JP2016511751A5 - - Google Patents
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- Publication number
- JP2016511751A5 JP2016511751A5 JP2015555353A JP2015555353A JP2016511751A5 JP 2016511751 A5 JP2016511751 A5 JP 2016511751A5 JP 2015555353 A JP2015555353 A JP 2015555353A JP 2015555353 A JP2015555353 A JP 2015555353A JP 2016511751 A5 JP2016511751 A5 JP 2016511751A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- follistatin
- protein
- seq
- recombinant follistatin
- 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
- 102000016970 Follistatin Human genes 0.000 claims 55
- 108010014612 Follistatin Proteins 0.000 claims 55
- 239000008194 pharmaceutical composition Substances 0.000 claims 40
- 125000003275 alpha amino acid group Chemical group 0.000 claims 22
- 108020001507 fusion proteins Proteins 0.000 claims 22
- 102000037865 fusion proteins Human genes 0.000 claims 22
- 108090000765 processed proteins & peptides Proteins 0.000 claims 13
- 101000931668 Homo sapiens Follistatin Proteins 0.000 claims 11
- 102000050536 human FST Human genes 0.000 claims 11
- 229920001184 polypeptide Polymers 0.000 claims 11
- 102000004196 processed proteins & peptides Human genes 0.000 claims 11
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 5
- 238000007910 systemic administration Methods 0.000 claims 5
- 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 4
- 230000037430 deletion Effects 0.000 claims 3
- 238000012217 deletion Methods 0.000 claims 3
- 210000004962 mammalian cell Anatomy 0.000 claims 3
- 210000003205 muscle Anatomy 0.000 claims 3
- 206010028320 muscle necrosis Diseases 0.000 claims 3
- 230000035772 mutation Effects 0.000 claims 3
- 108010059616 Activins Proteins 0.000 claims 2
- 102100039939 Growth/differentiation factor 8 Human genes 0.000 claims 2
- 102100026818 Inhibin beta E chain Human genes 0.000 claims 2
- 208000010428 Muscle Weakness Diseases 0.000 claims 2
- 206010028372 Muscular weakness Diseases 0.000 claims 2
- 108010056852 Myostatin Proteins 0.000 claims 2
- 101000690429 Panax ginseng Floral homeotic protein AGAMOUS Proteins 0.000 claims 2
- 101000828148 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) Transposon Ty3-G Gag polyprotein Proteins 0.000 claims 2
- 101000790437 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) Transposon Ty3-I Gag polyprotein Proteins 0.000 claims 2
- 239000000488 activin Substances 0.000 claims 2
- 125000000539 amino acid group Chemical group 0.000 claims 2
- 150000001413 amino acids Chemical class 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 230000004064 dysfunction Effects 0.000 claims 2
- 238000001727 in vivo Methods 0.000 claims 2
- 238000001990 intravenous administration Methods 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 210000002027 skeletal muscle Anatomy 0.000 claims 2
- 238000007920 subcutaneous administration Methods 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- 210000001519 tissue Anatomy 0.000 claims 2
- 206010069632 Bladder dysfunction Diseases 0.000 claims 1
- 208000031229 Cardiomyopathies Diseases 0.000 claims 1
- 208000032170 Congenital Abnormalities Diseases 0.000 claims 1
- 206010061619 Deformity Diseases 0.000 claims 1
- 208000019505 Deglutition disease Diseases 0.000 claims 1
- 201000000628 Gas Gangrene Diseases 0.000 claims 1
- 102100040898 Growth/differentiation factor 11 Human genes 0.000 claims 1
- 101710194452 Growth/differentiation factor 11 Proteins 0.000 claims 1
- 206010023201 Joint contracture Diseases 0.000 claims 1
- 206010028289 Muscle atrophy Diseases 0.000 claims 1
- 206010048654 Muscle fibrosis Diseases 0.000 claims 1
- 230000003110 anti-inflammatory effect Effects 0.000 claims 1
- 230000006741 behavioral dysfunction Effects 0.000 claims 1
- 230000017531 blood circulation Effects 0.000 claims 1
- 230000019771 cognition Effects 0.000 claims 1
- 208000010877 cognitive disease Diseases 0.000 claims 1
- 230000003111 delayed effect Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000003203 everyday effect Effects 0.000 claims 1
- 206010016256 fatigue Diseases 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 230000037431 insertion Effects 0.000 claims 1
- 238000003780 insertion Methods 0.000 claims 1
- 230000000968 intestinal effect Effects 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 230000001788 irregular Effects 0.000 claims 1
- 208000028867 ischemia Diseases 0.000 claims 1
- 230000009756 muscle regeneration Effects 0.000 claims 1
- 201000000585 muscular atrophy Diseases 0.000 claims 1
- 230000017074 necrotic cell death Effects 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 230000009325 pulmonary function Effects 0.000 claims 1
- 210000001139 rectus abdominis Anatomy 0.000 claims 1
- 230000000241 respiratory effect Effects 0.000 claims 1
- 206010039722 scoliosis Diseases 0.000 claims 1
- 230000000699 topical effect Effects 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361756996P | 2013-01-25 | 2013-01-25 | |
| US61/756,996 | 2013-01-25 | ||
| US201361915733P | 2013-12-13 | 2013-12-13 | |
| US61/915,733 | 2013-12-13 | ||
| PCT/US2014/012996 WO2014116981A1 (en) | 2013-01-25 | 2014-01-24 | Follistatin in treating duchenne muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018203613A Division JP2019059730A (ja) | 2013-01-25 | 2018-10-30 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016511751A JP2016511751A (ja) | 2016-04-21 |
| JP2016511751A5 true JP2016511751A5 (enExample) | 2017-02-23 |
| JP6475639B2 JP6475639B2 (ja) | 2019-02-27 |
Family
ID=50159515
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015555353A Expired - Fee Related JP6475639B2 (ja) | 2013-01-25 | 2014-01-24 | 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 |
| JP2018203613A Pending JP2019059730A (ja) | 2013-01-25 | 2018-10-30 | 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018203613A Pending JP2019059730A (ja) | 2013-01-25 | 2018-10-30 | 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 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US9957309B2 (enExample) |
| EP (1) | EP2948161B1 (enExample) |
| JP (2) | JP6475639B2 (enExample) |
| KR (1) | KR102253597B1 (enExample) |
| CN (1) | CN104902915B (enExample) |
| AU (2) | AU2014209178B2 (enExample) |
| CA (1) | CA2898121A1 (enExample) |
| EA (1) | EA201590719A1 (enExample) |
| ES (1) | ES2715710T3 (enExample) |
| IL (1) | IL240024B (enExample) |
| MX (1) | MX362105B (enExample) |
| WO (1) | WO2014116981A1 (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8128933B2 (en) | 2005-11-23 | 2012-03-06 | Acceleron Pharma, Inc. | Method of promoting bone growth by an anti-activin B antibody |
| TWI782836B (zh) | 2007-02-02 | 2022-11-01 | 美商艾瑟勒朗法瑪公司 | 衍生自ActRIIB的變體與其用途 |
| US20100008918A1 (en) | 2008-06-26 | 2010-01-14 | Acceleron Pharma Inc. | Methods for dosing an actriib antagonist and monitoring of treated patients |
| US8216997B2 (en) | 2008-08-14 | 2012-07-10 | Acceleron Pharma, Inc. | Methods for increasing red blood cell levels and treating anemia using a combination of GDF traps and erythropoietin receptor activators |
| 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 |
| EP2971039B1 (en) | 2013-03-14 | 2020-01-01 | Immusoft Corporation | Methods for in vitro memory b cell differentiation and transduction with vsv-g pseudotyped viral vectors |
| WO2015187977A1 (en) | 2014-06-04 | 2015-12-10 | Acceleron Pharma, Inc. | Methods and compositions for treatment of disorders with follistatin polypeptides |
| US10010498B2 (en) | 2014-06-04 | 2018-07-03 | Acceleron Pharma Inc. | Methods for treatment of amyotrophic lateral sclerosis with follistatin fusion proteins |
| WO2016154601A1 (en) * | 2015-03-26 | 2016-09-29 | Acceleron Pharma Inc. | Follistatin-related fusion proteins and uses thereof |
| EP4233887A1 (en) * | 2016-01-06 | 2023-08-30 | Stealth BioTherapeutics Inc. | Methods for the treatment of duchenne muscular dystrophy |
| KR20180137487A (ko) * | 2016-03-04 | 2018-12-27 | 샤이어 휴먼 지네틱 테라피즈 인크. | 재조합 폴리스타틴-fc 융합 단백질 및 듀시엔형 근이영양증 치료에서의 용도 |
| 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 |
| KR101822633B1 (ko) * | 2017-08-22 | 2018-01-26 | (주)진셀팜 | 폴리스타틴 유래 생리활성 펩타이드, 및 이의 용도 |
| WO2019046903A1 (en) * | 2017-09-08 | 2019-03-14 | Baker Heart and Diabetes Institute | THERAPEUTIC METHOD FOR INCREASING MUSCLE MASS IN A SUBJECT |
| JP2021515576A (ja) * | 2018-03-16 | 2021-06-24 | イミュソフト コーポレーション | フォリスタチンを分泌するように遺伝子操作されたb細胞ならびにフォリスタチン関連疾患、状態、障害を処置するために、ならびに筋肉の成長および強度を増強するためにこれを使用する方法 |
| WO2019191204A1 (en) * | 2018-03-28 | 2019-10-03 | Acceleron Pharma Inc. | Follistatin polypeptides for the treatment of muscle contracture |
| CA3094859A1 (en) * | 2020-10-01 | 2022-04-01 | Entos Pharmaceuticals Inc. | Proteolipid vesicles formulated with fusion associated small transmembrane proteins |
| KR102361657B1 (ko) | 2021-04-26 | 2022-02-14 | 주식회사 케이에스비튜젠 | 14-디옥시-11,12-디디히드로안드로그라폴라이드 석시네이트를 유효성분으로 함유하는 듀센형 근이영양증을 포함한 유전성 근육질환의 예방 또는 치료용 조성물 |
Family Cites Families (18)
| 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 |
| MXPA04008149A (es) | 2002-02-21 | 2005-06-17 | Wyeth Corp | Proteinas que contienen dominios de folistatina. |
| WO2005033134A2 (en) * | 2003-09-30 | 2005-04-14 | Regeneron Pharmaceuticals, Inc. | Secreted protein therapeutics and uses thereof |
| JP4688483B2 (ja) * | 2004-04-15 | 2011-05-25 | 株式会社テクノネットワーク四国 | フォリスタチン変異体ポリペプチド |
| 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 |
| NZ590326A (en) * | 2008-06-26 | 2013-05-31 | Acceleron Pharma Inc | Antagonists of activin-actriia and uses for increasing red blood cell levels |
| US20100008918A1 (en) | 2008-06-26 | 2010-01-14 | Acceleron Pharma Inc. | Methods for dosing an actriib antagonist and monitoring of treated patients |
| CA2773494A1 (en) * | 2009-09-09 | 2011-03-17 | Acceleron Pharma Inc. | Actriib antagonists and dosing and uses thereof |
| US9023890B2 (en) * | 2009-12-02 | 2015-05-05 | The Charlotte-Mecklenburg Hospital Authority | Nitrate esters and their use for the treatment of muscle and muscle related diseases |
| US8580922B2 (en) | 2011-03-04 | 2013-11-12 | Shire Human Genetic Therapies, Inc. | Peptide linkers for polypeptide compositions and methods for using same |
| WO2013186719A1 (en) | 2012-06-15 | 2013-12-19 | Pfizer Inc. | Improved antagonist antibodies against gdf-8 and uses therefor |
| LT3835310T (lt) | 2012-09-13 | 2024-04-25 | Bristol-Myers Squibb Company | Fibronektino pagrindo karkaso domeno baltymai, kurie rišasi prie miostatino |
| 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 |
| WO2016057975A2 (en) | 2014-10-10 | 2016-04-14 | Research Institute At Nationwide Children's Hospital | Guided injections for aav gene transfer to muscle |
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2014
- 2014-01-24 JP JP2015555353A patent/JP6475639B2/ja not_active Expired - Fee Related
- 2014-01-24 AU AU2014209178A patent/AU2014209178B2/en not_active Ceased
- 2014-01-24 KR KR1020157016986A patent/KR102253597B1/ko not_active Expired - Fee Related
- 2014-01-24 MX MX2015009631A patent/MX362105B/es active IP Right Grant
- 2014-01-24 WO PCT/US2014/012996 patent/WO2014116981A1/en not_active Ceased
- 2014-01-24 CA CA2898121A patent/CA2898121A1/en not_active Abandoned
- 2014-01-24 US US14/762,373 patent/US9957309B2/en not_active Expired - Fee Related
- 2014-01-24 EP EP14706362.2A patent/EP2948161B1/en active Active
- 2014-01-24 EA EA201590719A patent/EA201590719A1/ru unknown
- 2014-01-24 CN CN201480003715.0A patent/CN104902915B/zh not_active Expired - Fee Related
- 2014-01-24 ES ES14706362T patent/ES2715710T3/es active Active
-
2015
- 2015-07-20 IL IL240024A patent/IL240024B/en not_active IP Right Cessation
-
2018
- 2018-04-12 US US15/951,929 patent/US10723772B2/en not_active Expired - Fee Related
- 2018-10-30 JP JP2018203613A patent/JP2019059730A/ja active Pending
-
2019
- 2019-02-06 AU AU2019200797A patent/AU2019200797B2/en not_active Ceased