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Analysis

1.WO/2022/108832INCREASED VOLTAGE-GATED SODIUM CHANNEL ALPHA PROTEIN SUBUNIT EXPRESSION THROUGH VIRAL 2A-MEDIATED CO-EXPRESSION OF NAV BETA SUBUNITS
WO 27.05.2022
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/US2021/059044 Applicant MERCK SHARP & DOHME CORP. Inventor KRAUS, Richard, L.
A voltage-gated sodium channel expression system is described. The system comprises providing a polycistronic RNA message that encodes a polyprotein comprising a voltage-gated sodium channel alpha protein (Navα) subunit and one or more voltage-gated sodium channel beta protein (Navβ) subunits, each of said subunits being separated by a 2A self-cleaving peptide. During translation, the polyprotein is cleaved into individual subunit proteins which can assemble into a voltage-gated sodium channel. Host cells and lipoparticles comprising the sodium channel expression system are also provided.
2.WO/2022/109435TRNA FRAGMENTS AND METHODS OF USE
WO 27.05.2022
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/US2021/060430 Applicant THOMAS JEFFERSON UNIVERSITY Inventor KIRINO, Yohei
The present invention provides fragments of tRNA molecules and methods of use thereof to modulate toll like receptor (TLR) signaling, for immunotherapy and for other therapeutic applications.
3.WO/2022/109171RIBONUCLEIC ACID PURIFICATION
WO 27.05.2022
Int.Class C07H 1/06
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
1Processes for the preparation of sugar derivatives
06Separation; Purification
Appl.No PCT/US2021/059944 Applicant MODERNATX, INC. Inventor KELLEHER, Bill
The present invention relates to compositions and methods for the purification of ribonucleic acids including a polyA tail. The compositions and methods include a polystyrene divinylbenzene particle, a linker, and a poly-deoxythymidine oligonucleotide.
4.WO/2022/108839SYNTHETIC METHANOL INDUCIBLE PROMOTERS AND USES THEREOF
WO 27.05.2022
Int.Class C12N 15/81
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
80for fungi
81for yeasts
Appl.No PCT/US2021/059135 Applicant GINKGO BIOWORKS, INC. Inventor SRINIVAS, Swaminath
This application describes synthetic promoters capable of facilitating the high-yield synthesis of proteins and molecules.
5.WO/2022/109139COMPOUNDS AND METHODS FOR MODULATING ANGIOTENSINOGEN EXPRESSION
WO 27.05.2022
Int.Class C07H 21/04
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
04with deoxyribosyl as saccharide radical
Appl.No PCT/US2021/059896 Applicant IONIS PHARMACEUTICALS, INC. Inventor MULLICK, Adam
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of AGT RNA in a cell or subject, and in certain instances reducing the amount of AGT in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a cardiovascular disease. Such compound and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a RAAS pathway-related disease or disorder. Such symptoms and hallmarks include hypertension, chronic kidney disease, stroke, myocardial infarction, heart failure, valvular heart disease, aneurysms of the blood vessels, peripheral artery disease, and organ damage. Such cardiovascular diseases include hypertension, resistant hypertension, Marfan syndrome, and heart failure.
6.20220153775COMPOUNDS AND METHODS FOR MODULATING ANGIOTENSINOGEN EXPRESSION
US 19.05.2022
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 17529897 Applicant Ionis Pharmaceuticals, Inc. Inventor Adam Mullick

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of AGT RNA in a cell or subject, and in certain instances reducing the amount of AGT in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a cardiovascular disease. Such compound and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a RAAS pathway-related disease or disorder. Such symptoms and hallmarks include hypertension, chronic kidney disease, stroke, myocardial infarction, heart failure, valvular heart disease, aneurysms of the blood vessels, peripheral artery disease, and organ damage. Such cardiovascular diseases include hypertension, resistant hypertension, Marfan syndrome, and heart failure.

7.WO/2022/101512THERAPEUTIC-OLIGONUCLEOTIDES ACTIVATED BY NUCLEASES
WO 19.05.2022
Int.Class A61K 47/50
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
Appl.No PCT/EP2021/081891 Applicant HERNANDEZ, Frank J. Inventor HERNANDEZ, Frank J.
The present invention refers to a drug delivery system which comprises a polynucleotide sequence susceptible of being cleaved by a specific nuclease derived from a bacterium or a mammalian cell, wherein the polynucleotide sequence, from (upstream to downstream) 5' to 3', comprises i) an oligonucleotide sequence of RNase-resistant modified RNA bases and/or nuclease-resistant DNA bases, acting as a resistant moiety, directly linked to ii) a polynucleotide sequence susceptible of being cleaved by a specific nuclease derived from a bacterium or a mammalian cell which is in turn directly linked to iii) a pharmacological active ingredient, wherein the polynucleotide sequence when cleaved at the cleaving site, releases the pharmacological active ingredient.
8.WO/2022/104197METHODS OF OLIGONUCLEOTIDE-BASED AFFINITY CHROMATOGRAPHY
WO 19.05.2022
Int.Class C07H 21/00
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
Appl.No PCT/US2021/059363 Applicant PURILOGICS, LLC Inventor ZHOU, Jinxiang
Disclosed are methods for purifying polynucleotides from a feed solution using oligonucleotide affinity columns at flowrates between 0.5 CV/min to 1000 CV/min. The methods can include several steps including loading a mixture containing the target polynucleotide onto a chromatography media, e.g., a column, that carries a macroporous support with an oligonucleotide affinity ligand bonded to a surface. The affinity ligand can hybridize the targeted polynucleotide and allow for separation and purification of the target.
9.WO/2022/103842OLIGONUCLEOTIDES, REAGENTS AND PREPARATION THEREOF
WO 19.05.2022
Int.Class C07H 21/00
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
Appl.No PCT/US2021/058786 Applicant BIOGEN MA INC. Inventor YAN, Wuming
The present disclosure describes novel reagents and processes for preparing oligonucleotides, which have two or more nucleotides. In one embodiment, the reagent is represented by Formula I' or B.
10.WO/2022/098840MODIFIED ANTISENSE OLIGONUCLEOTIDES TARGETING SPLICING FACTORS
WO 12.05.2022
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/US2021/058010 Applicant THE JACKSON LABORATORY Inventor ANCZUKÓW-CAMARDA, Olga
Provided herein are chemically modified antisense oligonucleotides that bind to sequences on mRNAs encoding the splicing factor ΤΚΑ2β, associated with cancer.