JP2005506340A5 - - Google Patents
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- Publication number
- JP2005506340A5 JP2005506340A5 JP2003535542A JP2003535542A JP2005506340A5 JP 2005506340 A5 JP2005506340 A5 JP 2005506340A5 JP 2003535542 A JP2003535542 A JP 2003535542A JP 2003535542 A JP2003535542 A JP 2003535542A JP 2005506340 A5 JP2005506340 A5 JP 2005506340A5
- Authority
- JP
- Japan
- Prior art keywords
- therapeutic agent
- targeted therapeutic
- igf
- amino acids
- targeted
- 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.)
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- 239000003814 drug Substances 0.000 claims 39
- 150000001413 amino acids Chemical class 0.000 claims 19
- 102000004218 Insulin-like growth factor I Human genes 0.000 claims 14
- 108090000723 Insulin-like growth factor I Proteins 0.000 claims 14
- 102100013307 IGF2R Human genes 0.000 claims 5
- 102000014429 Insulin-like growth factor Human genes 0.000 claims 5
- 108050003490 Insulin-like growth factor Proteins 0.000 claims 5
- 229920001184 polypeptide Polymers 0.000 claims 5
- 230000001225 therapeutic Effects 0.000 claims 5
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical compound CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 claims 4
- 210000001218 Blood-Brain Barrier Anatomy 0.000 claims 3
- 102000004190 Enzymes Human genes 0.000 claims 3
- 108090000790 Enzymes Proteins 0.000 claims 3
- 108010031792 IGF Type 2 Receptor Proteins 0.000 claims 3
- 102400000022 Insulin-Like Growth Factor II Human genes 0.000 claims 3
- 108090001117 Insulin-Like Growth Factor II Proteins 0.000 claims 3
- 210000003712 Lysosomes Anatomy 0.000 claims 3
- 230000002132 lysosomal Effects 0.000 claims 3
- 230000001868 lysosomic Effects 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 210000004899 C-terminal region Anatomy 0.000 claims 2
- 101710032496 IGF2R Proteins 0.000 claims 2
- 125000000998 L-alanino group Chemical group [H]N([*])[C@](C([H])([H])[H])([H])C(=O)O[H] 0.000 claims 2
- 230000002209 hydrophobic Effects 0.000 claims 2
- 210000003169 Central Nervous System Anatomy 0.000 claims 1
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N 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 1
- 150000001768 cations Chemical class 0.000 claims 1
- 210000004027 cells Anatomy 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
Claims (22)
リソソーム中で治療学的に活性な治療剤および血液脳関門を通過するための手段
を含み、ここで、該血液脳関門を通過するための手段は、インシュリン様増殖因子（ＩＧＦ）部分を含む、標的化治療剤。 A targeted therapeutic agent comprising:
A therapeutically active therapeutic agent in lysosomes and means for crossing the blood brain barrier, wherein the means for crossing the blood brain barrier comprises an insulin-like growth factor (IGF) moiety, Targeted therapeutic agent.
リソソーム中で治療学的に活性な治療剤および
中枢神経系に標的化されるために、ヒトＩＧＦ−ＩのＡドメイン、Ｂドメイン、ＣドメインもしくはＤドメイン、またはＣ末端領域あるいはそれらの一部分のうちの少なくとも１つを十分に複製した配列を含むポリペプチドを含む標的化部分
を含む、標的化治療剤。 A targeted therapeutic agent comprising:
Of therapeutic agents active in lysosomes and for targeting to the central nervous system, the A domain, B domain, C domain or D domain of human IGF-I, or the C-terminal region or part thereof A targeted therapeutic comprising a targeting moiety comprising a polypeptide comprising a sequence that sufficiently replicates at least one of the above.
およびヒトＩＧＦまたはヒトＩＧＦ−Ｉの配列のムテインの配列を含むポリペプチドを含む標的化部分
を含み、ここで、該配列は、以下：
（ｉ） ヒトＩＧＦ−Ｉのアミノ酸１〜２５；
（ｉｉ） ヒトＩＧＦ−Ｉのアミノ酸２５〜４０；
（ｉｉｉ）ヒトＩＧＦ−Ｉのアミノ酸４０〜６５；および
（ｉｖ） ヒトＩＧＦ−Ｉのアミノ酸６５〜７０
からなる群より選択される、治療剤。 A targeted therapeutic agent comprising a targeting moiety comprising a therapeutic agent that is therapeutically active in human lysosomes and a polypeptide comprising a sequence of a human IGF or human IGF-I sequence mutein. Where the sequence is:
(I) amino acids 1-25 of human IGF-I;
(Ii) amino acids 25-40 of human IGF-I;
(Iii) amino acids 40-65 of human IGF-I; and (iv) amino acids 65-70 of human IGF-I.
A therapeutic agent selected from the group consisting of:
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US32965001P | 2001-10-16 | 2001-10-16 | |
| US10/136,639 US20030072761A1 (en) | 2001-10-16 | 2002-04-30 | Methods and compositions for targeting proteins across the blood brain barrier |
| US10/136,841 US7396811B2 (en) | 2001-04-30 | 2002-04-30 | Subcellular targeting of therapeutic proteins |
| US38445202P | 2002-05-29 | 2002-05-29 | |
| US38601902P | 2002-06-05 | 2002-06-05 | |
| US40881602P | 2002-09-06 | 2002-09-06 | |
| PCT/US2002/032968 WO2003032727A1 (en) | 2001-10-16 | 2002-10-16 | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2009143436A Division JP2009203241A (en) | 2001-10-16 | 2009-06-16 | Method and composition for targeting underglycosylated proteins across blood brain barrier |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2005506340A JP2005506340A (en) | 2005-03-03 |
| JP2005506340A5 true JP2005506340A5 (en) | 2006-01-12 |
Family
ID=27558195
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2003535542A Pending JP2005506340A (en) | 2001-10-16 | 2002-10-16 | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
| JP2009143436A Pending JP2009203241A (en) | 2001-10-16 | 2009-06-16 | Method and composition for targeting underglycosylated proteins across blood brain barrier |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2009143436A Pending JP2009203241A (en) | 2001-10-16 | 2009-06-16 | Method and composition for targeting underglycosylated proteins across blood brain barrier |
Country Status (6)
| Country | Link |
|---|---|
| EP (1) | EP1446007A4 (en) |
| JP (2) | JP2005506340A (en) |
| AU (3) | AU2002362930A2 (en) |
| CA (1) | CA2463473A1 (en) |
| IL (1) | IL161352A0 (en) |
| WO (2) | WO2003032727A1 (en) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| ATE384736T1 (en) | 2001-04-30 | 2008-02-15 | Zystor Therapeutics Inc | SUB-CELLULAR TARGETING OF THERAPEUTIC PROTEINS |
| US7560424B2 (en) | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| WO2003032727A1 (en) * | 2001-10-16 | 2003-04-24 | Symbiontics Inc. | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
| CA2487815A1 (en) * | 2002-05-29 | 2003-12-11 | Symbiontics, Inc. | Targeted therapeutic proteins |
| JP4914224B2 (en) * | 2004-02-10 | 2012-04-11 | バイオマリン ファーマシューティカル インコーポレイテッド | Acid α-glucosidase and its fragments |
| EP2997976A1 (en) | 2007-07-27 | 2016-03-23 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
| CN105126099A (en) * | 2007-12-21 | 2015-12-09 | 弗·哈夫曼-拉罗切有限公司 | Antibody formulation |
| AU2009244148B2 (en) | 2008-05-07 | 2014-10-09 | Biomarin Pharmaceutical Inc. | Lysosomal targeting peptides and uses thereof |
| EP3075386B1 (en) | 2009-06-17 | 2019-10-16 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| KR102094094B1 (en) | 2010-06-25 | 2020-03-27 | 샤이어 휴먼 지네틱 테라피즈 인크. | Methods and compositions for cns delivery of arylsulfatase a |
| MX2013000324A (en) * | 2010-06-25 | 2013-02-01 | Shire Human Genetic Therapies | Treatment of sanfilippo syndrome type b. |
| MX2020001395A (en) | 2010-06-25 | 2022-03-24 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of iduronate-2-sulfatas e. |
| CA2805413A1 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of heparan n-sulfatase |
| ES2650689T3 (en) | 2010-06-25 | 2018-01-19 | Shire Human Genetic Therapies, Inc. | Administration of therapeutic agents to the central nervous system |
| HUE055963T2 (en) | 2010-06-25 | 2022-01-28 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of iduronate-2-sulfatase |
| US20150037311A1 (en) * | 2011-12-01 | 2015-02-05 | Angiochem Inc. | Targeted lysosomal enzyme compounds |
| EP2793922B1 (en) | 2011-12-23 | 2019-10-30 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| EP2885318A4 (en) | 2012-08-14 | 2016-03-30 | Angiochem Inc | Peptide-dendrimer conjugates and uses thereof |
| KR102262882B1 (en) | 2012-11-27 | 2021-06-10 | 바이오마린 파머수티컬 인크. | Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof |
| US10537638B2 (en) * | 2013-03-15 | 2020-01-21 | Glaxosmithkline Intellectual Property (No.2) Limited | Low concentration antibody formulations |
| LT2970413T (en) | 2014-04-01 | 2018-10-10 | Swedish Orphan Biovitrum Ab (Publ) | Modified sulfamidase and production thereof |
| DK3307326T3 (en) | 2015-06-15 | 2020-10-19 | Angiochem Inc | METHODS FOR THE TREATMENT OF LEPTOMENINGAL CARCINOMATOSIS |
| KR20180114199A (en) | 2016-02-24 | 2018-10-17 | 바이오마린 파머수티컬 인크. | Targeted therapeutic lysozyme enzyme fusion proteins, related formulations and uses thereof |
| JP2021521851A (en) | 2018-04-30 | 2021-08-30 | アミカス セラピューティックス インコーポレイテッド | Gene therapy constructs and usage |
| SG11202103640VA (en) | 2018-10-10 | 2021-05-28 | Amicus Therapeutics Inc | Disulfide bond stabilized polypeptide compositions and methods of use |
| WO2020132452A1 (en) | 2018-12-20 | 2020-06-25 | Shire Human Genetic Therapies, Inc. | Purification of iduronate-2-sulfatase immunoglobulin fusion protein |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US5672683A (en) * | 1989-09-07 | 1997-09-30 | Alkermes, Inc. | Transferrin neuropharmaceutical agent fusion protein |
| CA2025907A1 (en) * | 1989-09-21 | 1991-03-22 | Franklin D. Collins | Method of transporting compositions across the blood brain barrier |
| US6472140B1 (en) * | 1997-09-05 | 2002-10-29 | The General Hospital Corporation | α-2- macroglobulin therapies and drug screening methods for Alzheimer's disease. |
| ATE384736T1 (en) * | 2001-04-30 | 2008-02-15 | Zystor Therapeutics Inc | SUB-CELLULAR TARGETING OF THERAPEUTIC PROTEINS |
| WO2003032727A1 (en) * | 2001-10-16 | 2003-04-24 | Symbiontics Inc. | Methods and compositions for targeting underglycosylated proteins across the blood brain barrier |
| CA2487815A1 (en) * | 2002-05-29 | 2003-12-11 | Symbiontics, Inc. | Targeted therapeutic proteins |
-
2002
- 2002-10-16 WO PCT/US2002/032968 patent/WO2003032727A1/en active Application Filing
- 2002-10-16 JP JP2003535542A patent/JP2005506340A/en active Pending
- 2002-10-16 AU AU2002362930A patent/AU2002362930A2/en not_active Abandoned
- 2002-10-16 IL IL16135202A patent/IL161352A0/en unknown
- 2002-10-16 EP EP02801739A patent/EP1446007A4/en not_active Withdrawn
- 2002-10-16 WO PCT/US2002/032996 patent/WO2003032913A2/en not_active Application Discontinuation
- 2002-10-16 AU AU2002347910A patent/AU2002347910A1/en not_active Abandoned
- 2002-10-16 CA CA002463473A patent/CA2463473A1/en not_active Abandoned
-
2009
- 2009-06-16 JP JP2009143436A patent/JP2009203241A/en active Pending
-
2010
- 2010-08-25 AU AU2010214643A patent/AU2010214643A1/en not_active Abandoned
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