SG11202101288TA - Modified oligonucleotides targeting snps - Google Patents

Modified oligonucleotides targeting snps

Info

Publication number
SG11202101288TA
SG11202101288TA SG11202101288TA SG11202101288TA SG11202101288TA SG 11202101288T A SG11202101288T A SG 11202101288TA SG 11202101288T A SG11202101288T A SG 11202101288TA SG 11202101288T A SG11202101288T A SG 11202101288TA SG 11202101288T A SG11202101288T A SG 11202101288TA
Authority
SG
Singapore
Prior art keywords
modified oligonucleotides
oligonucleotides targeting
targeting snps
snps
modified
Prior art date
Application number
SG11202101288TA
Inventor
Anastasia Khvorova
Julia Alterman
Faith Conroy
Edith Pfister
Neil Aronin
Ken Yamada
Original Assignee
Univ Massachusetts
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 Massachusetts filed Critical Univ Massachusetts
Publication of SG11202101288TA publication Critical patent/SG11202101288TA/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
    • 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
    • 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
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • 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
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses
    • 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
    • C12N2330/30Production chemically synthesised

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
SG11202101288TA 2018-08-10 2019-08-09 Modified oligonucleotides targeting snps SG11202101288TA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862717287P 2018-08-10 2018-08-10
US201962825429P 2019-03-28 2019-03-28
PCT/US2019/046013 WO2020033899A1 (en) 2018-08-10 2019-08-09 Modified oligonucleotides targeting snps

Publications (1)

Publication Number Publication Date
SG11202101288TA true SG11202101288TA (en) 2021-03-30

Family

ID=69414459

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202101288TA SG11202101288TA (en) 2018-08-10 2019-08-09 Modified oligonucleotides targeting snps

Country Status (12)

Country Link
US (2) US11827882B2 (en)
EP (1) EP3833763A4 (en)
JP (1) JP2021533762A (en)
KR (1) KR20210093227A (en)
CN (1) CN112805383A (en)
AU (1) AU2019316640A1 (en)
BR (1) BR112021002440A2 (en)
CA (1) CA3109133A1 (en)
IL (1) IL280724A (en)
MX (1) MX2021001590A (en)
SG (1) SG11202101288TA (en)
WO (1) WO2020033899A1 (en)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
WO2018031933A2 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
KR20210093227A (en) 2018-08-10 2021-07-27 유니버시티 오브 매사추세츠 Modified oligonucleotides targeting SNPs
JP2022528840A (en) * 2019-03-26 2022-06-16 ユニバーシティ・オブ・マサチューセッツ Modified oligonucleotides with increased stability
EP4017504A4 (en) * 2019-08-23 2024-01-24 Univ Massachusetts O-methyl rich fully stabilized oligonucleotides
CN115698291A (en) * 2020-05-28 2023-02-03 马萨诸塞大学 Oligonucleotides for SARS-CoV-2 modulation
TW202305134A (en) 2021-03-29 2023-02-01 美商艾拉倫製藥股份有限公司 Huntingtin (htt) irna agent compositions and methods of use thereof
EP4359539A1 (en) 2021-06-23 2024-05-01 University Of Massachusetts Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
WO2024002007A1 (en) * 2022-06-27 2024-01-04 大睿生物医药科技(上海)有限公司 Double-stranded rna comprising nucleotide analog capable of reducing off-target toxicity

Family Cites Families (147)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US5240848A (en) 1988-11-21 1993-08-31 Monsanto Company Dna sequences encoding human vascular permeability factor having 189 amino acids
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5332671A (en) 1989-05-12 1994-07-26 Genetech, Inc. Production of vascular endothelial cell growth factor and DNA encoding same
US5219739A (en) 1989-07-27 1993-06-15 Scios Nova Inc. DNA sequences encoding bVEGF120 and hVEGF121 and methods for the production of bovine and human vascular endothelial cell growth factors, bVEGF120 and hVEGF121
US5194596A (en) 1989-07-27 1993-03-16 California Biotechnology Inc. Production of vascular endothelial cell growth factor
DE69031951T2 (en) 1989-11-16 1998-08-13 Du Pont Transformation of animal skin cells with the help of particles
US5672697A (en) 1991-02-08 1997-09-30 Gilead Sciences, Inc. Nucleoside 5'-methylene phosphonates
US6291438B1 (en) 1993-02-24 2001-09-18 Jui H. Wang Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use
US5858988A (en) 1993-02-24 1999-01-12 Wang; Jui H. Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use
TW360548B (en) 1993-04-08 1999-06-11 Powderject Res Ltd Products for therapeutic use
DE69514351T2 (en) 1994-06-01 2000-08-10 Hybridon Inc Branched oligonucleotides as pathogen-inhibiting agents
US6383814B1 (en) 1994-12-09 2002-05-07 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
WO1996033761A1 (en) 1995-04-28 1996-10-31 Medtronic, Inc. Intraparenchymal infusion catheter system
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
US5735814A (en) 1996-04-30 1998-04-07 Medtronic, Inc. Techniques of treating neurodegenerative disorders by brain infusion
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6042820A (en) 1996-12-20 2000-03-28 Connaught Laboratories Limited Biodegradable copolymer containing α-hydroxy acid and α-amino acid units
US6472375B1 (en) 1998-04-16 2002-10-29 John Wayne Cancer Institute DNA vaccine and methods for its use
EP1016726A1 (en) 1998-12-30 2000-07-05 Introgene B.V. Gene therapy to promote angiogenesis
US7608681B2 (en) 1999-12-24 2009-10-27 Genentech, Inc. Methods and compositions for prolonging elimination half-times of bioactive compounds
US20070026394A1 (en) 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
CA2462144C (en) 2001-09-28 2016-09-20 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Micro-rna molecules
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US20050096284A1 (en) 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
CA2496253C (en) 2002-07-19 2016-05-10 Beth Israel Deaconess Medical Center, Inc. Methods of diagnosing and treating pre-eclampsia or eclampsia
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003295387A1 (en) 2002-11-05 2004-06-03 Isis Parmaceuticals, Inc. Modified oligonucleotides for use in rna interference
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
US6913890B2 (en) 2002-12-18 2005-07-05 Palo Alto Research Center Incorporated Process for preparing albumin protein conjugated oligonucleotide probes
AU2003299864A1 (en) 2002-12-27 2004-07-29 P. Radhakrishnan Sirna compounds and methods for the downregulation of gene expression
US20040198640A1 (en) 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
EP2664672A1 (en) 2003-04-17 2013-11-20 Alnylam Pharmaceuticals Inc. Modified iRNA agents
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
ES2712695T3 (en) 2003-06-02 2019-05-14 Univ Massachusetts Methods and compositions to control the efficiency of RNA silencing
ES2864206T3 (en) 2003-06-02 2021-10-13 Univ Massachusetts Methods and compositions to improve the efficacy and specificity of RNAi
MXPA06006892A (en) 2003-12-19 2008-02-13 Novartis Vaccines & Diagnostic Cell transfecting formulations of small interfering rna, related compositions and methods of making and use.
US20050176045A1 (en) * 2004-02-06 2005-08-11 Dharmacon, Inc. SNP discriminatory siRNA
WO2005077333A2 (en) 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
ES2423060T3 (en) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. IRNA agents that target VEGF
MXPA06012076A (en) 2004-04-20 2007-01-25 Nastech Pharm Co Methods and compositions for enhancing delivery of double-stranded rna or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells.
AU2005248147A1 (en) 2004-05-11 2005-12-08 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
AU2005252662B2 (en) 2004-06-03 2011-08-18 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
EP1937066A4 (en) 2005-08-18 2008-12-24 Alnylam Pharmaceuticals Inc Methods and compositions for treating neurological disease
WO2007051045A2 (en) 2005-10-28 2007-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of huntingtin gene
AU2006308765B2 (en) 2005-11-02 2013-09-05 Arbutus Biopharma Corporation Modified siRNA molecules and uses thereof
CA2628113A1 (en) 2005-11-04 2007-05-18 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
US20070259827A1 (en) 2006-01-25 2007-11-08 University Of Massachusetts Compositions and methods for enhancing discriminatory RNA interference
US9198981B2 (en) 2006-02-01 2015-12-01 The University Of Kentucky Modulation of angiogenesis
WO2007091269A2 (en) 2006-02-08 2007-08-16 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
JPWO2007094218A1 (en) 2006-02-16 2009-07-02 国立大学法人岐阜大学 Modified oligonucleotide
EP1986697B1 (en) 2006-02-17 2016-06-29 GE Healthcare Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
US20090306178A1 (en) 2006-03-27 2009-12-10 Balkrishen Bhat Conjugated double strand compositions for use in gene modulation
CA2662704A1 (en) 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
US20080039415A1 (en) 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
AU2007299705B2 (en) 2006-09-22 2012-09-06 Dharmacon, Inc. Duplex oligonucleotide complexes and methods for gene silencing by RNA interference
WO2008049078A1 (en) 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof
US8906874B2 (en) 2006-11-09 2014-12-09 Gradalis, Inc. Bi-functional shRNA targeting Stathmin 1 and uses thereof
CA2664271A1 (en) 2006-11-27 2008-06-12 Enzon Pharmaceuticals, Inc. Polymeric short interfering rna conjugates
EP2126081A2 (en) 2007-03-02 2009-12-02 MDRNA, Inc. Nucleic acid compounds for inhibiting hif1a gene expression and uses thereof
WO2008154482A2 (en) 2007-06-08 2008-12-18 Sirnaomics, Inc. Sirna compositions and methods of use in treatment of ocular diseases
EP2173358B1 (en) 2007-06-22 2015-10-21 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
CN101842381A (en) 2007-08-27 2010-09-22 波士顿生物医药公司 Composition of asymmetric RNA duplex as microrna mimetic or inhibitor
CN101199858A (en) 2007-10-18 2008-06-18 广州拓谱基因技术有限公司 Multiple target point small interference RNA cocktail agent for treating ophthalmic disease and preparing method thereof
TW200927177A (en) 2007-10-24 2009-07-01 Nat Inst Of Advanced Ind Scien Lipid-modified double-stranded RNA having potent RNA interference effect
EP2231195B1 (en) 2007-12-04 2017-03-29 Arbutus Biopharma Corporation Targeting lipids
US9029524B2 (en) 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
US20110081362A1 (en) 2008-01-31 2011-04-07 The Brigham And Women's Hospital, Inc. Treatment of cancer
CN104975020B (en) 2008-02-11 2020-01-17 菲奥医药公司 Modified RNAi polynucleotides and uses thereof
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
CA2731779A1 (en) 2008-07-24 2010-01-28 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
CA2746527A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Rna interference in skin indications
WO2010048585A2 (en) 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
CA2750561C (en) 2009-01-26 2017-10-10 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
KR101141544B1 (en) 2009-03-13 2012-05-03 한국과학기술원 Multi-conjugate of siRNA and preparing method thereof
WO2010118263A1 (en) 2009-04-08 2010-10-14 University Of Massachusetts Single-nucleotide polymorphism (snp) targeting therapies for the treatment of huntington's disease
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
GB0910723D0 (en) 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
WO2011031520A1 (en) 2009-08-27 2011-03-17 Idera Pharmaceuticals, Inc. Composition for inhibiting gene expression and uses thereof
US20150025122A1 (en) 2009-10-12 2015-01-22 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro
DK2534248T3 (en) 2010-02-08 2018-11-19 Ionis Pharmaceuticals Inc SELECTIVE REDUCTION OF ALLELVARIANS
WO2011109698A1 (en) 2010-03-04 2011-09-09 Rxi Pharmaceuticals Corporation Formulations and methods for targeted delivery to phagocyte cells
AU2011232365A1 (en) 2010-03-24 2012-10-25 Rxi Pharmaceuticals Corporation RNA interference in dermal and fibrotic indications
EP2550001B1 (en) 2010-03-24 2019-05-22 Phio Pharmaceuticals Corp. Rna interference in ocular indications
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
CN103154014B (en) 2010-04-28 2015-03-25 Isis制药公司 Modified nucleosides, modified nucleosides-like and oligomeric compounds prepared therefrom
WO2012005898A2 (en) 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
US8586301B2 (en) 2010-06-30 2013-11-19 Stratos Genomics, Inc. Multiplexed identification of nucleic acid sequences
WO2012037254A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. MODIFIED iRNA AGENTS
US10378016B2 (en) 2010-12-06 2019-08-13 Children's Medical Center Corporation Composition and method for oligonucleotide delivery
RU2013117288A (en) 2010-12-17 2015-01-27 Эрроухэд Рисерч Корпорейшн CONTAINING GALACTOSY CLUSTER A TARGETING GROUP FOR miRNA MODULATING FORMKOKINETIC PROPERTIES
CA2828544A1 (en) 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
GB201105137D0 (en) * 2011-03-28 2011-05-11 Isis Innovation Therapeutic molecules for use in the suppression of Parkinson's disease
AU2012272908A1 (en) 2011-06-21 2013-12-19 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
EP3358013B1 (en) 2012-05-02 2020-06-24 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
EP2853597B1 (en) 2012-05-22 2018-12-26 Olix Pharmaceuticals, Inc. Rna-interference-inducing nucleic acid molecule able to penetrate into cells, and use therefor
AU2013288738A1 (en) 2012-07-10 2015-01-29 Baseclick Gmbh Anandamide-modified nucleic acid molecules
WO2014043544A1 (en) 2012-09-14 2014-03-20 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
WO2014047649A1 (en) 2012-09-24 2014-03-27 The Regents Of The University Of California Methods for arranging and packing nucleic acids for unusual resistance to nucleases and targeted delivery for gene therapy
CN104837996A (en) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 Anti APOB antisense conjugate compounds
US20140200124A1 (en) 2013-01-15 2014-07-17 Karen Michelle JAMES Physical Therapy Device and Methods for Use Thereof
JP6387084B2 (en) 2013-05-01 2018-09-05 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. Compositions and methods for modulating apolipoprotein C-III expression
EP3007705A4 (en) 2013-06-12 2017-02-15 Oncoimmunin, Inc. Systemic in vivo delivery of oligonucleotides
CN105324119A (en) 2013-06-16 2016-02-10 国立大学法人东京医科齿科大学 Double-stranded antisense nucleic acid with exon-skipping effect
US9862350B2 (en) 2013-08-12 2018-01-09 Tk Holdings Inc. Dual chambered passenger airbag
WO2015057847A1 (en) 2013-10-16 2015-04-23 Cerulean Pharma Inc. Conjugates, particles, compositions, and related methods
EP3066219B1 (en) 2013-11-08 2018-12-26 Ionis Pharmaceuticals, Inc. Methods for detecting oligonucleotides
EP3099795A4 (en) 2014-01-27 2018-01-17 The Board of Trustees of the Leland Stanford Junior University Oligonucleotides and methods for treatment of cardiomyopathy using rna interference
WO2015161184A1 (en) 2014-04-18 2015-10-22 University Of Massachusetts Exosomal loading using hydrophobically modified oligonucleotides
WO2015168661A1 (en) * 2014-05-01 2015-11-05 Smith Larry J METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
KR102410687B1 (en) 2014-05-22 2022-06-22 알닐람 파마슈티칼스 인코포레이티드 Angiotensinogen (agt) irna compositions and methods of use thereof
US10588980B2 (en) 2014-06-23 2020-03-17 Novartis Ag Fatty acids and their use in conjugation to biomolecules
AU2015329974B2 (en) 2014-10-10 2019-06-20 F. Hoffmann-La Roche Ag GaINAc phosphoramidites, nucleic acid conjugates thereof and their use
PL3277815T3 (en) 2015-04-03 2022-03-21 University Of Massachusetts Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
CN116004624A (en) 2015-04-03 2023-04-25 马萨诸塞大学 Oligonucleotide compounds for targeting huntingtin mRNA
MX2017013022A (en) 2015-04-10 2018-03-15 Hudsonalpha Inst For Biotechnology METHOD FOR BLOCKING miRNA.
KR20180039621A (en) 2015-06-15 2018-04-18 엠펙 엘에이, 엘엘씨 The defined multijunctional oligonucleotides
MA43072A (en) 2015-07-22 2018-05-30 Wave Life Sciences Ltd COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
EP3334499A4 (en) 2015-08-14 2019-04-17 University of Massachusetts Bioactive conjugates for oligonucleotide delivery
CN105194689A (en) 2015-10-10 2015-12-30 湖南中楚博生物科技有限公司 SiRNA-based double-coupled compound
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
MA45328A (en) 2016-04-01 2019-02-06 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
WO2017174572A1 (en) 2016-04-04 2017-10-12 F. Hoffmann-La Roche Ag Nucleic acid sample preparation methods
US10036024B2 (en) * 2016-06-03 2018-07-31 Purdue Research Foundation siRNA compositions that specifically downregulate expression of a variant of the PNPLA3 gene and methods of use thereof for treating a chronic liver disease or alcoholic liver disease (ALD)
WO2018031933A2 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
PL3507366T3 (en) 2016-09-01 2021-05-04 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts Two-tailed self-delivering sirna and related methods
WO2019035038A1 (en) 2017-08-18 2019-02-21 The Procter & Gamble Company Cleaning agent
KR20210093227A (en) 2018-08-10 2021-07-27 유니버시티 오브 매사추세츠 Modified oligonucleotides targeting SNPs
EP3911747A4 (en) 2019-01-18 2023-05-24 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors

Also Published As

Publication number Publication date
WO2020033899A1 (en) 2020-02-13
AU2019316640A1 (en) 2021-03-18
EP3833763A4 (en) 2023-07-19
US20200123543A1 (en) 2020-04-23
US20240132888A1 (en) 2024-04-25
JP2021533762A (en) 2021-12-09
BR112021002440A2 (en) 2021-05-04
EP3833763A1 (en) 2021-06-16
KR20210093227A (en) 2021-07-27
CA3109133A1 (en) 2020-02-13
CN112805383A (en) 2021-05-14
IL280724A (en) 2021-03-25
MX2021001590A (en) 2021-07-02
US11827882B2 (en) 2023-11-28

Similar Documents

Publication Publication Date Title
IL268422A (en) Targeted oligonucleotides
SG11202101288TA (en) Modified oligonucleotides targeting snps
IL274442A (en) Rna sequence adaptation
HK1258758A1 (en) Branched oligonucleotides
IL261327A (en) Trans-replicating rna
IL268894A (en) Therapeutic rna
IL257091B (en) Targeted oligonucleotides
HK1252970A1 (en) Defined multi-conjugate oligonucleotides
EP3664817A4 (en) Chemically modified oligonucleotides
EP3314027A4 (en) Therapeutic oligonucleotides
GB201503408D0 (en) Oligonucleotides
GB201904709D0 (en) Chemically modified oligonucleotides
ZA202001649B (en) Rna molecules
RS62619B1 (en) Gene therapy
GB201504124D0 (en) Oligonucleotides
EP3584319A4 (en) Single-stranded oligonucleotide
EP3449002A4 (en) G-quadruplex-containing antisense oligonucleotides
EP3660153A4 (en) Single-stranded oligonucleotide
IL290329A (en) Chemically modified oligonucleotides targeting snps
EP3532617A4 (en) Antisense oligonucleotides
EP4041248A4 (en) Modified oligonucleotides
IL263852B (en) Oligonucleotides comprising modified nucleosides
GB201805865D0 (en) Genes
GB201701968D0 (en) Gene therapy
GB201714330D0 (en) Oligonucleotides