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Analysis

1.WO/2025/131119CANCER VACCINES AND USES THEREOF
WO 26.06.2025
Int.Class C12N 15/62
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
62DNA sequences coding for fusion proteins
Appl.No PCT/CN2024/141468 Applicant EVEREST MEDICINES (CHINA) CO., LTD. Inventor HU, Gang
Provided herein are nucleic acids, pharmaceutical compositions and vaccines that express a cancer antigen (e.g., PRAME). Also provided herein are methods for treating a cancer.
2.WO/2025/137275NOVEL CAS NUCLEASES AND POLYNUCLEOTIDES ENCODING THE SAME
WO 26.06.2025
Int.Class C12N 9/22
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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
Appl.No PCT/US2024/061026 Applicant MODERNATX, INC. Inventor AZMI, Ishara
The present disclosure provides novel CRISPR-associated (Cas) nucleases, as well as polynucleotides (e.g., DNA and RNA polynucleotides) encoding the same, host cells containing such nucleases and polynucleotides, and methods of using the foregoing compositions to effectuate genome editing.
3.WO/2025/132839INFLUENZA VIRUS VACCINES
WO 26.06.2025
Int.Class A61K 39/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
Appl.No PCT/EP2024/087475 Applicant GLAXOSMITHKLINE BIOLOGICALS SA Inventor BITTNER-SCHRADER, Zsófia
The present invention is inter alia directed to immunogenic compositions comprising: (a) a first hemagglutinin (HA) antigen or a first nucleic acid, suitably mRNA, encoding the first HA antigen wherein the first HA antigen is derived from a strain of Influenza virus; and (b) a second HA antigen or a second nucleic acid, suitably mRNA, encoding the second HA antigen wherein the second HA antigen is derived from a strain of Influenza virus, wherein (a) and (b) are different, and wherein the ratio of (a):(b) is greater than 5:1. The present invention is also directed to vaccines and kits or kits-of-parts comprising such. Immunogenic compositions, vaccines and kits-of-parts provided herein are suitable for use as a medicament, in particular, for use in the treatment or prophylaxis of an infection with an Influenza virus, suitably an Influenza A and/or Influenza B.
4.WO/2025/133951IONIZABLE LIPIDS SUITABLE FOR LIPID NANOPARTICLES
WO 26.06.2025
Int.Class C07F 7/18
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
FACYCLIC, CARBOCYCLIC, OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
7Compounds containing elements of Groups 4 or 14 of the Periodic Table
02Silicon compounds
08Compounds having one or more C-Si linkages
18Compounds having one or more C-Si linkages as well as one or more C-O-Si linkages
Appl.No PCT/IB2024/062841 Applicant GENEVANT SCIENCES GMBH Inventor CHEN, Kent Tsung Jeng
The present disclosure provides nitrogen-containing silicon ether ionizable lipid compounds and lipid nanoparticles including the ionizable lipid. The disclosure further relates to lipid nanoparticles including a provided ionizable lipid compound together with a phospholipid, e.g., a phospholipid that includes at least one unsaturated tail and a head group having a positively charged nitrogen. The provided materials are particularly beneficial in applications involving delivery of a nucleic acid. The disclosure also provides pharmaceutical compositions and methods including the provided ionizable lipids and/or lipid nanoparticles.
5.WO/2025/128901CIRCULAR RNA MOLECULES
WO 19.06.2025
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/US2024/059894 Applicant ARCTURUS THERAPEUTICS, INC. Inventor CHIVUKULA, Padmanabh
Provided herein are compositions and methods comprising a circular RNA that expresses an RNA-encoded polypeptide. Also provided herein are reaction mixtures i) comprising a DNA-dependent RNA polymerase, a plurality of nucleotide triphosphates, and a transcription template encoding an RNA and ii) an RNA and a ligase to produce a circular DNA.
6.WO/2025/129186CLOSTRIDIOIDES DIFFICILE VACCINE AND METHODS OF USE
WO 19.06.2025
Int.Class A61K 39/08
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
02Bacterial antigens
08Clostridium, e.g. Clostridium tetani
Appl.No PCT/US2024/060394 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor ALAMEH, Mohamad
The present disclosure provides compositions (e.g., pharmaceutical compositions, e.g., RNA-LNP vaccines) for delivery of C. difficile antigens and related technologies (e.g., components thereof and/or methods relating thereto).
7.WO/2025/129157GENE THERAPY FOR TREATMENT OF CANAVAN DISEASE
WO 19.06.2025
Int.Class C12N 15/86
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
86Viral vectors
Appl.No PCT/US2024/060265 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor HORDEAUX, Juliette
Compositions and methods for treatment of Canavan disease are provided. The compositions include recombinant adeno-associated virus (rAAV) vectors, lipid nanoparticles, a recombinant nucleic acid molecules having a nucleotide sequence encoding a functional aspartoacylase (ASPA). In one embodiment, the recombinant adeno-associated virus (rAAV) comprises an adeno-associated virus (AAV) capsid and a vector genome packaged therein, wherein the vector genome comprises an expression cassette comprising one or more regulatory sequences operably linked to a nucleotide sequence encoding a functional aspartoacylase (ASPA), and wherein the nucleotide sequence encoding the functional ASPA is SEQ ID NO: 1 or a nucleotide sequence at least 99% identical to SEQ ID NO: 1.
8.WO/2025/129106IMMUNOEVASIVE TARGETING MOLECULES FOR EFFICIENT TARGETED DELIVERY
WO 19.06.2025
Int.Class A61K 47/59
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY 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
56the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
59obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
Appl.No PCT/US2024/060185 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor PAPP, Tyler
The present invention relates to compositions and methods for effective delivery of a therapeutic agent to a subject using a delivery vehicle comprising a fusion construct comprising a targeting domain and a domain for evasion of the immune system. In some embodiments, the present invention relates to compositions and methods for targeted delivery of a therapeutic agent to a subject using a delivery vehicle comprising a fusion construct comprising a targeting domain for a specific cell type and a domain for evasion of the immune system. The invention also relates to methods of use of the compositions of the invention for the treatment of diseases and disorders, including the treatment of diseases and disorders in subjects having an inflammatory or autoimmune disease or disorder.
9.WO/2025/126019RNA MOLECULES
WO 19.06.2025
Int.Class A61K 39/095
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
02Bacterial antigens
095Neisseria
Appl.No PCT/IB2024/062402 Applicant PFIZER INC. Inventor MCMONAGLE, Patricia Michele
The present disclosure relates to RNA molecules encoding a Neisseria meningitidis polypeptide. The present disclosure further relates to compositions comprising the RNA molecules formulated in a lipid nanoparticle (RNA-LNP). The present disclosure further relates to the use of the RNA molecules, RNA-LNPs and compositions for the treatment and/or prevention of meningococcal disease.
10.WO/2025/125630METHOD FOR BIOPROTAC DESIGN
WO 19.06.2025
Int.Class C12N 15/10
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
10Processes for the isolation, preparation or purification of DNA or RNA
Appl.No PCT/EP2024/086358 Applicant MEDIMMUNE LIMITED Inventor HUNT, James
The present disclosure provides methods for identifying bioPROTACs capable of inducing degradation of target proteins. The disclosure further provides BIOPROTACs which have been generated according to these methods, including bioPROTACs capable of specifically causing degradation of c-Myc or K-Ras.