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1.WO/2025/140246MPOX VIRUS NUCLEIC ACID VACCINE
WO 03.07.2025
Int.Class C07K 19/00
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
19Hybrid peptides
Appl.No PCT/CN2024/142122 Applicant SUZHOU ABOGEN BIOSCIENCES CO., LTD. Inventor TIAN, Siyu
The present invention relates to the fields of biomedicine and virology, and in particular to a nucleic acid vaccine for preventing and/or treating mpox virus infections. In particular, provided in the present invention are a polypeptide or a polypeptide combination, a nucleic acid or a nucleic acid combination (in particular an mRNA or an mRNA combination), a composition, a pharmaceutical composition, a kit and the use thereof for preventing and/or treating mpox virus infections. Further provided in the present invention are an expression vector containing the nucleic acid or the nucleic acid combination and a host cell containing the nucleic acid or the nucleic acid combination or the expression vector.
2.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.
3.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.
4.20250207163METHODS AND APPARATUS FOR SYNTHESIZING NUCLEIC ACIDS
US 26.06.2025
Int.Class C12P 19/34
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
19Preparation of compounds containing saccharide radicals
26Preparation of nitrogen-containing carbohydrates
28N-glycosides
30Nucleotides
34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
Appl.No 18850185 Applicant Molecular Assemblies, Inc. Inventor Patrycja A. Hopkins

Methods for the synthesis of polynucleotides using polymerases nucleotide analogs with labile terminator groups that allow stepwise addition of nucleotides are described. These nucleotide analogs may have a modification on 3′, 2′, or 3′-2′-bridged hydroxyl groups or on nucleobase moieties and can be used in the synthesis of natural or modified polynucleotides suitable for use in biological systems. The labile terminator groups are removed under mild conditions or by enzymes. They are accepted as substrates by polymerases. The synthesis may be carried out on a surface.

5.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).
6.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.
7.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.
8.WO/2025/126071RNA MOLECULES
WO 19.06.2025
Int.Class C12N 15/67
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
67General methods for enhancing the expression
Appl.No PCT/IB2024/062510 Applicant PFIZER INC. Inventor BENNETT, Eric Matthew
The present disclosure relates to RNA molecules encoding a respiratory syncytial virus (RSV) 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 RSV infection-induced acute respiratory tract illness, including pneumonia and bronchitis.
9.2025092522脂質化合物及び脂質ナノ粒子組成物
JP 19.06.2025
Int.Class C07C 237/06
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
CACYCLIC OR CARBOCYCLIC COMPOUNDS
237Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
02having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
04the carbon skeleton being acyclic and saturated
06having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
Appl.No 2025050091 Applicant スージョウ・アボジェン・バイオサイエンシズ・カンパニー・リミテッド Inventor ボ・イン
【課題】本発明が解決しようとする課題は、ワクチン接種を含む治療または予防目的のために、インビトロ及びインビボの両方で、治療剤(例えば、ロック核酸(LNA)、ペプチド核酸(PNA)、及びモルホリノなどの核酸模倣物を含む核酸分子)を送達するための脂質ナノ粒子を形成するために、中性脂質、コレステロール及びポリマー共役脂質などの他の脂質構成要素と組み合わせて使用され得る脂質化合物を提供することである。
【解決手段】本発明は、式(I)の化合物、またはその薬学的に許容される塩、プロドラッグもしくは立体異性体に関する。
【選択図】なし
10.20250186624POLYNUCLEOTIDES CONTAINING A STABILIZING TAIL REGION
US 12.06.2025
Int.Class A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Appl.No 18901649 Applicant ModernaTX, Inc. Inventor Gabor BUTORA

The invention features polynucleotides encoding a polypeptide including a 3′-stabilizing region and having increased stability compared to wild-type polynucleotides.