WO2026055461A1 - Conjugués anticorps-oligonucléotide comprenant un agent polynucléotidique antisens conjugué à un anticorps cd33, et leurs procédés d'utilisation - Google Patents

Conjugués anticorps-oligonucléotide comprenant un agent polynucléotidique antisens conjugué à un anticorps cd33, et leurs procédés d'utilisation

Info

Publication number
WO2026055461A1
WO2026055461A1 PCT/US2025/045099 US2025045099W WO2026055461A1 WO 2026055461 A1 WO2026055461 A1 WO 2026055461A1 US 2025045099 W US2025045099 W US 2025045099W WO 2026055461 A1 WO2026055461 A1 WO 2026055461A1
Authority
WO
WIPO (PCT)
Prior art keywords
aoc
antibody
cell
disease
cells
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
PCT/US2025/045099
Other languages
English (en)
Inventor
Martin JACKO
Arventh VELUSAMY
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Aperture Therapeutics Inc
Original Assignee
Aperture Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Aperture Therapeutics Inc filed Critical Aperture Therapeutics Inc
Publication of WO2026055461A1 publication Critical patent/WO2026055461A1/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Hospice & Palliative Care (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente divulgation concerne des conjugués anticorps-oligonucléotide (AOC) comprenant un agent polynucléotidique antisens conjugué à un anticorps CD33, des procédés d'utilisation de ceux-ci, et des procédés de production.
PCT/US2025/045099 2024-09-05 2025-09-05 Conjugués anticorps-oligonucléotide comprenant un agent polynucléotidique antisens conjugué à un anticorps cd33, et leurs procédés d'utilisation Pending WO2026055461A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202463690860P 2024-09-05 2024-09-05
US63/690,860 2024-09-05

Publications (1)

Publication Number Publication Date
WO2026055461A1 true WO2026055461A1 (fr) 2026-03-12

Family

ID=97405845

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2025/045099 Pending WO2026055461A1 (fr) 2024-09-05 2025-09-05 Conjugués anticorps-oligonucléotide comprenant un agent polynucléotidique antisens conjugué à un anticorps cd33, et leurs procédés d'utilisation

Country Status (1)

Country Link
WO (1) WO2026055461A1 (fr)

Citations (161)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4816397A (en) 1983-03-25 1989-03-28 Celltech, Limited Multichain polypeptides or proteins and processes for their production
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US4845205A (en) 1985-01-08 1989-07-04 Institut Pasteur 2,N6 -disubstituted and 2,N6 -trisubstituted adenosine-3'-phosphoramidites
WO1990002809A1 (fr) 1988-09-02 1990-03-22 Protein Engineering Corporation Production et selection de proteines de liaison diversifiees de recombinaison
US4981957A (en) 1984-07-19 1991-01-01 Centre National De La Recherche Scientifique Oligonucleotides with modified phosphate and modified carbohydrate moieties at the respective chain termini
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
WO1991017271A1 (fr) 1990-05-01 1991-11-14 Affymax Technologies N.V. Procedes de triage de banques d'adn recombine
WO1992001047A1 (fr) 1990-07-10 1992-01-23 Cambridge Antibody Technology Limited Procede de production de chainon de paires a liaison specifique
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
WO1992009690A2 (fr) 1990-12-03 1992-06-11 Genentech, Inc. Methode d'enrichissement pour des variantes de l'hormone de croissance avec des proprietes de liaison modifiees
US5130300A (en) 1986-03-07 1992-07-14 Monsanto Company Method for enhancing growth of mammary parenchyma
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
WO1992015679A1 (fr) 1991-03-01 1992-09-17 Protein Engineering Corporation Phage de visualisation d'un determinant antigenique ameliore
WO1992018619A1 (fr) 1991-04-10 1992-10-29 The Scripps Research Institute Banques de recepteurs heterodimeres utilisant des phagemides
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
WO1992020791A1 (fr) 1990-07-10 1992-11-26 Cambridge Antibody Technology Limited Methode de production de chainons de paires de liaison specifique
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5177195A (en) 1991-01-08 1993-01-05 Imperial Chemical Industries Plc Disazo dyes
WO1993001288A1 (fr) 1991-07-08 1993-01-21 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Phagemide utile pour trier des anticorps
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5319080A (en) 1991-10-17 1994-06-07 Ciba-Geigy Corporation Bicyclic nucleosides, oligonucleotides, process for their preparation and intermediates
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5405939A (en) 1987-10-22 1995-04-11 Temple University Of The Commonwealth System Of Higher Education 2',5'-phosphorothioate oligoadenylates and their covalent conjugates with polylysine
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5446137A (en) 1993-12-09 1995-08-29 Syntex (U.S.A.) Inc. Oligonucleotides containing 4'-substituted nucleotides
US5445934A (en) 1989-06-07 1995-08-29 Affymax Technologies N.V. Array of oligonucleotides on a solid substrate
US5455233A (en) 1989-11-30 1995-10-03 University Of North Carolina Oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5466677A (en) 1993-03-06 1995-11-14 Ciba-Geigy Corporation Dinucleoside phosphinates and their pharmaceutical compositions
US5466786A (en) 1989-10-24 1995-11-14 Gilead Sciences 2'modified nucleoside and nucleotide compounds
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5514785A (en) 1990-05-11 1996-05-07 Becton Dickinson And Company Solid supports for nucleic acid hybridization assays
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5519126A (en) 1988-03-25 1996-05-21 University Of Virginia Alumni Patents Foundation Oligonucleotide N-alkylphosphoramidates
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5541316A (en) 1992-02-11 1996-07-30 Henkel Kommanditgesellschaft Auf Aktien Process for the production of polysaccharide-based polycarboxylates
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
US5552540A (en) 1987-06-24 1996-09-03 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
US5567811A (en) 1990-05-03 1996-10-22 Amersham International Plc Phosphoramidite derivatives, their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
US5576427A (en) 1993-03-30 1996-11-19 Sterling Winthrop, Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5596086A (en) 1990-09-20 1997-01-21 Gilead Sciences, Inc. Modified internucleoside linkages having one nitrogen and two carbon atoms
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5610300A (en) 1992-07-01 1997-03-11 Ciba-Geigy Corporation Carbocyclic nucleosides containing bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates
US5614617A (en) 1990-07-27 1997-03-25 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5639873A (en) 1992-02-05 1997-06-17 Centre National De La Recherche Scientifique (Cnrs) Oligothionucleotides
US5645620A (en) 1995-05-25 1997-07-08 Foster Wheeler Development Corp. System for separating particulates and condensable species from a gas stream
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5658873A (en) 1993-04-10 1997-08-19 Degussa Aktiengesellschaft Coated sodium percarbonate particles, a process for their production and detergent, cleaning and bleaching compositions containing them
US5663312A (en) 1993-03-31 1997-09-02 Sanofi Oligonucleotide dimers with amide linkages replacing phosphodiester linkages
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5677439A (en) 1990-08-03 1997-10-14 Sanofi Oligonucleotide analogues containing phosphate diester linkage substitutes, compositions thereof, and precursor dinucleotide analogues
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5677195A (en) 1991-11-22 1997-10-14 Affymax Technologies N.V. Combinatorial strategies for polymer synthesis
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5744305A (en) 1989-06-07 1998-04-28 Affymetrix, Inc. Arrays of materials attached to a substrate
US5750692A (en) 1990-01-11 1998-05-12 Isis Pharmaceuticals, Inc. Synthesis of 3-deazapurines
US5770722A (en) 1994-10-24 1998-06-23 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US5874219A (en) 1995-06-07 1999-02-23 Affymetrix, Inc. Methods for concurrently processing multiple biological chip assays
WO1999014226A2 (fr) 1997-09-12 1999-03-25 Exiqon A/S Analogues d'oligonucleotides
US6015886A (en) 1993-05-24 2000-01-18 Chemgenes Corporation Oligonucleotide phosphate esters
US6028188A (en) 1993-11-16 2000-02-22 Genta Incorporated Synthetic oligomers having chirally pure phosphonate internucleosidyl linkages mixed with non-phosphonate internucleosidyl linkages
US6124445A (en) 1994-11-23 2000-09-26 Isis Pharmaceuticals, Inc. Phosphotriester oligonucleotides, amidities and method of preparation
US6147200A (en) 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
US6160109A (en) 1995-10-20 2000-12-12 Isis Pharmaceuticals, Inc. Preparation of phosphorothioate and boranophosphate oligomers
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
US6169170B1 (en) 1994-03-18 2001-01-02 Lynx Therapeutics, Inc. Oligonucleotide N3′→N5′Phosphoramidate Duplexes
US6172209B1 (en) 1997-02-14 2001-01-09 Isis Pharmaceuticals Inc. Aminooxy-modified oligonucleotides and methods for making same
US6222025B1 (en) 1995-03-06 2001-04-24 Isis Pharmaceuticals, Inc. Process for the synthesis of 2′-O-substituted pyrimidines and oligomeric compounds therefrom
US6235887B1 (en) 1991-11-26 2001-05-22 Isis Pharmaceuticals, Inc. Enhanced triple-helix and double-helix formation directed by oligonucleotides containing modified pyrimidines
US6239265B1 (en) 1990-01-11 2001-05-29 Isis Pharmaceuticals, Inc. Oligonucleotides having chiral phosphorus linkages
US6268490B1 (en) 1997-03-07 2001-07-31 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogues
US6277603B1 (en) 1991-12-24 2001-08-21 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
US6326199B1 (en) 1991-12-24 2001-12-04 Isis Pharmaceuticals, Inc. Gapped 2′ modified oligonucleotides
US6346614B1 (en) 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
US6444423B1 (en) 1996-06-07 2002-09-03 Molecular Dynamics, Inc. Nucleosides comprising polydentate ligands
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US6528640B1 (en) 1997-11-05 2003-03-04 Ribozyme Pharmaceuticals, Incorporated Synthetic ribonucleic acids with RNAse activity
US6531590B1 (en) 1998-04-24 2003-03-11 Isis Pharmaceuticals, Inc. Processes for the synthesis of oligonucleotide compounds
US6534639B1 (en) 1999-07-07 2003-03-18 Isis Pharmaceuticals, Inc. Guanidinium functionalized oligonucleotides and method/synthesis
US6608035B1 (en) 1994-10-25 2003-08-19 Hybridon, Inc. Method of down-regulating gene expression
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US6639062B2 (en) 1997-02-14 2003-10-28 Isis Pharmaceuticals, Inc. Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom
US6670461B1 (en) 1997-09-12 2003-12-30 Exiqon A/S Oligonucleotide analogues
US6747014B2 (en) 1997-07-01 2004-06-08 Isis Pharmaceuticals, Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
US20040171570A1 (en) 2002-11-05 2004-09-02 Charles Allerson Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US6815432B2 (en) 1995-06-07 2004-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US6858225B2 (en) 1997-05-14 2005-02-22 Inex Pharmaceuticals Corporation Lipid-encapsulated polyanionic nucleic acid
US6858715B2 (en) 1999-02-04 2005-02-22 Isis Pharmaceuticals, Inc. Process for the synthesis of oligomeric compounds
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
US6878805B2 (en) 2002-08-16 2005-04-12 Isis Pharmaceuticals, Inc. Peptide-conjugated oligomeric compounds
US6998484B2 (en) 2000-10-04 2006-02-14 Santaris Pharma A/S Synthesis of purine locked nucleic acid analogues
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7273933B1 (en) 1998-02-26 2007-09-25 Isis Pharmaceuticals, Inc. Methods for synthesis of oligonucleotides
US7321029B2 (en) 2000-01-21 2008-01-22 Geron Corporation 2′-arabino-fluorooligonucleotide N3′→P5′ phosphoramidates: their synthesis and use
US20080039618A1 (en) 2002-11-05 2008-02-14 Charles Allerson Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US7399845B2 (en) 2006-01-27 2008-07-15 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
US7427672B2 (en) 2003-08-28 2008-09-23 Takeshi Imanishi Artificial nucleic acids of n-o bond crosslinkage type
US7495088B1 (en) 1989-12-04 2009-02-24 Enzo Life Sciences, Inc. Modified nucleotide compounds
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
WO2011005861A1 (fr) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. Coiffes d’extrémité d’oligonucléotides
US8030467B2 (en) 2006-05-11 2011-10-04 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
US8058069B2 (en) 2008-04-15 2011-11-15 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
US20110313020A1 (en) 2008-12-03 2011-12-22 Marina Biotech, Inc. UsiRNA Complexes
US8158601B2 (en) 2009-06-10 2012-04-17 Alnylam Pharmaceuticals, Inc. Lipid formulation
US20120157511A1 (en) 2009-07-07 2012-06-21 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
US8278426B2 (en) 2007-06-08 2012-10-02 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
US8278283B2 (en) 2007-07-05 2012-10-02 Isis Pharmaceuticals, Inc. 6-disubstituted or unsaturated bicyclic nucleic acid analogs
US8278425B2 (en) 2007-05-30 2012-10-02 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
US8314227B2 (en) 2007-05-22 2012-11-20 Marina Biotech, Inc. Hydroxymethyl substituted RNA oligonucleotides and RNA complexes
US20130011922A1 (en) 2007-03-02 2013-01-10 F/K/A Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
WO2013036868A1 (fr) 2011-09-07 2013-03-14 Marina Biotech Inc. Synthèse et utilisations de composés acides nucléiques comportant des monomères restreints de point de vue conformationnel
US20130190383A1 (en) 2010-04-26 2013-07-25 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
WO2019006280A1 (fr) * 2017-06-30 2019-01-03 Lentigen Technology, Inc. Anticorps monoclonaux humains spécifiques de cd33 et leurs procédés d'utilisation
WO2024046937A1 (fr) * 2022-08-29 2024-03-07 F. Hoffmann-La Roche Ag Oligonucléotides antisens d'acide nucléique de thréose et procédés associés

Patent Citations (181)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
US4816397A (en) 1983-03-25 1989-03-28 Celltech, Limited Multichain polypeptides or proteins and processes for their production
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
US4981957A (en) 1984-07-19 1991-01-01 Centre National De La Recherche Scientifique Oligonucleotides with modified phosphate and modified carbohydrate moieties at the respective chain termini
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US4845205A (en) 1985-01-08 1989-07-04 Institut Pasteur 2,N6 -disubstituted and 2,N6 -trisubstituted adenosine-3'-phosphoramidites
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683202B1 (fr) 1985-03-28 1990-11-27 Cetus Corp
US5130300A (en) 1986-03-07 1992-07-14 Monsanto Company Method for enhancing growth of mammary parenchyma
US5286717A (en) 1987-03-25 1994-02-15 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5552540A (en) 1987-06-24 1996-09-03 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US5405939A (en) 1987-10-22 1995-04-11 Temple University Of The Commonwealth System Of Higher Education 2',5'-phosphorothioate oligoadenylates and their covalent conjugates with polylysine
US5519126A (en) 1988-03-25 1996-05-21 University Of Virginia Alumni Patents Foundation Oligonucleotide N-alkylphosphoramidates
US5453496A (en) 1988-05-26 1995-09-26 University Patents, Inc. Polynucleotide phosphorodithioate
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
WO1990002809A1 (fr) 1988-09-02 1990-03-22 Protein Engineering Corporation Production et selection de proteines de liaison diversifiees de recombinaison
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
US5445934A (en) 1989-06-07 1995-08-29 Affymax Technologies N.V. Array of oligonucleotides on a solid substrate
US5744305A (en) 1989-06-07 1998-04-28 Affymetrix, Inc. Arrays of materials attached to a substrate
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5466786B1 (en) 1989-10-24 1998-04-07 Gilead Sciences 2' Modified nucleoside and nucleotide compounds
US5466786A (en) 1989-10-24 1995-11-14 Gilead Sciences 2'modified nucleoside and nucleotide compounds
US5455233A (en) 1989-11-30 1995-10-03 University Of North Carolina Oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US7495088B1 (en) 1989-12-04 2009-02-24 Enzo Life Sciences, Inc. Modified nucleotide compounds
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US6239265B1 (en) 1990-01-11 2001-05-29 Isis Pharmaceuticals, Inc. Oligonucleotides having chiral phosphorus linkages
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5750692A (en) 1990-01-11 1998-05-12 Isis Pharmaceuticals, Inc. Synthesis of 3-deazapurines
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5587469A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides containing N-2 substituted purines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5563253A (en) 1990-03-08 1996-10-08 Worcester Foundation For Biomedical Research Linear aminoalkylphosphoramidate oligonucleotide derivatives
US5536821A (en) 1990-03-08 1996-07-16 Worcester Foundation For Biomedical Research Aminoalkylphosphorothioamidate oligonucleotide deratives
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
WO1991017271A1 (fr) 1990-05-01 1991-11-14 Affymax Technologies N.V. Procedes de triage de banques d'adn recombine
US5567811A (en) 1990-05-03 1996-10-22 Amersham International Plc Phosphoramidite derivatives, their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
US5514785A (en) 1990-05-11 1996-05-07 Becton Dickinson And Company Solid supports for nucleic acid hybridization assays
WO1992001047A1 (fr) 1990-07-10 1992-01-23 Cambridge Antibody Technology Limited Procede de production de chainon de paires a liaison specifique
WO1992020791A1 (fr) 1990-07-10 1992-11-26 Cambridge Antibody Technology Limited Methode de production de chainons de paires de liaison specifique
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5614617A (en) 1990-07-27 1997-03-25 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5677439A (en) 1990-08-03 1997-10-14 Sanofi Oligonucleotide analogues containing phosphate diester linkage substitutes, compositions thereof, and precursor dinucleotide analogues
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
US5596086A (en) 1990-09-20 1997-01-21 Gilead Sciences, Inc. Modified internucleoside linkages having one nitrogen and two carbon atoms
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
WO1992009690A2 (fr) 1990-12-03 1992-06-11 Genentech, Inc. Methode d'enrichissement pour des variantes de l'hormone de croissance avec des proprietes de liaison modifiees
US5177195A (en) 1991-01-08 1993-01-05 Imperial Chemical Industries Plc Disazo dyes
WO1992015679A1 (fr) 1991-03-01 1992-09-17 Protein Engineering Corporation Phage de visualisation d'un determinant antigenique ameliore
WO1992018619A1 (fr) 1991-04-10 1992-10-29 The Scripps Research Institute Banques de recepteurs heterodimeres utilisant des phagemides
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
WO1993001288A1 (fr) 1991-07-08 1993-01-21 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Phagemide utile pour trier des anticorps
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5393878A (en) 1991-10-17 1995-02-28 Ciba-Geigy Corporation Bicyclic nucleosides, oligonucleotides, process for their preparation and intermediates
US5319080A (en) 1991-10-17 1994-06-07 Ciba-Geigy Corporation Bicyclic nucleosides, oligonucleotides, process for their preparation and intermediates
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5677195A (en) 1991-11-22 1997-10-14 Affymax Technologies N.V. Combinatorial strategies for polymer synthesis
US6380368B1 (en) 1991-11-26 2002-04-30 Isis Pharmaceuticals, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US6235887B1 (en) 1991-11-26 2001-05-22 Isis Pharmaceuticals, Inc. Enhanced triple-helix and double-helix formation directed by oligonucleotides containing modified pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US6326199B1 (en) 1991-12-24 2001-12-04 Isis Pharmaceuticals, Inc. Gapped 2′ modified oligonucleotides
US6277603B1 (en) 1991-12-24 2001-08-21 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
US5639873A (en) 1992-02-05 1997-06-17 Centre National De La Recherche Scientifique (Cnrs) Oligothionucleotides
US5541316A (en) 1992-02-11 1996-07-30 Henkel Kommanditgesellschaft Auf Aktien Process for the production of polysaccharide-based polycarboxylates
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5700920A (en) 1992-07-01 1997-12-23 Novartis Corporation Carbocyclic nucleosides containing bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates
US5610300A (en) 1992-07-01 1997-03-11 Ciba-Geigy Corporation Carbocyclic nucleosides containing bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates
US6683167B2 (en) 1992-07-23 2004-01-27 University Of Massachusetts Worcester Hybrid oligonucleotide phosphorothioates
US6346614B1 (en) 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
US5466677A (en) 1993-03-06 1995-11-14 Ciba-Geigy Corporation Dinucleoside phosphinates and their pharmaceutical compositions
US5576427A (en) 1993-03-30 1996-11-19 Sterling Winthrop, Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
US5663312A (en) 1993-03-31 1997-09-02 Sanofi Oligonucleotide dimers with amide linkages replacing phosphodiester linkages
US5658873A (en) 1993-04-10 1997-08-19 Degussa Aktiengesellschaft Coated sodium percarbonate particles, a process for their production and detergent, cleaning and bleaching compositions containing them
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US6015886A (en) 1993-05-24 2000-01-18 Chemgenes Corporation Oligonucleotide phosphate esters
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US6028188A (en) 1993-11-16 2000-02-22 Genta Incorporated Synthetic oligomers having chirally pure phosphonate internucleosidyl linkages mixed with non-phosphonate internucleosidyl linkages
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5446137A (en) 1993-12-09 1995-08-29 Syntex (U.S.A.) Inc. Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US6169170B1 (en) 1994-03-18 2001-01-02 Lynx Therapeutics, Inc. Oligonucleotide N3′→N5′Phosphoramidate Duplexes
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5770722A (en) 1994-10-24 1998-06-23 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
US6608035B1 (en) 1994-10-25 2003-08-19 Hybridon, Inc. Method of down-regulating gene expression
US6124445A (en) 1994-11-23 2000-09-26 Isis Pharmaceuticals, Inc. Phosphotriester oligonucleotides, amidities and method of preparation
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
US6222025B1 (en) 1995-03-06 2001-04-24 Isis Pharmaceuticals, Inc. Process for the synthesis of 2′-O-substituted pyrimidines and oligomeric compounds therefrom
US5645620A (en) 1995-05-25 1997-07-08 Foster Wheeler Development Corp. System for separating particulates and condensable species from a gas stream
US6815432B2 (en) 1995-06-07 2004-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5874219A (en) 1995-06-07 1999-02-23 Affymetrix, Inc. Methods for concurrently processing multiple biological chip assays
US6160109A (en) 1995-10-20 2000-12-12 Isis Pharmaceuticals, Inc. Preparation of phosphorothioate and boranophosphate oligomers
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US6444423B1 (en) 1996-06-07 2002-09-03 Molecular Dynamics, Inc. Nucleosides comprising polydentate ligands
US6172209B1 (en) 1997-02-14 2001-01-09 Isis Pharmaceuticals Inc. Aminooxy-modified oligonucleotides and methods for making same
US6639062B2 (en) 1997-02-14 2003-10-28 Isis Pharmaceuticals, Inc. Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
US6268490B1 (en) 1997-03-07 2001-07-31 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogues
US6858225B2 (en) 1997-05-14 2005-02-22 Inex Pharmaceuticals Corporation Lipid-encapsulated polyanionic nucleic acid
US6747014B2 (en) 1997-07-01 2004-06-08 Isis Pharmaceuticals, Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
US7034133B2 (en) 1997-09-12 2006-04-25 Exiqon A/S Oligonucleotide analogues
US6670461B1 (en) 1997-09-12 2003-12-30 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
WO1999014226A2 (fr) 1997-09-12 1999-03-25 Exiqon A/S Analogues d'oligonucleotides
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US6528640B1 (en) 1997-11-05 2003-03-04 Ribozyme Pharmaceuticals, Incorporated Synthetic ribonucleic acids with RNAse activity
US7273933B1 (en) 1998-02-26 2007-09-25 Isis Pharmaceuticals, Inc. Methods for synthesis of oligonucleotides
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
US6531590B1 (en) 1998-04-24 2003-03-11 Isis Pharmaceuticals, Inc. Processes for the synthesis of oligonucleotide compounds
USRE39464E1 (en) 1998-07-14 2007-01-09 Isis Pharmaceuticals Inc. Oligonucleolotides having site specific chiral phosphorothioate internucleoside linkages
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
US7041816B2 (en) 1999-02-04 2006-05-09 Isis Pharmaceuticals, Inc. Process for the synthesis of oligomeric compounds
US6858715B2 (en) 1999-02-04 2005-02-22 Isis Pharmaceuticals, Inc. Process for the synthesis of oligomeric compounds
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US6534639B1 (en) 1999-07-07 2003-03-18 Isis Pharmaceuticals, Inc. Guanidinium functionalized oligonucleotides and method/synthesis
US6147200A (en) 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
US7321029B2 (en) 2000-01-21 2008-01-22 Geron Corporation 2′-arabino-fluorooligonucleotide N3′→P5′ phosphoramidates: their synthesis and use
US6998484B2 (en) 2000-10-04 2006-02-14 Santaris Pharma A/S Synthesis of purine locked nucleic acid analogues
US6878805B2 (en) 2002-08-16 2005-04-12 Isis Pharmaceuticals, Inc. Peptide-conjugated oligomeric compounds
US20040171570A1 (en) 2002-11-05 2004-09-02 Charles Allerson Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US20080039618A1 (en) 2002-11-05 2008-02-14 Charles Allerson Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US7427672B2 (en) 2003-08-28 2008-09-23 Takeshi Imanishi Artificial nucleic acids of n-o bond crosslinkage type
US7399845B2 (en) 2006-01-27 2008-07-15 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
US20090012281A1 (en) 2006-01-27 2009-01-08 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
US7741457B2 (en) 2006-01-27 2010-06-22 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
US8022193B2 (en) 2006-01-27 2011-09-20 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
US8030467B2 (en) 2006-05-11 2011-10-04 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
US20130011922A1 (en) 2007-03-02 2013-01-10 F/K/A Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
US20130096289A1 (en) 2007-05-22 2013-04-18 Marina Biotech, Inc. Hydroxymethyl substituted rna oligonucleotides and rna complexes
US8314227B2 (en) 2007-05-22 2012-11-20 Marina Biotech, Inc. Hydroxymethyl substituted RNA oligonucleotides and RNA complexes
US8278425B2 (en) 2007-05-30 2012-10-02 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
US8278426B2 (en) 2007-06-08 2012-10-02 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
US8278283B2 (en) 2007-07-05 2012-10-02 Isis Pharmaceuticals, Inc. 6-disubstituted or unsaturated bicyclic nucleic acid analogs
US8058069B2 (en) 2008-04-15 2011-11-15 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
US20110313020A1 (en) 2008-12-03 2011-12-22 Marina Biotech, Inc. UsiRNA Complexes
US8158601B2 (en) 2009-06-10 2012-04-17 Alnylam Pharmaceuticals, Inc. Lipid formulation
US20120157511A1 (en) 2009-07-07 2012-06-21 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
WO2011005861A1 (fr) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. Coiffes d’extrémité d’oligonucléotides
US20130190383A1 (en) 2010-04-26 2013-07-25 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
WO2013036868A1 (fr) 2011-09-07 2013-03-14 Marina Biotech Inc. Synthèse et utilisations de composés acides nucléiques comportant des monomères restreints de point de vue conformationnel
WO2019006280A1 (fr) * 2017-06-30 2019-01-03 Lentigen Technology, Inc. Anticorps monoclonaux humains spécifiques de cd33 et leurs procédés d'utilisation
WO2024046937A1 (fr) * 2022-08-29 2024-03-07 F. Hoffmann-La Roche Ag Oligonucléotides antisens d'acide nucléique de thréose et procédés associés

Non-Patent Citations (105)

* Cited by examiner, † Cited by third party
Title
ANAMI, Y. ET AL.: "Glutamic acid-valine-citrulline linkers ensure stability and efficacy of antibody-drug conjugates in mice", NAT COMMUN, vol. 9, 2018, pages 2512
BACKSTROM, E. ET AL.: "Tissue pharmacokinetics of antisense oligonucleotides.", MOLECULAR THERAPY - NUCLEIC ACIDS, vol. 35, 2024, pages 102133
BAEK, YSINGH, N.ARUNKUMAR, A.ZYDNEY, A.: "Effects of Histidine and Sucrose on the Biophysical Properties of a Monoclonal Antibody", PHARMACEUTICAL RESEARCH, vol. 34, 2017
BALKA, K. R. ET AL.: "TBK1 and IKKε Act Redundantly to Mediate STING-Induced NF- B Responses in Myeloid Cells", CELL REPORTS, vol. 31, 2020, pages 107492
BARANY, PROC. NATL. ACAD. SCI. USA, vol. 88, 1991, pages 189 - 193
BARGH ET AL., CHEM. SOCIETY REV., vol. 48, 2019, pages 4361 - 4374
BARGH, J. D.ISIDRO-LLOBET, A.PARKER, J. S.SPRING, D. R.: "Cleavable linkers in antibody-drug conjugates", CHEM. SOC. REV., vol. 48, 2019, pages 4361 - 4374, XP009517547, DOI: 10.1039/c8cs00676h
BAXI, E. G. ET AL.: "Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines", NAT NEUROSCI, 2022
BENNETT ET AL., ANNU. REV. PHARMACOL. TOXICOL, vol. 61, 2021, pages 831 - 852
BENNETT, C. F.KORDASIEWICZ, H. B.CLEVELAND, D. W.: "Antisense Drugs Make Sense for Neurological Diseases", ANNU. REV. PHARMACOL. TOXICOL., vol. 61, 2021, pages 831 - 852, XP093105569, DOI: 10.1146/annurev-pharmtox-010919-023738
BERTRAM, C. ET AL.: "The differentiation/retrodifferentiation program of human U937 leukemia cells is accompanied by changes of VCP/p97", BMC CELL BIOL, vol. 9, 2008, pages 1 - 16
BEYCHOK: "Cells of Immunoglobulin Synthesis", 1979, ACADEMIC PRESS
BORGHERO, G. ET AL.: "TBK1 is associated with ALS and ALS-FTD in Sardinian patients", NEUROBIOLOGY OF AGING, vol. 43, 2016, XP029561330, DOI: 10.1016/j.neurobiolaging.2016.03.028
BOUCHER, L. E. ET AL.: "Stapling'' scFv for multispecific biotherapeutics of superior properties", MABS, vol. 15, 2023, pages 2195517, XP093216321, DOI: 10.1080/19420862.2023.2195517
BRENNER, D. ET AL.: "Heterozygous Tbk 1 loss has opposing effects in early and late stages of ALS in mice", JOURNAL OF EXPERIMENTAL MEDICINE, vol. 216, 2019, pages 267 - 278
BRIGHT, F. ET AL.: "Neuroinflammation in frontotemporal dementia", NAT REV NEUROL, vol. 15, 2019, pages 540 - 555, XP036875238, DOI: 10.1038/s41582-019-0231-z
BROWN, A.-L. ET AL.: "TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A", NATURE, 2022, pages 1 - 8
BROWN, K. M. ET AL.: "Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates", NAT BIOTECHNOL, vol. 40, 2022, pages 1500 - 1508, XP038002886, DOI: 10.1038/s41587-022-01334-x
CALIAS, P.BANKS, W. A.BEGLEY, D.SCARPA, M.DICKSON, P.: "Intrathecal delivery of protein therapeutics to the brain: A critical reassessment", PHARMACOLOGY & THERAPEUTICS, vol. 144, 2014, pages 114 - 122, XP029050993, DOI: 10.1016/j.pharmthera.2014.05.009
CATE, B. TEN ET AL.: "Targeted Elimination of Leukemia Stem Cells; a New Therapeutic Approach in Hemato-Oncology", CURRENT DRUG TARGETS, vol. 11, pages 95 - 110
CHATTOPADHYAYA ET AL., J. ORG. CHEM., vol. 74, 2009, pages 118 - 134
CHOTHIA ET AL., NATURE, vol. 342, 1989, pages 877 - 883
CHOTHIALESK, J. MOL. BIOL., vol. 196, 1987, pages 901 - 917
CLACKSON, NATURE, vol. 352, 1991, pages 624 - 628
COOPER-KNOCK, J. ET AL.: "A data-driven approach links microglia to pathology and prognosis in amyotrophic lateral sclerosis", ACTA NEUROPATHOLOGICA COMMUNICATIONS, vol. 5, 2017, pages 23
DEMING, Y. ET AL.: "The MS4A gene cluster is a key modulator of soluble TREM2 and Alzheimer's disease risk.", SCI. TRANSL. MED., vol. 11, 2019, pages 2291, XP093124733, DOI: 10.1126/scitranslmed.aau2291
DERMITZAKIS, I. ET AL.: "Origin and Emergence of Microglia in the CNS-An Interesting (Hi)story of an Eccentric Cell", CIMB, vol. 45, 2023, pages 2609 - 2628
DIAS, N. ET AL., MOL CANCER THER, vol. 1, 2002, pages 347 - 355
DIN ABDUL JABBAR, M. A. ET AL.: "Describing and characterising variability in ALS disease progression", AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION, vol. 25, 2024, pages 34 - 45
EHNINGER, A. ET AL.: "Distribution and levels of cell surface expression of CD33 and CD 123 in acute myeloid leukemia", BLOOD CANCER JOURNAL, vol. 4, 2014, pages e218 - e218, XP055705039, DOI: 10.1038/bcj.2014.39
ELMEN, J. ET AL., NUCLEIC ACIDS RESEARCH, vol. 33, no. 1, 2005, pages 439 - 447
ESTUS, S. ET AL.: "Evaluation of CD33 as a genetic risk factor for Alzheimer's disease.", ACTA NEUROPATHOL, vol. 138, 2019, pages 187 - 199, XP036968915, DOI: 10.1007/s00401-019-02000-4
FERGUSON, C. M. ET AL.: "Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep", JCI INSIGHT, vol. 6, 2021
FLUITER ET AL., MOL. BIOSYST., vol. 10, 2009, pages 1039
FU, Z.LI, SHAN, S.SHI, C.ZHANG, Y.: "Antibody drug conjugate: the ''biological missile'' for targeted cancer therapy", SIG TRANSDUCT TARGET THER, vol. 7, 2022, pages 1 - 25, XP093049353, DOI: 10.1038/s41392-022-00947-7
GAO, C.JIANG, J.TAN, Y.CHEN, S.: "Microglia in neurodegenerative diseases: mechanism and potential therapeutic targets.", SIG TRANSDUCT TARGET THER, vol. 8, 2023, pages 1 - 37
GBADAMOSI, M.MESHINCHI, S.LAMBA, J. K.: "Gemtuzumab Ozogamicin for Treatment of Newly Diagnosed CD33-Positive Acute Myeloid Leukemia", FUTURE ONCOL., vol. 14, 2018, pages 3199 - 3213, XP055756232, DOI: 10.2217/fon-2018-0325
GEARY, R. S.: "Antisense oligonucleotide pharmacokinetics and metabolism.", EXPERT OPINION ON DRUG METABOLISM & TOXICOLOGY, vol. 5, 2009, pages 381 - 391, XP009127048, DOI: 10.1517/17425250902877680
GERBINO, V. ET AL.: "The Loss of TBK1 Kinase Activity in Motor Neurons or in All Cell Types Differentially Impacts ALS Disease Progression in SOD1 Mice", NEURON, vol. 106, 2020, pages 789 - 805
GODINHO, B. M. D. C. ET AL.: "PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs", MOLECULAR THERAPY - NUCLEIC ACIDS, vol. 29, 2022, pages 116 - 132, XP093125875, DOI: 10.1016/j.omtn.2022.06.005
GREGORY, J. M.FAGEGALTIER, D.PHATNANI, H.HARMS, M. B.: "Genetics of Amyotrophic Lateral Sclerosis.", CURR GENET MED REP, vol. 8, 2020, pages 121 - 131
GRICIUC, A. ET AL.: "Alzheimer's Disease Risk Gene CD33 Inhibits Microglial Uptake of Amyloid Beta", NEURON, vol. 78, 2013, pages 631 - 643
GRICIUC, A. ET AL.: "TREM2 Acts Downstream of CD33 in Modulating Microglial Pathology in Alzheimer's Disease", NEURON, vol. 103, 2019, pages 820 - 835
GRUNWELLER, A. ET AL., NUCLEIC ACIDS RESEARCH, vol. 31, no. 12, 2003, pages 3185 - 3193
GUATELLI ET AL., PROC. NATL. ACAD. SCI. USA, vol. 87, 1990, pages 1874 - 1878
GUZMAN-MARTINEZ, L. ET AL.: "Neuroinflammation as a Common Feature of Neurodegenerative Disorders", FRONT. PHARMACOL., vol. 10, 2019, pages 1008
HILLARY, R. F. ET AL.: "Genome- and epigenome-wide studies of plasma protein biomarkers for Alzheimer's disease implicate TBCA and TREM2 in disease risk", ALZHEIMERS DEMENT (AMST, vol. 14, 2022, pages e12280
HUA, Y. ET AL.: "Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model", GENES DEV., vol. 24, 2010, pages 1634 - 1644, XP055038360, DOI: 10.1101/gad.1941310
HUA, YVICKERS, T. A.BAKER, B. F.BENNETT, C. F.KRAINER, A. R.: "Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the Exon.", PLOS BIOLOGY, vol. 5, 2007, pages e73
HUGHES, L. D.RAWLE, R. J.BOXER, S. G.: "Choose Your Label Wisely: Water-Soluble Fluorophores Often Interact with Lipid Bilayers", PLOS ONE, vol. 9, 2014, pages e87649
JOHNNSON, B. ET AL., ANAL. BIOCHEM., vol. 198, 1991, pages 268 - 277
JOHNSSON, B. ET AL., J. MOL. RECOGNIT., vol. 8, 1995, pages 125 - 131
JONSSON, T. ET AL.: "Variant of TREM2 Associated with the Risk of Alzheimer's Disease.", N ENGL J MED, vol. 368, 2013, pages 107 - 116, XP055101921, DOI: 10.1056/NEJMoa1211103
JÖNSSON, U. ET AL., BIOTECHNIQUES, vol. 11, 1991, pages 620 - 627
JÖNSSON, U., ANN. BIOL. CLIN., vol. 51, 1993, pages 19 - 26
KABAT ET AL., ANN. NY ACAD, SCI., vol. 190, 1971, pages 382 - 391
KOHLERMILSTEIN, NATURE, vol. 256, 1975, pages 495 - 499
KOROBEYNIKOV, V. A.LYASHCHENKO, A. K.BLANCO-REDONDO, B.JAFAR-NEJAD, P.SHNEIDER, N. A.: "Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis", NAT MED, 2022
KWOH ET AL., PROC. NATL. ACAD. SCI. USA, vol. 86, 1989, pages 1173 - 1177
KWON, H. S.KOH, S.-H.: "Neuroinflammation in neurodegenerative disorders: the roles of microglia and astrocytes", TRANSL NEURODEGENER, vol. 9, 2020, pages 42
LI HONG ET AL: "CD33-Targeted Lipid Nanoparticles (aCD33LNs) for Therapeutic Delivery of GTI-2040 to Acute Myelogenous Leukemia", MOLECULAR PHARMACEUTICS, vol. 12, no. 6, 28 April 2015 (2015-04-28), US, pages 2010 - 2018, XP093344938, ISSN: 1543-8384, DOI: 10.1021/mp5008212 *
LIZARDI ET AL., BIO/TECHNOLOGY, vol. 6, 1988, pages 1197
LOPES, K. DE P. ET AL.: "Genetic analysis of the human microglial transcriptome across brain regions, aging and disease pathologies", NAT GENET, vol. 54, 2022, pages 4 - 17, XP037663372, DOI: 10.1038/s41588-021-00976-y
MA, X. R. ET AL.: "TDP-43 represses cryptic exon inclusion in the FTD-ALS gene UNC13A", NATURE, 2022, pages 1 - 7
MACCALLUM, J MOL BIOL, vol. 262, no. 5, 1996, pages 732 - 45
MARKS ET AL., J. MOL. BIOL., vol. 222, 1991, pages 581 - 597
MARTIN ET AL., HELV. CHIM. ACTA, vol. 78, 1995, pages 486 - 504
MAZUR, C. ET AL.: "Brain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging", JCI INSIGHT, vol. 4, 2019, pages e129240
MCCAMPBELL, A. ET AL.: "Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models", JOURNAL OF CLINICAL INVESTIGATION, vol. 128, 2018, pages 3558 - 3567, XP055679194, DOI: 10.1172/JCI99081
MEJZINI, R. ET AL.: "ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?", FRONT. NEUROSCI., vol. 13, 2019, pages 1310
MILLER, T. ET AL.: "Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.", NEW ENGLAND JOURNAL OF MEDICINE, vol. 383, 2020, pages 109 - 119
MILLER, T. M. ET AL.: "Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.", NEW ENGLAND JOURNAL OF MEDICINE, vol. 387, 2022, pages 1099 - 1110, XP093284380, DOI: 10.1056/NEJMoa2204705
MOOK, OR. ET AL., MOL CANC THER, vol. 6, no. 3, 2007, pages 833 - 843
MORRISON ET AL., PROC. NATL. ACAD. SCI. U.S.A., vol. 81, 1985, pages 6851
MORRISON ET AL., PROC. NATL. ACAD. SCI. USA, vol. 81, 1984, pages 6851 - 55
MORTBERG, M. A. ET AL., A SINGLE-CELL MAP OF ANTISENSE OLIGONUCLEOTIDE ACTIVITY IN THE BRAIN., 2023, Retrieved from the Internet <URL:http://biorxiv.org/lookup/doi/10.1101/2023.02.14.528473>
MORTBERG, M. A. ET AL.: "A single-cell map of antisense oligonucleotide activity in the brain", NUCLEIC ACIDS RESEARCH, 2023
MUZIO, L., VIOTTI, A. & MARTINO, G.: " Microglia in Neuroinflammation and Neurodegeneration: From Understanding to Therapy", FRONT. NEUROSCI., vol. 15, 2021, pages 742065
NADKARNI, D. V.: "Antibody-Drug Conjugates: Methods and Protocols", 2020, SPRINGER US, article "Conjugations to Endogenous Cysteine Residues", pages: 37 - 49
NAJ, A. C. ET AL.: "Common variants at MS4A4/MS4A6E, CD2AP, CD33 and EPHA1 are associated with late-onset Alzheimer's disease", NAT GENET, vol. 43, 2011, pages 436 - 441, XP055945865, DOI: 10.1038/ng.801
NANNA, A. R. ET AL.: "Generation and validation of structurally defined antibody-siRNA conjugates", NUCLEIC ACIDS RESEARCH, vol. 48, 2020, pages 5281 - 5293, XP055876565, DOI: 10.1093/nar/gkaa286
NIELSEN ET AL., SCIENCE, vol. 254, 1991, pages 1497 - 1500
NUC. ACIDS SYMP. SERIES, vol. 52, 2008, pages 133 - 134
OSBORNE, O. M., NARANJO, O., HECKMANN, B. L., DYKXHOORN, D. & TOBOREK, M.: "Anti-amyloid: An antibody to cure Alzheimer's or an attitude", ISCIENCE, vol. 26, 2023, pages 107461
PADLAN, FASEB J., vol. 9, 1995, pages 133 - 139
PERO, J. KHAAS, E. M.THOMPSON, N. L.: "Size Dependence of Protein Diffusion Very Close to Membrane Surfaces: Measurement by Total Internal Reflection with Fluorescence Correlation Spectroscopy", J. PHYS. CHEM. B, vol. 110, 2006, pages 10910 - 10918
RANSOHOFF, R. M.: "How neuroinflammation contributes to neurodegeneration", SCIENCE, vol. 353, 2016, pages 777 - 783
REIDENBACH, A. G. ET AL.: "Characterization of the Prion Protein Binding Properties of Antisense Oligonucleotides", BIOMOLECULES, vol. 10, no. 1, 2020
ROBERTS ET AL., NAT. REV. DRUG DISCOV., vol. 19, 2020, pages 673 - 694
ROBERTS, T. C.LANGER, RWOOD, M. J. A.: "Advances in oligonucleotide drug delivery.", NAT REV DRUG DISCOV, vol. 19, 2020, pages 673 - 694, XP037256878, DOI: 10.1038/s41573-020-0075-7
ROMANO, R.BUCCI, C.: "Antisense therapy: a potential breakthrough in the treatment of neurodegenerative diseases", NEURAL REGENERATION RESEARCH, vol. 19, 2024, pages 1027 - 1035
ROMANOBUCCI, NEURAL REGEN. RES., vol. 19, 2024, pages 1027 - 1035
SANGHVI, Y S.: "dsRNA Research and Applications", 1993, CRC PRESS, pages: 276 - 278
SCOPES: "Protein Purification", 1982, SPRINGER VERLAG
SIOKAS, V. ET AL.: "CD33 rs3865444 as a risk factor for Parkinson's disease.", NEUROSCIENCE LETTERS, vol. 748, 2021, pages 135709
SMITH, SCIENCE, vol. 228, 1985, pages 1315 - 1317
SMUKOWSKI, S. N.: "Progress in Amyotrophic Lateral Sclerosis Gene Discovery: Reflecting on Classic Approaches and Leveraging Emerging Technologies", NEUROL GENET, vol. 8, 2022, pages e669
TAKEDA ET AL., NATURE, vol. 314, 1985, pages 452
THORNE, R. G.NICHOLSON, C.: "In vivo diffusion analysis with quantum dots and dextrans predicts the width of brain extracellular space", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 103, 2006, pages 5567 - 5572
WALKER, D. G. ET AL.: "Association of CD33 polymorphism rs3865444 with Alzheimer's disease pathology and CD33 expression in human cerebral cortex", NEUROBIOLOGY OF AGING, vol. 36, 2015, pages 571 - 582, XP055431146, DOI: 10.1016/j.neurobiolaging.2014.09.023
WINNAKER: "From Genes to Clones", 1987, VERLAGSGESELLSCHAFT
WONG, E. ET AL.: "HuM195 and its single-chain variable fragment increase Aβ phagocytosis in microglia via elimination of CD33 inhibitory signaling", MOL PSYCHIATRY, 2024
YOU, S.-F. ET AL., MS4A4A MODIFIES THE RISK OF ALZHEIMER DISEASE BY REGULATING LIPID METABOLISM AND IMMUNE RESPONSE IN A UNIQUE MICROGLIA STATE, 2023, Retrieved from the Internet <URL:http://medrxiv.org/lookup/doi/10.1101/2023.02.06.23285545>
YU, B.LIU, D.: "Gemtuzumab ozogamicin and novel antibody-drug conjugates in clinical trials for acute myeloid leukemia", BIOMARK RES, vol. 7, 2019, pages 1 - 13, XP093279222, DOI: 10.1186/s40364-019-0175-x
ZHAO LINGZHI: "CD33 in Alzheimer's Disease - Biology, Pathogenesis, and Therapeutics: A Mini-Review", GERONTOLOGY, 12 December 2018 (2018-12-12), CH, pages 323 - 331, XP055945986, ISSN: 0304-324X, Retrieved from the Internet <URL:https://www.karger.com/Article/Pdf/492596> [retrieved on 20251205], DOI: 10.1159/000492596 *

Similar Documents

Publication Publication Date Title
US12325753B2 (en) Method of using an anti-transferrin receptor antibody to deliver an oligonucleotide to a subject having facioscapulohumeral muscular dystrophy
AU2019314538B2 (en) Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US20230111212A1 (en) Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20230088865A1 (en) Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
AU2022345881A1 (en) Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
US11911484B2 (en) Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12319743B2 (en) Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US20230117883A1 (en) Muscle-targeting complexes and uses thereof in treating muscle atrophy
US20240148891A1 (en) Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12496352B2 (en) Muscle targeting complexes and uses thereof for treating myotonic dystrophy
CA3108313A1 (fr) Complexes de ciblage musculaire et leurs utilisations pour le traitement de l&#39;atrophie musculaire
CA3226301A1 (fr) Complexes de ciblage musculaire et utilisations associees pour le traitement de la dystrophie myotonique
KR20250036941A (ko) Btn1a1에 면역특이적으로 결합하는 항체 및 분자 및 이의 치료적 용도
KR20250034419A (ko) 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
JPWO2011118739A1 (ja) 新規修飾部位導入抗体および抗体フラグメント
US12528881B2 (en) Humanized antibody to digoxigenin and uses thereof
US12370264B1 (en) Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
KR20170007492A (ko) 장기에서의 허혈 재관류 손상 방지 방법 및 방지용 조성물
CN102471383B (zh) β细胞标记抗体
WO2026055461A1 (fr) Conjugués anticorps-oligonucléotide comprenant un agent polynucléotidique antisens conjugué à un anticorps cd33, et leurs procédés d&#39;utilisation
KR20260044219A (ko) 조혈 세포 표적화 접합체 및 관련 방법
WO2026076295A2 (fr) Agents arn ciblant zbtb7a et procédés associés
WO2026076314A1 (fr) Agents d&#39;arn ciblant bcl11a et méthodes associées
EA050720B1 (ru) Мышечно-специфические комплексы и их применение для лечения плече-лопаточно-лицевой мышечной дистрофии
EA050686B1 (ru) Мышечно-специфические комплексы и их применение для лечения дистрофинопатий

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 25791466

Country of ref document: EP

Kind code of ref document: A1