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SG11202105907QA - Compositions for drg-specific reduction of transgene expression - Google Patents

Compositions for drg-specific reduction of transgene expression

Info

Publication number
SG11202105907QA
SG11202105907QA SG11202105907QA SG11202105907QA SG11202105907QA SG 11202105907Q A SG11202105907Q A SG 11202105907QA SG 11202105907Q A SG11202105907Q A SG 11202105907QA SG 11202105907Q A SG11202105907Q A SG 11202105907QA SG 11202105907Q A SG11202105907Q A SG 11202105907QA
Authority
SG
Singapore
Prior art keywords
drg
compositions
transgene expression
specific reduction
reduction
Prior art date
Application number
SG11202105907QA
Inventor
Juliette Hordeaux
James Wilson
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of SG11202105907QA publication Critical patent/SG11202105907QA/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG11202105907QA 2018-12-21 2019-12-20 Compositions for drg-specific reduction of transgene expression SG11202105907QA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862783956P 2018-12-21 2018-12-21
US201962924970P 2019-10-23 2019-10-23
US201962934915P 2019-11-13 2019-11-13
PCT/US2019/067872 WO2020132455A1 (en) 2018-12-21 2019-12-20 Compositions for drg-specific reduction of transgene expression

Publications (1)

Publication Number Publication Date
SG11202105907QA true SG11202105907QA (en) 2021-07-29

Family

ID=71100588

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202105907QA SG11202105907QA (en) 2018-12-21 2019-12-20 Compositions for drg-specific reduction of transgene expression

Country Status (17)

Country Link
US (1) US20210077553A1 (en)
EP (1) EP3908326A4 (en)
JP (1) JP7660062B2 (en)
KR (1) KR20210107037A (en)
CN (1) CN113646005A (en)
AU (1) AU2019401314A1 (en)
BR (1) BR112021011143A2 (en)
CA (1) CA3123600A1 (en)
CL (1) CL2021001624A1 (en)
CO (1) CO2021008538A2 (en)
IL (1) IL284185B2 (en)
JO (1) JOP20210160A1 (en)
MX (1) MX2021007600A (en)
PE (1) PE20211581A1 (en)
PH (1) PH12021551341A1 (en)
SG (1) SG11202105907QA (en)
WO (1) WO2020132455A1 (en)

Families Citing this family (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020270421A1 (en) 2019-05-03 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
MX2022012752A (en) 2020-04-15 2023-01-16 Voyager Therapeutics Inc Tau binding compounds.
WO2021231579A1 (en) * 2020-05-12 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
EP4149955A1 (en) 2020-05-13 2023-03-22 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
JP2023531451A (en) 2020-06-17 2023-07-24 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 Compositions and methods for treatment of gene therapy patients
US20230270884A1 (en) 2020-07-13 2023-08-31 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
MX2023000815A (en) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency.
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
EP4229186A1 (en) 2020-10-18 2023-08-23 The Trustees of The University of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
MX2023006445A (en) 2020-12-01 2023-08-10 Univ Pennsylvania Compositions and uses thereof for treatment of angelman syndrome.
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240175050A1 (en) * 2021-03-19 2024-05-30 Mayo Foundation For Medical Education And Research Methods and materials for treating propionic acidemia
WO2023113806A1 (en) 2021-12-16 2023-06-22 Affinia Therapeutics, Inc. Recombinant aav for treatment of neural disease
EP4323531A1 (en) 2021-04-12 2024-02-21 Affinia Therapeutics Inc. Recombinant aav for treatment of neural disease
WO2022226263A1 (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
WO2022232267A1 (en) 2021-04-27 2022-11-03 The Trustees Of The University Of Pennsylvania Porcine-derived adeno-associated virus capsids and uses thereof
WO2023044483A2 (en) 2021-09-20 2023-03-23 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
EP4409010A1 (en) 2021-10-02 2024-08-07 The Trustees of The University of Pennsylvania Novel aav capsids and compositions containing same
US20240425882A1 (en) * 2021-10-18 2024-12-26 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
WO2023087019A2 (en) * 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20250034559A1 (en) 2021-11-17 2025-01-30 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
EP4433169A1 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
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
EP4469585A2 (en) 2022-01-25 2024-12-04 Voyager Therapeutics, Inc. Baculovirus expression system
TW202346599A (en) 2022-02-08 2023-12-01 美商航海家醫療公司 Aav capsid variants and uses thereof
CN119317645A (en) 2022-04-06 2025-01-14 宾夕法尼亚州大学信托人 Compositions and methods for treating HER2-positive metastatic breast cancer and other cancers
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
EP4522757A2 (en) 2022-05-13 2025-03-19 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023235791A1 (en) 2022-06-02 2023-12-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
EP4543473A1 (en) 2022-06-22 2025-04-30 Voyager Therapeutics, Inc. Tau binding compounds
AR129733A1 (en) 2022-06-28 2024-09-25 Voyager Therapeutics Inc AAV CAPSID VARIANTS AND THEIR USES
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024008950A1 (en) * 2022-07-08 2024-01-11 Ospedale San Raffaele S.R.L. Transgene cassettes
EP4565597A2 (en) 2022-08-03 2025-06-11 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
IL319121A (en) 2022-09-15 2025-04-01 Voyager Therapeutics Inc Tau binding compounds
WO2024073310A2 (en) * 2022-09-26 2024-04-04 Encoded Therapeutics, Inc. Elements for de-targeting gene expression in dorsal root ganglion and/or liver
TW202440939A (en) 2022-12-17 2024-10-16 賓州大學委員會 Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024145474A2 (en) 2022-12-29 2024-07-04 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
WO2024228943A1 (en) 2023-05-02 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2024229161A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2024229389A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
TW202444917A (en) 2023-05-04 2024-11-16 美商航海家醫療公司 Aav capsid variants and uses thereof
WO2024235159A1 (en) * 2023-05-12 2024-11-21 上海金珂博生物技术有限公司 Viral vector carrying smn gene expression cassette and use thereof
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025122530A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025122634A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1838870A2 (en) * 2004-12-29 2007-10-03 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF MICRORNAS AND THEIR TARGET MRNAs
DK2002003T3 (en) * 2005-05-27 2016-03-21 Ospedale San Raffaele Srl Gene vector comprising miRNA
US20100286044A1 (en) * 2005-12-29 2010-11-11 Exiqon A/S Detection of tissue origin of cancer
EP2215228A4 (en) * 2007-11-23 2012-01-11 Panagene Inc Microrna antisense pnas, compositions comprising the same, and methods for using and evaluating the same
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP3514232A1 (en) * 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
FR3004463A1 (en) 2013-04-11 2014-10-17 Genethon EXPRESSION SYSTEM FOR SELECTIVE GENE THERAPY
CA3012195A1 (en) * 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
JOP20190200A1 (en) * 2017-02-28 2019-08-27 Univ Pennsylvania Compositions useful in treatment of spinal muscular atrophy

Also Published As

Publication number Publication date
JP2022517174A (en) 2022-03-07
CO2021008538A2 (en) 2021-07-19
IL284185B2 (en) 2025-04-01
US20210077553A1 (en) 2021-03-18
WO2020132455A1 (en) 2020-06-25
JOP20210160A1 (en) 2023-01-30
IL284185A (en) 2021-08-31
PE20211581A1 (en) 2021-08-17
CA3123600A1 (en) 2020-06-25
CL2021001624A1 (en) 2021-11-26
AU2019401314A1 (en) 2021-06-24
EP3908326A4 (en) 2022-10-26
JP7660062B2 (en) 2025-04-10
CN113646005A (en) 2021-11-12
IL284185B1 (en) 2024-12-01
BR112021011143A2 (en) 2022-01-25
EP3908326A1 (en) 2021-11-17
PH12021551341A1 (en) 2021-12-13
MX2021007600A (en) 2021-08-11
KR20210107037A (en) 2021-08-31

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