AU2020260076A1 - Compositions and methods for treatment of cystic fibrosis - Google Patents

Compositions and methods for treatment of cystic fibrosis Download PDF

Info

Publication number
AU2020260076A1
AU2020260076A1 AU2020260076A AU2020260076A AU2020260076A1 AU 2020260076 A1 AU2020260076 A1 AU 2020260076A1 AU 2020260076 A AU2020260076 A AU 2020260076A AU 2020260076 A AU2020260076 A AU 2020260076A AU 2020260076 A1 AU2020260076 A1 AU 2020260076A1
Authority
AU
Australia
Prior art keywords
seq
polynucleotide
raav
sequence
variant
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
AU2020260076A
Other languages
English (en)
Inventor
John F. Engelhardt
Shen LIN
Yinghua Tang
Ziying Yan
Eric Yuen
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Iowa Research Foundation UIRF
Spirovant Sciences Inc
Original Assignee
University of Iowa Research Foundation UIRF
Spirovant Sciences Inc
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 University of Iowa Research Foundation UIRF, Spirovant Sciences Inc filed Critical University of Iowa Research Foundation UIRF
Publication of AU2020260076A1 publication Critical patent/AU2020260076A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2020260076A 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis Pending AU2020260076A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962833972P 2019-04-15 2019-04-15
US62/833,972 2019-04-15
US201962926308P 2019-10-25 2019-10-25
US62/926,308 2019-10-25
US202062967214P 2020-01-29 2020-01-29
US62/967,214 2020-01-29
PCT/US2020/028264 WO2020214668A1 (en) 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis

Publications (1)

Publication Number Publication Date
AU2020260076A1 true AU2020260076A1 (en) 2021-12-09

Family

ID=70847479

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020260076A Pending AU2020260076A1 (en) 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis

Country Status (14)

Country Link
US (1) US20220241436A1 (https=)
EP (1) EP3955971A1 (https=)
JP (1) JP7731801B2 (https=)
KR (1) KR20220044899A (https=)
CN (1) CN114641318A (https=)
AU (1) AU2020260076A1 (https=)
BR (1) BR112021020708A2 (https=)
CA (1) CA3137015A1 (https=)
CL (1) CL2021002702A1 (https=)
IL (1) IL287260A (https=)
MX (1) MX2021012681A (https=)
PH (1) PH12021552610A1 (https=)
SG (1) SG11202111334SA (https=)
WO (1) WO2020214668A1 (https=)

Families Citing this family (8)

* 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
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
BR112021020706A2 (pt) * 2019-04-15 2022-03-15 Spirovant Sciences Inc Métodos e composições para expressão de transgene
WO2022006253A2 (en) * 2020-06-30 2022-01-06 University Of Iowa Research Foundation Methods and compositions for administering recombinant viral vectors
WO2022221684A1 (en) 2021-04-15 2022-10-20 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis
WO2026072721A1 (en) * 2024-09-25 2026-04-02 Carbon Biosciences, Inc. Parvovirus compositions and methods for gene therapy

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5656785A (en) 1995-08-07 1997-08-12 The Charles Stark Draper Laboratory, Inc. Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor
AU5445998A (en) 1996-11-19 1998-06-10 Surgx Corporation A transient voltage protection device and method of making same
JP2001506133A (ja) 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの産生における使用のための、ａａｖスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株
AU2004227358B8 (en) 2003-03-31 2009-12-10 Targeted Genetics Corporation Compounds and methods to enhance rAAV transduction
US9233131B2 (en) * 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US7407801B2 (en) * 2003-12-05 2008-08-05 University Of Iowa Research Foundation Truncated CMV promoters and vectors containing same
RS57739B1 (sr) * 2013-03-14 2018-12-31 Translate Bio Inc Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
WO2014168953A1 (en) * 2013-04-08 2014-10-16 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
US11000597B2 (en) * 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
CA3016985C (en) * 2016-03-07 2023-07-04 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
BR112021020706A2 (pt) * 2019-04-15 2022-03-15 Spirovant Sciences Inc Métodos e composições para expressão de transgene

Also Published As

Publication number Publication date
JP7731801B2 (ja) 2025-09-01
CL2021002702A1 (es) 2022-11-11
KR20220044899A (ko) 2022-04-12
JP2022529457A (ja) 2022-06-22
CA3137015A1 (en) 2020-10-22
IL287260A (en) 2021-12-01
EP3955971A1 (en) 2022-02-23
SG11202111334SA (en) 2021-11-29
BR112021020708A2 (pt) 2022-03-15
US20220241436A1 (en) 2022-08-04
PH12021552610A1 (en) 2022-06-27
MX2021012681A (es) 2022-03-25
CN114641318A (zh) 2022-06-17
WO2020214668A1 (en) 2020-10-22

Similar Documents

Publication Publication Date Title
JP7731801B2 (ja) 嚢胞性線維症の治療のための組換えアデノ随伴ウイルス
CN105431170B (zh) 嵌合腺相关病毒/博卡病毒细小病毒载体
US20220195461A1 (en) Methods and compositions for transgene expression
JP6495273B2 (ja) 変異ａａｖ、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
US8137962B2 (en) Compositions for treating cystic fibrosis
JP2021510528A (ja) 遺伝子治療のための修飾ｒＡＡＶキャプシドタンパク質
US20230242941A1 (en) Methods and compositions for administering recombinant viral vectors
CN115960921B (zh) 一组肝靶向新型腺相关病毒的获得及其应用
JP4863874B2 (ja) 関節リウマチのインビボ遺伝子治療のためのａａｖベクター
EA050636B1 (ru) Композиции и способы лечения муковисцидоза
US20240115738A1 (en) Methods and compositions for treatment of cystic fibrosis
WO2025259575A9 (en) Methods and compositions for treatment of duchenne muscular dystrophy
HK1217916B (zh) 嵌合腺相關病毒/博卡病毒細小病毒載體