAM) |
| AU (1) | AU2012245280A1 (OSRAM) |
| BR (1) | BR112013026976A2 (OSRAM) |
| CA (1) | CA2833371A1 (OSRAM) |
| CL (1) | CL2013003010A1 (OSRAM) |
| CO (1) | CO6811810A2 (OSRAM) |
| CR (1) | CR20130555A (OSRAM) |
| EC (1) | ECSP13013036A (OSRAM) |
| GT (1) | GT201300252A (OSRAM) |
| IL (1) | IL228871A0 (OSRAM) |
| MA (1) | MA35125B1 (OSRAM) |
| MX (1) | MX2013012345A (OSRAM) |
| NI (1) | NI201300110A (OSRAM) |
| PE (1) | PE20140617A1 (OSRAM) |
| PH (1) | PH12013502149A1 (OSRAM) |
| RU (1) | RU2013151875A (OSRAM) |
| SG (2) | SG194486A1 (OSRAM) |
| TN (1) | TN2013000427A1 (OSRAM) |
| WO (1) | WO2012145644A1 (OSRAM) |
| ZA (1) | ZA201307696B (OSRAM) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014130723A1 (en) | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Methods and compositions for treatment of pompe disease |
| DK3201320T5 (da) | 2014-09-30 | 2024-10-14 | Amicus Therapeutics Inc | Højpotent sur alfa-glucosidase med forbedrede carbohydrater |
| JP2018538008A (ja) * | 2015-11-06 | 2018-12-27 | 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 | リソソーム酵素の取り込みを中和する抗体または他の因子の検出のための細胞ベースアッセイ |
| MX394968B (es) | 2015-12-30 | 2025-03-24 | Amicus Therapeutics Inc | Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe. |
| CA3019128A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| KR102343162B1 (ko) * | 2016-03-30 | 2021-12-23 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 방법 |
| EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293260A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| MX2019002842A (es) | 2016-09-12 | 2019-08-29 | Genethon | Variantes de alfa-glucosidasa acida y usos de las mismas. |
| EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| SMT202300187T1 (it) | 2017-05-15 | 2023-07-20 | Amicus Therapeutics Inc | Alfa-glucosidasi acida umana ricombinante |
| CN110809583A (zh) | 2017-06-07 | 2020-02-18 | 瑞泽恩制药公司 | 用于内化酶的组合物和方法 |
| KR101942093B1 (ko) * | 2018-01-05 | 2019-01-24 | 인하대학교 산학협력단 | 만노시다제 억제제를 포함하는 고만노즈 타입 n-당쇄를 갖는 인간 리소좀 효소 생산용 조성물 |
| KR20200118151A (ko) | 2018-02-07 | 2020-10-14 | 리제너론 파마슈티칼스 인코포레이티드 | 치료 단백질 전달을 위한 방법 및 조성물 |
| MX2020012350A (es) | 2018-05-17 | 2021-01-29 | Regeneron Pharma | Anticuerpos anti-cd63, conjugados y usos de estos. |
| US20220331408A1 (en) | 2019-07-09 | 2022-10-20 | Genethon | Treatment of glycogen storage disease (gsd) |
| AU2020388634A1 (en) * | 2019-11-19 | 2022-06-23 | AskBio Inc. | Therapeutic adeno-associated virus comprising liver-specific promoters for treating Pompe disease and lysosomal disorders |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6071890A (en) | 1994-12-09 | 2000-06-06 | Genzyme Corporation | Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy |
| US5952516A (en) | 1997-05-08 | 1999-09-14 | Genzyme Corporation | Cationic amphiphiles containing multiplesteroid lipophilic groups |
| EP1027069B1 (en) | 1997-10-29 | 2006-07-26 | Genzyme Corporation | Gene therapy for gaucher disease |
| US6287857B1 (en) | 1998-02-09 | 2001-09-11 | Genzyme Corporation | Nucleic acid delivery vehicles |
| GB9807464D0 (en) * | 1998-04-07 | 1998-06-10 | Pharming Bv | Purification of human acid µ-glucosidase |
| WO2000034451A1 (en) * | 1998-12-07 | 2000-06-15 | Pharming Intellectual Property B.V. | Treatment of pompe's disease |
| US7138262B1 (en) | 2000-08-18 | 2006-11-21 | Shire Human Genetic Therapies, Inc. | High mannose proteins and methods of making high mannose proteins |
| US7723296B2 (en) | 2001-01-18 | 2010-05-25 | Genzyme Corporation | Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof |
| US7001994B2 (en) | 2001-01-18 | 2006-02-21 | Genzyme Corporation | Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins |
| JP4742191B2 (ja) * | 2001-06-14 | 2011-08-10 | 独立行政法人産業技術総合研究所 | 糖蛋白質およびその製造方法 |
| BR122015016313B8 (pt) | 2001-07-16 | 2021-05-25 | Genzyme Corp | composto semelhante à ceramida e método de preparar um composto |
| US6835831B2 (en) | 2001-11-26 | 2004-12-28 | Genzyme Corporation | Diastereoselective synthesis of UDP-glucose: N-acylsphingosine glucosyltransferase inhibitors |
| US6916802B2 (en) | 2002-04-29 | 2005-07-12 | Genzyme Corporation | Amino ceramide-like compounds and therapeutic methods of use |
| US20040248262A1 (en) * | 2003-01-22 | 2004-12-09 | Koeberl Dwight D. | Constructs for expressing lysomal polypeptides |
| US20100196345A1 (en) | 2003-04-27 | 2010-08-05 | Protalix | Production of high mannose proteins in plant culture |
| CN101942442B (zh) | 2003-06-24 | 2013-10-09 | 建新公司 | 新的β-肌动蛋白和RPS21启动子及其应用 |
| ES2344302T3 (es) * | 2004-02-10 | 2010-08-24 | Zystor Therapeutics , Inc. | Alfa glucosidasa acida y fragmentos de la misma. |
| JP2010509344A (ja) * | 2006-11-13 | 2010-03-25 | ザイストール セラピューティクス, インコーポレイテッド | ポンペ病を治療するための方法 |
| BR122020010601B8 (pt) | 2008-12-16 | 2021-07-27 | Genzyme Corp | conjugados de proteína-oligossacarídeo, seus usos, e composições farmacêuticas |
| US8466340B2 (en) * | 2009-02-18 | 2013-06-18 | Amicus Therapeutics, Inc. | Mouse model for pompe disease and methods of use thereof |
-
2012
- 2012-04-20 PE PE2013002377A patent/PE20140617A1/es not_active Application Discontinuation
- 2012-04-20 RU RU2013151875/10A patent/RU2013151875A/ru not_active Application Discontinuation
- 2012-04-20 KR KR1020137029936A patent/KR20140037082A/ko not_active Withdrawn
- 2012-04-20 AU AU2012245280A patent/AU2012245280A1/en not_active Abandoned
- 2012-04-20 WO PCT/US2012/034479 patent/WO2012145644A1/en not_active Ceased
- 2012-04-20 JP JP2014506579A patent/JP2014513952A/ja active Pending
- 2012-04-20 PH PH1/2013/502149A patent/PH12013502149A1/en unknown
- 2012-04-20 SG SG2013076526A patent/SG194486A1/en unknown
- 2012-04-20 US US14/113,360 patent/US20140186326A1/en not_active Abandoned
- 2012-04-20 EP EP12717025.6A patent/EP2699676A1/en not_active Withdrawn
- 2012-04-20 CN CN201280030662.2A patent/CN103797115A/zh active Pending
- 2012-04-20 SG SG10201605874TA patent/SG10201605874TA/en unknown
- 2012-04-20 BR BR112013026976A patent/BR112013026976A2/pt not_active IP Right Cessation
- 2012-04-20 MX MX2013012345A patent/MX2013012345A/es not_active Application Discontinuation
- 2012-04-20 CA CA2833371A patent/CA2833371A1/en not_active Abandoned
-
2013
- 2013-10-14 IL IL228871A patent/IL228871A0/en unknown
- 2013-10-16 ZA ZA2013/07696A patent/ZA201307696B/en unknown
- 2013-10-16 NI NI201300110A patent/NI201300110A/es unknown
- 2013-10-17 CL CL2013003010A patent/CL2013003010A1/es unknown
- 2013-10-18 GT GT201300252A patent/GT201300252A/es unknown
- 2013-10-18 TN TNP2013000427A patent/TN2013000427A1/fr unknown
- 2013-10-29 CR CR20130555A patent/CR20130555A/es unknown
- 2013-11-14 MA MA36431A patent/MA35125B1/fr unknown
- 2013-11-20 CO CO13272849A patent/CO6811810A2/es not_active Application Discontinuation
- 2013-11-21 EC ECSP13013036 patent/ECSP13013036A/es unknown
-
2016
- 2016-09-23 JP JP2016185075A patent/JP2017035091A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2012245280A1 (en) | 2013-11-07 |
| WO2012145644A1 (en) | 2012-10-26 |
| CA2833371A1 (en) | 2012-10-26 |
| MA35125B1 (fr) | 2014-05-02 |
| PE20140617A1 (es) | 2014-05-28 |
| CR20130555A (es) | 2013-12-09 |
| US20140186326A1 (en) | 2014-07-03 |
| NI201300110A (es) | 2014-02-28 |
| SG10201605874TA (en) | 2016-09-29 |
| KR20140037082A (ko) | 2014-03-26 |
| IL228871A0 (en) | 2013-12-31 |
| MX2013012345A (es) | 2015-05-07 |
| RU2013151875A (ru) | 2015-05-27 |
| GT201300252A (es) | 2015-02-09 |
| TN2013000427A1 (en) | 2015-03-30 |
| PH12013502149A1 (en) | 2019-09-02 |
| BR112013026976A2 (pt) | 2019-09-24 |
| CN103797115A (zh) | 2014-05-14 |
| EP2699676A1 (en) | 2014-02-26 |
| SG194486A1 (en) | 2013-12-30 |
| CO6811810A2 (es) | 2013-12-16 |
| ECSP13013036A (es) | 2015-04-30 |
| JP2014513952A (ja) | 2014-06-19 |
| ZA201307696B (en) | 2014-07-30 |
| JP2017035091A (ja) | 2017-02-16 |
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