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 (https=) |
| EP (1) | EP4162059A1 (https=) |
| JP (1) | JP2023526310A (https=) |
| KR (1) | KR20230010670A (https=) |
| CN (1) | CN115803064A (https=) |
| AU (1) | AU2021270447A1 (https=) |
| BR (1) | BR112022022212A2 (https=) |
| CA (1) | CA3177407A1 (https=) |
| CO (1) | CO2022017959A2 (https=) |
| IL (1) | IL298138A (https=) |
| MX (1) | MX2022014258A (https=) |
| WO (1) | WO2021231579A1 (https=) |
Families Citing this family (9)
| 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 |
| EP4409010A1 (en) | 2021-10-02 | 2024-08-07 | 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 |
| JP2025512969A (ja) | 2022-04-06 | 2025-04-22 | 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 | Ｈｅｒ２陽性転移性乳がん及び他のがんを治療するための組成物及び方法 |
| US20250250326A1 (en) | 2022-04-06 | 2025-08-07 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| MA71383A (fr) * | 2022-07-08 | 2025-04-30 | Sensorion | Séquences régulatrices comprenant des sites cibles de micro-arn |
| 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 |
| AR133098A1 (es) | 2023-06-29 | 2025-08-27 | Univ Pennsylvania | Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen |
| 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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|---|---|---|---|---|
| PT1916258E (pt) | 1999-08-09 | 2014-07-29 | Genzyme Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 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 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| PL2002003T3 (pl) * | 2005-05-27 | 2016-06-30 | Ospedale San Raffaele Srl | Wektor genetyczny zawierający mi-RNA |
| 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 |
| CA2891996A1 (en) | 2005-10-18 | 2007-04-26 | Duke University | Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity |
| 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 |
| DK2982695T3 (da) | 2008-07-09 | 2019-05-13 | Biogen Ma Inc | Sammensætninger, der omfatter antistoffer mod lingo eller fragmenter deraf |
| DK3211075T3 (en) | 2008-07-14 | 2019-01-21 | Prec Biosciences Inc | RECOGNITION SEQUENCES FOR I-CREI-DERIVED MECHANUCLEASES AND APPLICATIONS THEREOF |
| ES2646630T3 (es) | 2008-11-07 | 2017-12-14 | Massachusetts Institute Of Technology | Lipidoides aminoalcohólicos y usos de los mismos |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | 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 |
| PL3586861T3 (pl) | 2011-06-08 | 2022-05-23 | Translate Bio, Inc. | Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna |
| EA037448B1 (ru) | 2012-06-08 | 2021-03-30 | Этрис Гмбх | Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция |
| 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 |
| SI2984166T1 (sl) | 2013-03-15 | 2020-09-30 | The Trustees Of The University Of Pennsylvania | Sestavki za zdravljenje MPSI |
| FR3004463A1 (fr) * | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| RS60902B1 (sr) | 2014-03-09 | 2020-11-30 | Univ Pennsylvania | Kompozicije korisne u lečenju nedostatka ornitinske transkarbamilaze (otc) |
| 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 |
| ES2876409T3 (es) | 2014-04-25 | 2021-11-12 | Univ Pennsylvania | Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol |
| BR112017005892A2 (pt) | 2014-09-24 | 2017-12-12 | Hope City | variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma |
| HK1257801A1 (zh) | 2015-10-28 | 2019-11-01 | 宾夕法尼亚州大学信托人 | 用於基因治疗的腺伴随病毒载体的鞘内给药 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| BR112018011975A2 (pt) | 2015-12-14 | 2018-12-11 | Univ Pennsylvania | composições úteis no tratamento de atrofia muscular espinhal |
| CA3007330A1 (en) | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Composition for treatment of crigler-najjar syndrome |
| US20180363000A1 (en) | 2015-12-14 | 2018-12-20 | The Trustees Of The University Of Pennsylvania | Aav-anti pcsk9 antibody constructs and uses thereof |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| IL305449B2 (en) | 2016-04-15 | 2026-01-01 | Univ Pennsylvania | Gene therapy for the treatment of mucocutaneous type ii diabetes |
| KR102450833B1 (ko) | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| EP3442597A1 (en) | 2016-04-15 | 2019-02-20 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia b |
| JP7455579B2 (ja) | 2017-02-28 | 2024-03-26 | 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 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| EP3612205B9 (en) | 2017-04-21 | 2023-10-04 | Precision Biosciences, Inc. | Engineered meganucleases specific for recognition sequences in the pcsk9 gene |
| KR102719222B1 (ko) | 2017-05-11 | 2024-10-21 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 신경 세로이드 지질갈색소증에 대한 유전자 요법 |
| US11680275B2 (en) * | 2017-06-06 | 2023-06-20 | University Of Massachusetts | Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome |
| KR20200104864A (ko) | 2017-11-30 | 2020-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 뮤코다당류증 iiib형에 대한 유전자 요법 |
| KR102823833B1 (ko) | 2017-11-30 | 2025-06-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 뮤코다당류증 iiia형에 대한 유전자 요법 |
| 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 |
| BR112021021908A2 (pt) * | 2019-05-03 | 2022-02-01 | Univ Pennsylvania | Composições úteis no tratamento da leucodistrofia metacromática |
| 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 US US17/998,328 patent/US20230304034A1/en active Pending
- 2021-05-12 IL IL298138A patent/IL298138A/en unknown
- 2021-05-12 EP EP21733864.9A patent/EP4162059A1/en active Pending
- 2021-05-12 CN CN202180049076.1A patent/CN115803064A/zh active Pending
- 2021-05-12 MX MX2022014258A patent/MX2022014258A/es unknown
- 2021-05-12 JP JP2022569265A patent/JP2023526310A/ja active Pending
- 2021-05-12 CA CA3177407A patent/CA3177407A1/en active Pending
- 2021-05-12 BR BR112022022212A patent/BR112022022212A2/pt unknown
- 2021-05-12 KR KR1020227042727A patent/KR20230010670A/ko active Pending
- 2021-05-12 AU AU2021270447A patent/AU2021270447A1/en active Pending
- 2021-05-12 WO PCT/US2021/032003 patent/WO2021231579A1/en not_active Ceased
-
2022
- 2022-12-12 CO CONC2022/0017959A patent/CO2022017959A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230010670A (ko) | 2023-01-19 |
| WO2021231579A1 (en) | 2021-11-18 |
| CO2022017959A2 (es) | 2023-03-07 |
| EP4162059A1 (en) | 2023-04-12 |
| CN115803064A (zh) | 2023-03-14 |
| JP2023526310A (ja) | 2023-06-21 |
| AU2021270447A1 (en) | 2023-01-05 |
| IL298138A (en) | 2023-01-01 |
| CA3177407A1 (en) | 2021-11-18 |
| MX2022014258A (es) | 2023-02-22 |
| US20230304034A1 (en) | 2023-09-28 |
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