BR112021020706A2 - Métodos e composições para expressão de transgene - Google Patents

Métodos e composições para expressão de transgene

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Publication number
BR112021020706A2
BR112021020706A2 BR112021020706A BR112021020706A BR112021020706A2 BR 112021020706 A2 BR112021020706 A2 BR 112021020706A2 BR 112021020706 A BR112021020706 A BR 112021020706A BR 112021020706 A BR112021020706 A BR 112021020706A BR 112021020706 A2 BR112021020706 A2 BR 112021020706A2
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BR
Brazil
Prior art keywords
methods
transgene
cell
compositions
transgene expression
Prior art date
Application number
BR112021020706A
Other languages
English (en)
Inventor
Eric Yuen
f engelhardt John
Shen Lin
Yinghua Tang
Ziying Yan
Original Assignee
Spirovant Sciences Inc
Univ Iowa Res Found
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Spirovant Sciences Inc, Univ Iowa Res Found filed Critical Spirovant Sciences Inc
Publication of BR112021020706A2 publication Critical patent/BR112021020706A2/pt

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/69—Boron compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/704—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/05—Dipeptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/06—Tripeptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/07—Tetrapeptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Physics & Mathematics (AREA)
  • Pulmonology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)

Abstract

métodos e composições e métodos para expressão de transgene. a divulgação fornece métodos para expressar um transgene em uma célula, métodos para tratar distúrbios em um sujeito em necessidade dos mesmos e composições farmacêuticas. em particular, os métodos envolvem contatar uma célula (por exemplo, uma célula de um sujeito sofrendo de um distúrbio como fibrose cística) com um vírus adenoassociado recombinante (raav) que inclui, em uma modalidade, uma proteína de capsídeo av.tl65 e um polinucleotídeo que inclui um transgene em combinação com um aumentador de transdução de aav, desse modo expressando o transgene na célula. a divulgação também fornece composições farmacêuticas que incluem um raav que inclui, em uma modalidade, uma proteína de capsídeo av.tl65 e um polinucleotídeo incluindo um transgene em combinação com um ou mais aumentadores.
BR112021020706A 2019-04-15 2020-04-15 Métodos e composições para expressão de transgene BR112021020706A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962833979P 2019-04-15 2019-04-15
US201962926317P 2019-10-25 2019-10-25
US202062967219P 2020-01-29 2020-01-29
PCT/US2020/028269 WO2020214672A1 (en) 2019-04-15 2020-04-15 Methods and compositions for transgene expression

Publications (1)

Publication Number Publication Date
BR112021020706A2 true BR112021020706A2 (pt) 2022-03-15

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ID=70779846

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021020706A BR112021020706A2 (pt) 2019-04-15 2020-04-15 Métodos e composições para expressão de transgene

Country Status (12)

Country Link
US (1) US20220195461A1 (pt)
EP (1) EP3955970A1 (pt)
JP (2) JP2022529470A (pt)
KR (1) KR20220047538A (pt)
CN (1) CN114340683A (pt)
AU (1) AU2020257182A1 (pt)
BR (1) BR112021020706A2 (pt)
CA (1) CA3137078A1 (pt)
IL (1) IL287262A (pt)
MX (1) MX2021012682A (pt)
SG (1) SG11202111353QA (pt)
WO (1) WO2020214672A1 (pt)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3016985C (en) 2016-03-07 2023-07-04 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
KR20220044899A (ko) * 2019-04-15 2022-04-12 유니버시티 오브 아이오와 리써치 파운데이션 낭성 섬유증의 치료를 위한 조성물 및 방법

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WO1995013392A1 (en) 1993-11-09 1995-05-18 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
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KR20220044899A (ko) * 2019-04-15 2022-04-12 유니버시티 오브 아이오와 리써치 파운데이션 낭성 섬유증의 치료를 위한 조성물 및 방법

Also Published As

Publication number Publication date
WO2020214672A1 (en) 2020-10-22
SG11202111353QA (en) 2021-11-29
EP3955970A1 (en) 2022-02-23
IL287262A (en) 2021-12-01
AU2020257182A1 (en) 2021-12-09
CN114340683A (zh) 2022-04-12
JP2023126658A (ja) 2023-09-07
CA3137078A1 (en) 2020-10-22
KR20220047538A (ko) 2022-04-18
MX2021012682A (es) 2022-03-25
US20220195461A1 (en) 2022-06-23
JP2022529470A (ja) 2022-06-22

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