JP2017529326A5 - - Google Patents
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- Publication number
- JP2017529326A5 JP2017529326A5 JP2017507791A JP2017507791A JP2017529326A5 JP 2017529326 A5 JP2017529326 A5 JP 2017529326A5 JP 2017507791 A JP2017507791 A JP 2017507791A JP 2017507791 A JP2017507791 A JP 2017507791A JP 2017529326 A5 JP2017529326 A5 JP 2017529326A5
- Authority
- JP
- Japan
- Prior art keywords
- targeted therapeutic
- seq
- therapeutic agent
- fusion protein
- acid sequence
- 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.)
- Granted
Links
- 239000003814 drug Substances 0.000 claims description 34
- 229940124597 therapeutic agent Drugs 0.000 claims description 32
- 108020001507 fusion proteins Proteins 0.000 claims description 24
- 102000037865 fusion proteins Human genes 0.000 claims description 24
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 22
- 210000004027 cell Anatomy 0.000 claims description 22
- 230000002132 lysosomal effect Effects 0.000 claims description 20
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 16
- 230000008685 targeting Effects 0.000 claims description 13
- 108090000790 Enzymes Proteins 0.000 claims description 12
- 102000004190 Enzymes Human genes 0.000 claims description 12
- 108090000623 proteins and genes Proteins 0.000 claims description 11
- 230000001225 therapeutic effect Effects 0.000 claims description 11
- 102000004169 proteins and genes Human genes 0.000 claims description 10
- 210000004962 mammalian cell Anatomy 0.000 claims description 8
- 150000007523 nucleic acids Chemical class 0.000 claims description 8
- 239000008194 pharmaceutical composition Substances 0.000 claims description 7
- 230000004988 N-glycosylation Effects 0.000 claims description 6
- 108020004707 nucleic acids Proteins 0.000 claims description 6
- 102000039446 nucleic acids Human genes 0.000 claims description 6
- 229940126585 therapeutic drug Drugs 0.000 claims description 6
- 208000015439 Lysosomal storage disease Diseases 0.000 claims description 5
- 108010044965 UDP-N-acetylglucosamine-lysosomal-enzyme N-acetylglucosaminephosphotransferase Proteins 0.000 claims description 4
- 210000003712 lysosome Anatomy 0.000 claims description 4
- 230000001868 lysosomic effect Effects 0.000 claims description 4
- 208000025820 Sanfilippo syndrome type B Diseases 0.000 claims description 3
- 239000003937 drug carrier Substances 0.000 claims description 3
- 238000004519 manufacturing process Methods 0.000 claims description 3
- 208000036709 mucopolysaccharidosis type 3B Diseases 0.000 claims description 3
- 208000012227 mucopolysaccharidosis type IIIB Diseases 0.000 claims description 3
- 101000588395 Bacillus subtilis (strain 168) Beta-hexosaminidase Proteins 0.000 claims description 2
- 241000894006 Bacteria Species 0.000 claims description 2
- 241000238631 Hexapoda Species 0.000 claims description 2
- 108090000581 Leukemia inhibitory factor Proteins 0.000 claims description 2
- 102000004058 Leukemia inhibitory factor Human genes 0.000 claims description 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 2
- 102100036197 Prosaposin Human genes 0.000 claims description 2
- 101710152403 Prosaposin Proteins 0.000 claims description 2
- 240000004808 Saccharomyces cerevisiae Species 0.000 claims description 2
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims description 2
- 238000012258 culturing Methods 0.000 claims description 2
- 239000012634 fragment Substances 0.000 claims description 2
- 210000005260 human cell Anatomy 0.000 claims description 2
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 238000003860 storage Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 4
- 230000004936 stimulating effect Effects 0.000 description 1
- 108700026220 vif Genes Proteins 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462036082P | 2014-08-11 | 2014-08-11 | |
| US62/036,082 | 2014-08-11 | ||
| PCT/US2015/044713 WO2016025519A1 (en) | 2014-08-11 | 2015-08-11 | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017529326A JP2017529326A (ja) | 2017-10-05 |
| JP2017529326A5 true JP2017529326A5 (enExample) | 2018-09-13 |
| JP6623213B2 JP6623213B2 (ja) | 2019-12-18 |
Family
ID=54035297
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017507791A Active JP6623213B2 (ja) | 2014-08-11 | 2015-08-11 | リソソーム酵素に融合したマンノース−６−リン酸含有ペプチド |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US10722559B2 (enExample) |
| EP (1) | EP3185889B1 (enExample) |
| JP (1) | JP6623213B2 (enExample) |
| CN (1) | CN106573039A (enExample) |
| AU (1) | AU2015301809A1 (enExample) |
| BR (1) | BR112017002741A2 (enExample) |
| CA (1) | CA2956469A1 (enExample) |
| EA (1) | EA201790178A1 (enExample) |
| MX (1) | MX2017001898A (enExample) |
| WO (1) | WO2016025519A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220133863A1 (en) * | 2010-06-25 | 2022-05-05 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
| CN104857504A (zh) | 2010-06-25 | 2015-08-26 | 夏尔人类遗传性治疗公司 | 芳基硫酸酯酶a cns递送的方法和组合物 |
| MA50746A (fr) | 2017-10-02 | 2020-08-12 | Denali Therapeutics Inc | Protéines de fusion comprenant des enzymes d'enzymothérapie substitutive |
| AU2019316103B2 (en) | 2018-08-02 | 2026-02-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| 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 |
| EP3994253A1 (en) * | 2019-07-02 | 2022-05-11 | M6P Therapeutics (Switzerland) LLC | Vector compositions and methods of using same for treatment of lysosomal storage disorders |
| CN111455004B (zh) * | 2020-02-29 | 2023-05-05 | 浙江农林大学 | 一种脂肪酶催化在线合成香豆素-3-羧酸-6’-o-d-甘露糖酯的方法 |
| WO2022026906A2 (en) * | 2020-07-30 | 2022-02-03 | The Board Of Trustees Of The Leland Stanford Junior University | Lysosomal targeting molecules comprising knottin peptides and related compositions and methods |
| 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 |
| WO2023150387A1 (en) * | 2022-02-07 | 2023-08-10 | M6P Therapeutics, Inc. | Compositions comprising acid alpha glucosidase and methods of use thereof |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of myotonic dystrophy |
| WO2025238106A1 (en) * | 2024-05-15 | 2025-11-20 | M6P Therapeutics (Switzerland) GmbH | Lysosome-targeting degradation fusion design |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040142474A1 (en) * | 2000-09-14 | 2004-07-22 | Expression Genetics, Inc. | Novel cationic lipopolymer as a biocompatible gene delivery agent |
| US7560424B2 (en) * | 2001-04-30 | 2009-07-14 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| JP2006506317A (ja) * | 2002-01-11 | 2006-02-23 | 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 | 治療用リソソーム酵素の送達のための酵素送達系としてのｐ９７の使用 |
| CA2705160A1 (en) * | 2007-11-09 | 2009-05-14 | Anaphore, Inc. | Fusion proteins of mannose binding lectins for treatment of disease |
| US20110223147A1 (en) * | 2008-05-07 | 2011-09-15 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| NZ702801A (en) | 2010-06-25 | 2016-08-26 | Shire Human Genetic Therapies | Treatment of sanfilippo syndrome type b |
| UA115648C2 (uk) * | 2010-06-25 | 2017-12-11 | Шае Хюмен Дженетік Терапіс, Інк. | Доставка терапевтичних агентів до цнс |
| WO2012012742A2 (en) * | 2010-07-22 | 2012-01-26 | Shire Human Genetic Therapies, Inc. | Crystal structure of human alpha-n-acetylglucosaminidase |
| US20140219988A1 (en) * | 2011-05-19 | 2014-08-07 | Tokyo Metropolitan Institute Of Medical Science | Rrecombinant human saposin b protein containing phosphorylated glucose ring and use thereof |
| RS56913B1 (sr) * | 2011-05-27 | 2018-05-31 | Amicus Therapeutics Inc | Postupci za kuplovanje peptida za ciljanje na rekombinantne lizosomalne encime za poboljšanje tretmana lizosomalnih bolesti taloženja |
| AU2012322811A1 (en) * | 2011-10-12 | 2014-05-01 | Synageva Biopharma Corp. | Recombinant human NaGlu protein and uses thereof |
| DK3115372T3 (da) * | 2012-11-27 | 2019-06-11 | Biomarin Pharm Inc | Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf |
-
2015
- 2015-08-11 BR BR112017002741-0A patent/BR112017002741A2/pt not_active Application Discontinuation
- 2015-08-11 US US15/503,223 patent/US10722559B2/en active Active
- 2015-08-11 EP EP15757368.4A patent/EP3185889B1/en active Active
- 2015-08-11 WO PCT/US2015/044713 patent/WO2016025519A1/en not_active Ceased
- 2015-08-11 JP JP2017507791A patent/JP6623213B2/ja active Active
- 2015-08-11 MX MX2017001898A patent/MX2017001898A/es unknown
- 2015-08-11 EA EA201790178A patent/EA201790178A1/ru unknown
- 2015-08-11 AU AU2015301809A patent/AU2015301809A1/en not_active Abandoned
- 2015-08-11 CN CN201580043154.1A patent/CN106573039A/zh active Pending
- 2015-08-11 CA CA2956469A patent/CA2956469A1/en not_active Abandoned
-
2020
- 2020-06-18 US US16/904,943 patent/US20200376095A1/en not_active Abandoned
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