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CHEM:(UTAIYTHAJQNQDW-KQYNXXCUSA-N)

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Analysis

1 / 472
1.WO/2024/154048VACCINES AGAINST RESPIRATORY DISEASES
WO - 25.07.2024
Int.Class A61K 39/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
 Appl.No PCT/IB2024/050404 Applicant PFIZER INC. Inventor BERMAN, Damon Andrew Hollands
The present disclosure relates to hMPV F, PIV3 F and PIV1 F protein mutants, nucleic acids or vectors encoding a hMPV F, PIV3 F and PIV1 F protein mutant, compositions comprising a hMPV F, PIV3 F and PIV1 F protein mutant or nucleic acid, and uses of the hMPV F, PIV3 F and PIV1 F protein mutants, nucleic acids or vectors, and compositions.
2.WO/2024/155739POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR TREATMENT OF NEURODEGENERATIVE DISEASES
WO - 25.07.2024
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/US2024/011890 Applicant APTAH BIO, INC. Inventor BRUNO QUINTA DE SOUZA LEAL, Caio
This disclosure concerns an engineered polynucleotide that interacts with a pre-mRNA and a spliceosome to regulate gene expression. The engineered polynucleotide may have stem- loop structure that recruits the spliceosome and targeting sequences that are complementary to a target sequence at an exon-intron splice junction and may include nucleotides with 2' modifications and phosphorothioate linkages. The engineered polynucleotide can be administered to a subject to treat a neurodegenerative disease.
3.WO/2024/155740POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR TREATMENT OF CANCER
WO - 25.07.2024
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/US2024/011891 Applicant APTAH BIO, INC. Inventor BRUNO QUINTA DE SOUZA LEAL, Caio
This disclosure provides an engineered polynucleotide that interacts with a pre-mRNA and a spliceosome to regulate gene expression. The engineered polynucleotide may have stem¬ loop structure that recruits the spliceosome and targeting sequences that are complementary to a target sequence at an exon-intron splice junction and may include nucleotides with 2' modifications and phosphorothioate linkages. The engineered polynucleotide can be administered to a subject to treat a cancer.
4.WO/2024/155770COMPOSITIONS FOR MODULATING KRAS EXPRESSION AND USES THEREOF
WO - 25.07.2024
Int.Class C12N 15/85
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
85for animal cells
 Appl.No PCT/US2024/011938 Applicant MOLECULAR AXIOM, LLC Inventor CHEN, Lishan
Described herein are compounds, compositions, and methods for modulating KRAS expression, such as mutated KRAS expression, and/or downstream signaling pathways. Also described herein are compounds, compositions, and methods for treating a disease or condition associated with mutated KRAS. In some embodiments, the compound comprises at least one antisense oligonucleotide that, upon being delivered into a cell, hybridizes to an endogenous KRAS mRNA, which leads to the degradation of the KRAS mRNA. In some embodiments, the antisense oligonucleotide hybridizes to an mRNA encoding a mutated KRAS protein, such as a KRAS protein comprising a G12C mutation, a G12V mutation, a G12D mutation, or a G12A mutation.
5.WO/2024/155741PRIME EDITING-MEDIATED READTHROUGH OF PREMATURE TERMINATION CODONS (PERT)
WO - 25.07.2024
Int.Class C12N 15/11
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
 Appl.No PCT/US2024/011892 Applicant THE BROAD INSTITUTE, INC. Inventor LIU, David, R.
Aspects of the disclosure relate to methods, compositions, and systems for editing an endogenous tRNA into a suppressor tRNA, or alternatively, replacing said endogenous tRNA with a suppressor tRNA with using prime editing. Additional aspects relate to compositions comprising the prime editing machinery, pegRNAs, and/or complexes comprising the prime editor and pegRNA that are capable of editing and/or replacing an endogenous tRNA to yield a suppressor tRNA. In some aspects, the disclosure further relates to polynucleotides encoding one or more nucleic acid sequences encoding the prime editor and/or pegRNA, cells comprising the polynucleotides and complexes comprising the prime editor and pegRNA, kits comprising any one of the compositions, complexes, polynucleotides, vectors, and/or cells disclosed herein, and/or delivery systems for administering any one of the compositions, complexes, polynucleotides, vectors to a subject in need thereof. Additional aspects relate to methods for inserting a new suppressor tRNA gene into a target site in a genome (e.g., a safe harbor locus site) using prime editing.
6.WO/2024/154052IMMUNOGENIC COMPOSITIONS AND METHODS OF INDUCING AN IMMUNE RESPONSE AGAINST VARICELLA ZOSTER VIRUS
WO - 25.07.2024
Int.Class A61K 39/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
 Appl.No PCT/IB2024/050411 Applicant PFIZER INC. Inventor ALLEN, Pirada Suphaphiphat
The present disclosure provides for methods of inducing an immune response against varicella zoster virus (VZV) in a human subject. The methods provided herein comprise administering immunogenic compositions (e.g., vaccines) comprising RNA molecules formulated in a lipid nanoparticle to a human subject. The present disclosure further relates to the use of immunogenic compositions for preventing or treating of herpes zoster in a human subject.
7.20240238210POLYNUCLEOTIDES ENCODING CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS
US - 18.07.2024
Int.Class A61K 9/51
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules
51Nanocapsules
 Appl.No 18471617 Applicant Moderna TX, Inc. Inventor Kerry Benenato

The invention relates to mRNA therapy for the treatment of cystic fibrosis. mRNAs for use in the invention, when administered in vivo, encode cystic fibrosis transmembrane conductance regulator (CFTR), isoforms thereof, functional fragments thereof, and fusion proteins comprising CFTR. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of CFTR expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient CFTR activity in subjects.

8.20240238402METHOD FOR PRODUCING RNA COMPOSITIONS
US - 18.07.2024
Int.Class A61K 39/145
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
145Orthomyxoviridae, e.g. influenza virus
 Appl.No 18349294 Applicant CureVac Manufacturing GmbH Inventor Thorsten MUTZKE

The present invention relates to a method for producing a liquid composition comprising a nanoparticle comprising at least one RNA and at least one cationic or polycationic compound, advantageously on a large scale suitable for pharmaceutical applications. The present invention further concerns the use of the inventive method in the manufacture of a medicament or a vaccine. Furthermore, the invention relates to compositions containing the RNA-comprising nanoparticle, and to pharmaceutical compositions comprising the same.

9.WO/2024/151811PERSONALIZED CANCER VACCINES
WO - 18.07.2024
Int.Class A61K 39/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
 Appl.No PCT/US2024/011156 Applicant MODERNATX, INC. Inventor BROWN, Michelle
Personalized mRNA cancer vaccines, as well as methods for their optimization and for their use in subjects are provided. In some embodiments, personalized mRNA cancer vaccines and their uses are provided for subjects having particular characteristics, e.g., the presence of certain biomarkers.
10.WO/2024/151501NOVEL IMIDAZOPYRIMIDINE COMPOUND AND USES THEREOF
WO - 18.07.2024
Int.Class A61K 31/519
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
495having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
519ortho- or peri-condensed with heterocyclic rings
 Appl.No PCT/US2024/010632 Applicant THE CHILDREN'S MEDICAL CENTER CORPORATION Inventor DOWLING, David, J.
The present disclosure provides Compound (1), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, and compositions thereof. Compound (1) is used as an enhancer and/or modifier of an immune response (e.g., innate and/or adaptive immune response), and is useful in treating and/or preventing a disease, as an adjuvant in a vaccine for a disease, (e.g., a proliferative disease, an inflammatory disease, an autoimmune disease, an infectious disease, an allergy, a fibrotic disease, a cardiovascular disease, a graft rejection, graft- versus-host disease, chronic disease, or addiction), or as stand alone anti-infective or immune response modifying agents. Also provided in the present disclosure are vaccines, pharmaceutical compositions, kits, methods, and uses including or using Compound (1).
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