JP2010509344A5 - - Google Patents
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- Publication number
- JP2010509344A5 JP2010509344A5 JP2009536345A JP2009536345A JP2010509344A5 JP 2010509344 A5 JP2010509344 A5 JP 2010509344A5 JP 2009536345 A JP2009536345 A JP 2009536345A JP 2009536345 A JP2009536345 A JP 2009536345A JP 2010509344 A5 JP2010509344 A5 JP 2010509344A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- human
- fusion protein
- targeting domain
- gaa
- 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.)
- Pending
Links
- 108020001507 fusion proteins Proteins 0.000 claims description 49
- 102000037865 fusion proteins Human genes 0.000 claims description 49
- 230000008685 targeting Effects 0.000 claims description 48
- 230000002132 lysosomal effect Effects 0.000 claims description 46
- 239000000203 mixture Substances 0.000 claims description 42
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims description 40
- 102000045921 human GAA Human genes 0.000 claims description 40
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 claims description 38
- 150000001413 amino acids Chemical class 0.000 claims description 32
- 101001076292 Homo sapiens Insulin-like growth factor II Proteins 0.000 claims description 24
- 239000012634 fragment Substances 0.000 claims description 24
- 102000057877 human IGF2 Human genes 0.000 claims description 24
- 239000008194 pharmaceutical composition Substances 0.000 claims description 18
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims description 16
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims description 16
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims description 16
- 201000004502 glycogen storage disease II Diseases 0.000 claims description 16
- 101001028831 Homo sapiens Cation-independent mannose-6-phosphate receptor Proteins 0.000 claims description 11
- 102000048143 Insulin-Like Growth Factor II Human genes 0.000 claims description 10
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 claims description 10
- 229920002527 Glycogen Polymers 0.000 claims description 6
- 229940096919 glycogen Drugs 0.000 claims description 6
- 210000003205 muscle Anatomy 0.000 claims description 5
- 230000037396 body weight Effects 0.000 claims description 4
- 125000006850 spacer group Chemical group 0.000 claims description 4
- 239000002253 acid Substances 0.000 claims description 3
- 150000001768 cations Chemical class 0.000 claims description 3
- 210000003712 lysosome Anatomy 0.000 claims description 3
- 230000001868 lysosomic effect Effects 0.000 claims description 3
- 208000031229 Cardiomyopathies Diseases 0.000 claims description 2
- 208000021642 Muscular disease Diseases 0.000 claims description 2
- 201000009623 Myopathy Diseases 0.000 claims description 2
- 102000016679 alpha-Glucosidases Human genes 0.000 claims description 2
- 108010028144 alpha-Glucosidases Proteins 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 230000000694 effects Effects 0.000 claims description 2
- 210000002027 skeletal muscle Anatomy 0.000 claims description 2
- 108010031792 IGF Type 2 Receptor Proteins 0.000 claims 4
- 102000019218 Mannose-6-phosphate receptors Human genes 0.000 claims 4
- -1 cation mannose-6-phosphate Chemical class 0.000 claims 2
- 230000008878 coupling Effects 0.000 claims 2
- 238000010168 coupling process Methods 0.000 claims 2
- 238000005859 coupling reaction Methods 0.000 claims 2
- 210000003462 vein Anatomy 0.000 claims 2
- 230000003442 weekly effect Effects 0.000 claims 2
- 230000004927 fusion Effects 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 238000000034 method Methods 0.000 description 32
- 230000003203 everyday effect Effects 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 229910052698 phosphorus Inorganic materials 0.000 description 1
- 239000011574 phosphorus Substances 0.000 description 1
- 108020003175 receptors Proteins 0.000 description 1
- 102000005962 receptors Human genes 0.000 description 1
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US85851406P | 2006-11-13 | 2006-11-13 | |
| US87925507P | 2007-01-05 | 2007-01-05 | |
| US90018707P | 2007-02-07 | 2007-02-07 | |
| PCT/US2007/023881 WO2008063511A2 (en) | 2006-11-13 | 2007-11-13 | Methods for treating pompe disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2010509344A JP2010509344A (ja) | 2010-03-25 |
| JP2010509344A5 true JP2010509344A5 (enExample) | 2011-06-23 |
Family
ID=39333078
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2009536345A Pending JP2010509344A (ja) | 2006-11-13 | 2007-11-13 | ポンペ病を治療するための方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US20090117091A1 (enExample) |
| EP (1) | EP2099523A2 (enExample) |
| JP (1) | JP2010509344A (enExample) |
| AU (1) | AU2007322123A1 (enExample) |
| CA (1) | CA2669347A1 (enExample) |
| IL (1) | IL198692A0 (enExample) |
| WO (1) | WO2008063511A2 (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2572148T3 (es) | 2005-05-17 | 2016-05-30 | Amicus Therapeutics Inc | Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados |
| US8536148B2 (en) | 2009-09-04 | 2013-09-17 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Disabling autophagy as a treatment for lysosomal storage diseases |
| WO2012061597A1 (en) * | 2010-11-03 | 2012-05-10 | The United States Of America As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer, National Institutes Of Health | Alpha-glucosidase binders and methods of their use |
| PE20140617A1 (es) * | 2011-04-22 | 2014-05-28 | Genzyme Corp | Alfa glucosidasa acida modificada con procesamiento acelerado |
| KR20140135222A (ko) | 2012-03-07 | 2014-11-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
| 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 |
| 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 |
| 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 |
| KR20200118151A (ko) | 2018-02-07 | 2020-10-14 | 리제너론 파마슈티칼스 인코포레이티드 | 치료 단백질 전달을 위한 방법 및 조성물 |
| BR112020021962A2 (pt) | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| MX2020012350A (es) | 2018-05-17 | 2021-01-29 | Regeneron Pharma | Anticuerpos anti-cd63, conjugados y usos de estos. |
| EP3863663A2 (en) | 2018-10-10 | 2021-08-18 | Amicus Therapeutics, Inc. | Disulfide bond stabilized polypeptide compositions and methods of use |
| BR112021021720A2 (pt) * | 2019-04-30 | 2021-12-28 | Univ Pennsylvania | Composições úteis para tratamento de doença de pompe |
| EP3871687A1 (en) | 2020-02-27 | 2021-09-01 | eleva GmbH | Enzyme replacement therapy for treating pompe disease |
| WO2021231863A1 (en) * | 2020-05-14 | 2021-11-18 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of pompe disease |
| JP2023159049A (ja) * | 2022-04-19 | 2023-10-31 | Ｊｃｒファーマ株式会社 | マンノース－６－リン酸の数を減少させた糖蛋白質 |
| WO2025137213A1 (en) | 2023-12-20 | 2025-06-26 | Denali Therapeutics Inc. | Fusion proteins comprising acid alpha-glucosidase enzymes and methods thereof |
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| US4309776A (en) * | 1980-05-13 | 1982-01-12 | Ramon Berguer | Intravascular implantation device and method of using the same |
| US4749570A (en) * | 1981-12-31 | 1988-06-07 | The Governors Of The University Of Alberta | Targeting conjugates of albumin and therapeutic agents |
| US4522811A (en) * | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| WO1986000619A1 (en) * | 1984-07-13 | 1986-01-30 | Chiron Corporation | Prepro insulin-like growth factors i and ii |
| US4902505A (en) * | 1986-07-30 | 1990-02-20 | Alkermes | Chimeric peptides for neuropeptide delivery through the blood-brain barrier |
| US4801575A (en) * | 1986-07-30 | 1989-01-31 | The Regents Of The University Of California | Chimeric peptides for neuropeptide delivery through the blood-brain barrier |
| US5470828A (en) * | 1987-12-24 | 1995-11-28 | Gropep Pty. Ltd. | Peptide analogs of insulin-like growth factor II |
| US5817623A (en) * | 1995-03-06 | 1998-10-06 | Univ Colorado State Res Found | Method for treating diabetic peripheral neuropathy with IGF-I or IGF-II |
| US6451600B1 (en) * | 1989-12-22 | 2002-09-17 | Genzyme Corporation | Enzymatically active recombinant glucocerebrosidase |
| US5549892A (en) * | 1988-12-23 | 1996-08-27 | Genzyme Corporation | Enhanced in vivo uptake of glucocerebrosidase |
| US5236838A (en) * | 1988-12-23 | 1993-08-17 | Genzyme Corporation | Enzymatically active recombinant glucocerebrosidase |
| US5399346A (en) * | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| US5977307A (en) * | 1989-09-07 | 1999-11-02 | Alkermes, Inc. | Transferrin receptor specific ligand-neuropharmaceutical agent fusion proteins |
| US5356804A (en) * | 1990-10-24 | 1994-10-18 | Mount Sinai School Of Medicine Of The City Of New York | Cloning and expression of biologically active human α-galactosidase A |
| US5401650A (en) * | 1990-10-24 | 1995-03-28 | The Mount Sinai School Of Medicine Of The City University Of New York | Cloning and expression of biologically active α-galactosidase A |
| US5633235A (en) * | 1991-04-19 | 1997-05-27 | Regents Of The University Of Michigan | Triciribine and analogs as antiviral drugs |
| EP0596969A1 (en) * | 1991-07-29 | 1994-05-18 | British Bio-Technology Limited | Igf-ii analogues |
| US6270989B1 (en) * | 1991-11-05 | 2001-08-07 | Transkaryotic Therapies, Inc. | Protein production and delivery |
| US5258453A (en) * | 1992-01-21 | 1993-11-02 | University Of Utah | Drug delivery system for the simultaneous delivery of drugs activatable by enzymes and light |
| AU4528493A (en) * | 1992-06-04 | 1994-01-04 | Regents Of The University Of California, The | In vivo gene therapy with intron-free sequence of interest |
| US5981194A (en) * | 1992-07-10 | 1999-11-09 | University Of British Columbia | Use of p97 and iron binding proteins as diagnostic and therapeutic agents |
| US5476779A (en) * | 1992-09-30 | 1995-12-19 | University Of Maryland At College Park | DNA encoding insulin-like growth factor II isolated from rainbow trout |
| US5633234A (en) * | 1993-01-22 | 1997-05-27 | The Johns Hopkins University | Lysosomal targeting of immunogens |
| US5798366A (en) * | 1993-05-13 | 1998-08-25 | Monsanto Company | Method for treatment of CNS-involved lysosomal storage diseases |
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| ATE384736T1 (de) * | 2001-04-30 | 2008-02-15 | Zystor Therapeutics Inc | Subzelluläres targeting von therapeutischen proteinen |
| US7629309B2 (en) * | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| AU2003224880A1 (en) * | 2002-04-05 | 2003-10-27 | Genzyme Corporation | Methods of enhancing lysosomal storage disease therapy |
| US20040248262A1 (en) * | 2003-01-22 | 2004-12-09 | Koeberl Dwight D. | Constructs for expressing lysomal polypeptides |
| 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 |
| ES2344302T3 (es) * | 2004-02-10 | 2010-08-24 | Zystor Therapeutics , Inc. | Alfa glucosidasa acida y fragmentos de la misma. |
-
2007
- 2007-11-13 JP JP2009536345A patent/JP2010509344A/ja active Pending
- 2007-11-13 WO PCT/US2007/023881 patent/WO2008063511A2/en not_active Ceased
- 2007-11-13 AU AU2007322123A patent/AU2007322123A1/en not_active Abandoned
- 2007-11-13 US US11/985,360 patent/US20090117091A1/en not_active Abandoned
- 2007-11-13 CA CA002669347A patent/CA2669347A1/en not_active Abandoned
- 2007-11-13 EP EP07867437A patent/EP2099523A2/en not_active Withdrawn
-
2009
- 2009-05-11 IL IL198692A patent/IL198692A0/en not_active IP Right Cessation
-
2011
- 2011-10-07 US US13/268,369 patent/US20120093794A1/en not_active Abandoned
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