MX2022014258A - Composiciones para la reducción específica de un fármaco de la expresión de transgén. - Google Patents
Composiciones para la reducción específica de un fármaco de la expresión de transgén.Info
- Publication number
- MX2022014258A MX2022014258A MX2022014258A MX2022014258A MX2022014258A MX 2022014258 A MX2022014258 A MX 2022014258A MX 2022014258 A MX2022014258 A MX 2022014258A MX 2022014258 A MX2022014258 A MX 2022014258A MX 2022014258 A MX2022014258 A MX 2022014258A
- Authority
- MX
- Mexico
- Prior art keywords
- gene product
- mir
- drg
- vector genome
- compositions
- Prior art date
Links
- 108700019146 Transgenes Proteins 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 abstract 5
- 108091026890 Coding region Proteins 0.000 abstract 2
- 108091070501 miRNA Proteins 0.000 abstract 2
- 210000003594 spinal ganglia Anatomy 0.000 abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 108091023796 miR-182 stem-loop Proteins 0.000 abstract 1
- 108091029500 miR-183 stem-loop Proteins 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
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
-
- 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
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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
- 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)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (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)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Se proporciona un AAV recombinante (rAAV) para la entrega de un producto génico a un paciente que lo necesita, que reprime específicamente la expresión del producto génico en los ganglios de la raíz dorsal (DRG). El rAAV comprende una cápside de AAV que tiene empaquetado en ella un genoma vectorial, en donde el genoma vectorial comprende: (a) una secuencia codificadora para el producto génico bajo el control de secuencias reguladoras que dirigen la expresión del producto génico en una célula que contiene el genoma del vector; y (b) al menos ocho secuencias diana de miR, en donde cada secuencia diana es específica para miR-183 o miR-182, y en donde las al menos ocho secuencias diana de miR están vinculadas operativamente al extremo 3' de la secuencia codificadora. También se proporcionan métodos y usos de los rAAV descritos para la entrega de un producto genético a un paciente que lo necesite.
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 |
|---|---|
| MX2022014258A true MX2022014258A (es) | 2023-02-22 |
Family
ID=76532231
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2022014258A MX2022014258A (es) | 2020-05-12 | 2021-05-12 | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
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 |
|---|---|---|---|---|
| AR125406A1 (es) | 2021-04-23 | 2023-07-12 | Univ Pennsylvania | Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen |
| TW202325845A (zh) | 2021-10-02 | 2023-07-01 | 賓州大學委員會 | 新穎aav衣殼及含其之組成物 |
| 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 |
| 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 |
| 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 |
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| JP4827353B2 (ja) | 1999-08-09 | 2011-11-30 | ターゲティッド ジェネティクス コーポレイション | 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大 |
| SG157224A1 (en) | 2001-11-13 | 2009-12-29 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| DK2359867T3 (en) | 2005-04-07 | 2015-01-05 | Univ Pennsylvania | A method for increasing an AAV vector function |
| KR20130114758A (ko) * | 2005-05-27 | 2013-10-17 | 오스페달레 산 라파엘 에스.알.엘. | Ｍｉ-ｒｎａ를 포함하는 유전자 벡터 |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| US20070027438A1 (en) | 2005-07-26 | 2007-02-01 | Frieder Loesel | System and method for compensating a corneal dissection |
| JP5937292B2 (ja) | 2005-10-18 | 2016-06-22 | デューク大学 | 配列特異性およびｄｎａ−結合親和度が変更された、合理設計メガヌクレアーゼ |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| DK2215223T3 (da) | 2007-10-31 | 2013-07-22 | Prec Biosciences Inc | Rationelt konstruerede enkeltkæde-meganucleaser med ikke-palindrome genkendelsessekvenser |
| WO2010005570A2 (en) | 2008-07-09 | 2010-01-14 | Biogen Idec Ma Inc. | Compositions comprising antibodies to lingo or fragments thereof |
| CA2730921A1 (en) | 2008-07-14 | 2010-01-21 | Precision Biosciences, Inc. | Recognition sequences for i-crei-derived meganucleases and uses thereof |
| JP6087504B2 (ja) | 2008-11-07 | 2017-03-01 | マサチューセッツ インスティテュート オブ テクノロジー | アミノアルコールリピドイドおよびその使用 |
| CA2793633A1 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| JP6184945B2 (ja) | 2011-06-08 | 2017-08-23 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | ｍＲＮＡ送達のための脂質ナノ粒子組成物および方法 |
| BR112014029807A2 (pt) | 2012-06-08 | 2017-06-27 | Ethris Gmbh | administração pulmonar de rna mensageiro |
| 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 |
| EP3747998A1 (en) | 2013-03-15 | 2020-12-09 | The Trustees of the University of Pennsylvania | Compositions for treating mpsi |
| EP3044318B1 (en) | 2013-09-13 | 2019-05-01 | California Institute of Technology | Selective recovery |
| HUE051311T2 (hu) | 2014-03-09 | 2021-03-01 | Univ Pennsylvania | Ornitin transzkarbamiláz (TC) deficiencia kezelésében alkalmas készítmények |
| 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 |
| JP6741590B2 (ja) | 2014-04-25 | 2020-08-19 | 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 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| WO2016049230A1 (en) | 2014-09-24 | 2016-03-31 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| BR112018008407A2 (pt) | 2015-10-28 | 2018-11-27 | Univ Pennsylvania | administração intratecal de vetores virais adenoassociados para terapia genética |
| WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| WO2017106326A1 (en) | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Aav-anti pcsk9 antibody constructs and uses thereof |
| CN109072254A (zh) | 2015-12-14 | 2018-12-21 | 宾夕法尼亚州大学信托人 | 用于治疗脊髓性肌萎缩的腺相关病毒载体 |
| AU2016370590B2 (en) | 2015-12-14 | 2023-11-02 | The Trustees Of The University Of Pennsylvania | Composition for treatment of Crigler-Najjar syndrome |
| SG11201806270XA (en) | 2016-02-03 | 2018-08-30 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| IL305449A (en) | 2016-04-15 | 2023-10-01 | Univ Pennsylvania | Gene therapy for the treatment of type II mucositis |
| CA3019425A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia a |
| WO2017180861A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsulvania | Gene therapy for treating hemophilia b |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| HRP20240257T1 (hr) | 2017-02-28 | 2024-05-24 | The Trustees Of The University Of Pennsylvania | Adeno-povezani virusni (aav) clade f vektor i njegova uporaba |
| CN110769845B (zh) | 2017-04-21 | 2023-08-15 | 精密生物科学公司 | 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶 |
| KR20200023280A (ko) | 2017-05-11 | 2020-03-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 신경 세로이드 지질갈색소증에 대한 유전자 요법 |
| EP3717652A4 (en) | 2017-11-30 | 2021-11-24 | The Trustees of the University of Pennsylvania | GENE THERAPY FOR MUCOPOLY SACCHARIDOSIS IIIB |
| CA3083416A1 (en) | 2017-11-30 | 2019-06-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis iii a |
| JOP20210160A1 (ar) * | 2018-12-21 | 2023-01-30 | Univ Pennsylvania | تركيبات لخفض التعبير عن الجين الناقل بشكل انتقائي لـ drg |
| CN114026236A (zh) * | 2019-04-24 | 2022-02-08 | 宾夕法尼亚州大学信托人 | 可用于治疗雷特综合征的组合物 |
| WO2020227166A1 (en) * | 2019-05-03 | 2020-11-12 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| CA3155154A1 (en) * | 2019-10-23 | 2021-04-29 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
-
2021
- 2021-05-12 JP JP2022569265A patent/JP2023526310A/ja active Pending
- 2021-05-12 BR BR112022022212A patent/BR112022022212A2/pt unknown
- 2021-05-12 EP EP21733864.9A patent/EP4162059A1/en active Pending
- 2021-05-12 KR KR1020227042727A patent/KR20230010670A/ko active Search and Examination
- 2021-05-12 CN CN202180049076.1A patent/CN115803064A/zh active Pending
- 2021-05-12 US US17/998,328 patent/US20230304034A1/en active Pending
- 2021-05-12 AU AU2021270447A patent/AU2021270447A1/en active Pending
- 2021-05-12 CA CA3177407A patent/CA3177407A1/en active Pending
- 2021-05-12 IL IL298138A patent/IL298138A/en unknown
- 2021-05-12 MX MX2022014258A patent/MX2022014258A/es unknown
- 2021-05-12 WO PCT/US2021/032003 patent/WO2021231579A1/en active Application Filing
-
2022
- 2022-12-12 CO CONC2022/0017959A patent/CO2022017959A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| BR112022022212A2 (pt) | 2022-12-13 |
| AU2021270447A1 (en) | 2023-01-05 |
| CO2022017959A2 (es) | 2023-03-07 |
| KR20230010670A (ko) | 2023-01-19 |
| JP2023526310A (ja) | 2023-06-21 |
| US20230304034A1 (en) | 2023-09-28 |
| EP4162059A1 (en) | 2023-04-12 |
| CA3177407A1 (en) | 2021-11-18 |
| WO2021231579A1 (en) | 2021-11-18 |
| CN115803064A (zh) | 2023-03-14 |
| IL298138A (en) | 2023-01-01 |
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