[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

SG11202101072UA - Variant rnai against alpha-synuclein - Google Patents

Variant rnai against alpha-synuclein

Info

Publication number
SG11202101072UA
SG11202101072UA SG11202101072UA SG11202101072UA SG11202101072UA SG 11202101072U A SG11202101072U A SG 11202101072UA SG 11202101072U A SG11202101072U A SG 11202101072UA SG 11202101072U A SG11202101072U A SG 11202101072UA SG 11202101072U A SG11202101072U A SG 11202101072UA
Authority
SG
Singapore
Prior art keywords
synuclein
rnai against
against alpha
variant
variant rnai
Prior art date
Application number
SG11202101072UA
Inventor
Bradford Elmer
Brenda Richards
Martine Latta-Mahieu
Maria Carmen Obinu
Vã©Ronique Taupin
Vã©Ronique Blanchard
Original Assignee
Genzyme Corp
Sanofi Aventis Rech & Developpement
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 Genzyme Corp, Sanofi Aventis Rech & Developpement filed Critical Genzyme Corp
Publication of SG11202101072UA publication Critical patent/SG11202101072UA/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
    • C12N15/11DNA 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
    • 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
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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 N.A.
    • 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
    • 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
    • C12N2830/38Vector systems having a special element relevant for transcription being a stuffer
    • 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
    • C12N2830/42Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
SG11202101072UA 2018-08-03 2019-08-02 Variant rnai against alpha-synuclein SG11202101072UA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862714616P 2018-08-03 2018-08-03
PCT/US2019/044924 WO2020028816A1 (en) 2018-08-03 2019-08-02 Variant rnai against alpha-synuclein

Publications (1)

Publication Number Publication Date
SG11202101072UA true SG11202101072UA (en) 2021-03-30

Family

ID=67587976

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202101072UA SG11202101072UA (en) 2018-08-03 2019-08-02 Variant rnai against alpha-synuclein

Country Status (15)

Country Link
US (1) US20210309999A1 (en)
EP (1) EP3830265A1 (en)
JP (1) JP2021534814A (en)
KR (1) KR20210062627A (en)
CN (1) CN112805382A (en)
AR (1) AR114540A1 (en)
AU (1) AU2019314529A1 (en)
BR (1) BR112021001980A2 (en)
CA (1) CA3108526A1 (en)
CO (1) CO2021002637A2 (en)
IL (1) IL280581A (en)
MX (1) MX2021001395A (en)
SG (1) SG11202101072UA (en)
TW (1) TW202022116A (en)
WO (1) WO2020028816A1 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020005569A2 (en) * 2017-09-22 2020-10-06 Genzyme Corporation interference rna (rnai) variant
CA3171757A1 (en) * 2020-03-18 2021-09-23 Anastasia Khvorova Oligonucleotides for snca modulation
EP4359525A1 (en) * 2021-06-21 2024-05-01 uniQure biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
WO2023196862A1 (en) * 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2024073609A2 (en) * 2022-09-29 2024-04-04 Atalanta Therapeutics, Inc. Sirna compositions and methods targeting alpha-synuclein nucleic acids

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DE60039766D1 (en) 1999-08-09 2008-09-18 Targeted Genetics Corp TEROLOGIC NUCLEOTIDE SEQUENCE FROM A RECOMBINANT VIRAL VECTOR BY STRUCTURE OF THE SEQUENCE IN A WAY THAT BASE COUNTERATIONS ARISE WITHIN THE SEQUENCE
WO2001092551A2 (en) 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
ES2439515T3 (en) 2001-11-13 2014-01-23 The Trustees Of The University Of Pennsylvania Serotype rh10 of adeno-associated virus (AAV)
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
EP2164967B1 (en) 2007-05-31 2015-07-08 University of Iowa Research Foundation Reduction of off-target rna interference toxicity
GB0816778D0 (en) * 2008-09-12 2008-10-22 Isis Innovation Gene silencing
ES2605305T3 (en) 2010-04-23 2017-03-13 University Of Massachusetts AAV vectors that target the CNS and their methods of use
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP2640853B1 (en) * 2010-11-17 2018-12-26 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CN103502458B (en) 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 For changing tissue specificity and improving compositions and the method for the gene transfer that AAV9-mediates
CN114231563B (en) 2014-05-02 2024-08-16 建新公司 AAV vectors for retinal and CNS gene therapy
PE20171382A1 (en) * 2015-02-10 2017-09-15 Genzyme Corp ARNi VARIANT

Also Published As

Publication number Publication date
US20210309999A1 (en) 2021-10-07
KR20210062627A (en) 2021-05-31
CN112805382A (en) 2021-05-14
IL280581A (en) 2021-03-25
JP2021534814A (en) 2021-12-16
TW202022116A (en) 2020-06-16
CO2021002637A2 (en) 2021-07-30
MX2021001395A (en) 2021-08-11
BR112021001980A2 (en) 2021-05-04
EP3830265A1 (en) 2021-06-09
AR114540A1 (en) 2020-09-16
AU2019314529A1 (en) 2021-03-25
WO2020028816A1 (en) 2020-02-06
CA3108526A1 (en) 2020-02-06

Similar Documents

Publication Publication Date Title
IL280581A (en) Variant rnai against alpha-synuclein
IL273394A (en) Variant rnai
GB201918689D0 (en) n
PL3456736T3 (en) Antibody variants
IL273351A (en) Antibody variants
ZA202100322B (en) Polytunnel structure
IL272951A (en) Antibody variants
GB201813574D0 (en) A1
GB2576916B (en) Polytunnel structure
GB201820913D0 (en) St
GB2575634B (en) Polytunnel structure
GB201813992D0 (en) St
GB201813486D0 (en) st
GB201813476D0 (en) st
GB201813347D0 (en) St
GB201813206D0 (en) St
GB201811251D0 (en) St
GB201809615D0 (en) St
GB201809583D0 (en) St
GB201809510D0 (en) St
GB201809503D0 (en) St
GB201818676D0 (en) Polytunnel structure
GB201808169D0 (en) St
GB201807984D0 (en) St
GB201805817D0 (en) St