SG11201909777YA - Modulatory polynucleotides - Google Patents

Modulatory polynucleotides

Info

Publication number
SG11201909777YA
SG11201909777YA SG11201909777YA SG11201909777YA SG 11201909777Y A SG11201909777Y A SG 11201909777YA SG 11201909777Y A SG11201909777Y A SG 11201909777YA SG 11201909777Y A SG11201909777Y A SG 11201909777YA
Authority
SG
Singapore
Prior art keywords
international
cambridge
street
sidney
sidney street
Prior art date
Application number
Other languages
English (en)
Inventor
Jinzhao Hou
Xin Wang
Pengcheng Zhou
xiao-qin Ren
Dinah Sah
Original Assignee
Voyager Therapeutics 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 Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of SG11201909777YA publication Critical patent/SG11201909777YA/en

Links

Classifications

    • 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2310/11Antisense
    • 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]
    • 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • 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
    • C12N2830/20Vector systems having a special element relevant for transcription transcription of more than one cistron

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG11201909777Y 2017-05-05 2018-05-04 Modulatory polynucleotides SG11201909777YA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762501787P 2017-05-05 2017-05-05
US201762507923P 2017-05-18 2017-05-18
US201762520093P 2017-06-15 2017-06-15
PCT/US2018/031108 WO2018204797A1 (en) 2017-05-05 2018-05-04 Modulatory polynucleotides

Publications (1)

Publication Number Publication Date
SG11201909777YA true SG11201909777YA (en) 2019-11-28

Family

ID=64016722

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201909777Y SG11201909777YA (en) 2017-05-05 2018-05-04 Modulatory polynucleotides

Country Status (9)

Country Link
US (2) US20200270635A1 (enExample)
EP (1) EP3619310A4 (enExample)
JP (2) JP2020518266A (enExample)
CN (2) CN118910166A (enExample)
AU (1) AU2018260998B2 (enExample)
CA (1) CA3061365A1 (enExample)
SG (1) SG11201909777YA (enExample)
TW (1) TWI897844B (enExample)
WO (1) WO2018204797A1 (enExample)

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING DEFICIENCY IN A HUMAN SUBJECT
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
AU2019299861A1 (en) * 2018-07-02 2021-01-14 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
EP3861113A1 (en) 2018-10-04 2021-08-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
EP3861107A1 (en) 2018-10-05 2021-08-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
JP2022505106A (ja) 2018-10-15 2022-01-14 ボイジャー セラピューティクス インコーポレイテッド バキュロウイルス/Sf9システムにおけるrAAVの大規模産生のための発現ベクター
CN113631225A (zh) 2019-01-18 2021-11-09 沃雅戈治疗公司 用于生产aav颗粒的方法和系统
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
WO2020223280A1 (en) * 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4022070A1 (en) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2021168362A1 (en) 2020-02-21 2021-08-26 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
CN111450083A (zh) * 2020-04-28 2020-07-28 天津大学 一种肝癌靶向治疗纳米颗粒的合成方法
GB202010981D0 (en) * 2020-07-16 2020-09-02 Ucl Business Ltd Gene therapy for neuromuscular and neuromotor disorders
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
US20230295656A1 (en) 2020-08-06 2023-09-21 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2022072324A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Isl1 and lhx3 vector
US20240269322A1 (en) * 2020-10-21 2024-08-15 Chan Zuckerberg Biohub, Inc. Adeno-associated virus virions and methods of use thereof
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CA3272944A1 (en) 2022-12-29 2024-07-04 Voyager Therapeutics Inc MAPPING COMPOSITIONS AND REGULATION METHODS
WO2024226790A1 (en) * 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202449167A (zh) 2023-04-26 2024-12-16 美商航海家醫療公司 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法
WO2025122644A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0755454B1 (en) * 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
US20080318210A1 (en) * 2003-08-27 2008-12-25 Rosetta Genomics Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof
WO2005017127A2 (en) * 2003-02-21 2005-02-24 The Penn State Research Foundation Rna interference compositions and methods
AU2004239114B2 (en) * 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
US20050064489A1 (en) * 2003-09-24 2005-03-24 Zhang Fang Liang Engineered U6 and H1 promoters
AU2005307737C1 (en) * 2004-11-18 2013-08-29 The Board Of Trustees Of The University Of Illinois Multicistronic siRNA constructs to inhibit tumors
WO2008134646A2 (en) * 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
WO2010078536A1 (en) * 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
WO2010140862A2 (ko) * 2009-06-05 2010-12-09 Seol Dai-Wu 단일 또는 멀티 표적 유전자를 억제하는 멀티-시스트로닉 shRNA 발현 카세트
DK3119797T3 (da) * 2014-03-18 2021-03-15 Univ Massachusetts Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose
MX2017006216A (es) * 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
KR102584655B1 (ko) * 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US20190224339A1 (en) * 2016-04-29 2019-07-25 Voyager Therapeutics, Inc. Compositions for the treatment of disease

Also Published As

Publication number Publication date
CN110914427A (zh) 2020-03-24
AU2018260998A1 (en) 2019-11-28
JP7768928B2 (ja) 2025-11-12
AU2018260998A2 (en) 2020-01-16
EP3619310A1 (en) 2020-03-11
CA3061365A1 (en) 2018-11-08
TWI897844B (zh) 2025-09-21
AU2018260998B2 (en) 2025-09-11
TW201905200A (zh) 2019-02-01
CN118910166A (zh) 2024-11-08
EP3619310A4 (en) 2021-01-27
US20200270635A1 (en) 2020-08-27
JP2020518266A (ja) 2020-06-25
US20220333131A1 (en) 2022-10-20
WO2018204797A1 (en) 2018-11-08
JP2023087019A (ja) 2023-06-22
CN110914427B (zh) 2024-08-09

Similar Documents

Publication Publication Date Title
SG11201909777YA (en) Modulatory polynucleotides
SG11201909868YA (en) Compositions and methods of treating huntington's disease
SG11201809643UA (en) Compositions and methods of treating huntington's disease
SG11201909870SA (en) Compositions and methods of treating amyotrophic lateral sclerosis (als)
SG11201909949XA (en) Targeted immunotolerance
SG11201804729RA (en) Methods and compositions for treating a serpinc1-associated disorder
SG11201809699XA (en) Modulatory polynucleotides
SG11201807573VA (en) Methods for providing single-stranded rna
SG11201808990QA (en) Compositions for topical application of compounds
SG11201811431VA (en) Multispecific antibodies against cd40 and cd137
SG11201909011PA (en) Niraparib compositions
SG11201909955XA (en) Formulations of anti-lag3 antibodies and co-formulations of anti-lag3 antibodies and anti-pd-1 antibodies
SG11201908813QA (en) Anti-sirp alpha antibodies
SG11201907162TA (en) Methods for cell-type specific profiling to identify drug targets
SG11201804443UA (en) Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
SG11201909864TA (en) Tcr and peptides
SG11201807912SA (en) Vaccine against rsv
SG11201803593QA (en) Engineered nucleic-acid targeting nucleic acids
SG11201809344QA (en) Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde
SG11201804696RA (en) Techniques for metadata processing
SG11201909856XA (en) Mesenchymal lineage precursor or stem cells with enhanced immunosuppression
SG11201804704PA (en) Compositions and methods for decreasing tau expression
SG11201907889YA (en) Glycan-interacting compounds and methods of use
SG11201909957TA (en) Engineered ligase variants
SG11201810525XA (en) Anti-gitr antibodies and uses thereof