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
- 108700019146 Transgenes Proteins 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 5
- 210000003594 spinal ganglia Anatomy 0.000 abstract 3
- 108091070501 miRNA Proteins 0.000 abstract 2
- 108091026890 Coding region Proteins 0.000 abstract 1
- 210000000234 capsid Anatomy 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
-
- 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
-
- 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 nucleic acids [NA]
- 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)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (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)
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 (enExample) |
| EP (1) | EP4162059A1 (enExample) |
| JP (1) | JP2023526310A (enExample) |
| KR (1) | KR20230010670A (enExample) |
| CN (1) | CN115803064A (enExample) |
| AU (1) | AU2021270447A1 (enExample) |
| BR (1) | BR112022022212A2 (enExample) |
| CA (1) | CA3177407A1 (enExample) |
| CO (1) | CO2022017959A2 (enExample) |
| IL (1) | IL298138A (enExample) |
| MX (1) | MX2022014258A (enExample) |
| WO (1) | WO2021231579A1 (enExample) |
Families Citing this family (9)
| 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 |
| EP4409010A1 (en) | 2021-10-02 | 2024-08-07 | 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 |
| 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 |
| EP4504797A1 (en) | 2022-04-06 | 2025-02-12 | The Trustees of The University of Pennsylvania | Compositions and methods for treating her2 positive metastatic breast cancer and other cancers |
| AU2023303882A1 (en) * | 2022-07-08 | 2025-01-23 | Sensorion | REGULATORY SEQUENCES COMPRISING MicroRNA TARGET SITES |
| US20260021205A1 (en) | 2022-07-15 | 2026-01-22 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| IL326390A (en) | 2023-08-10 | 2026-04-01 | Univ Pennsylvania | Compositions and methods for treating spinal muscular atrophy |
Family Cites Families (47)
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| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| CA2609142C (en) * | 2005-05-27 | 2016-02-09 | Fondazione Centro San Raffaele Del Monte Tabor | Therapeutic gene vectors comprising mirna target sequences |
| 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 |
| ES2582091T3 (es) | 2005-10-18 | 2016-09-09 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| EP2215223B1 (en) | 2007-10-31 | 2013-05-01 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| CA2729961C (en) | 2008-07-09 | 2018-05-01 | Biogen Idec Ma Inc. | Li113, li62 variant co2, anti-lingo antibodies |
| EP2313498B1 (en) | 2008-07-14 | 2017-03-15 | Precision Biosciences, Inc. | Recognition sequences for i-crei-derived meganucleases and uses thereof |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | 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 |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of 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 |
| AU2014235096B2 (en) | 2013-03-15 | 2020-03-19 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| FR3004463A1 (fr) * | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| SMT202000531T1 (it) | 2014-03-09 | 2020-11-10 | Univ Pennsylvania | Composizioni utili in trattamento di deficit di ornitina transcarbamilasi(otc) |
| EP3134431B1 (en) | 2014-04-25 | 2021-04-07 | The Trustees Of The University Of Pennsylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| WO2015164723A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| CN108779167A (zh) | 2015-10-28 | 2018-11-09 | 宾夕法尼亚州大学信托人 | 鞘内施用腺伴随病毒载体用于基因治疗 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| MX2018007234A (es) | 2015-12-14 | 2018-11-09 | Univ Pennsylvania | Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal. |
| US20180363000A1 (en) | 2015-12-14 | 2018-12-20 | The Trustees Of The University Of Pennsylvania | Aav-anti pcsk9 antibody constructs and uses thereof |
| JP7061067B2 (ja) | 2015-12-14 | 2022-04-27 | 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 | クリグラー・ナジャー症候群の処置のための組成物 |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| US11191847B2 (en) | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
| MX2018012537A (es) | 2016-04-15 | 2019-02-25 | Univ Pennsylvania | Terapia de genes para tratar hemofilia a. |
| SG10201912761UA (en) | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| IL301059B2 (en) | 2017-04-21 | 2026-04-01 | Prec Biosciences Inc | Engineered nucleosides specific for recognition sequences in the PCSK9 gene |
| IL316926A (en) | 2017-05-11 | 2025-01-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinosis |
| US11680275B2 (en) * | 2017-06-06 | 2023-06-20 | University Of Massachusetts | Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome |
| CN111989396A (zh) | 2017-11-30 | 2020-11-24 | 宾夕法尼亚州大学信托人 | 用于iiib型粘多糖贮积病的基因疗法 |
| AU2018375164B2 (en) | 2017-11-30 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for Mucopolysaccharidosis IIIA |
| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CA3133889A1 (en) * | 2019-04-24 | 2020-10-29 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| KR20220020261A (ko) * | 2019-05-03 | 2022-02-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 이염성 백질이영양증의 치료에 유용한 조성물 |
| WO2021081217A1 (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 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 EP EP21733864.9A patent/EP4162059A1/en active Pending
- 2021-05-12 US US17/998,328 patent/US20230304034A1/en active Pending
- 2021-05-12 JP JP2022569265A patent/JP2023526310A/ja active Pending
- 2021-05-12 IL IL298138A patent/IL298138A/en unknown
- 2021-05-12 CA CA3177407A patent/CA3177407A1/en active Pending
- 2021-05-12 WO PCT/US2021/032003 patent/WO2021231579A1/en not_active Ceased
- 2021-05-12 BR BR112022022212A patent/BR112022022212A2/pt unknown
- 2021-05-12 KR KR1020227042727A patent/KR20230010670A/ko active Pending
-
2022
- 2022-12-12 CO CONC2022/0017959A patent/CO2022017959A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230010670A (ko) | 2023-01-19 |
| IL298138A (en) | 2023-01-01 |
| WO2021231579A1 (en) | 2021-11-18 |
| EP4162059A1 (en) | 2023-04-12 |
| CO2022017959A2 (es) | 2023-03-07 |
| AU2021270447A1 (en) | 2023-01-05 |
| JP2023526310A (ja) | 2023-06-21 |
| CN115803064A (zh) | 2023-03-14 |
| CA3177407A1 (en) | 2021-11-18 |
| US20230304034A1 (en) | 2023-09-28 |
| MX2022014258A (es) | 2023-02-22 |
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