JPWO2020014471A5 - - Google Patents
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- JPWO2020014471A5 JPWO2020014471A5 JP2021500538A JP2021500538A JPWO2020014471A5 JP WO2020014471 A5 JPWO2020014471 A5 JP WO2020014471A5 JP 2021500538 A JP2021500538 A JP 2021500538A JP 2021500538 A JP2021500538 A JP 2021500538A JP WO2020014471 A5 JPWO2020014471 A5 JP WO2020014471A5
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- capsid protein
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- 102000004040 Capsid Proteins Human genes 0.000 claims description 26
- 108090000565 Capsid Proteins Proteins 0.000 claims description 26
- 210000004027 cells Anatomy 0.000 claims description 16
- 239000000203 mixture Substances 0.000 claims description 15
- 150000001413 amino acids Chemical class 0.000 claims description 10
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 6
- 238000002347 injection Methods 0.000 claims description 6
- 239000007924 injection Substances 0.000 claims description 6
- 206010003591 Ataxia Diseases 0.000 claims description 4
- 210000004556 Brain Anatomy 0.000 claims description 4
- 210000003594 Ganglia, Spinal Anatomy 0.000 claims description 4
- 206010053643 Neurodegenerative disease Diseases 0.000 claims description 4
- 239000002246 antineoplastic agent Substances 0.000 claims description 4
- 230000000875 corresponding Effects 0.000 claims description 4
- 102000037240 fusion proteins Human genes 0.000 claims description 4
- 108020001507 fusion proteins Proteins 0.000 claims description 4
- 238000007913 intrathecal administration Methods 0.000 claims description 4
- 210000002569 neurons Anatomy 0.000 claims description 4
- 108020004707 nucleic acids Proteins 0.000 claims description 4
- 150000007523 nucleic acids Chemical class 0.000 claims description 4
- 201000010874 syndrome Diseases 0.000 claims description 4
- 230000001225 therapeutic Effects 0.000 claims description 4
- 206010028640 Myopathy Diseases 0.000 claims description 3
- 201000010770 muscular disease Diseases 0.000 claims description 3
- 201000009623 myopathy Diseases 0.000 claims description 3
- 206010001897 Alzheimer's disease Diseases 0.000 claims description 2
- 206010002026 Amyotrophic lateral sclerosis Diseases 0.000 claims description 2
- 206010067608 Canavan disease Diseases 0.000 claims description 2
- 206010050389 Cerebral ataxia Diseases 0.000 claims description 2
- 210000003703 Cisterna Magna Anatomy 0.000 claims description 2
- 206010015037 Epilepsy Diseases 0.000 claims description 2
- IRSCQMHQWWYFCW-UHFFFAOYSA-N Ganciclovir Chemical compound O=C1NC(N)=NC2=C1N=CN2COC(CO)CO IRSCQMHQWWYFCW-UHFFFAOYSA-N 0.000 claims description 2
- 229960002963 Ganciclovir Drugs 0.000 claims description 2
- 210000002216 Heart Anatomy 0.000 claims description 2
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 claims description 2
- 201000001971 Huntington's disease Diseases 0.000 claims description 2
- 210000003712 Lysosomes Anatomy 0.000 claims description 2
- 210000003205 Muscles Anatomy 0.000 claims description 2
- 210000004940 Nucleus Anatomy 0.000 claims description 2
- 206010061536 Parkinson's disease Diseases 0.000 claims description 2
- 210000000278 Spinal Cord Anatomy 0.000 claims description 2
- 201000001203 Wilson disease Diseases 0.000 claims description 2
- 238000009825 accumulation Methods 0.000 claims description 2
- 201000005216 brain cancer Diseases 0.000 claims description 2
- 239000003292 glue Substances 0.000 claims description 2
- 238000001361 intraarterial administration Methods 0.000 claims description 2
- 238000007918 intramuscular administration Methods 0.000 claims description 2
- 238000007912 intraperitoneal administration Methods 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims description 2
- 230000001868 lysosomic Effects 0.000 claims description 2
- 201000011442 metachromatic leukodystrophy Diseases 0.000 claims description 2
- 201000006417 multiple sclerosis Diseases 0.000 claims description 2
- 210000000663 muscle cells Anatomy 0.000 claims description 2
- 230000003387 muscular Effects 0.000 claims description 2
- 230000002107 myocardial Effects 0.000 claims description 2
- 238000007920 subcutaneous administration Methods 0.000 claims description 2
- 210000001519 tissues Anatomy 0.000 claims description 2
- 206010013801 Duchenne muscular dystrophy Diseases 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000006011 modification reaction Methods 0.000 description 1
Description
他の実施形態
本発明を、その詳細な説明と併せて記載しているが、上記の記載は、添付の特許請求の
範囲により画定される本発明の範囲を例示することが意図されており限定することは意図
されていないことを理解しなければならない。他の態様、利点、および改変は、下記の特
許請求の範囲内である。
本発明の様々な実施形態を以下に示す。
１．配列ＴＶＳＡＬＦＫ（配列番号８）；ＴＶＳＡＬＫ（配列番号４）；ＫＬＡＳＶＴ（配列番号８３）；またはＫＦＬＡＳＶＴ（配列番号８４）からの少なくとも４個の連続するアミノ酸を含むアミノ酸配列を含むＡＡＶカプシドタンパク質。
２．配列ＴＶＳＡＬＫ（配列番号４）；ＴＶＳＡＬＦＫ（配列番号８）；ＫＬＡＳＶＴ（配列番号８３）；またはＫＦＬＡＳＶＴ（配列番号８４）からの少なくとも５個の連続するアミノ酸を含むアミノ酸配列を含む上記１に記載のＡＡＶカプシドタンパク質。
３．配列ＴＶＳＡＬＫ（配列番号４）；ＴＶＳＡＬＦＫ（配列番号８）；ＫＬＡＳＶＴ（配列番号８３）；またはＫＦＬＡＳＶＴ（配列番号８４）からの少なくとも６個の連続するアミノ酸を含むアミノ酸配列を含む上記１に記載のＡＡＶカプシドタンパク質。
４．前記ＡＡＶは、ＡＡＶ９である、上記１～３のいずれかに記載のＡＡＶカプシドタンパク質。
５．ＡＡＶ９ ＶＰ１を含む上記１～４のいずれかに記載のＡＡＶカプシドタンパク質。
６．配列番号８５のアミノ酸５８８および５８９に対応する位置にターゲティング配列が挿入されている、上記５に記載のＡＡＶカプシドタンパク質。
７．上記１～６のいずれかに記載のＡＡＶカプシドタンパク質をコードする核酸。
８．上記１～６のいずれかに記載のカプシドタンパク質を含むＡＡＶ。
９．導入遺伝子、好ましくは治療用導入遺伝子をさらに含む上記８に記載のＡＡＶ。
１０．Ｖ［Ｓ／ｐ］［Ａ／ｍ／ｔ／］Ｌ（配列番号７９）、ＴＶ［Ｓ／ｐ］［Ａ／ｍ／ｔ／］Ｌ（配列番号８０）、ＴＶ［Ｓ／ｐ］［Ａ／ｍ／ｔ／］ＬＫ（配列番号８１）、またはＴＶ［Ｓ／ｐ］［Ａ／ｍ／ｔ／］ＬＦＫ．（配列番号８２）を含むターゲティング配列。
１１．前記ターゲティング配列は、ＶＰＡＬＲ（配列番号１）；ＶＳＡＬＫ（配列番号２）；ＴＶＰＡＬＲ（配列番号３）；ＴＶＳＡＬＫ（配列番号４）；ＴＶＰＭＬＫ（配列番号１２）；ＴＶＰＴＬＫ（配列番号１３）；ＦＴＶＳＡＬＫ（配列番号５）；ＬＴＶＳＡＬＫ（配列番号６）；ＴＶＳＡＬＦＫ（配列番号８）；ＴＶＰＡＬＦＲ（配列番号９）；ＴＶＰＭＬＦＫ（配列番号１０）、またはＴＶＰＴＬＦＫ（配列番号１１）を含む、上記１０に記載のターゲティング配列。
１２．上記１０または１１に記載のターゲティング配列と、異種配列とを含む融合タンパク質。
１３．上記１０または１１に記載のターゲティング配列を含むＡＡＶカプシドタンパク質。
１４．ＡＡＶ９ ＶＰ１を含む上記１３に記載のＡＡＶカプシドタンパク質。
１５．前記ターゲティング配列は、配列番号のアミノ酸５８８および５８９に対応する位置に挿入されている、上記１４に記載のＡＡＶカプシドタンパク質。
１６．上記１０～１６のいずれかに記載のターゲティング配列、融合タンパク質、またはＡＡＶカプシドタンパク質をコードする核酸。
１７．上記１３～１５のいずれかに記載のカプシドタンパク質を含むＡＡＶ。
１８．導入遺伝子、好ましくは治療用導入遺伝子をさらに含む上記１７に記載のＡＡＶ。
１９．細胞に導入遺伝子を送達する方法であって、前記細胞と、上記１～９、１７、または１８に記載のＡＡＶとを接触させることを含む方法。
２０．前記細胞は、神経細胞（任意選択で後根神経節神経細胞）、星状膠細胞、心筋細胞、または筋細胞である、上記１９に記載の方法。
２１．前記細胞は、生きている対象中に存在する、上記１９に記載の方法。
２２．前記対象は、哺乳類対象である、上記１９に記載の方法。
２３．前記細胞は、脳、脊髄、後根神経節、心臓、または筋肉、およびこれらの組み合わせから選択される組織中に存在する、上記２０～２２のいずれかに記載の方法。
２４．前記対象は、神経変性疾患、てんかん；脳卒中；脊髄小脳失調症；キャナヴァン病；異染性白質ジストロフィー；脊髄性筋萎縮症；フリートライヒ運動失調症；Ｘ連鎖中心核ミオパチー；リソソーム蓄積症；バース症候群；デュシェンヌ型筋ジストロフィー；ウィルソン病；またはクリグラー・ナジャー症候群１型を有する、上記２３に記載の方法。
２５．前記神経変性疾患は、パーキンソン病；アルツハイマー病；ハンチントン病；筋萎縮性側索硬化症；および多発性硬化症である、上記２４に記載の方法。
２６．前記対象は、脳がんを有し、前記方法は、抗がん剤をコードするＡＡＶを投与することを含む、上記２３に記載の方法。
２７．前記抗がん剤は、ＨＳＶ．ＴＫ１であり、前記方法は、ガンシクロビルを投与することをさらに含む、上記２６に記載の方法。
２８．前記細胞は、前記対象の脳中に存在し、前記ＡＡＶを、非経口送達；脳内；または髄腔内送達により投与する、上記２２～２７のいずれかに記載の方法。
２９．前記非経口送達は、静脈内送達、動脈内送達、皮下送達、腹腔内送達、または筋肉内送達による、上記２８に記載の方法。
３０．前記髄腔内送達は、腰椎注射、大槽注射、または実質内注射による、上記２８に記載の方法。
Other Embodiments The invention is described in conjunction with a detailed description thereof, but the above description is intended to illustrate and limit the scope of the invention as defined by the appended claims. You must understand that what you do is not intended. Other aspects, advantages, and modifications are within the claims below.
Various embodiments of the present invention are shown below.
1. 1. AAV capsid protein comprising an amino acid sequence comprising at least 4 contiguous amino acids from the sequence TVSALFK (SEQ ID NO: 8); TVSALK (SEQ ID NO: 4); KLASVT (SEQ ID NO: 83); or KFLASVT (SEQ ID NO: 84).
2. 2. AAV according to 1 above, which comprises an amino acid sequence comprising at least 5 contiguous amino acids from the sequence TVSALK (SEQ ID NO: 4); TVSALFK (SEQ ID NO: 8); KLASVT (SEQ ID NO: 83); or KFLASVT (SEQ ID NO: 84). Capsid protein.
3. 3. AAV according to 1 above, comprising an amino acid sequence comprising at least 6 contiguous amino acids from the sequence TVSALK (SEQ ID NO: 4); TVSALFK (SEQ ID NO: 8); KLASVT (SEQ ID NO: 83); or KFLASVT (SEQ ID NO: 84). Capsid protein.
4. The AAV capsid protein according to any one of 1 to 3 above, wherein the AAV is AAV9.
5. The AAV capsid protein according to any one of 1 to 4 above, which comprises AAV9 VP1.
6. The AAV capsid protein according to 5 above, wherein the targeting sequence is inserted at the position corresponding to the amino acids 588 and 589 of SEQ ID NO: 85.
7. A nucleic acid encoding the AAV capsid protein according to any one of 1 to 6 above.
8. AAV containing the capsid protein according to any one of 1 to 6 above.
9. 8. The AAV according to 8 above, further comprising a transgene, preferably a therapeutic transgene.
10. V [S / p] [A / m / t /] L (SEQ ID NO: 79), TV [S / p] [A / m / t /] L (SEQ ID NO: 80), TV [S / p] [A / M / t /] LK (SEQ ID NO: 81), or TV [S / p] [A / m / t /] LFK. A targeting sequence comprising (SEQ ID NO: 82).
11. The targeting sequence is VPARR (SEQ ID NO: 1); VSALK (SEQ ID NO: 2); TVPARR (SEQ ID NO: 3); TVSALK (SEQ ID NO: 4); TVPMLK (SEQ ID NO: 12); TVPTLK (SEQ ID NO: 13); FTVSALK (SEQ ID NO: 3). The targeting sequence according to 10 above, comprising: LTVSALK (SEQ ID NO: 6); TVSALFK (SEQ ID NO: 8); TVPALFR (SEQ ID NO: 9); TVPMLFK (SEQ ID NO: 10), or TVPTLFK (SEQ ID NO: 11).
12. A fusion protein comprising the targeting sequence according to 10 or 11 above and a heterologous sequence.
13. AAV capsid protein comprising the targeting sequence according to 10 or 11 above.
14. The AAV capsid protein according to 13 above, which comprises AAV9 VP1.
15. The AAV capsid protein according to 14 above, wherein the targeting sequence is inserted at positions corresponding to amino acids 588 and 589 of SEQ ID NO: 14.
16. Nucleic acid encoding the targeting sequence, fusion protein, or AAV capsid protein according to any of 10-16 above.
17. AAV comprising the capsid protein according to any one of 13 to 15 above.
18. 17. The AAV according to 17 above, further comprising a transgene, preferably a therapeutic transgene.
19. A method of delivering a transgene to a cell, comprising contacting the cell with the AAV according to 1-9, 17, or 18.
20. 19. The method of 19 above, wherein the cells are nerve cells (optionally dorsal root ganglion neurons), stellate glue cells, myocardial cells, or muscle cells.
21. 19. The method of 19 above, wherein the cells are present in a living object.
22. 19. The method according to 19 above, wherein the subject is a mammalian subject.
23. The method according to any of 20 to 22 above, wherein the cells are present in the brain, spinal cord, dorsal root ganglion, heart, or muscle, and tissues selected from combinations thereof.
24. The subjects were neurodegenerative diseases, epilepsy; stroke; spinal cerebral ataxia; Canavan's disease; metachromatic leukodystrophy; spinal muscular ataxia; Friedrich ataxia; X-chain central nucleus myopathy; lysosome accumulation; Bath syndrome. 23. The method of 23 above, having Duchenne's muscular dystrophy; Wilson's disease; or Krigler-Nager syndrome type 1.
25. 24. The method of 24 above, wherein the neurodegenerative diseases are Parkinson's disease; Alzheimer's disease; Huntington's disease; amyotrophic lateral sclerosis; and multiple sclerosis.
26. 23. The method of 23 above, wherein the subject has brain cancer, wherein the method comprises administering AAV encoding an anti-cancer agent.
27. The anticancer agent is HSV. 26. The method of 26 above, wherein the method is TK1 and further comprises administering ganciclovir.
28. 22-27. The method of any of 22-27 above, wherein the cells are present in the subject's brain and the AAV is administered by parenteral delivery; intracerebral delivery; or intrathecal delivery.
29. 28. The method of 28 above, wherein the parenteral delivery is by intravenous delivery, intraarterial delivery, subcutaneous delivery, intraperitoneal delivery, or intramuscular delivery.
30. 28. The method according to 28 above, wherein the intrathecal delivery is by lumbar injection, cisterna magna injection, or intraparenchymal injection.
Claims (30)
28. The composition of claim 28, wherein the intrathecal delivery is by lumbar injection, cisterna magna injection, or intraparenchymal injection.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862696422P | 2018-07-11 | 2018-07-11 | |
| US62/696,422 | 2018-07-11 | ||
| PCT/US2019/041386 WO2020014471A1 (en) | 2018-07-11 | 2019-07-11 | Methods and compositions for delivery of agents across the blood-brain barrier |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021530218A JP2021530218A (en) | 2021-11-11 |
| JPWO2020014471A5 true JPWO2020014471A5 (en) | 2022-07-19 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021500538A Pending JP2021530218A (en) | 2018-07-11 | 2019-07-11 | Methods and compositions for the delivery of drugs across the blood-brain barrier |
Country Status (6)
| Country | Link |
|---|---|
| US (3) | US20210277066A1 (en) |
| EP (1) | EP3820885A4 (en) |
| JP (1) | JP2021530218A (en) |
| CN (1) | CN112703198A (en) |
| CA (1) | CA3105381A1 (en) |
| WO (1) | WO2020014471A1 (en) |
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| US20210277066A1 (en) | 2018-07-11 | 2021-09-09 | The Brigham And Women`S Hospital, Inc. | Methods and compositions for delivery of agents across the blood-brain barrier |
| US20210277418A1 (en) | 2018-08-03 | 2021-09-09 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| CN113966399A (en) | 2018-09-26 | 2022-01-21 | 加州理工学院 | Adeno-associated virus compositions for targeted gene therapy |
| JP2021534809A (en) | 2018-10-02 | 2021-12-16 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. | Redirection of AAV capsid orientation |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| US20220143214A1 (en) | 2019-01-30 | 2022-05-12 | The Broad Institute, Inc. | Systems for evolved adeno-associated viruses (aavs) for targeted delivery |
| WO2020210655A1 (en) | 2019-04-11 | 2020-10-15 | California Institute Of Technology | Virus compositions with enhanced specificity in the brain |
| WO2021025995A1 (en) | 2019-08-02 | 2021-02-11 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
-
2019
- 2019-07-11 US US17/258,846 patent/US20210277066A1/en active Pending
- 2019-07-11 WO PCT/US2019/041386 patent/WO2020014471A1/en unknown
- 2019-07-11 EP EP19833958.2A patent/EP3820885A4/en active Pending
- 2019-07-11 CN CN201980059342.1A patent/CN112703198A/en active Pending
- 2019-07-11 CA CA3105381A patent/CA3105381A1/en active Pending
- 2019-07-11 JP JP2021500538A patent/JP2021530218A/en active Pending
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2021
- 2021-12-02 US US17/457,337 patent/US11518787B2/en active Active
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2022
- 2022-10-21 US US18/048,716 patent/US20230077490A1/en active Pending
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