BR112023026862A2 - Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos - Google Patents

Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos

Info

Publication number
BR112023026862A2
BR112023026862A2 BR112023026862A BR112023026862A BR112023026862A2 BR 112023026862 A2 BR112023026862 A2 BR 112023026862A2 BR 112023026862 A BR112023026862 A BR 112023026862A BR 112023026862 A BR112023026862 A BR 112023026862A BR 112023026862 A2 BR112023026862 A2 BR 112023026862A2
Authority
BR
Brazil
Prior art keywords
treatment
angiogenic disorders
flt1
eclampsia
oligonucleotide compounds
Prior art date
Application number
BR112023026862A
Other languages
English (en)
Portuguese (pt)
Inventor
Ananth Karumanchi
Anastasia Khvorova
Annabelle Biscans
Sarah Davis
Narayan Hariharan Vignesh
Original Assignee
Beth Israel Deaconess Medical Ct Inc
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 Beth Israel Deaconess Medical Ct Inc, Univ Massachusetts filed Critical Beth Israel Deaconess Medical Ct Inc
Publication of BR112023026862A2 publication Critical patent/BR112023026862A2/pt

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
    • C12N15/1138Non-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 against receptors or cell surface proteins
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular 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
    • 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
    • C12N2310/30Chemical 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
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • 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
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
BR112023026862A 2021-06-23 2022-06-22 Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos BR112023026862A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163214224P 2021-06-23 2021-06-23
PCT/US2022/034461 WO2022271786A1 (en) 2021-06-23 2022-06-22 Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders

Publications (1)

Publication Number Publication Date
BR112023026862A2 true BR112023026862A2 (pt) 2024-03-05

Family

ID=84544941

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023026862A BR112023026862A2 (pt) 2021-06-23 2022-06-22 Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos

Country Status (13)

Country Link
US (2) US11702659B2 (enExample)
EP (1) EP4359539A4 (enExample)
JP (2) JP7758907B2 (enExample)
KR (1) KR20240040729A (enExample)
CN (1) CN117677699A (enExample)
AR (1) AR126207A1 (enExample)
AU (1) AU2022299169A1 (enExample)
BR (1) BR112023026862A2 (enExample)
CA (1) CA3174095A1 (enExample)
IL (1) IL309334A (enExample)
MX (1) MX2023015523A (enExample)
TW (1) TW202315943A (enExample)
WO (1) WO2022271786A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) * 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
CA3033368A1 (en) * 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ 2テイル自己デリバリー型siRNAおよび関連方法
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
WO2025015189A1 (en) 2023-07-13 2025-01-16 Comanche Biopharma Corp. Formulations of nucleic acid compounds and uses thereof

Family Cites Families (163)

* 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
US5194596A (en) 1989-07-27 1993-03-16 California Biotechnology Inc. Production of vascular endothelial cell growth factor
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
ES2113371T3 (es) 1989-11-16 1998-05-01 Univ Duke Transformacion de celulas epidermicas de tejidos animales con la ayuda de particulas.
US5985558A (en) 1997-04-14 1999-11-16 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the inibition of c-Jun and c-Fos
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
EP0763050B1 (en) 1994-06-01 2000-01-05 Hybridon, Inc. Branched oligonucleotide as pathogen-inhibitory 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
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
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
EP1240337B1 (en) 1999-12-24 2006-08-23 Genentech, Inc. Methods and compositions for prolonging elimination half-times of bioactive compounds
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
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
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
EP2447370B1 (en) 2001-09-28 2018-07-18 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
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)
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)
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
KR101215701B1 (ko) 2002-07-19 2012-12-26 베스 이스라엘 데코니스 메디칼 센터 자간전증 또는 자간의 진단 및 치료 방법
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)
AU2003291755A1 (en) 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
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
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
EP1633890B2 (en) 2003-06-02 2020-11-18 University of Massachusetts METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi
PT1633767T (pt) 2003-06-02 2019-02-27 Univ Massachusetts Métodos e composições para controlar a eficácia do silenciamento de arn
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2008005562A2 (en) 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
EP2256201A3 (en) 2003-09-18 2012-07-04 Isis Pharmaceuticals, Inc. Modulation of eIF4E expression
EP1709195B1 (en) 2003-12-19 2014-01-22 Novartis Vaccines and Diagnostics, Inc. Cell transfecting formulations of small interfering rna, related compositions and methods of making and use
WO2005077333A2 (en) 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
US20060014289A1 (en) 2004-04-20 2006-01-19 Nastech Pharmaceutical Company Inc. Methods and compositions for enhancing delivery of double-stranded RNA or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells
WO2005116204A1 (ja) 2004-05-11 2005-12-08 Rnai Co., Ltd. Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
EP1937066A4 (en) 2005-08-18 2008-12-24 Alnylam Pharmaceuticals Inc METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES
EP1931780B1 (en) 2005-08-29 2016-01-06 Regulus Therapeutics Inc. Antisense compounds having enhanced anti-microrna activity
JP5111385B2 (ja) 2005-10-28 2013-01-09 アルナイラム ファーマシューティカルズ, インコーポレイテッド ハンチンチン遺伝子の発現を抑制するための組成物および方法
CN101346393B (zh) 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
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
EP1989307B1 (en) 2006-02-08 2012-08-08 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
WO2007094218A1 (ja) 2006-02-16 2007-08-23 Gifu University 修飾オリゴヌクレオチド
WO2007095387A2 (en) 2006-02-17 2007-08-23 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
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
CA2666657A1 (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
US20100015706A1 (en) 2007-03-02 2010-01-21 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
WO2009002944A1 (en) 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
EP3889261A1 (en) 2007-08-27 2021-10-06 1Globe Health Institute LLC Compositions of asymmetric interfering rna and uses thereof
CN101199858A (zh) 2007-10-18 2008-06-18 广州拓谱基因技术有限公司 治疗眼科疾病的多靶点小干扰rna鸡尾酒制剂及制备方法
TW200927177A (en) 2007-10-24 2009-07-01 Nat Inst Of Advanced Ind Scien Lipid-modified double-stranded RNA having potent RNA interference effect
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
US9029524B2 (en) 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
WO2009099991A2 (en) 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
CA2715289C (en) 2008-02-11 2019-12-24 Rxi Pharmaceuticals Corporation 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
US20110251258A1 (en) 2008-07-24 2011-10-13 Rxi Pharmaceuticals Corporation Rnai constructs and uses thereof
EP2321414B1 (en) 2008-07-25 2018-01-10 Alnylam Pharmaceuticals, Inc. Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
ES2794401T3 (es) 2008-10-15 2020-11-18 Axolabs Gmbh Método de detección de oligonucleótidos
EP2447274B1 (en) 2008-10-24 2017-10-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds and methods
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
US9023820B2 (en) 2009-01-26 2015-05-05 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein C-III expression
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
WO2010118263A1 (en) 2009-04-08 2010-10-14 University Of Massachusetts Single-nucleotide polymorphism (snp) targeting therapies for the treatment of huntington's disease
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
GB0910723D0 (en) 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
EP2470656B1 (en) 2009-08-27 2015-05-06 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
US8877439B2 (en) 2009-12-17 2014-11-04 Sirna Therapeutics, Inc. Method for rapidly evaluating performance of short interfering RNA with novel chemical modifications
WO2011109698A1 (en) 2010-03-04 2011-09-09 Rxi Pharmaceuticals Corporation Formulations and methods for targeted delivery to phagocyte cells
CN103200945B (zh) 2010-03-24 2016-07-06 雷克西制药公司 眼部症候中的rna干扰
EP3560503B1 (en) 2010-03-24 2021-11-17 Phio Pharmaceuticals Corp. Rna interference in dermal and fibrotic indications
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides 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
WO2012003330A2 (en) 2010-06-30 2012-01-05 Stratos Genomics, Inc Multiplexed identification of nucleic acid sequences
CA2812046A1 (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
AU2011343664B2 (en) 2010-12-17 2015-10-08 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for siRNA
CA2828544A1 (en) 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
US20140275211A1 (en) 2011-06-21 2014-09-18 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
US20150299696A1 (en) 2012-05-02 2015-10-22 Sirna Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
KR101567576B1 (ko) 2012-05-22 2015-11-10 올릭스 주식회사 세포 내 관통능을 가지고 rna 간섭을 유도하는 핵산 분자 및 그 용도
US9480752B2 (en) 2012-07-10 2016-11-01 Baseclick Gmbh Anandamide-modified nucleic acid molecules
US20150247141A1 (en) 2012-09-14 2015-09-03 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 (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
IL292159B1 (en) 2012-12-05 2026-04-01 Alnylam Pharmaceuticals Inc PCSK9 iRNA compositions and methods of using them
US20140200124A1 (en) 2013-01-15 2014-07-17 Karen Michelle JAMES Physical Therapy Device and Methods for Use Thereof
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
AU2014282666A1 (en) 2013-06-16 2016-01-07 National University Corporation Tokyo Medical And Dental University Double-stranded antisense nucleic acid with exon-skipping effect
US9862350B2 (en) 2013-08-12 2018-01-09 Tk Holdings Inc. Dual chambered passenger airbag
GB201315141D0 (en) 2013-08-23 2013-10-09 Logicor R & D Ltd Improvements to electric heating systems and method of use thereof
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
US10513710B2 (en) 2014-04-18 2019-12-24 University Of Massachusetts Exosomal loading using hydrophobically modified oligonucleotides
US20170051286A1 (en) 2014-05-01 2017-02-23 Larry J. Smith METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
AU2015264038B2 (en) 2014-05-22 2021-02-11 Alnylam Pharmaceuticals, Inc. 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
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
CN106795200B (zh) 2014-10-10 2020-06-19 豪夫迈·罗氏有限公司 Galnac亚磷酰胺、其核酸缀合物及其用途
WO2016083623A1 (en) * 2014-11-28 2016-06-02 Silence Therapeutics Gmbh Means for the treatment of pre-eclampsia
CA2979703A1 (en) 2015-03-17 2016-09-22 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of factor xii
PL3277814T3 (pl) 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
RS62672B1 (sr) 2015-04-03 2021-12-31 Univ Massachusetts Oligonukleotidna jedinjenja za tretman preeklampsije i drugih angiogenskih poremećaja
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2016164866A1 (en) 2015-04-10 2016-10-13 Hudsonalpha Institute For Biotechnology METHOD FOR BLOCKING miRNA
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
EP3331573A4 (en) 2015-08-06 2019-02-27 City of Hope THERAPEUTIC CELL-INTERNALIZING CONJUGATES
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
IL295755A (en) 2015-10-09 2022-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
CN105194689A (zh) 2015-10-10 2015-12-30 湖南中楚博生物科技有限公司 一种基于siRNA的双偶联复合物
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
EP3440222B1 (en) 2016-04-04 2025-02-19 F. Hoffmann-La Roche AG Nucleic acid sample preparation methods
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
DK3607069T3 (da) 2017-04-05 2022-11-21 Silence Therapeutics Gmbh Produkter og sammensætninger
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ 2テイル自己デリバリー型siRNAおよび関連方法
WO2019035038A1 (en) 2017-08-18 2019-02-21 The Procter & Gamble Company Cleaning agent
EP3550021A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions for inhibiting expression of a target gene
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
US11820985B2 (en) 2019-03-26 2023-11-21 University Of Massachusetts Modified oligonucleotides with increased stability
WO2021041247A1 (en) 2019-08-23 2021-03-04 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
GB201914296D0 (en) * 2019-10-03 2019-11-20 Univ Oxford Innovation Ltd Treatment
JP7837560B2 (ja) 2020-03-26 2026-03-31 ユニバーシティー オブ マサチューセッツ 安定性の向上を有する修飾オリゴヌクレオチドの合成
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
US20220228141A1 (en) 2020-11-23 2022-07-21 University Of Massachusetts Oligonucleotides for dgat2 modulation

Also Published As

Publication number Publication date
AU2022299169A1 (en) 2024-02-08
US20240067967A1 (en) 2024-02-29
CN117677699A (zh) 2024-03-08
KR20240040729A (ko) 2024-03-28
AR126207A1 (es) 2023-09-27
JP7758907B2 (ja) 2025-10-23
US20230078622A1 (en) 2023-03-16
JP2024523509A (ja) 2024-06-28
IL309334A (en) 2024-02-01
CA3174095A1 (en) 2022-12-29
WO2022271786A1 (en) 2022-12-29
MX2023015523A (es) 2024-03-11
US11702659B2 (en) 2023-07-18
TW202315943A (zh) 2023-04-16
EP4359539A1 (en) 2024-05-01
EP4359539A4 (en) 2025-05-14
JP2026009427A (ja) 2026-01-20

Similar Documents

Publication Publication Date Title
BR112023026862A2 (pt) Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos
CA3197311A1 (en) Oligonucleotide compositions and methods thereof
MX2023008469A (es) Oligonucleotidos de doble hebra modificados.
BR112021017583A2 (pt) Compostos, composições e métodos para o tratamento de doença
EP3995581A3 (en) Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
MX2022004101A (es) Composiciones de oligonucleotidos y metodos de uso de las mismas.
BR112022004451A2 (pt) Antagonistas de hpk1 e usos dos mesmos
BR112021022624A2 (pt) Compostos para o tratamento da doença de huntington
MX2023006467A (es) Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos.
BR112022000503A2 (pt) Compostos úteis para tratar infecções por vírus influenza
BRPI0617165B1 (pt) Compostos inibidores mek, composições farmacêuticas que os contem e métodos de uso dos mesmos
BR112023015210A2 (pt) Inibidores de cdk2 e métodos de uso dos mesmos
BRPI0606542A8 (pt) métodos para aumentar o tempo de progressão de uma doença (ttp)
AR107579A1 (es) Composiciones y métodos para disminuir la expresión de tau
BR112022002691A2 (pt) Construtos de vesícula-aso extracelular visando stat6
BRPI0511078A (pt) compostos de pirimidinona cìclicos, composição compreendendo os mesmos, método de inibição e uso
BR112021025645A2 (pt) Degradação de proteína alvo de parp14 para uso em terapia
MX2025008039A (es) Uso de oligonucleótidos antisentido multisegmentados modificados
BRPI0410669A (pt) inibidores de caspase e usos dos mesmo
BR112022012283A2 (pt) Combinações
PE20211238A1 (es) Tratamiento antisentido del sindrome de angelman
EP3283502A1 (en) Methods for reactivating genes on the inactive x chromosome
WO2024077023A3 (en) Cereblon-recruiting bcl-xl/bcl-2 dual degraders
BR112022001365A2 (pt) Formas cristalinas de um inibidor de cd73
BR112022006476A2 (pt) Oligonucleotídeos com análogos de nucleosídeos

Legal Events

Date Code Title Description
B154 Notification of filing of divisional application [chapter 15.50 patent gazette]

Free format text: O PEDIDO FOI DIVIDIDO NO BR122025012809-0 PROTOCOLO 870250051990 EM 20/06/2025 17:12.O PEDIDO FOI DIVIDIDO NO BR122025012809-0 PROTOCOLO 870250051990 EM 20/06/2025 17:12.