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Analysis

1 / 283
1.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.
2.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.
3.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.
4.20240238210POLYNUCLEOTIDES 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.

5.20240238402METHOD 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.

6.WO/2024/151975DETECTING DEGRADATION OF A POLYNUCLEOTIDE BY RAMAN SPECTROSCOPY
WO - 18.07.2024
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/US2024/011439 Applicant LEDNEV, Igor Inventor LEDNEV, Igor
Provided is a method of detecting degradation of a polynucleotide, including irradiating the polynucleotide with ultraviolet light having a wavelength within a range of from about 200 nm to about 280 nm, and detecting Raman scattering by the polynucleotide. A wavenumber shift of a Raman band or change in peak intensity of a Raman band may constitute detection of polynucleotide degradation. In an example the polynucleotide is in a lipid nanoparticle.
7.WO/2024/151685RECOMBINANT NUCLEIC ACID MOLECULES AND THEIR USE IN WOUND HEALING
WO - 18.07.2024
Int.Class A61L 15/18
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION, OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS, OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS, OR SURGICAL ARTICLES
15Chemical aspects of, or use of materials for, bandages, dressings or absorbent pads
16Bandages, dressings or absorbent pads for physiological fluids such as urine or blood, e.g. sanitary towels, tampons
18containing inorganic materials
 Appl.No PCT/US2024/010955 Applicant BETH ISRAEL DEACONESS MEDICAL CENTER, INC. Inventor VEVES, Aristidis
In certain aspects, the disclosure relates to devices and methods for treating wounds wherein an acetylcholinesterase inhibitor is delivered to a wound to promote wound healing. In further aspects, metrifonate can be delivered to the wound by time release from a wound dressing. In further aspects a sequence of wound dressings can be applied to the wound to treat different phases of wound healing.
8.WO/2024/151687GENETIC SWITCHES AND THEIR USE IN TREATING CANCER
WO - 18.07.2024
Int.Class C07K 14/47
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
 Appl.No PCT/US2024/010957 Applicant FLAGSHIP PIONEERING INNOVATIONS V, INC. Inventor SHALER, Christopher, Ryan
In certain aspects, the disclosure relates to a nucleic acid molecule encoding a cytokine (e.g., IL-12) and one or more genetic switches encoding thanotransmission polypeptides (e.g., TRIP, Gasdermin E, or variants thereof). Vectors (e.g., engineered viruses, plasmids and transposons), cells and pharmaceutical compositions comprising one or more nucleic acid molecules encoding one or more thanotransmission polypeptides are also disclosed. Methods of promoting thanotransmission by a target cell, methods of promoting an immune response in a subject, and methods of treating cancer in a subject are further disclosed.
9.20240238438CD-90 Targeted Lipid Nanoparticles
US - 18.07.2024
Int.Class A61K 47/68
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
51the non-active ingredient being a modifying agent
68the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
 Appl.No 18558014 Applicant The Trustees of the University of Pennsylvania Inventor Hamideh Parhiz

The present invention relates to compositions and methods for effective delivery of an agent to a stem cell using a delivery vehicle, such as a lipid nanoparticle (LNP), comprising a CD90 targeting domain.

10.20240238473RECOMBINANT NUCLEIC ACID MOLECULES AND THEIR USE IN WOUND HEALING
US - 18.07.2024
Int.Class A61L 15/32
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION, OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS, OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS, OR SURGICAL ARTICLES
15Chemical aspects of, or use of materials for, bandages, dressings or absorbent pads
16Bandages, dressings or absorbent pads for physiological fluids such as urine or blood, e.g. sanitary towels, tampons
22containing macromolecular materials
32Proteins, polypeptides; Degradation products or derivatives thereof, e.g. albumin, collagen, fibrin, gelatin
 Appl.No 18408498 Applicant Beth Israel Deaconess Medical Center, Inc. Inventor Aristidis Veves

In certain aspects, the disclosure relates to a recombinant nucleic acid molecule (e.g. RNA molecule) encoding one or more polypeptides selected from the group consisting of chitinase-3-like protein 1 (CHI3L1), a fibroblast growth factor (FGF), interleukin-2 ORF7 (IL-2 ORF7), interleukin-2 (IL-2), an interleukin-17 (IL-17) protein, and an IL-17 receptor, wherein the recombinant RNA molecule comprises at least one chemical modification. Cells, pharmaceutical compositions and wound dressings comprising one or more of the recombinant nucleic acid molecules are also disclosed. Methods of promoting wound healing in a subject are further disclosed.

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