JP2004502456A5 - - Google Patents
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- Publication number
- JP2004502456A5 JP2004502456A5 JP2002509470A JP2002509470A JP2004502456A5 JP 2004502456 A5 JP2004502456 A5 JP 2004502456A5 JP 2002509470 A JP2002509470 A JP 2002509470A JP 2002509470 A JP2002509470 A JP 2002509470A JP 2004502456 A5 JP2004502456 A5 JP 2004502456A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- iduronidase
- composition according
- human
- alpha
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Withdrawn
Links
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 claims description 7
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 6
- 102000056929 human IDUA Human genes 0.000 claims description 6
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims description 5
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 5
- 102000008100 Human Serum Albumin Human genes 0.000 claims description 3
- 108091006905 Human Serum Albumin Proteins 0.000 claims description 3
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 20
- 108010003381 Iduronidase Proteins 0.000 claims 11
- 102000004627 Iduronidase Human genes 0.000 claims 11
- 238000000034 method Methods 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 5
- 239000012530 fluid Substances 0.000 claims 5
- 239000000203 mixture Substances 0.000 claims 5
- 229920002684 Sepharose Polymers 0.000 claims 4
- PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 claims 3
- 239000000872 buffer Substances 0.000 claims 3
- 230000000694 effects Effects 0.000 claims 3
- 239000012634 fragment Substances 0.000 claims 3
- 238000001802 infusion Methods 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 239000000243 solution Substances 0.000 claims 3
- 206010020751 Hypersensitivity Diseases 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 238000001914 filtration Methods 0.000 claims 2
- 238000003306 harvesting Methods 0.000 claims 2
- 230000001404 mediated effect Effects 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 claims 2
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 claims 2
- 229920000053 polysorbate 80 Polymers 0.000 claims 2
- 229940068968 polysorbate 80 Drugs 0.000 claims 2
- 239000011780 sodium chloride Substances 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- 102100035028 Alpha-L-iduronidase Human genes 0.000 claims 1
- RYGMFSIKBFXOCR-UHFFFAOYSA-N Copper Chemical compound [Cu] RYGMFSIKBFXOCR-UHFFFAOYSA-N 0.000 claims 1
- JPVYNHNXODAKFH-UHFFFAOYSA-N Cu2+ Chemical compound [Cu+2] JPVYNHNXODAKFH-UHFFFAOYSA-N 0.000 claims 1
- 208000015204 Hurler-Scheie syndrome Diseases 0.000 claims 1
- 206010021079 Hypopnoea Diseases 0.000 claims 1
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 1
- 230000002378 acidificating effect Effects 0.000 claims 1
- 230000001154 acute effect Effects 0.000 claims 1
- 208000026935 allergic disease Diseases 0.000 claims 1
- 230000007815 allergy Effects 0.000 claims 1
- 208000008784 apnea Diseases 0.000 claims 1
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical compound [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims 1
- 239000001045 blue dye Substances 0.000 claims 1
- YKCWQPZFAFZLBI-UHFFFAOYSA-N cibacron blue Chemical compound C1=2C(=O)C3=CC=CC=C3C(=O)C=2C(N)=C(S(O)(=O)=O)C=C1NC(C=C1S(O)(=O)=O)=CC=C1NC(N=1)=NC(Cl)=NC=1NC1=CC=CC=C1S(O)(=O)=O YKCWQPZFAFZLBI-UHFFFAOYSA-N 0.000 claims 1
- 229910052802 copper Inorganic materials 0.000 claims 1
- 239000010949 copper Substances 0.000 claims 1
- 229910001431 copper ion Inorganic materials 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000010790 dilution Methods 0.000 claims 1
- 239000012895 dilution Substances 0.000 claims 1
- 239000002552 dosage form Substances 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 230000029142 excretion Effects 0.000 claims 1
- 239000012467 final product Substances 0.000 claims 1
- 238000009472 formulation Methods 0.000 claims 1
- 239000001963 growth medium Substances 0.000 claims 1
- 230000004217 heart function Effects 0.000 claims 1
- 230000002209 hydrophobic effect Effects 0.000 claims 1
- 238000011081 inoculation Methods 0.000 claims 1
- 210000003127 knee Anatomy 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 230000002132 lysosomal effect Effects 0.000 claims 1
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 1
- 229910052760 oxygen Inorganic materials 0.000 claims 1
- 239000001301 oxygen Substances 0.000 claims 1
- 230000010412 perfusion Effects 0.000 claims 1
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims 1
- 239000008055 phosphate buffer solution Substances 0.000 claims 1
- 229920000136 polysorbate Polymers 0.000 claims 1
- 229950008882 polysorbate Drugs 0.000 claims 1
- 238000005086 pumping Methods 0.000 claims 1
- 238000011084 recovery Methods 0.000 claims 1
- 230000004044 response Effects 0.000 claims 1
- 239000012064 sodium phosphate buffer Substances 0.000 claims 1
- 210000000952 spleen Anatomy 0.000 claims 1
- 210000002700 urine Anatomy 0.000 claims 1
- 238000005406 washing Methods 0.000 claims 1
- 230000003442 weekly effect Effects 0.000 claims 1
- 238000003556 assay Methods 0.000 description 2
- 238000003745 diagnosis Methods 0.000 description 1
- 208000035475 disorder Diseases 0.000 description 1
- 210000000265 leukocyte Anatomy 0.000 description 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US09/439,923 US6426208B1 (en) | 1999-11-12 | 1999-11-12 | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| PCT/US2000/031293 WO2002004616A1 (en) | 1999-11-12 | 2000-11-09 | RECOMBINANT α-L-IDURONIDASE, METHODS FOR PRODUCING AND PURIFYING THE SAME AND METHODS FOR TREATING DISEASES CAUSED BY DEFICIENCIES THEREOF |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2004242343A Division JP3857705B2 (ja) | 1999-11-12 | 2004-08-23 | 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠失により引き起こされる疾病の処理方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2004502456A JP2004502456A (ja) | 2004-01-29 |
| JP2004502456A5 true JP2004502456A5 (OSRAM) | 2005-11-17 |
Family
ID=23746702
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2002509470A Withdrawn JP2004502456A (ja) | 1999-11-12 | 2000-11-09 | 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠失により引き起こされる疾病の処理方法 |
| JP2004242343A Expired - Lifetime JP3857705B2 (ja) | 1999-11-12 | 2004-08-23 | 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠失により引き起こされる疾病の処理方法 |
| JP2006215076A Pending JP2006312648A (ja) | 1999-11-12 | 2006-08-07 | 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠失により引き起こされる疾病の処理方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2004242343A Expired - Lifetime JP3857705B2 (ja) | 1999-11-12 | 2004-08-23 | 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠失により引き起こされる疾病の処理方法 |
| JP2006215076A Pending JP2006312648A (ja) | 1999-11-12 | 2006-08-07 | 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠失により引き起こされる疾病の処理方法 |
Country Status (14)
| Country | Link |
|---|---|
| US (3) | US6426208B1 (OSRAM) |
| EP (2) | EP1332206B1 (OSRAM) |
| JP (3) | JP2004502456A (OSRAM) |
| CN (2) | CN1420924B (OSRAM) |
| AT (2) | ATE342964T1 (OSRAM) |
| AU (2) | AU773663B2 (OSRAM) |
| BR (1) | BRPI0015533B8 (OSRAM) |
| CA (1) | CA2391098C (OSRAM) |
| DE (2) | DE60031456T2 (OSRAM) |
| ES (2) | ES2324727T3 (OSRAM) |
| IL (3) | IL149568A0 (OSRAM) |
| MX (1) | MXPA02004742A (OSRAM) |
| WO (1) | WO2002004616A1 (OSRAM) |
| ZA (1) | ZA200203619B (OSRAM) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6569661B1 (en) * | 1999-11-12 | 2003-05-27 | Biomarin Pharmaceutical Inc. | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| US6585971B1 (en) * | 1999-11-12 | 2003-07-01 | Harbor-Ucla Research And Education Institute | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof |
| EP1368039A4 (en) * | 2001-02-20 | 2006-03-29 | Uab Research Foundation | AMINOGLYCOSIDE TREATMENT FOR LYSOSOMAL STORAGE DISEASES |
| 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 |
| US7485314B2 (en) * | 2002-05-06 | 2009-02-03 | Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center | Induction of antigen specific immunologic tolerance |
| US20050026823A1 (en) * | 2003-06-20 | 2005-02-03 | Biomarin Pharmaceutical Inc. | Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues |
| EP1668110A2 (en) * | 2003-08-21 | 2006-06-14 | Q Therapeutics, Inc. | Genetically modified somatic cells for sustained secretion of lysosomal proenzymes deficient in lysosomal storage disorders |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| MXPA03011987A (es) | 2003-12-19 | 2005-06-23 | Osio Sancho Alberto | Metodo para el tratamiento de la presbicia induciendo cambios en el poder y fisiologia corneal. |
| US9062126B2 (en) | 2005-09-16 | 2015-06-23 | Raptor Pharmaceuticals Inc. | Compositions comprising receptor-associated protein (RAP) variants specific for CR-containing proteins and uses thereof |
| US8105788B2 (en) * | 2005-12-08 | 2012-01-31 | The University Of British Columbia | Mucopolysaccharidosis (MPS) diagnostic methods, systems, kits and assays associated therewith |
| WO2008116171A1 (en) * | 2007-03-21 | 2008-09-25 | Raptor Pharmaceutical Inc. | Cyclic receptor-associated protein (rap) peptides |
| HUE037162T2 (hu) | 2007-07-09 | 2018-08-28 | Genentech Inc | Diszulfidkötés redukciójának megelõzése polipeptidek rekombináns elõállítása során |
| ES2387251T3 (es) * | 2008-02-21 | 2012-09-19 | Fiat Group Automobiles S.P.A. | Conjunto de soporte de vidrio para una puerta de vehículo a motor con un borde superior sin armazón |
| ES2629853T3 (es) | 2008-05-07 | 2017-08-16 | Biomarin Pharmaceutical Inc. | Péptidos de dirección lisosómica y usos de los mismos |
| EP2475376B1 (en) | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| WO2012101671A1 (en) * | 2011-01-25 | 2012-08-02 | Jcr Pharmaceuticals Co., Ltd. | Method for production of human recombinant iduronate 2-sulfatase |
| DE102011102170B4 (de) | 2011-05-20 | 2024-06-06 | Vitesco Technologies GmbH | Einspritzvorrichtung zur Einspritzung eines Fluids |
| SMT202000445T1 (it) | 2012-06-19 | 2020-09-10 | Univ Florida | Composizioni e metodi per trattare patologie |
| ES2994608T3 (en) | 2012-08-10 | 2025-01-27 | Osio Corp D/B/A Yolia Health | Contact lens use in the treatment of an ophthalmologic condition |
| JP6591956B2 (ja) | 2013-03-15 | 2019-10-16 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｍｐｓ１を治療するための組成物および方法 |
| AU2017215211C1 (en) | 2016-02-03 | 2023-11-16 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| MA47436A (fr) * | 2017-01-31 | 2019-12-11 | Regenxbio Inc | Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylée entièrement humaine (idua) |
| EP3676385A1 (en) | 2017-07-06 | 2020-07-08 | The Trustees of The University of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| JP7449223B2 (ja) | 2017-09-22 | 2024-03-13 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ムコ多糖症ｉｉ型を治療するための遺伝子療法 |
| CN107699590A (zh) * | 2017-10-13 | 2018-02-16 | 成都中医药大学 | 一种制备重组人α‑L‑艾杜糖醛酸酶的方法 |
| US20220023434A1 (en) | 2018-12-19 | 2022-01-27 | The Board Of Trustees Of The Leland Stanford Junior University | Bifunctional Molecules for Lysosomal Targeting and Related Compositions and Methods |
| CN110577559B (zh) * | 2019-08-19 | 2023-02-17 | 江西科技师范大学 | α-L-艾杜糖醛酸酶测定用荧光糖苷酶底物的合成方法 |
| JP7791517B2 (ja) * | 2021-01-04 | 2025-12-24 | 国立大学法人徳島大学 | ライソゾームを標的とした新規ｄｄｓの開発 |
| WO2022145449A1 (ja) * | 2021-01-04 | 2022-07-07 | 国立大学法人徳島大学 | ライソゾームを標的とした新規ｄｄｓの開発 |
| CN115109790A (zh) * | 2021-03-23 | 2022-09-27 | 北京据德医药科技有限公司 | 一种重组a-L-艾杜糖醛酸普酶及其制备方法 |
| JP2024524614A (ja) | 2021-07-14 | 2024-07-05 | ライシア セラピューティクス， インコーポレイテッド | Ａｓｇｐｒ細胞表面受容体結合化合物及びコンジュゲート |
| CN114181318B (zh) * | 2021-11-08 | 2023-08-01 | 四川大学 | 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用 |
| CN115161284B (zh) * | 2022-07-04 | 2024-07-09 | 无锡多宁生物科技有限公司 | 一种lmh悬浮细胞复苏培养方法及其应用 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NL281225A (OSRAM) | 1961-07-20 | |||
| GB1029548A (en) | 1963-07-17 | 1966-05-11 | Amagasaki Iron & Steel Mfg Co | Corrugated roofing sheet |
| US3472931A (en) | 1969-01-17 | 1969-10-14 | Foster Milburn Co | Percutaneous absorption with lower alkyl amides |
| US3891757A (en) | 1971-11-11 | 1975-06-24 | Alza Corp | Anaesthetic topical and percutaneous administration with selected promoters |
| US5270051A (en) | 1991-10-15 | 1993-12-14 | Harris Donald H | Enzyme-orthokeratology |
| WO1993010244A1 (en) | 1991-11-14 | 1993-05-27 | Women's And Children's Hospital | Synthetic alpha-l-iduronidase and genetic sequences encoding same |
| US6149909A (en) | 1995-06-23 | 2000-11-21 | Women's And Children's Hospital | Synthetic α-L-iduronidase and genetic sequences encoding same |
| US5278051A (en) * | 1991-12-12 | 1994-01-11 | New York University | Construction of geometrical objects from polynucleotides |
| AU7071196A (en) | 1995-09-14 | 1997-04-01 | Croptech Development Corporation | Production of lysosomal enzymes in plant-based expression systems |
| GB9807464D0 (en) | 1998-04-07 | 1998-06-10 | Pharming Bv | Purification of human acid µ-glucosidase |
| WO1999058691A2 (en) | 1998-05-13 | 1999-11-18 | Harbor-Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
-
1999
- 1999-11-12 US US09/439,923 patent/US6426208B1/en not_active Expired - Lifetime
-
2000
- 2000-11-09 MX MXPA02004742A patent/MXPA02004742A/es active IP Right Grant
- 2000-11-09 WO PCT/US2000/031293 patent/WO2002004616A1/en not_active Ceased
- 2000-11-09 BR BRPI0015533A patent/BRPI0015533B8/pt not_active IP Right Cessation
- 2000-11-09 DE DE60031456T patent/DE60031456T2/de not_active Expired - Lifetime
- 2000-11-09 EP EP00993857A patent/EP1332206B1/en not_active Expired - Lifetime
- 2000-11-09 JP JP2002509470A patent/JP2004502456A/ja not_active Withdrawn
- 2000-11-09 DE DE60042137T patent/DE60042137D1/de not_active Expired - Lifetime
- 2000-11-09 AU AU84635/01A patent/AU773663B2/en not_active Expired
- 2000-11-09 CA CA002391098A patent/CA2391098C/en not_active Expired - Lifetime
- 2000-11-09 AT AT00993857T patent/ATE342964T1/de not_active IP Right Cessation
- 2000-11-09 IL IL14956800A patent/IL149568A0/xx unknown
- 2000-11-09 ES ES06011595T patent/ES2324727T3/es not_active Expired - Lifetime
- 2000-11-09 CN CN008181705A patent/CN1420924B/zh not_active Expired - Lifetime
- 2000-11-09 EP EP06011595A patent/EP1792983B1/en not_active Expired - Lifetime
- 2000-11-09 ES ES00993857T patent/ES2273748T3/es not_active Expired - Lifetime
- 2000-11-09 CN CNA2008101440588A patent/CN101407796A/zh active Pending
- 2000-11-09 AT AT06011595T patent/ATE430193T1/de not_active IP Right Cessation
-
2002
- 2002-05-07 ZA ZA200203619A patent/ZA200203619B/en unknown
- 2002-05-09 IL IL149568A patent/IL149568A/en active IP Right Grant
- 2002-07-25 US US10/206,443 patent/US7041487B2/en not_active Expired - Lifetime
-
2004
- 2004-08-23 JP JP2004242343A patent/JP3857705B2/ja not_active Expired - Lifetime
- 2004-09-03 AU AU2004208651A patent/AU2004208651A1/en not_active Abandoned
-
2006
- 2006-05-05 US US11/429,422 patent/US20060286087A1/en not_active Abandoned
- 2006-08-07 JP JP2006215076A patent/JP2006312648A/ja active Pending
-
2008
- 2008-06-11 IL IL192078A patent/IL192078A/en active IP Right Grant
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