JP2019516719A5 - - Google Patents
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- JP2019516719A5 JP2019516719A5 JP2018560595A JP2018560595A JP2019516719A5 JP 2019516719 A5 JP2019516719 A5 JP 2019516719A5 JP 2018560595 A JP2018560595 A JP 2018560595A JP 2018560595 A JP2018560595 A JP 2018560595A JP 2019516719 A5 JP2019516719 A5 JP 2019516719A5
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- 239000008194 pharmaceutical composition Substances 0.000 claims 16
- 150000001875 compounds Chemical class 0.000 claims 8
- 108020004999 Messenger RNA Proteins 0.000 claims 6
- 150000002632 lipids Chemical class 0.000 claims 6
- 229920002106 messenger RNA Polymers 0.000 claims 6
- 239000000203 mixture Substances 0.000 claims 6
- RWQNBRDOKXIBIV-UHFFFAOYSA-N Thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 4
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 4
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 4
- 239000003795 chemical substances by application Substances 0.000 claims 4
- 239000002105 nanoparticle Substances 0.000 claims 4
- 150000003839 salts Chemical class 0.000 claims 3
- 239000011780 sodium chloride Substances 0.000 claims 3
- 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 2
- 201000005603 Fabry disease Diseases 0.000 claims 2
- 229920001320 Leader sequence (mRNA) Polymers 0.000 claims 2
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 2
- 229920000401 Three prime untranslated region Polymers 0.000 claims 2
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 2
- 125000000217 alkyl group Chemical group 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- 229920001223 polyethylene glycol Polymers 0.000 claims 2
- 229920001184 polypeptide Polymers 0.000 claims 2
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims 1
- RGICCULPCWNRAB-UHFFFAOYSA-N 2-[2-(2-hexoxyethoxy)ethoxy]ethanol Chemical compound CCCCCCOCCOCCOCCO RGICCULPCWNRAB-UHFFFAOYSA-N 0.000 claims 1
- JRYMOPZHXMVHTA-DAGMQNCNSA-N 2-amino-7-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-1H-pyrrolo[2,3-d]pyrimidin-4-one Chemical compound C1=CC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O JRYMOPZHXMVHTA-DAGMQNCNSA-N 0.000 claims 1
- BGTXMQUSDNMLDW-AEHJODJJSA-N 2-amino-9-[(2R,3S,4R,5R)-3-fluoro-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-3H-purin-6-one Chemical compound C1=2NC(N)=NC(=O)C=2N=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@]1(O)F BGTXMQUSDNMLDW-AEHJODJJSA-N 0.000 claims 1
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-Methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- KELXHQACBIUYSE-UHFFFAOYSA-N 5-methoxy-1H-pyrimidine-2,4-dione Chemical compound COC1=CNC(=O)NC1=O KELXHQACBIUYSE-UHFFFAOYSA-N 0.000 claims 1
- HCAJQHYUCKICQH-VPENINKCSA-N 8-Oxo-7,8-dihydro-2'-deoxyguanosine Chemical compound C1=2NC(N)=NC(=O)C=2NC(=O)N1[C@H]1C[C@H](O)[C@@H](CO)O1 HCAJQHYUCKICQH-VPENINKCSA-N 0.000 claims 1
- 241000180579 Arca Species 0.000 claims 1
- 101700021786 CAP2 Proteins 0.000 claims 1
- 229940107161 Cholesterol Drugs 0.000 claims 1
- NYHBQMYGNKIUIF-PXMDKTAGSA-N Guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1[C@H]1O[C@@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-PXMDKTAGSA-N 0.000 claims 1
- 229940029575 Guanosine Drugs 0.000 claims 1
- UGQMRVRMYYASKQ-KMPDEGCQSA-N Inosine Natural products O[C@H]1[C@H](O)[C@@H](CO)O[C@@H]1N1C(N=CNC2=O)=C2N=C1 UGQMRVRMYYASKQ-KMPDEGCQSA-N 0.000 claims 1
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims 1
- 101700016759 PARK7 Proteins 0.000 claims 1
- 101700026796 PRSS8 Proteins 0.000 claims 1
- 101710044691 TMPRSS4 Proteins 0.000 claims 1
- ZEMGGZBWXRYJHK-UHFFFAOYSA-N Thiouracil Chemical compound O=C1C=CNC(=S)N1 ZEMGGZBWXRYJHK-UHFFFAOYSA-N 0.000 claims 1
- 229940113082 Thymine Drugs 0.000 claims 1
- 229940035893 Uracil Drugs 0.000 claims 1
- 125000003342 alkenyl group Chemical group 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 125000001072 heteroaryl group Chemical group 0.000 claims 1
- 125000005349 heteroarylcycloalkyl group Chemical group 0.000 claims 1
- 125000000592 heterocycloalkyl group Chemical group 0.000 claims 1
- 229960003786 inosine Drugs 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 150000003904 phospholipids Chemical class 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 235000000346 sugar Nutrition 0.000 claims 1
- 150000008163 sugars Chemical class 0.000 claims 1
Claims (15)
- α−ガラクトシダーゼＡ（ＧＬＡ）ポリペプチドをコードするオープンリーディングフレーム（ＯＲＦ）を含むｍＲＮＡと、式（ＩＩ）
またはその塩もしくは立体異性体を有する化合物を含む送達剤と
を含む医薬組成物であって、式中、
ｌは、１、２、３、４、及び５から選択され、
Ｍ１は、結合またはＭ’であり、
Ｒ４は、非置換Ｃ１−３アルキル、または−（ＣＨ２）ｎＱであり、式中、ｎは、２、３、または４であり、Ｑは、ＯＨ、−ＮＨＣ（Ｓ）Ｎ（Ｒ）２、または−ＮＨＣ（Ｏ）Ｎ（Ｒ）２、−Ｎ（Ｒ）Ｃ（Ｏ）Ｒ、−Ｎ（Ｒ）Ｓ（Ｏ）２Ｒ、−Ｎ（Ｒ）Ｒ８、−ＮＨＣ（＝ＮＲ９）Ｎ（Ｒ）２、−ＮＨＣ（＝ＣＨＲ９）Ｎ（Ｒ）２、−ＯＣ（Ｏ）Ｎ（Ｒ）２、−Ｎ（Ｒ）Ｃ（Ｏ）ＯＲ、ヘテロアリールまたはヘテロシクロアルキルであり、 Ｍ及びＭ’は独立して、−Ｃ（Ｏ）Ｏ−、−ＯＣ（Ｏ）−、−Ｃ（Ｏ）Ｎ（Ｒ’）−、−Ｐ（Ｏ）（ＯＲ’）Ｏ−、−Ｓ−Ｓ−、アリール基、及びヘテロアリール基から選択され、
Ｒ２及びＲ３は独立して、Ｈ、Ｃ１−１４アルキル、及びＣ２−１４アルケニルからなる群から選択される、医薬組成物。 - 前記化合物は、化合物１〜化合物１４７、それらの塩及び立体異性体、ならびにそれらの任意の組み合わせからなる群から選択される、請求項１に記載の医薬組成物。
- 前記化合物は、化合物１８、その塩もしくは立体異性体、またはそれらの任意の組み合わせである、請求項２に記載の医薬組成物。
- 前記ｍＲＮＡは、脂質ナノ粒子として製剤化されている、請求項１〜３のいずれか１項に記載の医薬組成物。
- 前記脂質ナノ粒子または前記送達剤は、ＰＥＧ脂質をさらに含む、請求項１〜４のいずれか１項に記載の医薬組成物。
- 前記ＰＥＧ脂質は、化合物４２８である、請求項５に記載の医薬組成物。
- 前記脂質ナノ粒子または前記送達剤は、リン脂質をさらに含む、請求項１〜６のいずれか１項に記載の医薬組成物。
- 前記脂質ナノ粒子または前記送達剤は、化合物１８、ＤＳＰＣ、コレステロール、及び化合物４２８を約５０：１０：３８．５：１．５のモル比で含む、請求項１〜７のいずれか１項に記載の医薬組成物。
- 前記ｍＲＮＡは３’ＵＴＲを含み、必要に応じて、前記３’ＵＴＲは、配列番号５１〜７５、８１〜８２、８８、１０３、１０６〜１１３、１１８、及び１６１〜１７０、ならびにそれらの任意の組み合わせからなる群から選択される核酸配列を含む、請求項１〜８のいずれか１項に記載の医薬組成物。
- 前記ｍＲＮＡは５’ＵＴＲを含み、必要に応じて、前記５’ＵＴＲが、配列番号３３〜５０、７７、及び１１５〜１１７、ならびにそれらの任意の組み合わせからなる群から選択される配列を含む、請求項１〜９のいずれか１項に記載の医薬組成物。
- 前記ｍＲＮＡは、５’末端キャップをさらに含み、必要に応じて、前記５’末端キャップは、Ｃａｐ０、Ｃａｐ１、ＡＲＣＡ、イノシン、Ｎ１−メチル−グアノシン、２’−フルオロ−グアノシン、７−デアザ−グアノシン、８−オキソ−グアノシン、２−アミノ−グアノシン、ＬＮＡ−グアノシン、２−アジドグアノシン、Ｃａｐ２、Ｃａｐ４、５’メチルＧキャップ、またはそれらの類似体を含む、請求項１〜１０のいずれか１項に記載の医薬組成物。
- 前記ｍＲＮＡは、少なくとも１つの化学修飾されたヌクレオ塩基、糖、骨格、またはそれらの任意の組み合わせを含み、必要に応じて、
（ａ）前記少なくとも１つの化学修飾されたヌクレオ塩基は、シュードウラシル（ψ）、Ｎ１−メチルシュードウラシル（ｍ１ψ）、１−エチルシュードウラシル、２−チオウラシル（ｓ２Ｕ）、４’−チオウラシル、５−メチルシトシン、５−メチルウラシル、５−メトキシウラシル、及びそれらの任意の組み合わせからなる群から選択され、かつ／または
（ｂ）ウラシルまたはチミンの少なくとも約２５％、少なくとも約３０％、少なくとも約４０％、少なくとも約５０％、少なくとも約６０％、少なくとも約７０％、少なくとも約８０％、少なくとも約９０％、少なくとも約９５％、少なくとも約９９％、または１００％が化学修飾されている、
請求項１〜１１のいずれか１項に記載の医薬組成物。 - 前記ＧＬＡポリペプチドは、ＧＬＡ活性を有する、野生型、バリアント、または変異体であり、必要に応じて、前記ＯＲＦは、配列番号３〜２７、７９〜８０、及び１４１〜１５９からなる群から選択される核酸配列を含む、請求項１〜１２のいずれか１項に記載の医薬組成物。
- ファブリー病に対する治療または予防を必要とするヒト対象を治療するための方法における使用のための、請求項１〜１３のいずれか１項に記載の医薬組成物。
- 前記対象においてファブリー病の徴候または症状の発症を治療、予防または遅延させるための方法における使用のための、請求項１〜１４のいずれか１項に記載の医薬組成物。
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022119527A JP2022136230A (ja) | 2016-05-18 | 2022-07-27 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662338354P | 2016-05-18 | 2016-05-18 | |
| US62/338,354 | 2016-05-18 | ||
| PCT/US2017/033398 WO2017201328A1 (en) | 2016-05-18 | 2017-05-18 | POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022119527A Division JP2022136230A (ja) | 2016-05-18 | 2022-07-27 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019516719A JP2019516719A (ja) | 2019-06-20 |
| JP2019516719A5 true JP2019516719A5 (ja) | 2020-06-25 |
| JP7114485B2 JP7114485B2 (ja) | 2022-08-08 |
Family
ID=59021568
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018560595A Active JP7114485B2 (ja) | 2016-05-18 | 2017-05-18 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
| JP2022119527A Pending JP2022136230A (ja) | 2016-05-18 | 2022-07-27 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022119527A Pending JP2022136230A (ja) | 2016-05-18 | 2022-07-27 | ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド |
Country Status (6)
| Country | Link |
|---|---|
| US (5) | US20190000932A1 (ja) |
| EP (2) | EP3896164A1 (ja) |
| JP (2) | JP7114485B2 (ja) |
| AU (1) | AU2017266932B2 (ja) |
| CA (1) | CA3024507A1 (ja) |
| WO (1) | WO2017201328A1 (ja) |
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2017
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- 2017-05-18 AU AU2017266932A patent/AU2017266932B2/en active Active
- 2017-05-18 CA CA3024507A patent/CA3024507A1/en active Pending
- 2017-05-18 EP EP17728702.6A patent/EP3458590B9/en active Active
- 2017-05-18 JP JP2018560595A patent/JP7114485B2/ja active Active
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2018
- 2018-08-23 US US16/110,788 patent/US20190000932A1/en not_active Abandoned
- 2018-08-23 US US16/110,309 patent/US10519455B2/en active Active
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-
2019
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2022
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2023
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