JP2019509735A5 - - Google Patents
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- Publication number
- JP2019509735A5 JP2019509735A5 JP2018546471A JP2018546471A JP2019509735A5 JP 2019509735 A5 JP2019509735 A5 JP 2019509735A5 JP 2018546471 A JP2018546471 A JP 2018546471A JP 2018546471 A JP2018546471 A JP 2018546471A JP 2019509735 A5 JP2019509735 A5 JP 2019509735A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- recombinant follistatin
- amino acid
- composition
- fusion protein
- 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
- 102000016970 Follistatin Human genes 0.000 claims description 129
- 108010014612 Follistatin Proteins 0.000 claims description 129
- 229920001184 polypeptide Polymers 0.000 claims description 64
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 64
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 64
- 102000037865 fusion proteins Human genes 0.000 claims description 63
- 108020001507 fusion proteins Proteins 0.000 claims description 63
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 44
- 239000000203 mixture Substances 0.000 claims description 35
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 30
- 150000001413 amino acids Chemical class 0.000 claims description 28
- 238000000034 method Methods 0.000 claims description 26
- 210000003205 muscle Anatomy 0.000 claims description 17
- 108090000623 proteins and genes Proteins 0.000 claims description 14
- 102000004169 proteins and genes Human genes 0.000 claims description 14
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 10
- 102100039939 Growth/differentiation factor 8 Human genes 0.000 claims description 9
- 108010056852 Myostatin Proteins 0.000 claims description 9
- 238000010494 dissociation reaction Methods 0.000 claims description 8
- 230000005593 dissociations Effects 0.000 claims description 8
- 102000040430 polynucleotide Human genes 0.000 claims description 8
- 239000002157 polynucleotide Substances 0.000 claims description 8
- 108091033319 polynucleotide Proteins 0.000 claims description 8
- 102100028726 Bone morphogenetic protein 10 Human genes 0.000 claims description 6
- 101710118482 Bone morphogenetic protein 10 Proteins 0.000 claims description 6
- 108010090290 Growth Differentiation Factor 2 Proteins 0.000 claims description 6
- 102100040892 Growth/differentiation factor 2 Human genes 0.000 claims description 6
- HTTJABKRGRZYRN-UHFFFAOYSA-N Heparin Chemical compound OC1C(NC(=O)C)C(O)OC(COS(O)(=O)=O)C1OC1C(OS(O)(=O)=O)C(O)C(OC2C(C(OS(O)(=O)=O)C(OC3C(C(O)C(O)C(O3)C(O)=O)OS(O)(=O)=O)C(CO)O2)NS(O)(=O)=O)C(C(O)=O)O1 HTTJABKRGRZYRN-UHFFFAOYSA-N 0.000 claims description 6
- 229960002897 heparin Drugs 0.000 claims description 6
- 229920000669 heparin Polymers 0.000 claims description 6
- 238000007911 parenteral administration Methods 0.000 claims description 6
- 238000006467 substitution reaction Methods 0.000 claims description 6
- 102220639700 Leptin_R78L_mutation Human genes 0.000 claims description 4
- 208000010428 Muscle Weakness Diseases 0.000 claims description 4
- 206010048654 Muscle fibrosis Diseases 0.000 claims description 4
- 206010028372 Muscular weakness Diseases 0.000 claims description 4
- 108010023082 activin A Proteins 0.000 claims description 4
- 210000004027 cell Anatomy 0.000 claims description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 4
- 208000035475 disorder Diseases 0.000 claims description 4
- 239000003814 drug Substances 0.000 claims description 4
- 239000013604 expression vector Substances 0.000 claims description 4
- 238000001990 intravenous administration Methods 0.000 claims description 4
- 206010028320 muscle necrosis Diseases 0.000 claims description 4
- 239000002773 nucleotide Substances 0.000 claims description 4
- 125000003729 nucleotide group Chemical group 0.000 claims description 4
- 239000008194 pharmaceutical composition Substances 0.000 claims description 4
- 210000002027 skeletal muscle Anatomy 0.000 claims description 4
- 238000007920 subcutaneous administration Methods 0.000 claims description 4
- 229940124597 therapeutic agent Drugs 0.000 claims description 4
- 238000002560 therapeutic procedure Methods 0.000 claims description 4
- 230000003442 weekly effect Effects 0.000 claims description 4
- 230000002401 inhibitory effect Effects 0.000 claims description 3
- 208000024891 symptom Diseases 0.000 claims description 3
- 206010069632 Bladder dysfunction Diseases 0.000 claims description 2
- 208000031229 Cardiomyopathies Diseases 0.000 claims description 2
- 208000032170 Congenital Abnormalities Diseases 0.000 claims description 2
- 206010061619 Deformity Diseases 0.000 claims description 2
- 208000019505 Deglutition disease Diseases 0.000 claims description 2
- 102220605043 Histone H4-like protein type G_K76A_mutation Human genes 0.000 claims description 2
- 102100026120 IgG receptor FcRn large subunit p51 Human genes 0.000 claims description 2
- 101710177940 IgG receptor FcRn large subunit p51 Proteins 0.000 claims description 2
- 206010061218 Inflammation Diseases 0.000 claims description 2
- 102220477772 Interferon-inducible GTPase 5_K82A_mutation Human genes 0.000 claims description 2
- 206010023201 Joint contracture Diseases 0.000 claims description 2
- 206010028289 Muscle atrophy Diseases 0.000 claims description 2
- 102220587527 Putative uncharacterized protein FLJ13197_K81D_mutation Human genes 0.000 claims description 2
- 102220591431 Relaxin receptor 1_K81A_mutation Human genes 0.000 claims description 2
- 102220481747 Zinc finger SWIM domain-containing protein 3_C66S_mutation Human genes 0.000 claims description 2
- 230000003542 behavioural effect Effects 0.000 claims description 2
- 230000017531 blood circulation Effects 0.000 claims description 2
- 102220371048 c.225A>C Human genes 0.000 claims description 2
- 102200087803 c.241A>G Human genes 0.000 claims description 2
- 230000019771 cognition Effects 0.000 claims description 2
- 208000010877 cognitive disease Diseases 0.000 claims description 2
- 238000012258 culturing Methods 0.000 claims description 2
- 229960001145 deflazacort Drugs 0.000 claims description 2
- FBHSPRKOSMHSIF-GRMWVWQJSA-N deflazacort Chemical compound C1CC2=CC(=O)C=C[C@]2(C)[C@@H]2[C@@H]1[C@@H]1C[C@H]3OC(C)=N[C@@]3(C(=O)COC(=O)C)[C@@]1(C)C[C@@H]2O FBHSPRKOSMHSIF-GRMWVWQJSA-N 0.000 claims description 2
- 230000003111 delayed effect Effects 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 230000004064 dysfunction Effects 0.000 claims description 2
- 239000012634 fragment Substances 0.000 claims description 2
- 238000001415 gene therapy Methods 0.000 claims description 2
- 210000004408 hybridoma Anatomy 0.000 claims description 2
- 230000004054 inflammatory process Effects 0.000 claims description 2
- 230000000968 intestinal effect Effects 0.000 claims description 2
- 238000007918 intramuscular administration Methods 0.000 claims description 2
- 208000028867 ischemia Diseases 0.000 claims description 2
- 230000004199 lung function Effects 0.000 claims description 2
- 230000009756 muscle regeneration Effects 0.000 claims description 2
- 201000000585 muscular atrophy Diseases 0.000 claims description 2
- 229960004618 prednisone Drugs 0.000 claims description 2
- XOFYZVNMUHMLCC-ZPOLXVRWSA-N prednisone Chemical compound O=C1C=C[C@]2(C)[C@H]3C(=O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 XOFYZVNMUHMLCC-ZPOLXVRWSA-N 0.000 claims description 2
- 102000005962 receptors Human genes 0.000 claims description 2
- 210000001139 rectus abdominis Anatomy 0.000 claims description 2
- 230000000241 respiratory effect Effects 0.000 claims description 2
- 102220017540 rs118203356 Human genes 0.000 claims description 2
- 102220311820 rs1376867262 Human genes 0.000 claims description 2
- 102220269398 rs1555403432 Human genes 0.000 claims description 2
- 102200058951 rs17560 Human genes 0.000 claims description 2
- 102220050493 rs193921030 Human genes 0.000 claims description 2
- 102220077293 rs193922450 Human genes 0.000 claims description 2
- 102220226509 rs63750437 Human genes 0.000 claims description 2
- 102200105315 rs748277951 Human genes 0.000 claims description 2
- 102200065571 rs762020512 Human genes 0.000 claims description 2
- 102200127051 rs9511510 Human genes 0.000 claims description 2
- 206010039722 scoliosis Diseases 0.000 claims description 2
- 230000007103 stamina Effects 0.000 claims description 2
- 230000000699 topical effect Effects 0.000 claims description 2
- 229940121710 HMGCoA reductase inhibitor Drugs 0.000 claims 2
- 102000007056 Recombinant Fusion Proteins Human genes 0.000 claims 2
- 108010008281 Recombinant Fusion Proteins Proteins 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662303954P | 2016-03-04 | 2016-03-04 | |
| US62/303,954 | 2016-03-04 | ||
| PCT/US2017/020723 WO2017152090A2 (en) | 2016-03-04 | 2017-03-03 | Recombinant follistatin-fc fusion proteins and use in treating duchenne muscular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019509735A JP2019509735A (ja) | 2019-04-11 |
| JP2019509735A5 true JP2019509735A5 (enExample) | 2020-04-09 |
Family
ID=58358939
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018546471A Pending JP2019509735A (ja) | 2016-03-04 | 2017-03-03 | デュシェンヌ型筋ジストロフィー治療における組換えフォリスタチン−Ｆｃ融合タンパク質及び使用 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20200247856A1 (enExample) |
| EP (1) | EP3423479A2 (enExample) |
| JP (1) | JP2019509735A (enExample) |
| KR (1) | KR20180137487A (enExample) |
| CN (1) | CN109153708A (enExample) |
| AU (1) | AU2017228475A1 (enExample) |
| BR (1) | BR112018067747A2 (enExample) |
| CA (1) | CA3016576A1 (enExample) |
| EA (1) | EA201891990A1 (enExample) |
| IL (1) | IL261397A (enExample) |
| MA (1) | MA43588A (enExample) |
| MX (1) | MX2018010470A (enExample) |
| SG (1) | SG11201807400TA (enExample) |
| WO (1) | WO2017152090A2 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20180362604A1 (en) * | 2017-05-12 | 2018-12-20 | Shire Human Genetic Therapies, Inc. | Recombinant Follistatin-FC Fusion Proteins and Use in Treating Duchenne Muscular Dystrophy |
| TW201936201A (zh) | 2017-12-14 | 2019-09-16 | 美商堅固生物科技公司 | 基因之非病毒生產及遞送 |
| CN108303860B (zh) * | 2018-03-26 | 2019-11-08 | 西安电子科技大学 | 一种用于无掩模光刻扫描的分布式曝光方法 |
| WO2019191204A1 (en) * | 2018-03-28 | 2019-10-03 | Acceleron Pharma Inc. | Follistatin polypeptides for the treatment of muscle contracture |
| AU2020216361A1 (en) | 2019-01-29 | 2021-08-12 | Takeda Pharmaceutical Company Limited | Parathyroid hormone variants |
| EP4540273A1 (en) | 2022-06-15 | 2025-04-23 | UCB Biopharma SRL | Fusion protein for the prevention, treatment or amelioration of kidney diseases |
| JP2025520393A (ja) | 2022-06-15 | 2025-07-03 | ユーシービー バイオファルマ エスアールエル | フォリスタチン－ｆｃ融合タンパク質 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6277375B1 (en) | 1997-03-03 | 2001-08-21 | Board Of Regents, The University Of Texas System | Immunoglobulin-like domains with increased half-lives |
| PT1355919E (pt) | 2000-12-12 | 2011-03-02 | Medimmune Llc | Moléculas com semivida longa, composições que as contêm e suas utilizações |
| MXPA04008150A (es) * | 2002-02-21 | 2005-06-17 | Wyeth Corp | Gasp1: una proteina que contiene dominio de folistatina. |
| EP1896503B1 (en) | 2005-05-31 | 2014-10-29 | Board of Regents, The University of Texas System | IgG1 ANTIBODIES WITH MUTATED Fc PORTION FOR INCREASED BINDING TO FcRn RECEPTOR AND USES TEHEREOF |
| WO2008030367A2 (en) * | 2006-09-01 | 2008-03-13 | The General Hospital Corporation | Selective myostatin inhibitors |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| KR20160044598A (ko) * | 2011-03-29 | 2016-04-25 | 로슈 글리카트 아게 | 항체 Fc 변이체 |
| WO2013192131A1 (en) * | 2012-06-21 | 2013-12-27 | Indiana University Research And Technology Corporation | Incretin receptor ligand polypeptide fc-region fusion polypeptides and conjugates with altered fc-effector function |
| EP3626735A1 (en) * | 2012-12-18 | 2020-03-25 | Novartis AG | Stabilized insulin-like growth factor polypeptides |
| JP6475639B2 (ja) * | 2013-01-25 | 2019-02-27 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | デュシェンヌ筋ジストロフィーの治療におけるフォリスタチン |
| WO2014187807A1 (en) * | 2013-05-21 | 2014-11-27 | Arcarios B.V. | Follistatin derivatives |
| WO2015187977A1 (en) * | 2014-06-04 | 2015-12-10 | Acceleron Pharma, Inc. | Methods and compositions for treatment of disorders with follistatin polypeptides |
-
2017
- 2017-03-03 KR KR1020187028662A patent/KR20180137487A/ko not_active Withdrawn
- 2017-03-03 EP EP17711939.3A patent/EP3423479A2/en not_active Ceased
- 2017-03-03 CN CN201780025363.2A patent/CN109153708A/zh active Pending
- 2017-03-03 EA EA201891990A patent/EA201891990A1/ru unknown
- 2017-03-03 CA CA3016576A patent/CA3016576A1/en not_active Abandoned
- 2017-03-03 MX MX2018010470A patent/MX2018010470A/es unknown
- 2017-03-03 SG SG11201807400TA patent/SG11201807400TA/en unknown
- 2017-03-03 US US16/082,070 patent/US20200247856A1/en not_active Abandoned
- 2017-03-03 WO PCT/US2017/020723 patent/WO2017152090A2/en not_active Ceased
- 2017-03-03 MA MA043588A patent/MA43588A/fr unknown
- 2017-03-03 AU AU2017228475A patent/AU2017228475A1/en not_active Abandoned
- 2017-03-03 JP JP2018546471A patent/JP2019509735A/ja active Pending
- 2017-03-03 BR BR112018067747A patent/BR112018067747A2/pt not_active Application Discontinuation
-
2018
- 2018-08-27 IL IL261397A patent/IL261397A/en unknown
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