ATE333893T1 - Gentherapie für gaucher-krankheit - Google Patents
Gentherapie für gaucher-krankheitInfo
- Publication number
- ATE333893T1 ATE333893T1 AT98956290T AT98956290T ATE333893T1 AT E333893 T1 ATE333893 T1 AT E333893T1 AT 98956290 T AT98956290 T AT 98956290T AT 98956290 T AT98956290 T AT 98956290T AT E333893 T1 ATE333893 T1 AT E333893T1
- Authority
- AT
- Austria
- Prior art keywords
- gene therapy
- gaucher disease
- biologically active
- cells
- vector
- Prior art date
Links
- 208000015872 Gaucher disease Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 108090000790 Enzymes Proteins 0.000 abstract 3
- 102000004190 Enzymes Human genes 0.000 abstract 3
- 108700019146 Transgenes Proteins 0.000 abstract 2
- 230000002132 lysosomal effect Effects 0.000 abstract 2
- 230000002950 deficient Effects 0.000 abstract 1
Classifications
-
- 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
-
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- 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)
- C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- 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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US6352797P | 1997-10-29 | 1997-10-29 |
Publications (1)
Publication Number | Publication Date |
---|---|
ATE333893T1 true ATE333893T1 (de) | 2006-08-15 |
Family
ID=22049811
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
AT98956290T ATE333893T1 (de) | 1997-10-29 | 1998-10-29 | Gentherapie für gaucher-krankheit |
Country Status (10)
Country | Link |
---|---|
US (3) | US6066626A (de) |
EP (2) | EP1027069B1 (de) |
JP (2) | JP2002522509A (de) |
AT (1) | ATE333893T1 (de) |
AU (1) | AU734290B2 (de) |
CA (1) | CA2305768A1 (de) |
DE (1) | DE69835367T2 (de) |
ES (1) | ES2268799T3 (de) |
IL (3) | IL135578A0 (de) |
WO (1) | WO2000009153A1 (de) |
Families Citing this family (82)
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DE69835367T2 (de) * | 1997-10-29 | 2007-08-02 | Genzyme Corp., Framingham | Gentherapie für gaucher-krankheit |
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US20010036454A1 (en) * | 2000-02-17 | 2001-11-01 | Chester Li | Genetic modification of the lung as a portal for gene delivery |
AU2001236713A1 (en) * | 2000-02-17 | 2001-08-27 | Genzyme Corporation | Methods for treatment of lysosomal storage diseases |
US20040086420A1 (en) * | 2000-03-23 | 2004-05-06 | Macphee Martin J. | Methods for sterilizing serum or plasma |
US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
US20020095135A1 (en) | 2000-06-19 | 2002-07-18 | David Meeker | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
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 |
EP1368039A4 (de) * | 2001-02-20 | 2006-03-29 | Uab Research Foundation | Aminoglycosid-behandlung für lysosomale speicherkrankheiten |
US6682695B2 (en) * | 2001-03-23 | 2004-01-27 | Clearant, Inc. | Methods for sterilizing biological materials by multiple rates |
US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
US7629309B2 (en) * | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
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ATE384736T1 (de) * | 2001-04-30 | 2008-02-15 | Zystor Therapeutics Inc | Subzelluläres targeting von therapeutischen proteinen |
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US6696060B2 (en) * | 2001-06-14 | 2004-02-24 | Clearant, Inc. | Methods for sterilizing preparations of monoclonal immunoglobulins |
US6946098B2 (en) | 2001-08-10 | 2005-09-20 | Clearant, Inc. | Methods for sterilizing biological materials |
US20030031584A1 (en) * | 2001-08-10 | 2003-02-13 | Wilson Burgess | Methods for sterilizing biological materials using dipeptide stabilizers |
US7252799B2 (en) * | 2001-08-31 | 2007-08-07 | Clearant, Inc. | Methods for sterilizing preparations containing albumin |
US6749851B2 (en) | 2001-08-31 | 2004-06-15 | Clearant, Inc. | Methods for sterilizing preparations of digestive enzymes |
US20030095890A1 (en) * | 2001-09-24 | 2003-05-22 | Shirley Miekka | Methods for sterilizing biological materials containing non-aqueous solvents |
US6783968B2 (en) | 2001-09-24 | 2004-08-31 | Clearant, Inc. | Methods for sterilizing preparations of glycosidases |
US20030185702A1 (en) * | 2002-02-01 | 2003-10-02 | Wilson Burgess | Methods for sterilizing tissue |
US20110091353A1 (en) * | 2001-09-24 | 2011-04-21 | Wilson Burgess | Methods for Sterilizing Tissue |
US20030072761A1 (en) * | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
US20030124023A1 (en) * | 2001-12-21 | 2003-07-03 | Wilson Burgess | Method of sterilizing heart valves |
US20030180181A1 (en) * | 2002-02-01 | 2003-09-25 | Teri Greib | Methods for sterilizing tissue |
US7090836B2 (en) * | 2002-06-21 | 2006-08-15 | Institut Pasteur | Vector for expressing α-L-iduronidase and method of treating MPS I by stereotactic injection into the brain of a mammal |
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PT2444102E (pt) * | 2003-01-31 | 2015-09-17 | Sinai School Medicine | Terapia combinada para o tratamento de distúrbios de deficiência proteica |
US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
US20040258666A1 (en) * | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
WO2005046612A2 (en) * | 2003-11-12 | 2005-05-26 | Amicus Therapeutics Inc. | Hydroxy piperidine derivatives to treat gaucher disease |
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WO2006007560A2 (en) | 2004-07-01 | 2006-01-19 | University Of Pennsylvania | Targeted protein replacement for the treatment of lysosomal storage disorders |
US7935336B2 (en) | 2005-11-18 | 2011-05-03 | Tokyo Metropolitan Organization For Medical Research | Highly functional enzyme having α-galactosidase activity |
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CN101631794B (zh) | 2007-01-18 | 2013-01-02 | 建新公司 | 包含氨基氧基基团的寡糖及其轭合物 |
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EP2675472A4 (de) | 2011-02-15 | 2014-09-17 | Synageva Biopharma Corp | Verfahren zur behandlung von mangel an lysosomaler saurer lipase |
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RU2013151875A (ru) | 2011-04-22 | 2015-05-27 | Джензим Корпорейшн | Модифицированная кислая альфа глюкозидаза с ускоренным процессингом |
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EP3679923B1 (de) | 2012-06-01 | 2021-08-04 | Icahn School of Medicine at Mount Sinai | Ceramidspiegel bei der behandlung und vorbeugung von infektionen |
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US20150366997A1 (en) | 2012-12-07 | 2015-12-24 | Shire Human Genetics Therapies, Inc. | COMPOSITIONS AND METHODS FOR mRNA DELIVERY |
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TW202332774A (zh) | 2013-10-23 | 2023-08-16 | 美商健臻公司 | 重組醣蛋白及其用途 |
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US20220001027A1 (en) * | 2018-11-15 | 2022-01-06 | President And Fellows Of Harvard College | Genetically Engineered Skin Cells for the Systemic In Vivo Treatment of Deficient Enzymes, Factors or Proteins |
EP3972653A1 (de) | 2019-05-22 | 2022-03-30 | Massachusetts Institute of Technology | Zirkuläre rns-zusammensetzungen und verfahren |
CN114786710A (zh) * | 2019-10-17 | 2022-07-22 | 西湖生物医药科技(杭州)有限公司 | 工程改造红细胞用于治疗溶酶体贮积病 |
EP3920976B1 (de) | 2019-12-04 | 2023-07-19 | Orna Therapeutics, Inc. | Zirkuläre rns-zusammensetzungen und verfahren |
CN116034114A (zh) | 2020-03-20 | 2023-04-28 | 奥纳治疗公司 | 环状rna组合物和方法 |
CN116322788A (zh) | 2020-05-19 | 2023-06-23 | 奥纳治疗公司 | 环状rna组合物和方法 |
TW202220648A (zh) | 2020-07-24 | 2022-06-01 | 美商健臻公司 | 包含芬谷司他(venglustat)之醫藥組合物 |
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US20240052049A1 (en) | 2022-06-24 | 2024-02-15 | Orna Therapeutics, Inc. | Circular rna encoding chimeric antigen receptors targeting bcma |
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-
1998
- 1998-10-29 DE DE69835367T patent/DE69835367T2/de not_active Expired - Lifetime
- 1998-10-29 US US09/182,245 patent/US6066626A/en not_active Expired - Fee Related
- 1998-10-29 AU AU12847/99A patent/AU734290B2/en not_active Ceased
- 1998-10-29 IL IL13557898A patent/IL135578A0/xx active IP Right Review Request
- 1998-10-29 WO PCT/US1998/022886 patent/WO2000009153A1/en active Application Filing
- 1998-10-29 JP JP2000564655A patent/JP2002522509A/ja not_active Withdrawn
- 1998-10-29 CA CA002305768A patent/CA2305768A1/en not_active Abandoned
- 1998-10-29 EP EP98956290A patent/EP1027069B1/de not_active Expired - Lifetime
- 1998-10-29 AT AT98956290T patent/ATE333893T1/de not_active IP Right Cessation
- 1998-10-29 ES ES98956290T patent/ES2268799T3/es not_active Expired - Lifetime
- 1998-10-29 EP EP09175718A patent/EP2147681A1/de not_active Withdrawn
-
2000
- 2000-04-10 IL IL135578A patent/IL135578A/en not_active IP Right Cessation
-
2002
- 2002-09-13 US US10/244,700 patent/US20030087868A1/en not_active Abandoned
-
2006
- 2006-08-02 IL IL177228A patent/IL177228A0/en unknown
-
2008
- 2008-10-14 US US12/251,451 patent/US20100041151A1/en not_active Abandoned
-
2009
- 2009-09-28 JP JP2009222892A patent/JP2010031023A/ja not_active Withdrawn
Also Published As
Publication number | Publication date |
---|---|
DE69835367D1 (de) | 2006-09-07 |
IL135578A0 (en) | 2001-05-20 |
CA2305768A1 (en) | 2000-02-24 |
DE69835367T2 (de) | 2007-08-02 |
EP1027069A1 (de) | 2000-08-16 |
IL135578A (en) | 2007-03-08 |
IL177228A0 (en) | 2006-12-10 |
US20100041151A1 (en) | 2010-02-18 |
AU734290B2 (en) | 2001-06-07 |
US20030087868A1 (en) | 2003-05-08 |
AU1284799A (en) | 2000-03-06 |
EP1027069B1 (de) | 2006-07-26 |
JP2002522509A (ja) | 2002-07-23 |
US6066626A (en) | 2000-05-23 |
ES2268799T3 (es) | 2007-03-16 |
EP2147681A1 (de) | 2010-01-27 |
JP2010031023A (ja) | 2010-02-12 |
WO2000009153A1 (en) | 2000-02-24 |
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