JP2020015733A5 - - Google Patents
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- JP2020015733A5 JP2020015733A5 JP2019149672A JP2019149672A JP2020015733A5 JP 2020015733 A5 JP2020015733 A5 JP 2020015733A5 JP 2019149672 A JP2019149672 A JP 2019149672A JP 2019149672 A JP2019149672 A JP 2019149672A JP 2020015733 A5 JP2020015733 A5 JP 2020015733A5
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- JP
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- Prior art keywords
- pharmaceutical composition
- composition according
- polypeptide
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- fgfr3
- Prior art date
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- 229920001184 polypeptide Polymers 0.000 claims description 29
- 239000008194 pharmaceutical composition Substances 0.000 claims description 20
- 102100020191 FGFR3 Human genes 0.000 claims description 16
- 108010081267 Type 3 Fibroblast Growth Factor Receptor Proteins 0.000 claims description 16
- 208000008919 Achondroplasia Diseases 0.000 claims description 8
- 206010008723 Chondrodystrophy Diseases 0.000 claims description 8
- 125000000511 arginine group Chemical group N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims description 2
- 125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 claims description 2
- 150000001413 amino acids Chemical class 0.000 claims 2
- 230000037396 body weight Effects 0.000 claims 2
- 210000000845 Cartilage Anatomy 0.000 claims 1
- 210000001612 Chondrocytes Anatomy 0.000 claims 1
- 210000004349 Growth Plate Anatomy 0.000 claims 1
- 102000018358 Immunoglobulins Human genes 0.000 claims 1
- 108060003951 Immunoglobulins Proteins 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 230000001747 exhibiting Effects 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 239000011159 matrix material Substances 0.000 claims 1
- 239000012466 permeate Substances 0.000 claims 1
- 238000007920 subcutaneous administration Methods 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 230000002588 toxic Effects 0.000 claims 1
- 231100000331 toxic Toxicity 0.000 claims 1
- 201000010099 disease Diseases 0.000 description 13
- 206010053759 Growth retardation Diseases 0.000 description 8
- 231100000001 growth retardation Toxicity 0.000 description 8
- 210000000988 Bone and Bones Anatomy 0.000 description 5
- 230000002265 prevention Effects 0.000 description 4
- 239000003937 drug carrier Substances 0.000 description 3
- 206010058314 Dysplasia Diseases 0.000 description 2
- 208000003351 Melanosis Diseases 0.000 description 2
- 208000007326 Muenke Syndrome Diseases 0.000 description 2
- 239000004475 Arginine Substances 0.000 description 1
- 101700008564 CHIC2 Proteins 0.000 description 1
- 201000006526 Crouzon syndrome Diseases 0.000 description 1
- 102100018000 FGFR2 Human genes 0.000 description 1
Description
本発明はまた、治療的有効量のｓＦＧＦＲ３ポリペプチドまたはその機能的等価体および薬学的に許容される担体を含む医薬組成物を、それを必要とする対象に投与する工程を含む、骨格成長遅延障害の予防または処置のための方法を提供する。
本発明のさらなる態様を、以下に記載する：
［項１］
骨格成長遅延障害の予防または処置における使用のための、単離された可溶性線維芽細胞増殖因子受容体３（ｓＦＧＦＲ３）ポリペプチドまたはその機能的等価体。
［項２］
骨格成長遅延障害が特発性発育遅延障害である、項１に記載のポリペプチド。
［項３］
骨格成長遅延障害がＦＧＦＲ３関連骨系統疾患である、項１に記載のポリペプチド。
［項４］
ＦＧＦＲ３関連骨系統疾患が、タナトフォリック骨異形成症Ｉ型、タナトフォリック骨異形成症ＩＩ型、発育遅延および黒色表皮症を伴う重度の軟骨無形成症、軟骨低形成症、軟骨無形成症およびＦＧＦＲ３関連頭蓋骨縫合早期癒合症、例えばミュエンク症候群（Muenke syndrome）および黒色表皮症を伴うクルーゾン症候群、からなる群より選択される、項３に記載のポリペプチド。
［項５］
ＦＧＦＲ３関連骨系統疾患が軟骨無形成症である、項４に記載のポリペプチド。
［項６］
ＦＧＦＲ３関連骨系統疾患が、対象における構成的に活性化されたＦＧＦＲ３受容体変異体の発現に起因する、項３〜５のいずれか一項に記載のポリペプチド。
［項７］
ＦＧＦＲ３関連骨系統疾患が軟骨無形成症であり、構成的に活性化されたＦＧＦＲ３受容体変異体が、３８０位のグリシン残基がアルギニンで置換されている（すなわち、Ｇ３８０Ｒである）変異体である、項６に記載のポリペプチド。
［項８］
ポリペプチドが、配列番号１で定義されるポリペプチド配列によりコードされる、項１〜７のいずれか一項に記載のポリペプチド。
［項９］
ポリペプチドが、配列番号２で定義されるポリペプチド配列によりコードされる、項８に記載のポリペプチド。
［項１０］
単離されたｓＦＧＦＲ３ポリペプチドまたはその機能的等価体および薬学的に許容される担体を含む、医薬組成物。
［項１１］
単離されたｓＦＧＦＲ３ポリペプチドまたはその機能的等価体および薬学的に許容される担体を含む、骨格成長遅延障害の予防または処置における使用のための、医薬組成物。
［項１２］
治療的有効量のｓＦＧＦＲ３ポリペプチドまたはかかるポリペプチドを含む医薬組成物を、それを必要とする対象に投与する工程を含む、骨格成長遅延障害の予防または処置のための方法。
The present invention also comprises the step of administering to a subject in need of a pharmaceutical composition comprising a therapeutically effective amount of the sFGFR3 polypeptide or functional equivalent thereof and a pharmaceutically acceptable carrier, skeletal growth retardation. Provide methods for the prevention or treatment of disorders.
Further aspects of the invention are described below:
[Item 1]
An isolated soluble fibroblast growth factor receptor 3 (sFGFR3) polypeptide or functional equivalent thereof for use in the prevention or treatment of skeletal growth retardation disorders.
[Item 2]
Item 2. The polypeptide according to Item 1, wherein the skeletal growth retardation disorder is an idiopathic growth retardation disorder.
[Item 3]
Item 2. The polypeptide according to Item 1, wherein the skeletal growth retardation disorder is a FGFR3-related bone system disease.
[Item 4]
FGFR3-related bone system diseases are tanatophoric dysplasia type I, tanatophoric dysplasia type II, severe achondroplasia with growth retardation and melanosis, achondroplasia, achondroplasia Item 3. The polypeptide according to Item 3, wherein the polypeptide is selected from the group consisting of disease and FGFR3-related craniosynostosis, eg, Muenke syndrome and Crouzon syndrome with melanosis.
[Item 5]
Item 4. The polypeptide according to Item 4, wherein the FGFR3-related bone system disease is achondroplasia.
[Item 6]
Item 8. The polypeptide according to any one of Items 3 to 5, wherein the FGFR3-related bone system disease is caused by the expression of a constitutively activated FGFR3 receptor mutant in a subject.
[Item 7]
The FGFR3-related bone system disease is achondroplasia, and the constitutively activated FGFR3 receptor mutant is a mutant in which the glycine residue at position 380 is replaced with arginine (that is, G380R). Item 6. The polypeptide according to Item 6.
[Item 8]
Item 8. The polypeptide according to any one of Items 1 to 7, wherein the polypeptide is encoded by the polypeptide sequence defined in SEQ ID NO: 1.
[Item 9]
Item 8. The polypeptide according to Item 8, wherein the polypeptide is encoded by the polypeptide sequence defined in SEQ ID NO: 2.
[Item 10]
A pharmaceutical composition comprising an isolated sFGFR3 polypeptide or functional equivalent thereof and a pharmaceutically acceptable carrier.
[Item 11]
A pharmaceutical composition for use in the prevention or treatment of skeletal growth retardation disorders, comprising an isolated sFGFR3 polypeptide or functional equivalent thereof and a pharmaceutically acceptable carrier.
[Item 12]
A method for the prevention or treatment of skeletal growth retardation disorders, comprising the step of administering a therapeutically effective amount of a sFGFR3 polypeptide or a pharmaceutical composition comprising such a polypeptide to a subject in need thereof.
Claims (13)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019149672A JP2020015733A (en) | 2019-08-19 | 2019-08-19 | Soluble fibroblast growth factor receptor 3 (fgr3) polypeptide for use in prevention or treatment of skeletal growth retardation disorders |
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019149672A JP2020015733A (en) | 2019-08-19 | 2019-08-19 | Soluble fibroblast growth factor receptor 3 (fgr3) polypeptide for use in prevention or treatment of skeletal growth retardation disorders |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015553175A Division JP2016506914A (en) | 2013-01-16 | 2013-01-16 | Soluble fibroblast growth factor receptor 3 (FGR3) polypeptide for use in the prevention or treatment of skeletal growth retardation disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020015733A JP2020015733A (en) | 2020-01-30 |
| JP2020015733A5 true JP2020015733A5 (en) | 2020-09-10 |
Family
ID=69580027
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019149672A Pending JP2020015733A (en) | 2019-08-19 | 2019-08-19 | Soluble fibroblast growth factor receptor 3 (fgr3) polypeptide for use in prevention or treatment of skeletal growth retardation disorders |
Country Status (1)
| Country | Link |
|---|---|
| JP (1) | JP2020015733A (en) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110272900B (en) * | 2019-04-19 | 2024-03-26 | 中国人民解放军陆军军医大学 | sgRNA for preparing skeletal dysplasia pig model and application thereof |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK0836380T3 (en) * | 1995-06-12 | 2002-04-22 | Yeda Res & Dev | FGF9 as a specific ligand for FGFR3 |
| EP1423428B2 (en) * | 2001-06-20 | 2012-11-14 | Fibron Ltd. | Antibodies that block fgfr3 activation, methods of screening for and uses thereof |
| JP2003104908A (en) * | 2001-09-28 | 2003-04-09 | Ichikazu Nakao | Therapeutic drug for achondrogenesis |
| BRPI0203172B8 (en) * | 2001-09-28 | 2021-05-25 | Nakao Kazuwa | pharmaceutical composition for achondroplasia |
| IL156495A0 (en) * | 2003-06-17 | 2004-01-04 | Prochon Biotech Ltd | Use of fgfr3 antagonists for treating t cell mediated diseases |
| JP4970057B2 (en) * | 2004-02-24 | 2012-07-04 | アラーガン、インコーポレイテッド | Botulinum toxin screening assay |
| CA2614039C (en) * | 2005-07-22 | 2015-10-13 | Five Prime Therapeutics, Inc. | Compositions and methods of treating disease with fgfr fusion proteins |
| WO2008133873A2 (en) * | 2007-04-25 | 2008-11-06 | Aveo Pharmaceuticals, Inc. | Fgf-binding fusion proteins |
| CA2823066A1 (en) * | 2010-12-27 | 2012-07-05 | Alexion Pharma International Sarl | Compositions comprising natriuretic peptides and methods of use thereof |
-
2019
- 2019-08-19 JP JP2019149672A patent/JP2020015733A/en active Pending
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