JPWO2020061229A5 - - Google Patents

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JPWO2020061229A5
JPWO2020061229A5 JP2021539499A JP2021539499A JPWO2020061229A5 JP WO2020061229 A5 JPWO2020061229 A5 JP WO2020061229A5 JP 2021539499 A JP2021539499 A JP 2021539499A JP 2021539499 A JP2021539499 A JP 2021539499A JP WO2020061229 A5 JPWO2020061229 A5 JP WO2020061229A5
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capsid
seq
amino acid
acid sequence
polypeptide
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JP7344300B2 (en
JP2022502077A (en
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Claims (25)

組換えの内因性のＧａｇポリペプチド、および前記内因性のＧａｇポリペプチドに固有ではない異種のカーゴを含むカプシドであって、前記内因性のＧａｇポリペプチドがＡｒｃポリペプチドではない、カプシド。 A capsid comprising a recombinant endogenous Gag polypeptide and a heterologous cargo not native to said endogenous Gag polypeptide, wherein said endogenous Gag polypeptide is not an Arc polypeptide. 前記内因性のＧａｇポリペプチドは腫瘍随伴性のＭａ抗原ファミリーポリペプチドである、請求項１に記載のカプシド。 2. The capsid of claim 1 , wherein said endogenous Gag polypeptide is a tumor-associated Ma antigen family polypeptide . 前記腫瘍随伴性のＭａ抗原ファミリーポリペプチドは、The paraneoplastic Ma antigen family polypeptide comprises
ａ）ＳＥＱ ＩＤ ＮＯ：１６であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：１６に少なくとも９０％同一であるアミノ酸配列、 a) an amino acid sequence that is SEQ ID NO:16 or an amino acid sequence that is at least 90% identical to SEQ ID NO:16;
ｂ）ＳＥＱ ＩＤ ＮＯ：１７であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：１７に少なくとも９０％同一であるアミノ酸配列、 b) an amino acid sequence that is SEQ ID NO:17 or an amino acid sequence that is at least 90% identical to SEQ ID NO:17;
ｃ）ＳＥＱ ＩＤ ＮＯ：１８であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：１８に少なくとも９０％同一であるアミノ酸配列、 c) an amino acid sequence that is SEQ ID NO:18 or an amino acid sequence that is at least 90% identical to SEQ ID NO:18;
ｄ）ＳＥＱ ＩＤ ＮＯ：１９であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：１９に少なくとも９０％同一であるアミノ酸配列、あるいは、 d) an amino acid sequence that is SEQ ID NO:19 or is at least 90% identical to SEQ ID NO:19; or
ｅ）ＳＥＱ ＩＤ ＮＯ：２７であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：２７に少なくとも９０％同一であるアミノ酸配列、 e) an amino acid sequence that is SEQ ID NO:27 or an amino acid sequence that is at least 90% identical to SEQ ID NO:27;
を含む、請求項２に記載のカプシド。3. The capsid of claim 2, comprising: 前記異種のカーゴは核酸である、請求項２に記載のカプシド。3. The capsid of Claim 2, wherein said heterologous cargo is a nucleic acid. 前記異種のカーゴはＲＮＡである、請求項４に記載のカプシド。5. The capsid of claim 4, wherein said heterologous cargo is RNA. 前記異種のカーゴは治療剤である、請求項２に記載のカプシド。3. The capsid of Claim 2, wherein said heterologous cargo is a therapeutic agent. 前記異種のカーゴはポリペプチドである、請求項２に記載のカプシド。3. The capsid of Claim 2, wherein said heterologous cargo is a polypeptide. 前記異種のカーゴは遺伝子編集システムである、請求項２に記載のカプシド。3. The capsid of Claim 2, wherein said heterologous cargo is a gene editing system. 第２のポリペプチドをさらに含む、請求項２に記載のカプシド。3. The capsid of Claim 2, further comprising a second polypeptide. 前記第２のポリペプチドは抗体またはその結合フラグメントである、請求項９に記載のカプシド。10. The capsid of claim 9, wherein said second polypeptide is an antibody or binding fragment thereof. 前記第２のポリペプチドは標的部位に結合する、請求項９に記載のカプシド。10. The capsid of Claim 9, wherein said second polypeptide binds to a target site. マイクロベシクル、微粒子、リポソーム、またはミセルを含む送達成分をさらに含む、請求項２に記載のカプシド。3. The capsid of Claim 2, further comprising a delivery component comprising microvesicles, microparticles, liposomes, or micelles. 細胞外小胞を含む送達成分をさらに含む、請求項２に記載のカプシド。3. The capsid of Claim 2, further comprising a delivery component comprising extracellular vesicles. 前記内因性のＧａｇポリペプチドはレトロトランスポゾンＧａｇ様ファミリーポリペプチドである、請求項１に記載のカプシド。2. The capsid of claim 1, wherein said endogenous Gag polypeptide is a retrotransposon Gag-like family polypeptide. 前記レトロトランスポゾンＧａｇ様ファミリーポリペプチドは、The retrotransposon Gag-like family polypeptide comprises
ａ）ＳＥＱ ＩＤ ＮＯ：２０であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：２０に少なくとも９０％同一であるアミノ酸配列、a) an amino acid sequence that is SEQ ID NO:20 or an amino acid sequence that is at least 90% identical to SEQ ID NO:20;
ｂ）ＳＥＱ ＩＤ ＮＯ：２１であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：２１に少なくとも９０％同一であるアミノ酸配列、 b) an amino acid sequence that is SEQ ID NO:21 or an amino acid sequence that is at least 90% identical to SEQ ID NO:21;
ｃ）ＳＥＱ ＩＤ ＮＯ：２２であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：２２に少なくとも９０％同一であるアミノ酸配列、 c) an amino acid sequence that is SEQ ID NO:22 or an amino acid sequence that is at least 90% identical to SEQ ID NO:22;
ｄ）ＳＥＱ ＩＤ ＮＯ：２３であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：２３に少なくとも９０％同一であるアミノ酸配列、 d) an amino acid sequence that is SEQ ID NO:23 or an amino acid sequence that is at least 90% identical to SEQ ID NO:23;
ｅ）ＳＥＱ ＩＤ ＮＯ：２４であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：２４に少なくとも９０％同一であるアミノ酸配列、あるいは、 e) an amino acid sequence that is SEQ ID NO:24 or is at least 90% identical to SEQ ID NO:24; or
ｆ）ＳＥＱ ＩＤ ＮＯ：２８であるアミノ酸配列、またはＳＥＱ ＩＤ ＮＯ：２８に少なくとも９０％同一であるアミノ酸配列、 f) an amino acid sequence that is SEQ ID NO:28 or an amino acid sequence that is at least 90% identical to SEQ ID NO:28;
を含む、請求項１４に記載のカプシド。15. The capsid of claim 14, comprising 前記異種のカーゴは核酸である、請求項１４に記載のカプシド。15. The capsid of Claim 14, wherein said heterologous cargo is a nucleic acid. 前記異種のカーゴはＲＮＡである、請求項１６に記載のカプシド。17. The capsid of claim 16, wherein said heterologous cargo is RNA. 前記異種のカーゴは治療剤である、請求項１４に記載のカプシド。15. The capsid of Claim 14, wherein said heterologous cargo is a therapeutic agent. 前記異種のカーゴはポリペプチドである、請求項１４に記載のカプシド。15. The capsid of Claim 14, wherein said heterologous cargo is a polypeptide. 前記異種のカーゴは遺伝子編集システムである、請求項１４に記載のカプシド。15. The capsid of Claim 14, wherein said heterologous cargo is a gene editing system. 第２のポリペプチドをさらに含む、請求項１４に記載のカプシド。15. The capsid of Claim 14, further comprising a second polypeptide. 前記第２のポリペプチドは抗体またはその結合フラグメントである、請求項２１に記載のカプシド。22. The capsid of claim 21, wherein said second polypeptide is an antibody or binding fragment thereof. 前記第２のポリペプチドは標的部位に結合する、請求項２１に記載のカプシド。22. The capsid of Claim 21, wherein said second polypeptide binds to a target site. マイクロベシクル、微粒子、リポソーム、またはミセルを含む送達成分をさらに含む、請求項１４に記載のカプシド。15. The capsid of Claim 14, further comprising a delivery component comprising microvesicles, microparticles, liposomes, or micelles. 細胞外小胞を含む送達成分をさらに含む、請求項１４に記載のカプシド。15. The capsid of Claim 14, further comprising a delivery component comprising extracellular vesicles.
JP2021539499A 2018-09-18 2019-09-18 ARC-based capsids and their uses Active JP7344300B2 (en)

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