JP2019509735A5 - - Google Patents
Download PDFInfo
- 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 |
|---|---|---|---|---|
| AU2018266893A1 (en) * | 2017-05-12 | 2019-12-05 | Takeda Pharmaceutical Company Limited | 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 |
| KR20210121137A (ko) | 2019-01-29 | 2021-10-07 | 샤이어-엔피에스 파마슈티칼즈, 인크. | 부갑상선 호르몬 변이체 |
| KR20250024825A (ko) | 2022-06-15 | 2025-02-19 | 유씨비 바이오파마 에스알엘 | 폴리스타틴-fc 융합 단백질 |
| CA3259309A1 (en) | 2022-06-15 | 2023-12-21 | UCB Biopharma SRL | Fusion protein for the prevention, treatment or improvement of kidney disease |
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 |
| EP1355919B1 (en) | 2000-12-12 | 2010-11-24 | MedImmune, LLC | Molecules with extended half-lives, compositions and uses thereof |
| BR0307907A2 (pt) * | 2002-02-21 | 2011-07-05 | Wyeth Corp | gasp1: proteìna contendo domìnio de folistatina |
| WO2006130834A2 (en) | 2005-05-31 | 2006-12-07 | Board Of Regents, The University Of Texas System | IGGl ANTIBODIES WITH MUTATED FC PORTION FOR INCREASED BINDING TO FCRN RECEPTOR AND USES THEREOF |
| 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 |
| LT2691417T (lt) * | 2011-03-29 | 2018-10-25 | Roche Glycart Ag | Antikūno fc variantai |
| MX2014015557A (es) * | 2012-06-21 | 2015-02-24 | Univ Indiana Res & Tech Corp | Polipeptidos de fusion de region fc de polipeptido de ligando receptor de incretina y conjugados con función efectora fc alterada. |
| SG11201503782PA (en) * | 2012-12-18 | 2015-06-29 | Novartis Ag | Stabilized insulin-like growth factor polypeptides |
| CA2898121A1 (en) * | 2013-01-25 | 2014-07-31 | Shire Human Genetic Therapies, Inc. | Follistatin in treating duchenne muscular dystrophy |
| WO2014187807A1 (en) * | 2013-05-21 | 2014-11-27 | Arcarios B.V. | Follistatin derivatives |
| JP6568110B2 (ja) * | 2014-06-04 | 2019-08-28 | アクセルロン ファーマ， インコーポレイテッド | フォリスタチンポリペプチドによる障害の処置のための方法および組成物 |
-
2017
- 2017-03-03 BR BR112018067747A patent/BR112018067747A2/pt not_active Application Discontinuation
- 2017-03-03 US US16/082,070 patent/US20200247856A1/en not_active Abandoned
- 2017-03-03 EP EP17711939.3A patent/EP3423479A2/en not_active Ceased
- 2017-03-03 EA EA201891990A patent/EA201891990A1/ru unknown
- 2017-03-03 MA MA043588A patent/MA43588A/fr unknown
- 2017-03-03 KR KR1020187028662A patent/KR20180137487A/ko not_active Withdrawn
- 2017-03-03 MX MX2018010470A patent/MX2018010470A/es unknown
- 2017-03-03 CN CN201780025363.2A patent/CN109153708A/zh active Pending
- 2017-03-03 SG SG11201807400TA patent/SG11201807400TA/en unknown
- 2017-03-03 WO PCT/US2017/020723 patent/WO2017152090A2/en not_active Ceased
- 2017-03-03 AU AU2017228475A patent/AU2017228475A1/en not_active Abandoned
- 2017-03-03 JP JP2018546471A patent/JP2019509735A/ja active Pending
- 2017-03-03 CA CA3016576A patent/CA3016576A1/en not_active Abandoned
-
2018
- 2018-08-27 IL IL261397A patent/IL261397A/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020519291A5 (enExample) | ||
| JP2014158480A5 (enExample) | ||
| EP3576789B1 (en) | MONOMERIC HUMAN IgG1 Fc AND BISPECIFIC ANTIBODIES | |
| JP2016511751A5 (enExample) | ||
| JP2020509756A5 (enExample) | ||
| JP2021527063A5 (enExample) | ||
| IL292780A (en) | A new 4-1bbl trimer containing antigen-binding molecules | |
| JP2008542278A5 (enExample) | ||
| JP2024527324A5 (enExample) | ||
| JP2019527543A5 (enExample) | ||
| JP2023514407A5 (enExample) | ||
| JP2017101068A5 (enExample) | ||
| JP2021500029A5 (enExample) | ||
| JP2011520793A5 (enExample) | ||
| JP2017533694A5 (enExample) | ||
| JP2017500018A5 (enExample) | ||
| CN107106682A (zh) | 多特异性抗体平台和相关方法 | |
| JP2015501814A5 (enExample) | ||
| JP2016519569A5 (enExample) | ||
| JP2023523401A5 (enExample) | ||
| JP2021520393A5 (enExample) | ||
| JP2022549172A5 (enExample) | ||
| JP2018531622A5 (enExample) | ||
| JP2023530663A5 (enExample) | ||
| JP2022531556A5 (enExample) |