MX2012012356A - Enzima de enfermedad del almacenamiento lisosomico. - Google Patents
Enzima de enfermedad del almacenamiento lisosomico.Info
- Publication number
- MX2012012356A MX2012012356A MX2012012356A MX2012012356A MX2012012356A MX 2012012356 A MX2012012356 A MX 2012012356A MX 2012012356 A MX2012012356 A MX 2012012356A MX 2012012356 A MX2012012356 A MX 2012012356A MX 2012012356 A MX2012012356 A MX 2012012356A
- Authority
- MX
- Mexico
- Prior art keywords
- acid lipase
- lysosomal acid
- lysosomal storage
- storage disease
- human lysosomal
- Prior art date
Links
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
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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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
- C12N9/20—Triglyceride splitting, e.g. by means of lipase
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0278—Humanized animals, e.g. knockin
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- 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
- A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
- A61K31/19—Carboxylic acids, e.g. valproic acid
- A61K31/194—Carboxylic acids, e.g. valproic acid having two or more carboxyl groups, e.g. succinic, maleic or phthalic acid
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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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/38—Albumins
- A61K38/385—Serum albumin
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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)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- 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
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- 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
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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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
- A61P35/04—Antineoplastic agents specific for metastasis
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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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)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01013—Sterol esterase (3.1.1.13)
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
- A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/30—Bird
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/01—Animal expressing industrially exogenous proteins
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/10041—Use of virus, viral particle or viral elements as a vector
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/11011—Alpharetrovirus, e.g. avian leucosis virus
- C12N2740/11041—Use of virus, viral particle or viral elements as a vector
- C12N2740/11043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/11011—Alpharetrovirus, e.g. avian leucosis virus
- C12N2740/11041—Use of virus, viral particle or viral elements as a vector
- C12N2740/11045—Special targeting system for viral vectors
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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
- C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
- C12N2760/00011—Details
- C12N2760/20011—Rhabdoviridae
- C12N2760/20211—Vesiculovirus, e.g. vesicular stomatitis Indiana virus
- C12N2760/20222—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
Abstract
La presente invención proporciona composiciones de lipasa ácida lisosómica humana recombinante que tiene patrones de glicosilación particulares para internalización en células objetivo, un vector que contiene el ácido nucleico que codifica la lipasa ácida lisosomal humana, una célula huésped transformada con el vector, composiciones farmacéuticas que comprenden la lipasa ácida lisosomal humana recombinante y método para tratar condiciones asociadas con la deficiencia de lipasa ácida lisosomal.
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US34317710P | 2010-04-23 | 2010-04-23 | |
US39637610P | 2010-05-26 | 2010-05-26 | |
US40301110P | 2010-09-09 | 2010-09-09 | |
US45601410P | 2010-10-29 | 2010-10-29 | |
US201161432372P | 2011-01-13 | 2011-01-13 | |
PCT/US2011/033699 WO2011133960A2 (en) | 2010-04-23 | 2011-04-23 | Lysosomal storage disease enzyme |
Publications (2)
Publication Number | Publication Date |
---|---|
MX2012012356A true MX2012012356A (es) | 2013-04-11 |
MX338426B MX338426B (es) | 2016-04-15 |
Family
ID=44834852
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2016004772A MX362430B (es) | 2010-04-23 | 2011-04-23 | Enzima de enfermedad del almacenamiento lisosomico. |
MX2012012356A MX338426B (es) | 2010-04-23 | 2011-04-23 | Enzima de enfermedad del almacenamiento lisosomico. |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2016004772A MX362430B (es) | 2010-04-23 | 2011-04-23 | Enzima de enfermedad del almacenamiento lisosomico. |
Country Status (21)
Country | Link |
---|---|
US (4) | US20130209436A1 (es) |
EP (3) | EP3205351B1 (es) |
JP (4) | JP5925187B2 (es) |
KR (1) | KR101742346B1 (es) |
CN (2) | CN105214076B (es) |
AU (1) | AU2011242461B2 (es) |
BR (1) | BR112012027143B1 (es) |
CA (2) | CA2995446C (es) |
DK (2) | DK2561069T3 (es) |
ES (2) | ES2627535T3 (es) |
FI (1) | FI3205351T3 (es) |
HK (2) | HK1183059A1 (es) |
HU (2) | HUE033217T2 (es) |
IL (2) | IL222513A0 (es) |
LT (1) | LT3205351T (es) |
MX (2) | MX362430B (es) |
PL (2) | PL3205351T3 (es) |
PT (2) | PT2561069T (es) |
RU (2) | RU2015151189A (es) |
SI (2) | SI2561069T1 (es) |
WO (1) | WO2011133960A2 (es) |
Families Citing this family (15)
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ES2328446T5 (es) | 2000-02-04 | 2014-02-27 | Children's Hospital Research Foundation | Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas |
US20080064862A1 (en) * | 2004-12-29 | 2008-03-13 | Avigenics, Inc. | Transgene expression in a avians |
US20090297496A1 (en) * | 2005-09-08 | 2009-12-03 | Childrens Hospital Medical Center | Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases |
EP3205351B1 (en) | 2010-04-23 | 2023-04-12 | Alexion Pharmaceuticals, Inc. | Lysosomal storage disease enzyme |
MX365007B (es) | 2010-09-09 | 2019-05-20 | Alexion Pharmaceuticals Inc Star | Uso de acido lipasa lisosomal para tratar la dificiencia de acido lipasa lisosomal en pacientes. |
KR20140006037A (ko) | 2011-02-15 | 2014-01-15 | 시나게바 바이오파르마, 코포레이션 | 리소좀의 산 리파제 결핍을 치료하는 방법들 |
KR20140130443A (ko) * | 2012-03-02 | 2014-11-10 | 시나게바 바이오파르마, 코포레이션 | 절두된 리소좀 산 리파제 |
EP3133932B1 (en) * | 2014-04-23 | 2021-08-11 | Alexion Pharmaceuticals, Inc. | Egg white processing |
US11235036B2 (en) | 2016-06-17 | 2022-02-01 | Alexion Pharmaceuticals, Inc. | Lysosomal acid lipase deficiency compositions and methods |
WO2018039163A1 (en) | 2016-08-23 | 2018-03-01 | Alexion Pharmaceuticals, Inc. | Method of purifying a heterologous protein from an egg white |
WO2018064303A1 (en) | 2016-09-30 | 2018-04-05 | Alexion Pharmaceuticals, Inc. | Methods for reducing liver fibrosis and treating lysosomal acid lipase deficiency in patients based on ishak fibrosis stage |
US10633705B2 (en) | 2016-10-14 | 2020-04-28 | Alexion Pharmaceuticals, Inc. | N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods |
WO2019152343A1 (en) | 2018-02-01 | 2019-08-08 | Alexion Pharmaceuticals, Inc. | Methods of desensitizing against infusion associated reactions in lysosomal acid lipase deficiency (lal-d) patients treated with exogenous lysosomal acid lipase (lal |
KR20220069917A (ko) * | 2019-07-02 | 2022-05-27 | 엠6피 테라퓨틱스 (스위처랜드) 엘엘씨 | 리소좀 축적 장애의 치료를 위한 벡터 조성물 및 이의 사용 방법 |
CN114369620A (zh) * | 2021-12-31 | 2022-04-19 | 广州辉园苑医药科技有限公司 | 一种可持续分泌lal的载体、脐带间充质干细胞及其构建方法和应用 |
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