JPWO2021231579A5 - - Google Patents
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- JPWO2021231579A5 JPWO2021231579A5 JP2022569265A JP2022569265A JPWO2021231579A5 JP WO2021231579 A5 JPWO2021231579 A5 JP WO2021231579A5 JP 2022569265 A JP2022569265 A JP 2022569265A JP 2022569265 A JP2022569265 A JP 2022569265A JP WO2021231579 A5 JPWO2021231579 A5 JP WO2021231579A5
- Authority
- JP
- Japan
- Prior art keywords
- mir
- target sequences
- raav
- composition according
- gene product
- 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.)
- Pending
Links
- 108091070501 miRNA Proteins 0.000 claims 21
- 239000000203 mixture Substances 0.000 claims 18
- 108090000623 proteins and genes Proteins 0.000 claims 14
- 210000003594 spinal ganglia Anatomy 0.000 claims 9
- 108091026890 Coding region Proteins 0.000 claims 7
- 239000008194 pharmaceutical composition Substances 0.000 claims 7
- 125000006850 spacer group Chemical group 0.000 claims 7
- 239000013598 vector Substances 0.000 claims 7
- 108091023796 miR-182 stem-loop Proteins 0.000 claims 6
- 108091029500 miR-183 stem-loop Proteins 0.000 claims 6
- 108020005345 3' Untranslated Regions Proteins 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 108020003589 5' Untranslated Regions Proteins 0.000 claims 3
- 210000004027 cell Anatomy 0.000 claims 3
- 238000001476 gene delivery Methods 0.000 claims 3
- 210000002569 neuron Anatomy 0.000 claims 3
- 230000001105 regulatory effect Effects 0.000 claims 3
- 108700019146 Transgenes Proteins 0.000 claims 2
- 238000007913 intrathecal administration Methods 0.000 claims 2
- 239000002679 microRNA Substances 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 229920001661 Chitosan Polymers 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 108091061960 Naked DNA Proteins 0.000 claims 1
- 241000125945 Protoparvovirus Species 0.000 claims 1
- 210000003050 axon Anatomy 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 230000000747 cardiac effect Effects 0.000 claims 1
- 210000003169 central nervous system Anatomy 0.000 claims 1
- 230000007850 degeneration Effects 0.000 claims 1
- 238000009472 formulation Methods 0.000 claims 1
- 239000012537 formulation buffer Substances 0.000 claims 1
- 238000001415 gene therapy Methods 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 239000010954 inorganic particle Substances 0.000 claims 1
- 238000000185 intracerebroventricular administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000003205 muscle Anatomy 0.000 claims 1
- 230000004770 neurodegeneration Effects 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 239000002245 particle Substances 0.000 claims 1
- 229920000642 polymer Polymers 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 1
- 230000009885 systemic effect Effects 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241001430294 unidentified retrovirus Species 0.000 claims 1
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063023593P | 2020-05-12 | 2020-05-12 | |
| US63/023,593 | 2020-05-12 | ||
| US202063038488P | 2020-06-12 | 2020-06-12 | |
| US63/038,488 | 2020-06-12 | ||
| US202063043562P | 2020-06-24 | 2020-06-24 | |
| US63/043,562 | 2020-06-24 | ||
| US202063079299P | 2020-09-16 | 2020-09-16 | |
| US63/079,299 | 2020-09-16 | ||
| US202163152042P | 2021-02-22 | 2021-02-22 | |
| US63/152,042 | 2021-02-22 | ||
| PCT/US2021/032003 WO2021231579A1 (en) | 2020-05-12 | 2021-05-12 | Compositions for drg-specific reduction of transgene expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023526310A JP2023526310A (ja) | 2023-06-21 |
| JPWO2021231579A5 true JPWO2021231579A5 (es) | 2024-05-22 |
Family
ID=76532231
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022569265A Pending JP2023526310A (ja) | 2020-05-12 | 2021-05-12 | 導入遺伝子発現のｄｒｇ特異的低減のための組成物 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230304034A1 (es) |
| EP (1) | EP4162059A1 (es) |
| JP (1) | JP2023526310A (es) |
| KR (1) | KR20230010670A (es) |
| CN (1) | CN115803064A (es) |
| AU (1) | AU2021270447A1 (es) |
| BR (1) | BR112022022212A2 (es) |
| CA (1) | CA3177407A1 (es) |
| CO (1) | CO2022017959A2 (es) |
| IL (1) | IL298138A (es) |
| MX (1) | MX2022014258A (es) |
| WO (1) | WO2021231579A1 (es) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2023012513A (es) | 2021-04-23 | 2023-12-15 | Univ Pennsylvania | Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen. |
| WO2023056399A1 (en) | 2021-10-02 | 2023-04-06 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| WO2023133574A1 (en) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for treatment of c9orf72-mediated disorders |
| WO2023196893A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating her2 positive metastatic breast cancer and other cancers |
| WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| TW202409278A (zh) * | 2022-07-08 | 2024-03-01 | 法商聖索瑞恩公司 | 包含ＭｉｃｒｏＲＮＡ靶位點的調節序列 |
| WO2024015966A2 (en) | 2022-07-15 | 2024-01-18 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
Family Cites Families (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2308989T3 (es) | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios. |
| ATE520707T1 (de) | 2001-11-13 | 2011-09-15 | Univ Pennsylvania | Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen |
| EP2359869B1 (en) | 2001-12-17 | 2018-12-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP3409296A1 (en) | 2005-04-07 | 2018-12-05 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
| DK2002003T3 (en) * | 2005-05-27 | 2016-03-21 | Ospedale San Raffaele Srl | Gene vector comprising miRNA |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| US20070027438A1 (en) | 2005-07-26 | 2007-02-01 | Frieder Loesel | System and method for compensating a corneal dissection |
| EP2484758B1 (en) | 2005-10-18 | 2013-10-02 | Precision Biosciences | Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| EP3098309B1 (en) | 2007-10-31 | 2019-04-10 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| EP2315779A2 (en) | 2008-07-09 | 2011-05-04 | Biogen Idec MA Inc. | Compositions comprising antibodies to lingo or fragments thereof |
| ES2625941T3 (es) | 2008-07-14 | 2017-07-21 | Precision Biosciences, Inc. | Secuencias de reconocimiento para meganucleasas derivadas de i-crei y sus usos |
| CN104910025B (zh) | 2008-11-07 | 2019-07-16 | 麻省理工学院 | 氨基醇类脂质和其用途 |
| SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| CA3066596A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| KR102128248B1 (ko) | 2011-06-08 | 2020-07-01 | 샤이어 휴먼 지네틱 테라피즈 인크. | Mrna 전달을 위한 지질 나노입자 조성물 및 방법 |
| DK2858677T3 (da) | 2012-06-08 | 2020-08-31 | Ethris Gmbh | Pulmonær levering af messenger rna |
| WO2014125647A1 (ja) | 2013-02-18 | 2014-08-21 | 富士通オプティカルコンポーネンツ株式会社 | 光受信装置 |
| JP6591956B2 (ja) | 2013-03-15 | 2019-10-16 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｍｐｓ１を治療するための組成物および方法 |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| CA2939950C (en) | 2014-03-09 | 2023-08-22 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
| CA2946392A1 (en) | 2014-04-25 | 2015-10-29 | James M. Wilson | Ldlr variants and their use in compositions for reducing cholesterol levels |
| US10780182B2 (en) | 2014-04-25 | 2020-09-22 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| ES2856090T3 (es) | 2014-09-24 | 2021-09-27 | Hope City | Variantes de vectores de virus adenoasociados para la edición genómica de alta eficacia y sus métodos |
| WO2017075119A1 (en) | 2015-10-28 | 2017-05-04 | The Trustees Of The Univeresity Of Pennsylvania | Intrathecal administration of adeno-associated-viral vectors for gene therapy |
| WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| JP7082050B2 (ja) | 2015-12-14 | 2022-06-07 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 脊髄性筋萎縮症の処置において有用なアデノ－関連ウイルスベクター |
| WO2017106326A1 (en) | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Aav-anti pcsk9 antibody constructs and uses thereof |
| WO2017106345A1 (en) | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Composition for treatment of crigler-najjar syndrome |
| AU2017215211C1 (en) | 2016-02-03 | 2023-11-16 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| WO2017180861A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsulvania | Gene therapy for treating hemophilia b |
| KR20230125339A (ko) | 2016-04-15 | 2023-08-29 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| WO2017180857A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia a |
| SG11201907714UA (en) | 2017-02-28 | 2019-09-27 | Univ Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| JP2020517255A (ja) | 2017-04-21 | 2020-06-18 | プレシジョン バイオサイエンシズ，インク． | Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ |
| JP7273730B2 (ja) | 2017-05-11 | 2023-05-15 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 神経セロイドリポフスチン症のための遺伝子療法 |
| EP3717653A4 (en) | 2017-11-30 | 2021-12-01 | The Trustees of The University of Pennsylvania | GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS TYPE IIIA |
| WO2019108856A1 (en) | 2017-11-30 | 2019-06-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis iiib |
| AU2019401314A1 (en) * | 2018-12-21 | 2021-06-24 | The Trustees Of The University Of Pennsylvania | Compositions for DRG-specific reduction of transgene expression |
| KR20220003553A (ko) * | 2019-04-24 | 2022-01-10 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 레트 증후군의 치료에 유용한 조성물 |
| CN114072129A (zh) * | 2019-05-03 | 2022-02-18 | 宾夕法尼亚州大学信托人 | 可用于治疗异染性脑白质营养不良的组合物 |
| US20220389457A1 (en) * | 2019-10-23 | 2022-12-08 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
-
2021
- 2021-05-12 US US17/998,328 patent/US20230304034A1/en active Pending
- 2021-05-12 CA CA3177407A patent/CA3177407A1/en active Pending
- 2021-05-12 EP EP21733864.9A patent/EP4162059A1/en active Pending
- 2021-05-12 WO PCT/US2021/032003 patent/WO2021231579A1/en active Application Filing
- 2021-05-12 BR BR112022022212A patent/BR112022022212A2/pt unknown
- 2021-05-12 MX MX2022014258A patent/MX2022014258A/es unknown
- 2021-05-12 CN CN202180049076.1A patent/CN115803064A/zh active Pending
- 2021-05-12 AU AU2021270447A patent/AU2021270447A1/en active Pending
- 2021-05-12 IL IL298138A patent/IL298138A/en unknown
- 2021-05-12 JP JP2022569265A patent/JP2023526310A/ja active Pending
- 2021-05-12 KR KR1020227042727A patent/KR20230010670A/ko active Search and Examination
-
2022
- 2022-12-12 CO CONC2022/0017959A patent/CO2022017959A2/es unknown
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