[go: up one dir, main page]

MX2022001769A - Construcciones de vesícula extracelular-oligonucleótido antisentido (aso) que se dirigen a la proteína beta de unión al potenciador ccaat (cebp)/beta. - Google Patents

Construcciones de vesícula extracelular-oligonucleótido antisentido (aso) que se dirigen a la proteína beta de unión al potenciador ccaat (cebp)/beta.

Info

Publication number
MX2022001769A
MX2022001769A MX2022001769A MX2022001769A MX2022001769A MX 2022001769 A MX2022001769 A MX 2022001769A MX 2022001769 A MX2022001769 A MX 2022001769A MX 2022001769 A MX2022001769 A MX 2022001769A MX 2022001769 A MX2022001769 A MX 2022001769A
Authority
MX
Mexico
Prior art keywords
aso
cebp
beta
extracellular vesicle
constructs targeting
Prior art date
Application number
MX2022001769A
Other languages
English (en)
Inventor
Sriram Sathyanarayanan
Ajay Verma
Dalia Burzyn
Sushrut Kamerkar
Adam T Boutin
Wendy Broom
Original Assignee
Codiak Biosciences 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 Codiak Biosciences Inc filed Critical Codiak Biosciences Inc
Publication of MX2022001769A publication Critical patent/MX2022001769A/es

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
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/10Drugs for disorders of the urinary system of the bladder
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/02Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N2310/30Chemical 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
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • 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
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Pulmonology (AREA)
  • Pain & Pain Management (AREA)
  • Dispersion Chemistry (AREA)
  • Dermatology (AREA)
  • Psychology (AREA)
  • Cardiology (AREA)

Abstract

La presente descripción se relaciona con vesículas extracelulares, p. ej., exosomas, que comprenden un oligonucleótido antisentido (ASO), en donde el ASO comprende una secuencia de nucleótidos contiguos de 10 a 30 nucleótidos de longitud que es complementaria a una secuencia de ácido nucleico dentro de un transcrito CEBP/. En la presente también se proporcionan métodos para producir los exosomas y métodos para usar los exosomas para tratar y/o prevenir enfermedades o trastornos.
MX2022001769A 2019-08-14 2020-08-14 Construcciones de vesícula extracelular-oligonucleótido antisentido (aso) que se dirigen a la proteína beta de unión al potenciador ccaat (cebp)/beta. MX2022001769A (es)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201962886930P 2019-08-14 2019-08-14
US201962900136P 2019-09-13 2019-09-13
US201962936220P 2019-11-15 2019-11-15
US201962944204P 2019-12-05 2019-12-05
US202062989540P 2020-03-13 2020-03-13
US202063023065P 2020-05-11 2020-05-11
US202063035357P 2020-06-05 2020-06-05
PCT/US2020/046563 WO2021030780A1 (en) 2019-08-14 2020-08-14 Extracellular vesicle-aso constructs targeting cebp/beta

Publications (1)

Publication Number Publication Date
MX2022001769A true MX2022001769A (es) 2022-06-09

Family

ID=72322528

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022001769A MX2022001769A (es) 2019-08-14 2020-08-14 Construcciones de vesícula extracelular-oligonucleótido antisentido (aso) que se dirigen a la proteína beta de unión al potenciador ccaat (cebp)/beta.

Country Status (11)

Country Link
US (1) US20230002764A1 (es)
EP (1) EP4013878A1 (es)
JP (1) JP2022544288A (es)
KR (1) KR20220070432A (es)
CN (1) CN114729366A (es)
AU (1) AU2020330133A1 (es)
BR (1) BR112022002690A2 (es)
CA (1) CA3147369A1 (es)
IL (1) IL290497A (es)
MX (1) MX2022001769A (es)
WO (1) WO2021030780A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021237100A1 (en) 2020-05-21 2021-11-25 Codiak Biosciences, Inc. Methods of targeting extracellular vesicles to lung
CA3220734A1 (en) * 2021-05-20 2022-11-24 Board Of Regents, The University Of Texas System Production of therapeutic mesenchymal stem cell-derived exosomes
IL311962A (en) 2021-10-14 2024-06-01 Lonza Sales Ag Modified producer cells for extracellular vesicle production
WO2024026345A2 (en) * 2022-07-27 2024-02-01 Lonza Sales Ag Extracellular vesicle-aso constructs targeting cebp/beta
CN116381234B (zh) * 2022-11-06 2025-07-15 复旦大学 Mmp14作为软脑膜转移瘤分子标志物和治疗靶点的应用

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1981001145A1 (en) 1979-10-18 1981-04-30 Univ Illinois Hydrolytic enzyme-activatible pro-drugs
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5773001A (en) 1994-06-03 1998-06-30 American Cyanamid Company Conjugates of methyltrithio antitumor agents and intermediates for their synthesis
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
CN1273478C (zh) 1999-02-12 2006-09-06 三共株式会社 新型核苷及低聚核苷酸类似物
JP2002543214A (ja) 1999-05-04 2002-12-17 エクシコン エ/エス L−リボ−lna類縁体
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US6271030B1 (en) * 2000-06-14 2001-08-07 Isis Pharmaceuticals, Inc. Antisense inhibition of C/EBP beta expression
US7091186B2 (en) 2001-09-24 2006-08-15 Seattle Genetics, Inc. p-Amidobenzylethers in drug delivery agents
US20030083304A1 (en) * 2001-10-09 2003-05-01 Smart Robert C. CCAAT/enhancer binding protein-beta (C/EBPbeta) is a molecular target for cancer treatment
WO2004046160A2 (en) 2002-11-18 2004-06-03 Santaris Pharma A/S Amino-lna, thio-lna and alpha-l-oxy-ln
AU2005216251B2 (en) 2004-02-23 2011-03-10 Genentech, Inc. Heterocyclic self-immolative linkers and conjugates
RU2402548C2 (ru) 2004-05-19 2010-10-27 Медарекс, Инк. Химические линкеры и их конъюгаты
TW200616604A (en) 2004-08-26 2006-06-01 Nicholas Piramal India Ltd Nitric oxide releasing prodrugs containing bio-cleavable linker
US20060269480A1 (en) 2005-05-31 2006-11-30 Ramot At Tel Aviv University Ltd. Multi-triggered self-immolative dendritic compounds
US8142781B2 (en) 2005-10-07 2012-03-27 Armagen Technologies, Inc. Fusion proteins for blood-brain barrier delivery
ES2516815T3 (es) 2006-01-27 2014-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en la posición 6
WO2007131237A2 (en) 2006-05-05 2007-11-15 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of ptp1b
ES2389737T3 (es) 2006-05-11 2012-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en 5'
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
WO2008054544A2 (en) 2006-05-22 2008-05-08 Immune Disease Institute, Inc. Method for delivery across the blood brain barrier
CA2660842C (en) 2006-08-18 2012-03-13 F. Hoffmann-La Roche Ag Polyconjugates for in vivo delivery of polynucleotides
WO2008033924A2 (en) 2006-09-12 2008-03-20 Board Of Regents Of The University Of Nebraska Methods and compositions for targeted delivery of therapeutic agents
TWI412367B (zh) 2006-12-28 2013-10-21 Medarex Llc 化學鏈接劑與可裂解基質以及其之綴合物
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
WO2009006478A2 (en) 2007-07-05 2009-01-08 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
US20130331294A1 (en) * 2007-11-09 2013-12-12 Fox Chase Cancer Center Egfr/nedd9/tgf-beta interactome and methods of use thereof for the identification of agents having efficacy in the treatment of hyperproliferative disorders
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
US8501805B2 (en) 2008-09-24 2013-08-06 Isis Pharmaceuticals, Inc. Substituted alpha-L-bicyclic nucleosides
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US9364495B2 (en) 2009-10-20 2016-06-14 Roche Innovation Center Copenhagen A/S Oral delivery of therapeutically effective LNA oligonucleotides
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
PL2646470T3 (pl) 2010-11-30 2017-07-31 F.Hoffmann-La Roche Ag Przeciwciała przeciw receptorowi transferyny o niskim powinowactwie oraz ich zastosowanie do przenoszenia terapeutycznego scfv przez barierę krew-mózg
WO2013036868A1 (en) 2011-09-07 2013-03-14 Marina Biotech Inc. Synthesis and uses of nucleic acid compounds with conformationally restricted monomers
US9056892B2 (en) 2011-09-09 2015-06-16 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
US9221864B2 (en) 2012-04-09 2015-12-29 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
EP2895200B1 (en) 2012-09-14 2019-11-06 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
WO2016077840A2 (en) 2014-11-14 2016-05-19 Ossianix, Inc. TfR SELECTIVE BINDING COMPOUNDS AND RELATED METHODS
CA2988585A1 (en) * 2015-06-10 2016-12-15 Raghu Kalluri Use of exosomes for the treatment of disease
CN109071597A (zh) * 2016-03-15 2018-12-21 科迪艾克生物科学公司 治疗性膜囊泡
JP2019535839A (ja) * 2016-11-29 2019-12-12 ピュアテック ヘルス エルエルシー 治療剤の送達のためのエクソソーム
US10195290B1 (en) 2017-08-25 2019-02-05 Codiak Biosciences, Inc. Preparation of therapeutic exosomes using membrane proteins
JP2021503300A (ja) 2017-11-17 2021-02-12 コディアック バイオサイエンシーズ, インコーポレイテッド 操作エクソソームの組成物及び内腔エクソソームペイロードの搭載方法
KR20200104347A (ko) * 2017-12-22 2020-09-03 로슈 이노베이션 센터 코펜하겐 에이/에스 포스포로디티오에이트 인터뉴클레오시드 연결을 포함하는 갭머 올리고뉴클레오티드
CN111655271A (zh) * 2017-12-28 2020-09-11 科迪亚克生物科学公司 用于免疫肿瘤学和抗炎疗法的外来体
CN119614501A (zh) * 2018-02-12 2025-03-14 隆萨销售股份公司 用于巨噬细胞极化的方法和组合物
CN112543809A (zh) * 2018-06-15 2021-03-23 米纳治疗有限公司 包含C/EBPα saRNA的组合疗法
PL3672614T3 (pl) * 2018-11-16 2022-03-07 Codiak Biosciences, Inc. Modyfikowane pęcherzyki zewnątrzkomórkowe i ich zastosowania
AU2020241903A1 (en) * 2019-03-21 2021-10-14 Lonza Sales Ag Extracellular vesicle conjugates and uses thereof
JP2022544290A (ja) * 2019-08-14 2022-10-17 コディアック バイオサイエンシーズ, インコーポレイテッド Krasを標的とするアンチセンスオリゴヌクレオチドを有する細胞外小胞
WO2021237100A1 (en) * 2020-05-21 2021-11-25 Codiak Biosciences, Inc. Methods of targeting extracellular vesicles to lung
WO2022216942A1 (en) * 2021-04-07 2022-10-13 Dana-Farber Cancer Institute, Inc. Compositions and methods for the treatment of cancer

Also Published As

Publication number Publication date
CN114729366A (zh) 2022-07-08
CA3147369A1 (en) 2021-02-18
BR112022002690A2 (pt) 2022-08-23
US20230002764A1 (en) 2023-01-05
IL290497A (en) 2022-04-01
KR20220070432A (ko) 2022-05-31
EP4013878A1 (en) 2022-06-22
AU2020330133A1 (en) 2022-03-17
JP2022544288A (ja) 2022-10-17
WO2021030780A1 (en) 2021-02-18

Similar Documents

Publication Publication Date Title
AU2020328611A8 (en) Extracellular vesicle-ASO constructs targeting STAT6
MX2022001769A (es) Construcciones de vesícula extracelular-oligonucleótido antisentido (aso) que se dirigen a la proteína beta de unión al potenciador ccaat (cebp)/beta.
CN107106592B (zh) 用于p21基因调节的rna干扰剂
US10538763B2 (en) Compounds and methods for modulation of DUX4
Wang et al. MicroRNAs in farm animals
FI4220360T3 (fi) Oligonukleotideja paternaalisen ube3a:n ilmentämiseksi
ES2671877T3 (es) Tratamiento de enfermedades relacionadas con la RNASA (H1) mediante inhibición del transcrito antisentido natural a RNASA H1
MX2021012126A (es) Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central.
FI3445388T3 (fi) Materiaaleja ja menetelmiä hemoglobinopatioiden hoitamiseksi
JP2018516091A5 (es)
JP2013226147A5 (es)
HRP20201200T1 (hr) OLIGONUKLEOTIDNI SPOJEVI ZA CILJANJE HUNTINGTIN mRNK
US20210147853A1 (en) Use of Trinucleotide Repeat RNAs To Treat Cancer
CA2921457A1 (en) Heterochromatin forming non-coding rnas
PE20211749A1 (es) Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos
WO2015099122A1 (ja) 遺伝子発現制御のための人工ミミックmiRNAおよびその用途
MX2024001181A (es) Oligomeros antisentido y metodos de uso de los mismos para el tratamiento de enfermedades asociadas con el gen alfa-glucosidasa acida.
CA3139005A1 (en) Exosome comprising stabilized rna therapeutics
JPWO2013073576A1 (ja) RNAi分子の活性を抑制するための核酸分子
JP2019089820A (ja) 化学修飾siRNA
US11786611B2 (en) Methods for treating VEGF-related conditions
WO2024026345A3 (en) Extracellular vesicle-aso constructs targeting cebp/beta
US20220168332A1 (en) Multiplex shRNA for Use in Vectors
TW201726919A (zh) 非對稱干擾rna及其組成物、用途或製備
US20150044196A1 (en) Compositions for Inhibiting Gene Expression and Uses Thereof