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Analysis

1 / 6,049
1.WO/2024/093907LIGAND FOR DELIVERING SIRNA TO EYE AND CENTRAL NERVOUS SYSTEM
WO - 10.05.2024
Int.Class C07D 265/30
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
265Heterocyclic compounds containing six-membered rings having one nitrogen atom and one oxygen atom as the only ring hetero atoms
281,4-Oxazines; Hydrogenated 1,4-oxazines
30not condensed with other rings
 Appl.No PCT/CN2023/127728 Applicant SHANGHAI RONA THERAPEUTICS CO., LTD. Inventor HUANG, Jinyu
Provided are a compound of formula (I) or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof. The compound of formula (I) is contained inside a nucleotide, or at a 5' end and/or a 3' end, and is used for improving the ability of a double-stranded RNA to pass through the blood-brain barrier. The present invention further relates to a test kit, a pharmaceutical composition, a cell, a carrier, a double-stranded RNA, or an oligonucleotide comprising the compound of formula (I).
2.WO/2024/093947PRODRUG FOR DELIVERING SIRNA INTO CELL
WO - 10.05.2024
Int.Class C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
 Appl.No PCT/CN2023/127941 Applicant SHANGHAI RONA THERAPEUTICS CO., LTD. Inventor HUANG, Jinyu
The present invention provides an oligonucleotide, comprising a compound of formula (Ia), or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof. The compound of formula (I) is contained inside the oligonucleotide or at the 5' terminal and/or the 3' terminal, and is used for improving the ability of a double-stranded RNA to pass through a cell membrane. The present invention also relates to a double-stranded RNA, a cell, a pharmaceutical composition and a kit comprising the oligonucleotide.
3.WO/2024/097262CHEMOPROTEOMIC CAPTURE OF RNA BINDING ACTIVITY IN LIVING CELLS
WO - 10.05.2024
Int.Class C12Q 1/68
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
 Appl.No PCT/US2023/036544 Applicant UNIVERSITY OF VIRGINIA PATENT FOUNDATION Inventor HSU, Ku-Lung
Methods of identifying RNA-binding sites in proteins are described. In some embodiments, the methods utilize clickable electrophilic purines (CEPs) to study protein-RNA interactions on proteins through photoaffinity competition with 4-thiouridine (4SU)-labeled RNA in cells. The method can be performed in live cells and can be used to identify druggable sites on RNA-binding proteins, including druggable allosteric sites on RNA-binding proteins.
4.WO/2024/097744DNA-COMPATIBLE WITTIG OLEFINATION OF ON-DNA PEPTIDYL-YLIDES FOR DEVELOPMENT OF ON-DNA PEPTIDOMIMETICS THROUGH DIVERSITY-ORIENTED SYNTHESIS (DOS)
WO - 10.05.2024
Int.Class C07H 21/02
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
02with ribosyl as saccharide radical
 Appl.No PCT/US2023/078334 Applicant THE ROCKEFELLER UNIVERSITY Inventor FLAJOLET, Marc
The present invention provides methods of generating diverse chemical structures on DNA through Wittig olefination of novel on-DNA phosphorane ylides and Homer- Wadsworth-Emmons reaction of on-DNA β-keto phosphonates. The methods of this invention provide access to DNA-encode libraries (DELs) of diverse peptides, peptidomimetics, chalcone-based molecules, and the like.
5.WO/2024/097894COMPOSITIONS AND METHODS OF RIBONUCLEIC ACID VACCINES ENCODING NYE-SO-1
WO - 10.05.2024
Int.Class A61K 31/7105
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
 Appl.No PCT/US2023/078543 Applicant RNAIMMUNE, INC. Inventor SHEN, Dong
Compositions and methods are provided for potent NYE-SO-1 vaccines for treatment of cancer. The compositions include a pharmaceutical composition containing RNA molecules encoding NYE-SO-1 derived peptides together with a pharmaceutically acceptable carrier. Methods for stimulating system immune responses and treatment are provided, including intratumoral, intravenous, intramuscular, intradermal, and subcutaneous injection.
6.WO/2024/097821NUCLEOTIDE-BASED ENHANCEMENT AGENT FOR RNA DELIVERY AND THERAPY
WO - 10.05.2024
Int.Class A61K 31/7115
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
 Appl.No PCT/US2023/078442 Applicant SANEGENE BIO USA INC. Inventor WANG, Weimin
The present disclosure provides a Nucleotide-Based Enhancement Agent being a compound or a pharmaceutically acceptable salt thereof, comprising from 2 to 30 nucleotides, wherein each nucleotide independently is of Formula (I) or (II): (I); or (II); pharmaceutically acceptable salts thereof, and related conjugates. The present disclosure also relates to uses of the Nucleotide-Based Enhancement Agents and conjugates, e.g., in delivering nucleic acid and/or treating or preventing diseases.
7.WO/2024/097737GENE THERAPY FOR BVES-RELATED DISORDERS
WO - 10.05.2024
Int.Class A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
 Appl.No PCT/US2023/078326 Applicant OHIO STATE INNOVATION FOUNDATION Inventor HAN, Renzhi
The present disclosure relates to compositions and methods for treating blood vessel epicardial substance (BVES) protein-related disorders.
8.WO/2024/098053POLYMER-NUCLEIC ACID NANOPARTICLES FOR GENE EDITING
WO - 10.05.2024
Int.Class A61K 9/16
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
14Particulate form, e.g. powders
16Agglomerates; Granulates; Microbeadlets
 Appl.No PCT/US2023/078797 Applicant THE JOHNS HOPKINS UNIVERSITY Inventor GREEN, Jordan J.
Provided are nanoparticles comprising a biodegradable synthetic polymer, one or more nucleic acids, and a stabilizing agent, such as PEGylated lipid and their use for delivering genes to the lung epithelium and other tissues for gene editing to correct genetic mutations in diseases, such as cystic fibrosis.
9.WO/2024/091286COMPOSITIONS AND METHODS FOR ANTI-SENSE OLIGONUCLEOTIDE (ASO) TREATMENT OF HUNTINGTON'S DISEASE
WO - 02.05.2024
Int.Class C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
 Appl.No PCT/US2023/017275 Applicant OPHIDION INC. Inventor MACNEIL, Douglas J.
A composition for treating Huntington's disease (HD) by reducing endogenous Huntingtin (HTT) levels in a target includes an ASO comprising an ASO sequence selected from SEQ ID NOs:18 to 86, and a carrier peptide comprising a peptide sequence of SEQ ID NO:1. The carrier peptide may target the nicotinic acetylcholine receptor (nAChR) of neuronal cells in order to deliver the ASO across the blood-brain barrier (BBB). In some embodiments, the ASO may be conjugated with the carrier peptide. A method of reducing endogenous HTT levels to treat HD in the target may include administering a composition including an ASO sequence selected from SEQ ID NOs: 18 to 86 to the target.
10.WO/2024/089953METHOD FOR PRODUCING OLIGONUCLEOTIDE
WO - 02.05.2024
Int.Class C07H 21/02
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
02with ribosyl as saccharide radical
 Appl.No PCT/JP2023/026108 Applicant SUMITOMO CHEMICAL COMPANY, LIMITED Inventor TANAKA, Yuki
The present invention provides a method for producing oligonucleotide by a solid-phase synthesis technique, wherein said method for producing oligonucleotide comprises a step for reacting, in the presence of a thiol, an acid with an oligonucleotide in which the hydroxyl group at the 5' end is protected by a protection group that is removable under acidic conditions, in order to remove the protection group on the hydroxyl group at the 5' end. The present invention also provides oligonucleotide in which the content ratio of N-1-mer in the oligonucleotide is not greater than a certain amount.
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