CO2023018002A2 - Antisense compounds and methods for targeting cug repeats - Google Patents
Antisense compounds and methods for targeting cug repeatsInfo
- Publication number
- CO2023018002A2 CO2023018002A2 CONC2023/0018002A CO2023018002A CO2023018002A2 CO 2023018002 A2 CO2023018002 A2 CO 2023018002A2 CO 2023018002 A CO2023018002 A CO 2023018002A CO 2023018002 A2 CO2023018002 A2 CO 2023018002A2
- Authority
- CO
- Colombia
- Prior art keywords
- methods
- antisense compounds
- cug repeats
- expanded
- compounds
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 5
- 230000000692 anti-sense effect Effects 0.000 title abstract 3
- 230000008685 targeting Effects 0.000 title 1
- 101000701517 Homo sapiens Putative protein ATXN8OS Proteins 0.000 abstract 2
- 102100030469 Putative protein ATXN8OS Human genes 0.000 abstract 2
- 201000010099 disease Diseases 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 201000009340 myotonic dystrophy type 1 Diseases 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 abstract 1
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 abstract 1
- 108010069514 Cyclic Peptides Proteins 0.000 abstract 1
- 102000001189 Cyclic Peptides Human genes 0.000 abstract 1
- 208000037140 Steinert myotonic dystrophy Diseases 0.000 abstract 1
- 125000004122 cyclic group Chemical group 0.000 abstract 1
Classifications
-
- 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
- C12N15/113—Non-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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61K47/62—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 a protein, peptide or polyamino acid
- A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
- A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
- A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
- C07K7/64—Cyclic peptides containing only normal peptide links
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
Abstract
Se proporcionan compuestos que comprenden un péptido cíclico, tal como un péptido penetrante de células cíclico, y un compuesto antisentido. El compuesto antisentido se une a un gen que tiene una repetición CTG expandida o un transcrito génico que tiene una repetición CUG expandida. Los compuestos pueden suministrarse a los sujetos para tratar enfermedades asociadas con repeticiones CTG·CUG expandidas, tales como la distrofia miotónica tipo 1 (DM1), la ataxia espinocerebelosa-8 (SCA8) y la enfermedad similar a Huntington-2 (HDL2).Provided are compounds comprising a cyclic peptide, such as a cyclic cell penetrating peptide, and an antisense compound. The antisense compound binds to a gene that has an expanded CTG repeat or a gene transcript that has an expanded CUG repeat. The compounds can be administered to subjects to treat diseases associated with expanded CTG·CUG repeats, such as myotonic dystrophy type 1 (DM1), spinocerebellar ataxia-8 (SCA8) and Huntington-like disease-2 (HDL2).
Applications Claiming Priority (16)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163213900P | 2021-06-23 | 2021-06-23 | |
US202163239671P | 2021-09-01 | 2021-09-01 | |
US202163239847P | 2021-09-01 | 2021-09-01 | |
US202163290892P | 2021-12-17 | 2021-12-17 | |
US202163290960P | 2021-12-17 | 2021-12-17 | |
US202263298565P | 2022-01-11 | 2022-01-11 | |
US202263305071P | 2022-01-31 | 2022-01-31 | |
US202263268577P | 2022-02-25 | 2022-02-25 | |
US202263314369P | 2022-02-26 | 2022-02-26 | |
US202263316634P | 2022-03-04 | 2022-03-04 | |
US202263317856P | 2022-03-08 | 2022-03-08 | |
US202263362295P | 2022-03-31 | 2022-03-31 | |
US202263326201P | 2022-03-31 | 2022-03-31 | |
US202263327179P | 2022-04-04 | 2022-04-04 | |
US202263339250P | 2022-05-06 | 2022-05-06 | |
PCT/US2022/034517 WO2022271818A1 (en) | 2021-06-23 | 2022-06-22 | Antisense compounds and methods for targeting cug repeats |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2023018002A2 true CO2023018002A2 (en) | 2024-02-05 |
Family
ID=82608300
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2023/0018002A CO2023018002A2 (en) | 2021-06-23 | 2024-01-09 | Antisense compounds and methods for targeting cug repeats |
Country Status (7)
Country | Link |
---|---|
EP (1) | EP4359006A1 (en) |
KR (1) | KR20240038967A (en) |
AU (1) | AU2022298774A1 (en) |
CA (1) | CA3222824A1 (en) |
CO (1) | CO2023018002A2 (en) |
IL (1) | IL309001A (en) |
WO (1) | WO2022271818A1 (en) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023219933A1 (en) * | 2022-05-09 | 2023-11-16 | Entrada Therapeutics, Inc. | Compositions and methods for delivery of nucleic acid therapeutics |
Family Cites Families (85)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
FR2567892B1 (en) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | NOVEL OLIGONUCLEOTIDES, THEIR PREPARATION PROCESS AND THEIR APPLICATIONS AS MEDIATORS IN DEVELOPING THE EFFECTS OF INTERFERONS |
US4987071A (en) | 1986-12-03 | 1991-01-22 | University Patents, Inc. | RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods |
CA1340323C (en) | 1988-09-20 | 1999-01-19 | Arnold E. Hampel | Rna catalyst for cleaving specific rna sequences |
JP3058686B2 (en) | 1989-08-31 | 2000-07-04 | シティ・オブ・ホープ | Chimeric DNA-RNA catalytically active sequence |
US5591722A (en) | 1989-09-15 | 1997-01-07 | Southern Research Institute | 2'-deoxy-4'-thioribonucleosides and their antiviral activity |
EP0942000B1 (en) | 1989-10-24 | 2004-06-23 | Isis Pharmaceuticals, Inc. | 2'-Modified oligonucleotides |
US5646265A (en) | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
US5670633A (en) | 1990-01-11 | 1997-09-23 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
ES2116977T3 (en) | 1990-05-11 | 1998-08-01 | Microprobe Corp | SOLID SUPPORTS FOR NUCLEIC ACID HYBRIDIZATION TESTS AND METHODS TO IMMOBILIZE OLIGONUCLEOTIDES IN A COVALENT WAY. |
US6365730B1 (en) | 1990-06-19 | 2002-04-02 | Gene Shears Pty. Limited | DNA-Armed ribozymes and minizymes |
DE552178T1 (en) | 1990-10-12 | 1994-02-03 | Max Planck Gesellschaft | MODIFIED RIBOZYMS. |
DE4216134A1 (en) | 1991-06-20 | 1992-12-24 | Europ Lab Molekularbiolog | SYNTHETIC CATALYTIC OLIGONUCLEOTIDE STRUCTURES |
DE59208572D1 (en) | 1991-10-17 | 1997-07-10 | Ciba Geigy Ag | Bicyclic nucleosides, oligonucleotides, processes for their preparation and intermediates |
US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
FR2687679B1 (en) | 1992-02-05 | 1994-10-28 | Centre Nat Rech Scient | OLIGOTHIONUCLEOTIDES. |
WO1993023569A1 (en) | 1992-05-11 | 1993-11-25 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for inhibiting viral replication |
EP0577558A2 (en) | 1992-07-01 | 1994-01-05 | Ciba-Geigy Ag | Carbocyclic nucleosides having bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates |
EP0786522A2 (en) | 1992-07-17 | 1997-07-30 | Ribozyme Pharmaceuticals, Inc. | Enzymatic RNA molecules for treatment of stenotic conditions |
EP0673559A1 (en) | 1992-12-14 | 1995-09-27 | Honeywell Inc. | Motor system with individually controlled redundant windings |
CA2159631A1 (en) | 1993-03-30 | 1994-10-13 | Sanofi | Acyclic nucleoside analogs and oligonucleotide sequences containing them |
DE4311944A1 (en) | 1993-04-10 | 1994-10-13 | Degussa | Coated sodium percarbonate particles, process for their preparation and detergent, cleaning and bleaching compositions containing them |
US5446137B1 (en) | 1993-12-09 | 1998-10-06 | Behringwerke Ag | Oligonucleotides containing 4'-substituted nucleotides |
US5519134A (en) | 1994-01-11 | 1996-05-21 | Isis Pharmaceuticals, Inc. | Pyrrolidine-containing monomers and oligomers |
US5631359A (en) | 1994-10-11 | 1997-05-20 | Ribozyme Pharmaceuticals, Inc. | Hairpin ribozymes |
US5627053A (en) | 1994-03-29 | 1997-05-06 | Ribozyme Pharmaceuticals, Inc. | 2'deoxy-2'-alkylnucleotide containing nucleic acid |
US5597909A (en) | 1994-08-25 | 1997-01-28 | Chiron Corporation | Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use |
AU3889595A (en) | 1994-10-05 | 1996-05-02 | Amgen, Inc. | Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein |
US5792747A (en) | 1995-01-24 | 1998-08-11 | The Administrators Of The Tulane Educational Fund | Highly potent agonists of growth hormone releasing hormone |
US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
JP3756313B2 (en) | 1997-03-07 | 2006-03-15 | 武 今西 | Novel bicyclonucleosides and oligonucleotide analogues |
IL135000A0 (en) | 1997-09-12 | 2001-05-20 | Exiqon As | Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues |
US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
US6043352A (en) | 1998-08-07 | 2000-03-28 | Isis Pharmaceuticals, Inc. | 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides |
WO2000032235A1 (en) | 1998-11-26 | 2000-06-08 | Pentapharm Ag | Transport system conjugate |
EP1152009B2 (en) | 1999-02-12 | 2017-09-06 | Daiichi Sankyo Company, Limited | Novel nucleosides and oligonucleotide analogues |
US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
KR100782896B1 (en) | 1999-05-04 | 2007-12-06 | 엑시콘 에이/에스 | L-Ribo-LNA analogues |
US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
JP4151751B2 (en) | 1999-07-22 | 2008-09-17 | 第一三共株式会社 | New bicyclonucleoside analogues |
WO2001038547A2 (en) | 1999-11-24 | 2001-05-31 | Mcs Micro Carrier Systems Gmbh | Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells |
EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
US7033597B2 (en) | 2000-10-13 | 2006-04-25 | Université de Lausanne | Intracellular delivery of biological effectors |
AU2002220979A1 (en) | 2000-10-13 | 2002-04-22 | Xigen Sa | Intracellular delivery of biological effectors by novel transporter peptide sequences |
US7569575B2 (en) | 2002-05-08 | 2009-08-04 | Santaris Pharma A/S | Synthesis of locked nucleic acid derivatives |
US20040219565A1 (en) | 2002-10-21 | 2004-11-04 | Sakari Kauppinen | Oligonucleotides useful for detecting and analyzing nucleic acids of interest |
AU2003291753B2 (en) | 2002-11-05 | 2010-07-08 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
CA2524255C (en) | 2003-03-21 | 2014-02-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
ES2382807T3 (en) | 2003-08-28 | 2012-06-13 | Takeshi Imanishi | New artificial nucleic acids of the N-O link type with cross-linking |
US7523096B2 (en) | 2003-12-03 | 2009-04-21 | Google Inc. | Methods and systems for personalized network searching |
AU2005252662B2 (en) | 2004-06-03 | 2011-08-18 | Isis Pharmaceuticals, Inc. | Double strand compositions comprising differentially modified strands for use in gene modulation |
AU2006237727B2 (en) | 2005-04-22 | 2012-06-28 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mRNA by interfering with the binding of SR proteins and by interfering with secondary RNA structure. |
KR20130042043A (en) | 2006-01-27 | 2013-04-25 | 아이시스 파마수티컬즈 인코포레이티드 | 6-modified bicyclic nucleic acid analogs |
EP1986697B1 (en) | 2006-02-17 | 2016-06-29 | GE Healthcare Dharmacon, Inc. | Compositions and methods for inhibiting gene silencing by rna interference |
WO2007123391A1 (en) | 2006-04-20 | 2007-11-01 | Academisch Ziekenhuis Leiden | Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene. |
EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
US8252755B2 (en) | 2006-09-22 | 2012-08-28 | Dharmacon, Inc. | Duplex oligonucleotide complexes and methods for gene silencing by RNA interference |
EP2410053B2 (en) | 2006-10-18 | 2020-07-15 | Ionis Pharmaceuticals, Inc. | Antisense compounds |
US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
EP2203173B1 (en) | 2007-10-26 | 2015-12-23 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
JP2012523225A (en) | 2009-04-10 | 2012-10-04 | アソシアシオン・アンスティテュ・ドゥ・ミオロジー | Tricyclo-DNA antisense oligonucleotides, compositions and methods for treatment of disease |
US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
KR102240217B1 (en) | 2012-09-25 | 2021-04-14 | 젠자임 코포레이션 | Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy |
US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
JP6552965B2 (en) | 2012-12-12 | 2019-07-31 | ザ・ブロード・インスティテュート・インコーポレイテッド | Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation |
ES2576128T3 (en) | 2012-12-12 | 2016-07-05 | The Broad Institute, Inc. | Modification by genetic technology and optimization of systems, methods and compositions for the manipulation of sequences with functional domains |
EP4234696A3 (en) | 2012-12-12 | 2023-09-06 | The Broad Institute Inc. | Crispr-cas component systems, methods and compositions for sequence manipulation |
US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
CN113528577A (en) | 2012-12-12 | 2021-10-22 | 布罗德研究所有限公司 | Engineering of systems, methods and optimized guide compositions for sequence manipulation |
JP6712948B2 (en) | 2013-12-12 | 2020-06-24 | ザ・ブロード・インスティテュート・インコーポレイテッド | Compositions and methods of using the CRISPR-cas system in nucleotide repeat disorders |
ES2905181T3 (en) | 2015-05-01 | 2022-04-07 | Prec Biosciences Inc | Precise deletion of chromosomal sequences in vivo |
AU2016335032B2 (en) | 2015-10-05 | 2022-04-14 | Proqr Therapeutics Ii B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
JP6657520B2 (en) | 2016-02-03 | 2020-03-04 | 株式会社神戸製鋼所 | Tire air filling mechanism and tire air filling method of tire testing device |
KR20170130253A (en) | 2016-05-18 | 2017-11-28 | 에스케이텔레콤 주식회사 | Method for providing of adaptive streaming service and apparatus therefor |
US10265249B2 (en) | 2016-09-29 | 2019-04-23 | The Procter & Gamble Company | Fibrous structures comprising glyceride copolymers |
EP3541932A4 (en) | 2016-11-16 | 2021-03-03 | The Regents of the University of California | Inhibitors of crispr-cas9 |
KR20180102871A (en) | 2017-03-08 | 2018-09-18 | 엘지전자 주식회사 | Mobile terminal and vehicle control method of mobile terminal |
EP3592365A4 (en) | 2017-03-10 | 2021-01-13 | The Board Of Regents Of The University Of Texas System | Treatment of fuchs' endothelial corneal dystrophy |
EP3755351A4 (en) * | 2018-02-22 | 2021-11-24 | Entrada Therapeutics, Inc. | Compositions and methods for treating mitochondrial neurogastrointestinal encephalopathy |
GB201812972D0 (en) * | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
WO2021127650A1 (en) * | 2019-12-19 | 2021-06-24 | Entrada Therapeutics, Inc. | Compositions for delivery of antisense compounds |
-
2022
- 2022-06-22 WO PCT/US2022/034517 patent/WO2022271818A1/en active Application Filing
- 2022-06-22 CA CA3222824A patent/CA3222824A1/en active Pending
- 2022-06-22 KR KR1020247002577A patent/KR20240038967A/en unknown
- 2022-06-22 EP EP22744043.5A patent/EP4359006A1/en active Pending
- 2022-06-22 AU AU2022298774A patent/AU2022298774A1/en active Pending
- 2022-06-22 IL IL309001A patent/IL309001A/en unknown
-
2024
- 2024-01-09 CO CONC2023/0018002A patent/CO2023018002A2/en unknown
Also Published As
Publication number | Publication date |
---|---|
WO2022271818A1 (en) | 2022-12-29 |
EP4359006A1 (en) | 2024-05-01 |
AU2022298774A1 (en) | 2023-12-14 |
KR20240038967A (en) | 2024-03-26 |
CA3222824A1 (en) | 2022-12-29 |
IL309001A (en) | 2024-02-01 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
CO2023018002A2 (en) | Antisense compounds and methods for targeting cug repeats | |
UY37881A (en) | IRN AGENTS AND COMPOSITIONS TO INHIBIT THE EXPRESSION OF ANGIOPOYETIN TYPE 3 (ANGPTL3) AND METHODS OF USE | |
AR112072A1 (en) | METHODS OF USE OF INFILTRATING TUMOR LYMPHOCYTES IN DOUBLE REFRACTORY MELANOMA | |
ECSP10010287A (en) | VIRUELA ONCOLITICAL VIRUS VECTORS | |
BR112020022546A8 (en) | extra-hepatic delivery | |
ECSP10010288A (en) | VIRUELA ONCOLITICAL VIRUS VECTORS | |
PE20170140A1 (en) | METHODS FOR TREATING CANCER WITH TIGIT INHIBITORS AND AGENTS AGAINST CANCER | |
UY31543A1 (en) | SYSTEM OF ADMINISTRATION OF RNAI OF INTERFERENCE AND USES OF THE SAME | |
CL2016001093A1 (en) | Assisted remote arc plasma discharge processes | |
AR062271A1 (en) | USE OF AN EFFECTIVE AMOUNT OF AT LEAST A CXCR4 INHIBITOR, AT LEAST A CXCR2 AND G-CSF AGONIST TO MOBILIZE PROGENITOR CELLS AND / OR MOTHER CELLS | |
PE20130558A1 (en) | ISOLATED BACTERIAL STRAIN OF THE GENUS BURKHOLDERIA AND PESTICIDED METABOLITES OF THE SAME | |
AR067980A1 (en) | PEPTIDES OF CDH3 AND AGENTS THAT UNDERSTAND THEM | |
DOP2010000269A (en) | PEPTIDIC ANTAGONIST CONJUGATES ANALOGS TO THE BOMB | |
AR092658A1 (en) | OLIGONUCLEOTIDOS ANTISENTIDO MORFOLINO UNITED TO PEPTIDE FOR THE TREATMENT OF MYOTONIC DYSTROPHY | |
DOP2019000132A (en) | COMPOSITIONS OF SERPINA1 RNAi AND ITS METHODS OF USE OF THE SAME. | |
BRPI0714495B8 (en) | Deficient lentivirus for pseudotyped recombinant replication | |
AR091539A1 (en) | MICRO-ARN miR-15 FAMILY INHIBITORS | |
CO2022002638A2 (en) | Methods for treating fabry disease in patients who have a mutation in the gla gene | |
NI200900165A (en) | STABILIZATION OF VITAMIN B12 | |
CO2020005351A2 (en) | Use of p38 inhibitors to reduce dux4 expression | |
AR084319A1 (en) | MicroRNA INHIBITORS (miRNA OR miR) THAT INCLUDE BLOCKED NUCLEOTIDES | |
CO2020010270A2 (en) | Recombinant vector for gene therapy for girdle muscular dystrophy type 2c | |
CO2023007900A2 (en) | Compositions and methods for targeting bcl11a | |
DOP2022000016A (en) | COMBINED THERAPY WITH ANTIBODIES AGAINST CLAUDIN 18.2 AND INHIBITORS OF THE IMMUNE CHECKPOINT FOR THE TREATMENT OF CANCER | |
AR122352A1 (en) | A C21-N-PYRAZOLIL 19-NOR C3,3 DISUBSTITUTED STEROID AND METHODS OF USE OF IT |