BR112022022212A2 - Composições para redução específica de drg de expressão de transgene - Google Patents
Composições para redução específica de drg de expressão de transgeneInfo
- Publication number
- BR112022022212A2 BR112022022212A2 BR112022022212A BR112022022212A BR112022022212A2 BR 112022022212 A2 BR112022022212 A2 BR 112022022212A2 BR 112022022212 A BR112022022212 A BR 112022022212A BR 112022022212 A BR112022022212 A BR 112022022212A BR 112022022212 A2 BR112022022212 A2 BR 112022022212A2
- Authority
- BR
- Brazil
- Prior art keywords
- gene product
- mir
- compositions
- drg
- vector genome
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- Dermatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- General Preparation And Processing Of Foods (AREA)
- Coloring Foods And Improving Nutritive Qualities (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
COMPOSIÇÕES PARA REDUÇÃO ESPECÍFICA DE DRG DE EXPRESSÃO DE TRANSGENE. É fornecido um AAV recombinante (rAAV) para distribuição de um produto genético a um paciente em necessidade do mesmo que reprime especificamente a expressão do produto genético nos gânglios da raiz dorsal (DRG). O rAAV compreende um capsídeo de AAV tendo empacotado nele um genoma de vetor, em que o genoma de vetor compreende: (a) uma sequência de codificação para o produto genético sob o controle de sequências regulatórias que direcionam a expressão do produto genético em uma célula contendo o genoma de vetor; e (b) pelo menos oito sequências-alvo de miR, em que cada sequência-alvo é específica para miR-183 ou miR-182, e em que as pelo menos oito sequências-alvo de miR estão operacionalmente ligadas à extremidade 3 da sequência de codificação. Também são fornecidos métodos e usos dos rAAVs descritos para distribuição de um produto genético a um paciente em necessidade do mesmo.
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063023593P | 2020-05-12 | 2020-05-12 | |
| US202063038488P | 2020-06-12 | 2020-06-12 | |
| US202063043562P | 2020-06-24 | 2020-06-24 | |
| US202063079299P | 2020-09-16 | 2020-09-16 | |
| US202163152042P | 2021-02-22 | 2021-02-22 | |
| PCT/US2021/032003 WO2021231579A1 (en) | 2020-05-12 | 2021-05-12 | Compositions for drg-specific reduction of transgene expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022022212A2 true BR112022022212A2 (pt) | 2022-12-13 |
Family
ID=76532231
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022022212A BR112022022212A2 (pt) | 2020-05-12 | 2021-05-12 | Composições para redução específica de drg de expressão de transgene |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230304034A1 (pt) |
| EP (1) | EP4162059A1 (pt) |
| JP (1) | JP2023526310A (pt) |
| KR (1) | KR20230010670A (pt) |
| CN (1) | CN115803064A (pt) |
| AU (1) | AU2021270447A1 (pt) |
| BR (1) | BR112022022212A2 (pt) |
| CA (1) | CA3177407A1 (pt) |
| CO (1) | CO2022017959A2 (pt) |
| IL (1) | IL298138A (pt) |
| MX (1) | MX2022014258A (pt) |
| WO (1) | WO2021231579A1 (pt) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3216004A1 (en) | 2021-04-23 | 2022-10-27 | The Trustees Of The University Of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| WO2023056399A1 (en) | 2021-10-02 | 2023-04-06 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| AR128239A1 (es) | 2022-01-10 | 2024-04-10 | Univ Pennsylvania | Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72 |
| 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 |
| 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 |
| WO2024008949A1 (en) * | 2022-07-08 | 2024-01-11 | Sensorion | REGULATORY SEQUENCES COMPRISING MicroRNA TARGET SITES |
| 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 |
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| CA2469785C (en) | 2001-12-17 | 2014-04-22 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| CN102199626B (zh) | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| CN101203613B (zh) | 2005-04-07 | 2012-12-12 | 宾夕法尼亚大学托管会 | 增强腺相关病毒载体功能的方法 |
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| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| US20070027438A1 (en) | 2005-07-26 | 2007-02-01 | Frieder Loesel | System and method for compensating a corneal dissection |
| EP2360244B1 (en) | 2005-10-18 | 2014-12-24 | 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 |
| WO2009059195A2 (en) | 2007-10-31 | 2009-05-07 | Precision Biosciences | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| NZ590605A (en) | 2008-07-09 | 2012-11-30 | Biogen Idec Inc | Compositions comprising antibodies to lingo or fragments thereof |
| ES2705756T3 (es) | 2008-07-14 | 2019-03-26 | Prec Biosciences Inc | Secuencias de reconocimiento para meganucleasas derivadas de I-Crel y sus usos |
| MX353900B (es) | 2008-11-07 | 2018-02-01 | Massachusetts Inst Technology | Lipidoides de aminoalcohol y usos de los mismos. |
| MX342858B (es) | 2010-03-29 | 2016-10-13 | The Trustees Of The Univ Of Pennsylvania * | Sistema de ablacion transgenica inducida farmacologicamente. |
| ES2698203T3 (es) | 2010-04-23 | 2019-02-01 | Univ Massachusetts | Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| NZ719345A (en) | 2011-06-08 | 2023-03-31 | Translate Bio Inc | Lipid nanoparticle compositions and methods for mrna delivery |
| KR20150020288A (ko) | 2012-06-08 | 2015-02-25 | 에트리스 게엠베하 | 메신저 rna의 폐전달 |
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| KR20180121899A (ko) | 2016-02-03 | 2018-11-09 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | I형 점액다당류증을 치료하기 위한 유전자 요법 |
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| SG10202009849WA (en) | 2016-04-15 | 2020-11-27 | Univ Pennsylvania | Gene therapy for treating hemophilia a |
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| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
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| CA3061655A1 (en) | 2017-05-11 | 2018-11-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
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| EP3717652A4 (en) | 2017-11-30 | 2021-11-24 | The Trustees of the University of Pennsylvania | GENE THERAPY FOR MUCOPOLY SACCHARIDOSIS IIIB |
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| KR20220003553A (ko) * | 2019-04-24 | 2022-01-10 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 레트 증후군의 치료에 유용한 조성물 |
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| EP4048785A4 (en) * | 2019-10-23 | 2024-03-27 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR DRG-SPECIFIC REDUCTION OF TRANSGENE EXPRESSION |
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2021
- 2021-05-12 AU AU2021270447A patent/AU2021270447A1/en active Pending
- 2021-05-12 KR KR1020227042727A patent/KR20230010670A/ko active Search and Examination
- 2021-05-12 IL IL298138A patent/IL298138A/en unknown
- 2021-05-12 JP JP2022569265A patent/JP2023526310A/ja active Pending
- 2021-05-12 MX MX2022014258A patent/MX2022014258A/es unknown
- 2021-05-12 WO PCT/US2021/032003 patent/WO2021231579A1/en active Application Filing
- 2021-05-12 EP EP21733864.9A patent/EP4162059A1/en active Pending
- 2021-05-12 BR BR112022022212A patent/BR112022022212A2/pt unknown
- 2021-05-12 CA CA3177407A patent/CA3177407A1/en active Pending
- 2021-05-12 CN CN202180049076.1A patent/CN115803064A/zh active Pending
- 2021-05-12 US US17/998,328 patent/US20230304034A1/en active Pending
-
2022
- 2022-12-12 CO CONC2022/0017959A patent/CO2022017959A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| JP2023526310A (ja) | 2023-06-21 |
| AU2021270447A1 (en) | 2023-01-05 |
| US20230304034A1 (en) | 2023-09-28 |
| WO2021231579A1 (en) | 2021-11-18 |
| CO2022017959A2 (es) | 2023-03-07 |
| CN115803064A (zh) | 2023-03-14 |
| MX2022014258A (es) | 2023-02-22 |
| EP4162059A1 (en) | 2023-04-12 |
| IL298138A (en) | 2023-01-01 |
| KR20230010670A (ko) | 2023-01-19 |
| CA3177407A1 (en) | 2021-11-18 |
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