JP2017513504A5 - - Google Patents
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- JP2017513504A5 JP2017513504A5 JP2016564200A JP2016564200A JP2017513504A5 JP 2017513504 A5 JP2017513504 A5 JP 2017513504A5 JP 2016564200 A JP2016564200 A JP 2016564200A JP 2016564200 A JP2016564200 A JP 2016564200A JP 2017513504 A5 JP2017513504 A5 JP 2017513504A5
- Authority
- JP
- Japan
- Prior art keywords
- hldlr
- modified
- amino acid
- raav vector
- expression
- 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.)
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- 101000356132 LDLR Proteins 0.000 claims 21
- 102000023738 human LDLR protein Human genes 0.000 claims 21
- 125000003275 alpha amino acid group Chemical group 0.000 claims 10
- 238000006467 substitution reaction Methods 0.000 claims 10
- 239000000203 mixture Substances 0.000 claims 8
- HVYWMOMLDIMFJA-DPAQBDIFSA-N (3β)-Cholest-5-en-3-ol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 229940107161 Cholesterol Drugs 0.000 claims 3
- 230000001105 regulatory Effects 0.000 claims 3
- 210000000234 Capsid Anatomy 0.000 claims 2
- 230000036499 Half live Effects 0.000 claims 2
- 210000003494 Hepatocytes Anatomy 0.000 claims 2
- 208000000563 Hyperlipoproteinemia Type II Diseases 0.000 claims 2
- 108060004326 LDLR Proteins 0.000 claims 2
- 102100012475 LDLR Human genes 0.000 claims 2
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims 2
- 239000003937 drug carrier Substances 0.000 claims 2
- 201000001386 familial hypercholesterolemia Diseases 0.000 claims 2
- 230000037361 pathway Effects 0.000 claims 2
- 241000432074 Adeno-associated virus Species 0.000 claims 1
- 102100019098 MYLIP Human genes 0.000 claims 1
- 101710017999 MYLIP Proteins 0.000 claims 1
- 101700000651 PCSK9 Proteins 0.000 claims 1
- 102100005352 PCSK9 Human genes 0.000 claims 1
- 101700000527 RH10 Proteins 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 230000004059 degradation Effects 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 230000002452 interceptive Effects 0.000 claims 1
Claims (18)
- 改変されたヒト低密度リポタンパク質受容体（ｈＬＤＬＲ）遺伝子を含んでなる発現カセットを有する組換えアデノ随伴ウイルス（ｒＡＡＶ）ベクターであって、前記ｈＬＤＬＲ遺伝子が発現後にコレステロールを低下させる改変されたｈＬＤＬＲをコードし、かつ、前記改変されたｈＬＤＬＲが：（ａ）野生型ｈＬＤＬＲ ＩＤＯＬ経路を妨害する１個若しくはそれ以上のアミノ酸置換；および／または
（ｂ）ＰＣＳＫ９経路を妨害することによりｈＬＤＬＲの分解に抵抗性である１個若しくはそれ以上のアミノ酸置換
を含んでなる、上記ベクター。 - 改変されたｈＬＤＬＲが、（ａ）配列番号１の番号付けに基づくＮ２９５、Ｈ３０６、Ｖ３０７、Ｎ３０９、Ｄ３１０および／若しくはＬ３１８の１個若しくはそれ以上のアミノ酸置換を含んでなる、請求項１に記載のｒＡＡＶベクター。
- ｈＬＤＬＲが、
（ａ） 配列番号１の番号付けに基づくＮ２９５Ｄ、Ｈ３０６Ｄ、Ｖ３０７Ｄ、Ｎ３０９Ａ、Ｄ３１０Ｎ、Ｌ３１８Ｈおよび／若しくはＬ３１８Ｄから選択される１個若しくはそれ以上のアミノ酸置換、および／若しくは
（ｂ） 配列番号１の番号付けに基づくＬ７６９Ｒ、Ｋ８０９Ｒおよび／若しくはＣ８１８Ａの最低１個のアミノ酸置換を含んでなる、
請求項１または２に記載のｒＡＡＶベクター。 - ＡＡＶ８、ｒｈ６４Ｒ１、ＡＡＶ９若しくはｒｈ１０から選択されるキャプシドを含んでなる、請求項１−３のいずれか１つに記載のｒＡＡＶベクター。
- 発現カセットが、肝細胞中の改変されたｈＬＤＬｒの発現を特異的に指図するプロモーターを含んでなる、請求項１−４のいずれか１つに記載のｒＡＡＶベクター。
- 改変されたｈＬＤＬＲ遺伝子を含んでなる発現カセットを有するｒＡＡＶベクターであって、前記ｈＬＤＬＲ遺伝子が、配列番号１の番号付けに基づくＬ３１８Ｄアミノ酸置換を含んでなる改変されたｈＬＤＬＲをコードする、上記ベクター。
- 改変されたｈＬＤＬＲが、配列番号１の番号付けに基づくＫ８０９Ｒおよび／若しくはＣ８１８Ａアミノ酸置換をさらに含んでなる、請求項６に記載のｒＡＡＶベクター。
- ベクターがＡＡＶ８、ｒｈ６４Ｒ１、ＡＡＶ９若しくはｒｈ１０から選択されるキャプシドを含んでなる、請求項６若しくは７に記載のｒＡＡＶベクター。
- 発現カセットが、肝細胞中での改変されたｈＬＤＬｒの発現を特異的に指図するプロモーターを含んでなる、請求項６−８のいずれか１つに記載のｒＡＡＶベクター。
- 製薬学的に許容できる担体および請求項１−９のいずれか１つに記載のｒＡＡＶベクターを含んでなる製薬学的組成物。
- 請求項１−９のいずれか１つに記載のｒＡＡＶベクターを含んでなる、それの必要な被験体における循環コレステロールレベルを低下させるための製薬学的組成物であって、前記発現カセットが、該被験体での改変されたｈＬＤＬＲの発現を指図する調節制御配列をさらに含んでなる、上記組成物。
- 請求項１−９のいずれか１つに記載のｒＡＡＶベクターを含んでなる、それの必要な被験体におけるｈＬＤＬＲの循環半減期を増大させるための製薬学的組成物であって、前記発現カセットが、該被験体での改変されたｈＬＤＬＲの発現を指図する調節制御配列をさらに含んでなる、上記組成物。
- それの必要な被験体における循環コレステロールレベルを低下させるための請求項１０に記載の組成物であって、前記発現カセットが、該被験体での改変されたｈＬＤＬＲの発現を指図する調節制御配列をさらに含んでなる、上記組成物。
- それの必要な被験体におけるｈＬＤＬＲの循環半減期を増大させるための請求項１０に記載の組成物であって、前記発現カセットが、該被験体での改変されたｈＬＤＬＲの発現を指図する調節制御配列をさらに含んでなる、上記組成物。
- 家族性高コレステロール血症を処置するためのレジメンでの使用のための、請求項１−９のいずれか１つに記載のｒＡＡＶベクターを含んでなる製薬学的組成物。
- 家族性高コレステロール血症を処置するためのレジメンでの使用のための、請求項１０に記載の組成物。
- （ａ）配列番号１の番号付けに基づくＮ２９５Ｄ、Ｈ３０６Ｄ、Ｖ３０７Ｄ、Ｎ３０９Ａ、Ｄ３１０Ｎ、Ｌ３１８Ｈおよび／若しくはＬ３１８Ｄの１個若しくはそれ以上のアミノ酸置換；または
（ｂ）配列番号１の番号付けに基づくＫ７６９Ｒ、Ｋ８０９Ｒおよび／若しくはＣ８１８Ａの最低１個のアミノ酸置換と組合せの（ａ）のいずれかのアミノ酸置換
を含んでなる、合成若しくは組換えｈＬＤＬＲ。 - 請求項１７に記載の合成若しくは組換えｈＬＤＬＲタンパク質および製薬学的に許容できる担体を含んでなる製薬学的組成物。
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461984620P | 2014-04-25 | 2014-04-25 | |
| US61/984,620 | 2014-04-25 | ||
| US201462022627P | 2014-07-09 | 2014-07-09 | |
| US62/022,627 | 2014-07-09 | ||
| PCT/US2015/027572 WO2015164778A1 (en) | 2014-04-25 | 2015-04-24 | Ldlr variants and their use in compositions for reducing cholesterol levels |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020074046A Division JP7280853B2 (ja) | 2014-04-25 | 2020-04-17 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017513504A JP2017513504A (ja) | 2017-06-01 |
| JP2017513504A5 true JP2017513504A5 (ja) | 2018-06-07 |
| JP6741590B2 JP6741590B2 (ja) | 2020-08-19 |
Family
ID=53055129
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016564200A Active JP6741590B2 (ja) | 2014-04-25 | 2015-04-24 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| JP2020074046A Active JP7280853B2 (ja) | 2014-04-25 | 2020-04-17 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| JP2023078988A Pending JP2023100960A (ja) | 2014-04-25 | 2023-05-12 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020074046A Active JP7280853B2 (ja) | 2014-04-25 | 2020-04-17 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| JP2023078988A Pending JP2023100960A (ja) | 2014-04-25 | 2023-05-12 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US11555059B2 (ja) |
| EP (2) | EP3134431B1 (ja) |
| JP (3) | JP6741590B2 (ja) |
| BR (1) | BR112016024379A2 (ja) |
| CA (1) | CA2946392A1 (ja) |
| ES (1) | ES2876409T3 (ja) |
| WO (1) | WO2015164778A1 (ja) |
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| WO2015164778A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennysylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
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| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| KR20190118163A (ko) | 2017-02-20 | 2019-10-17 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 |
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