PL2666476T3 - Dokomorowe dostarczanie enzymu dla lizosomalnych chorób spichrzeniowych - Google Patents
Dokomorowe dostarczanie enzymu dla lizosomalnych chorób spichrzeniowychInfo
- Publication number
- PL2666476T3 PL2666476T3 PL13173895T PL13173895T PL2666476T3 PL 2666476 T3 PL2666476 T3 PL 2666476T3 PL 13173895 T PL13173895 T PL 13173895T PL 13173895 T PL13173895 T PL 13173895T PL 2666476 T3 PL2666476 T3 PL 2666476T3
- Authority
- PL
- Poland
- Prior art keywords
- lysosomal storage
- storage diseases
- enzyme delivery
- intraventricular
- intraventricular enzyme
- Prior art date
Links
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)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- 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
- 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
- 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
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
- C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)
-
- 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)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
-
- 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)
-
- 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)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
-
- 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)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Dermatology (AREA)
- Obesity (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Urology & Nephrology (AREA)
- Pulmonology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Infusion, Injection, And Reservoir Apparatuses (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US76037806P | 2006-01-20 | 2006-01-20 | |
PCT/US2007/001566 WO2007084737A2 (en) | 2006-01-20 | 2007-01-22 | Intraventricular enzyme delivery for lysosomal storage diseases |
EP13173895.7A EP2666476B1 (en) | 2006-01-20 | 2007-01-22 | Intraventricular enzyme delivery for lysosomal storage diseases |
EP07716858.1A EP1984018B1 (en) | 2006-01-20 | 2007-01-22 | Intraventricular enzyme delivery for lysosomal storage diseases |
Publications (1)
Publication Number | Publication Date |
---|---|
PL2666476T3 true PL2666476T3 (pl) | 2019-06-28 |
Family
ID=38288294
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL13173895T PL2666476T3 (pl) | 2006-01-20 | 2007-01-22 | Dokomorowe dostarczanie enzymu dla lizosomalnych chorób spichrzeniowych |
PL07716858T PL1984018T3 (pl) | 2006-01-20 | 2007-01-22 | Dokomorowe dostarczanie enzymu w lizosomalnych chorobach spichrzeniowych |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL07716858T PL1984018T3 (pl) | 2006-01-20 | 2007-01-22 | Dokomorowe dostarczanie enzymu w lizosomalnych chorobach spichrzeniowych |
Country Status (19)
Country | Link |
---|---|
US (4) | US8926967B2 (pl) |
EP (3) | EP2666476B1 (pl) |
JP (1) | JP5681345B2 (pl) |
CN (1) | CN101431960A (pl) |
AR (2) | AR059089A1 (pl) |
BR (1) | BRPI0706699A2 (pl) |
CA (2) | CA2966155A1 (pl) |
DK (2) | DK2666476T3 (pl) |
ES (2) | ES2526710T3 (pl) |
HU (1) | HUE041840T2 (pl) |
IL (2) | IL192740A (pl) |
LT (1) | LT2666476T (pl) |
MX (1) | MX349749B (pl) |
PL (2) | PL2666476T3 (pl) |
PT (2) | PT1984018E (pl) |
RU (3) | RU2529830C2 (pl) |
SI (2) | SI2666476T1 (pl) |
TR (1) | TR201903855T4 (pl) |
WO (1) | WO2007084737A2 (pl) |
Families Citing this family (34)
Publication number | Priority date | Publication date | Assignee | Title |
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AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
LT1988823T (lt) | 2006-02-09 | 2018-12-10 | Genzyme Corporation | Lėtas intraventrikuliarinis pateikimas |
CA2684946C (en) | 2007-05-16 | 2015-03-31 | The Brigham And Women's Hospital, Inc. | Treatment of synucleinopathies |
CN105879047A (zh) * | 2008-12-16 | 2016-08-24 | 建新公司 | 寡糖-蛋白缀合物 |
LT3421603T (lt) | 2009-05-02 | 2022-01-10 | Genzyme Corporation | Genų terapija, skirta neurodegeneraciniams sutrikimams |
IL300625A (en) * | 2009-08-28 | 2023-04-01 | Icahn School Med Mount Sinai | Increasing dose replacement enzyme for the treatment of acid sphingomyelinase deficiency |
SI3205351T1 (sl) | 2010-04-23 | 2023-09-29 | Alexion Pharmaceuticals, Inc., | Encim za bolezen lizosomskega shranjevanja |
ES2754234T3 (es) | 2010-06-25 | 2020-04-16 | Shire Human Genetic Therapies | Métodos y composiciones para la administración SNC de iduronato-2-sulfatasa |
DK2588130T3 (en) * | 2010-06-25 | 2016-10-24 | Shire Human Genetic Therapies | Cns delivery of therapeutic agents |
US20110318324A1 (en) * | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of b-galactocerebrosidase |
RU2012154576A (ru) * | 2010-06-25 | 2014-07-27 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
LT2585104T (lt) * | 2010-06-25 | 2019-10-25 | Shire Human Genetic Therapies | Būdai ir kompozicijos, skirti arilsulfatazės a pristatymui į cns |
LT3626258T (lt) | 2010-06-25 | 2021-12-10 | Shire Human Genetic Therapies, Inc. | Būdai ir kompozicijos, skirti iduronato-2-sulfatazės įvedimui į cns |
JP2012062312A (ja) * | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
SG188456A1 (en) * | 2010-09-09 | 2013-04-30 | Synageva Biopharma Corp | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
US9387236B2 (en) * | 2011-06-10 | 2016-07-12 | Prothera Inc. | Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases |
WO2013096912A2 (en) * | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
EA039246B1 (ru) * | 2012-12-06 | 2021-12-22 | Шир Хьюман Дженетик Терапис, Инк. | Способ лечения когнитивных нарушений, связанных с синдромом хантера |
CN105189542A (zh) | 2013-02-20 | 2015-12-23 | 瓦莱里昂治疗有限责任公司 | 用于治疗庞贝氏症的方法和组合物 |
SG10201709925SA (en) | 2013-06-07 | 2017-12-28 | Genzyme Corp | Marker for acid sphingomyelinase disorders and uses thereof |
JP6616189B2 (ja) | 2013-12-25 | 2019-12-04 | Jcrファーマ株式会社 | 血液脳関門を通過する新規抗トランスフェリン受容体抗体 |
WO2015119989A1 (en) * | 2014-02-04 | 2015-08-13 | New York University | Progranulin (pgrn) and its derivatives for diagnosis and treatment of lysosomal storage diseases |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
AU2016283343B2 (en) | 2015-06-24 | 2022-05-19 | Jcr Pharmaceuticals Co., Ltd. | Anti-human transferrin receptor antibody permeating blood-brain barrier |
CA2990565A1 (en) | 2015-06-24 | 2016-12-29 | Jcr Pharmaceuticals Co., Ltd. | Fusion protein containing bdnf and anti-human transferrin receptor antibody |
US11390656B2 (en) * | 2015-08-04 | 2022-07-19 | New York University | Progranulin (PGRN) fragments and derivatives for treatment or alleviation of lysosomal storage diseases |
RU2634113C1 (ru) * | 2016-11-18 | 2017-10-24 | Государственное бюджетное учреждение "Академия наук Республики Саха (Якутия)" (ГБУ АН РС(Я)) | Способ фиксации головного мозга ископаемого позднеплейстоценового млекопитающего |
MX2019007719A (es) | 2016-12-26 | 2019-09-09 | Japan Chem Res | Anticuerpo anti-receptor de transferrina humana novedoso capaz de penetrar la barrera hematoencefalica. |
EP3561058A4 (en) | 2016-12-26 | 2020-07-22 | JCR Pharmaceuticals Co., Ltd. | FUSION PROTEIN INCLUDING BDNF |
JP2021500857A (ja) | 2017-10-02 | 2021-01-14 | デナリ セラピューティクス インコーポレイテッドDenali Therapeutics Inc. | 酵素補充療法用酵素を含む融合タンパク質 |
UY38238A (es) * | 2018-05-25 | 2019-12-31 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
AU2020215263A1 (en) | 2019-02-01 | 2021-09-09 | Oxyrane Uk Ltd | Glucocerebrosidase polypeptides |
RU2723187C1 (ru) * | 2019-10-04 | 2020-06-09 | федеральное государственное автономное образовательное учреждение высшего образования "Казанский (Приволжский) федеральный университет" (ФГАОУ ВО КФУ) | Генетическая кассета, содержащая кодон-оптимизированные нуклеотидные последовательности генов HEXA и HEXВ, и фармацевтическая композиция для лечения болезни Тея-Сакса |
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SE8303626D0 (sv) | 1983-06-23 | 1983-06-23 | Kabigen Ab | A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced |
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DK1445322T4 (da) | 1995-06-15 | 2012-09-03 | Crucell Holland Bv | Pakningssystemer for humant rekombinant adenovirus til anvendelse ved genterapi |
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US6689756B2 (en) | 2001-03-02 | 2004-02-10 | Integra Lifesciences Corporation | Treatment of neurological disease |
US20030163181A1 (en) | 2001-12-07 | 2003-08-28 | Neuron Therapeutics, Inc. | Protection of neurological tissue by direct CNS perfusion cooling |
WO2004047768A2 (en) | 2002-11-26 | 2004-06-10 | Seacoast Neuroscience, Inc. | Buoyant polymer particles delivering therapeutic agents |
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AR059088A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una proteina para esclerosis lateral amiotrofica |
AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
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-
2007
- 2007-01-19 AR ARP070100239A patent/AR059089A1/es not_active Application Discontinuation
- 2007-01-22 PL PL13173895T patent/PL2666476T3/pl unknown
- 2007-01-22 CA CA2966155A patent/CA2966155A1/en not_active Abandoned
- 2007-01-22 EP EP13173895.7A patent/EP2666476B1/en active Active
- 2007-01-22 BR BRPI0706699-6A patent/BRPI0706699A2/pt not_active Application Discontinuation
- 2007-01-22 EP EP18212320.8A patent/EP3517124A1/en not_active Withdrawn
- 2007-01-22 CA CA2636991A patent/CA2636991C/en not_active Expired - Fee Related
- 2007-01-22 EP EP07716858.1A patent/EP1984018B1/en active Active
- 2007-01-22 DK DK13173895.7T patent/DK2666476T3/en active
- 2007-01-22 TR TR2019/03855T patent/TR201903855T4/tr unknown
- 2007-01-22 ES ES07716858.1T patent/ES2526710T3/es active Active
- 2007-01-22 MX MX2013012372A patent/MX349749B/es unknown
- 2007-01-22 PT PT77168581T patent/PT1984018E/pt unknown
- 2007-01-22 SI SI200732098T patent/SI2666476T1/sl unknown
- 2007-01-22 WO PCT/US2007/001566 patent/WO2007084737A2/en active Application Filing
- 2007-01-22 HU HUE13173895A patent/HUE041840T2/hu unknown
- 2007-01-22 SI SI200731529T patent/SI1984018T1/sl unknown
- 2007-01-22 DK DK07716858.1T patent/DK1984018T3/da active
- 2007-01-22 PL PL07716858T patent/PL1984018T3/pl unknown
- 2007-01-22 PT PT13173895T patent/PT2666476T/pt unknown
- 2007-01-22 LT LTEP13173895.7T patent/LT2666476T/lt unknown
- 2007-01-22 JP JP2008551442A patent/JP5681345B2/ja not_active Expired - Fee Related
- 2007-01-22 ES ES13173895T patent/ES2716206T3/es active Active
- 2007-01-22 CN CNA2007800069255A patent/CN101431960A/zh active Pending
- 2007-01-22 RU RU2008134120/15A patent/RU2529830C2/ru not_active IP Right Cessation
-
2008
- 2008-07-10 IL IL192740A patent/IL192740A/en not_active IP Right Cessation
- 2008-07-18 US US12/175,610 patent/US8926967B2/en active Active
-
2014
- 2014-07-16 RU RU2014129319A patent/RU2665381C2/ru not_active IP Right Cessation
- 2014-12-03 US US14/559,851 patent/US10080783B2/en active Active
-
2017
- 2017-03-05 IL IL250937A patent/IL250937A0/en unknown
- 2017-07-27 AR ARP170102114A patent/AR109170A2/es unknown
-
2018
- 2018-08-15 RU RU2018129747A patent/RU2018129747A/ru not_active Application Discontinuation
- 2018-08-23 US US16/111,009 patent/US20190083582A1/en not_active Abandoned
-
2020
- 2020-12-29 US US17/137,060 patent/US20210228692A1/en not_active Abandoned
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