JP2020519291A5 - - Google Patents
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- JP2020519291A5 JP2020519291A5 JP2019562409A JP2019562409A JP2020519291A5 JP 2020519291 A5 JP2020519291 A5 JP 2020519291A5 JP 2019562409 A JP2019562409 A JP 2019562409A JP 2019562409 A JP2019562409 A JP 2019562409A JP 2020519291 A5 JP2020519291 A5 JP 2020519291A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- recombinant follistatin
- fusion protein
- amino acid
- item
- 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 150
- 108010014612 Follistatin Proteins 0.000 claims description 150
- 108020001507 fusion proteins Proteins 0.000 claims description 86
- 102000037865 fusion proteins Human genes 0.000 claims description 86
- 229920001184 polypeptide Polymers 0.000 claims description 56
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 56
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 56
- 238000000034 method Methods 0.000 claims description 48
- 150000001413 amino acids Chemical class 0.000 claims description 47
- 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
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 25
- 210000003205 muscle Anatomy 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 14
- 102000004169 proteins and genes Human genes 0.000 claims description 14
- 238000007920 subcutaneous administration Methods 0.000 claims description 14
- 238000006467 substitution reaction Methods 0.000 claims description 14
- 238000001990 intravenous administration Methods 0.000 claims description 13
- 125000000539 amino acid group Chemical group 0.000 claims description 12
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 9
- 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 8
- 238000010494 dissociation reaction Methods 0.000 claims description 8
- 230000005593 dissociations Effects 0.000 claims description 8
- 229960002897 heparin Drugs 0.000 claims description 8
- 229920000669 heparin Polymers 0.000 claims description 8
- 102100039939 Growth/differentiation factor 8 Human genes 0.000 claims description 7
- 108010056852 Myostatin Proteins 0.000 claims description 7
- 239000008194 pharmaceutical composition Substances 0.000 claims description 7
- 102000040430 polynucleotide Human genes 0.000 claims description 7
- 239000002157 polynucleotide Substances 0.000 claims description 7
- 108091033319 polynucleotide Proteins 0.000 claims description 7
- 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
- 239000003814 drug Substances 0.000 claims description 6
- 238000007911 parenteral administration Methods 0.000 claims description 6
- 229940124597 therapeutic agent Drugs 0.000 claims description 6
- 239000000203 mixture Substances 0.000 claims description 5
- 210000002027 skeletal muscle Anatomy 0.000 claims description 5
- 102220639700 Leptin_R78L_mutation Human genes 0.000 claims description 4
- 108010023082 activin A Proteins 0.000 claims description 4
- 210000004027 cell Anatomy 0.000 claims description 4
- 239000013604 expression vector Substances 0.000 claims description 4
- 230000007935 neutral effect Effects 0.000 claims description 4
- 230000004064 dysfunction Effects 0.000 claims description 3
- 239000002773 nucleotide Substances 0.000 claims description 3
- 125000003729 nucleotide group Chemical group 0.000 claims description 3
- 230000012743 protein tagging Effects 0.000 claims description 3
- 208000024891 symptom Diseases 0.000 claims description 3
- 208000008037 Arthrogryposis Diseases 0.000 claims description 2
- 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
- 206010023509 Kyphosis Diseases 0.000 claims description 2
- 208000010428 Muscle Weakness Diseases 0.000 claims description 2
- 206010028289 Muscle atrophy Diseases 0.000 claims description 2
- 206010028372 Muscular weakness Diseases 0.000 claims description 2
- -1 Pamrebulmab Chemical compound 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
- 230000006741 behavioral dysfunction 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
- 239000003937 drug carrier Substances 0.000 claims description 2
- 229950002185 edasalonexent Drugs 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
- 238000007913 intrathecal administration Methods 0.000 claims description 2
- 208000028867 ischemia Diseases 0.000 claims description 2
- 230000004199 lung function Effects 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 206010028320 muscle necrosis Diseases 0.000 claims description 2
- 230000009756 muscle regeneration Effects 0.000 claims description 2
- 201000000585 muscular atrophy Diseases 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- JQLBBYLGWHUHRW-KUBAVDMBSA-N n-[2-[[(4z,7z,10z,13z,16z,19z)-docosa-4,7,10,13,16,19-hexaenoyl]amino]ethyl]-2-hydroxybenzamide Chemical compound CC\C=C/C\C=C/C\C=C/C\C=C/C\C=C/C\C=C/CCC(=O)NCCNC(=O)C1=CC=CC=C1O JQLBBYLGWHUHRW-KUBAVDMBSA-N 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
- 230000007103 stamina Effects 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims 2
- GOJUJUVQIVIZAV-UHFFFAOYSA-N 2-amino-4,6-dichloropyrimidine-5-carbaldehyde Chemical group NC1=NC(Cl)=C(C=O)C(Cl)=N1 GOJUJUVQIVIZAV-UHFFFAOYSA-N 0.000 claims 1
- 230000007423 decrease Effects 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 230000001934 delay Effects 0.000 claims 1
- 210000003314 quadriceps muscle Anatomy 0.000 claims 1
- 108010059616 Activins Proteins 0.000 description 1
- 102100026818 Inhibin beta E chain Human genes 0.000 description 1
- 239000000488 activin Substances 0.000 description 1
- 230000003111 delayed effect Effects 0.000 description 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1
- 208000035475 disorder Diseases 0.000 description 1
- 230000002401 inhibitory effect Effects 0.000 description 1
- 230000000977 initiatory effect Effects 0.000 description 1
- 230000001105 regulatory effect Effects 0.000 description 1
- 230000001629 suppression Effects 0.000 description 1
- 230000004584 weight gain Effects 0.000 description 1
- 235000019786 weight gain Nutrition 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762505642P | 2017-05-12 | 2017-05-12 | |
| US62/505,642 | 2017-05-12 | ||
| US201862618376P | 2018-01-17 | 2018-01-17 | |
| US62/618,376 | 2018-01-17 | ||
| PCT/US2018/032332 WO2018209242A1 (en) | 2017-05-12 | 2018-05-11 | Recombinant follistatin-fc fusion proteins and use in treating duchenne muscular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020519291A JP2020519291A (ja) | 2020-07-02 |
| JP2020519291A5 true JP2020519291A5 (enExample) | 2021-07-26 |
Family
ID=62599687
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019562409A Pending JP2020519291A (ja) | 2017-05-12 | 2018-05-11 | 組み換えフォリスタチン−ｆｃ融合タンパク質およびデュシェンヌ型筋ジストロフィーの治療における使用 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20180362604A1 (enExample) |
| EP (1) | EP3621986A1 (enExample) |
| JP (1) | JP2020519291A (enExample) |
| CN (1) | CN110914294A (enExample) |
| AU (1) | AU2018266893A1 (enExample) |
| BR (1) | BR112019023860A2 (enExample) |
| MX (1) | MX2019013523A (enExample) |
| WO (1) | WO2018209242A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN106795224B (zh) | 2014-06-04 | 2021-05-18 | 阿塞勒隆制药公司 | 用促滤泡素抑制素多肽治疗病症的方法和组合物 |
| US10010498B2 (en) | 2014-06-04 | 2018-07-03 | Acceleron Pharma Inc. | Methods for treatment of amyotrophic lateral sclerosis with follistatin fusion proteins |
| WO2019191204A1 (en) * | 2018-03-28 | 2019-10-03 | Acceleron Pharma Inc. | Follistatin polypeptides for the treatment of muscle contracture |
| WO2021216372A1 (en) * | 2020-04-23 | 2021-10-28 | Eli Lilly And Company | Subcutaneous absorption and bioavailability of antibodies |
| WO2021222875A1 (en) | 2020-04-30 | 2021-11-04 | Keros Therapeutics, Inc. | Methods of using alk2 inhibitors |
| CA3094859A1 (en) * | 2020-10-01 | 2022-04-01 | Entos Pharmaceuticals Inc. | Proteolipid vesicles formulated with fusion associated small transmembrane proteins |
| WO2022098812A1 (en) | 2020-11-04 | 2022-05-12 | Keros Therapeutics, Inc. | Methods of treating iron overload |
Family Cites Families (8)
| 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 |
| CA2431600C (en) | 2000-12-12 | 2012-04-17 | Medimmune, Inc. | Molecules with extended half-lives, compositions and uses thereof |
| 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 |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| TW201817744A (zh) * | 2011-09-30 | 2018-05-16 | 日商中外製藥股份有限公司 | 具有促進抗原清除之ＦｃＲｎ結合域的治療性抗原結合分子 |
| EP2948161B1 (en) * | 2013-01-25 | 2018-12-12 | Shire Human Genetic Therapies, Inc. | Follistatin in treating duchenne muscular dystrophy |
| WO2014187807A1 (en) * | 2013-05-21 | 2014-11-27 | Arcarios B.V. | Follistatin derivatives |
| CN109153708A (zh) * | 2016-03-04 | 2019-01-04 | 夏尔人类遗传性治疗公司 | 重组卵泡抑素-fc融合蛋白及其在治疗杜氏肌营养不良症中的应用 |
-
2018
- 2018-05-11 EP EP18731246.7A patent/EP3621986A1/en not_active Withdrawn
- 2018-05-11 CN CN201880045571.3A patent/CN110914294A/zh active Pending
- 2018-05-11 AU AU2018266893A patent/AU2018266893A1/en not_active Abandoned
- 2018-05-11 MX MX2019013523A patent/MX2019013523A/es unknown
- 2018-05-11 US US15/977,635 patent/US20180362604A1/en not_active Abandoned
- 2018-05-11 BR BR112019023860-3A patent/BR112019023860A2/pt not_active IP Right Cessation
- 2018-05-11 WO PCT/US2018/032332 patent/WO2018209242A1/en not_active Ceased
- 2018-05-11 JP JP2019562409A patent/JP2020519291A/ja active Pending
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