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Analysis

1.20250207163METHODS 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.

2.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.
3.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.
4.20250188132NUCLEIC ACID VACCINES FOR VARICELLA ZOSTER VIRUS (VZV)
US 12.06.2025
Int.Class C07K 14/005
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005from viruses
Appl.No 18927762 Applicant ModernaTX, Inc. Inventor Giuseppe Ciaramella

Aspects of the disclosure relate to nucleic acid vaccines. The vaccines include at least one RNA polynucleotides having an open reading frame encoding at least one varicella zoster virus (VZV) antigen. Methods for preparing and using such vaccines are also described.

5.120129532稳定的脂质或类脂质纳米颗粒悬浮液
CN 10.06.2025
Int.Class A61K 47/10
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
06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
08containing oxygen
10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols ; Poloxamers; PEG/POE alkyl ethers
Appl.No 202380072045.7 Applicant 埃泽瑞斯公司 Inventor C·多曼
本发明提供了稳定的脂质纳米颗粒(LNP)/类脂质纳米颗粒(LiNP)制剂和LNP/LiNP悬浮液、其用途以及在治疗方法中的用途,其依据是发现在制剂或悬浮液中添加表面活性剂避免了聚集,使其具有惊人的长保质期和延长的振荡稳定性。根据本发明,所述聚集的减少降低了本发明制剂和悬浮液的副作用,特别是降低了由包括LNP和/或LiNP的疫苗制剂和抗癌制剂引起的副作用。
6.20250154504GUIDE OLIGONUCLEOTIDES FOR NUCLEIC ACID EDITING IN THE TREATMENT OF HYPERCHOLESTEROLEMIA
US 15.05.2025
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 18838201 Applicant ProQR Therapeutics II B.V. Inventor Gerardus Johannes PLATENBURG

The invention relates to guide oligonucleotides that can bring about specific changes to a target RNA or DNA molecule in a eukaryotic cell, preferably liver cells, for use in the treatment of hypercholesterolemia, cardiovascular disease, liver injury and/or alcohol-induced steatohepatitis in human subjects. More specifically, the invention relates to guide oligonucleotides and the use thereof in the editing of nucleic acids encoding an auto-cleavage site within the PCSK9 proprotein, thereby inhibiting, or inactivating the PCSK9 protein (or the matured PCSK9 complex) in its ability to cause LDL receptor protein degradation.

7.WO/2025/096809OLIGONUCLEOTIDES COMPRISING PHOSPHORAMIDATE INTERNUCLEOTIDE LINKAGES
WO 08.05.2025
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/US2024/053930 Applicant KORRO BIO, INC. Inventor ALAWNEH, Ayman
Provided herein are oligonucleotides comprising phosporamidite internucleotide linkages of the following formula: (I), wherein R1 is isopropyl, isobutyl, sec-butyl, C1-6 haloalkyl, C2-6 hydroxyalkyl, C2-8 alkylene-N(RN)2, C0-2alkylene-C3-8cycloalkyl, 4-10 membered heterocycloalkyl having 1-3 ring heteroatoms selected from 0, N, and S, or 5-10 membered heteroaryl having 1-3 ring heteroatoms selected from 0, N, and S, with the proviso that the heterocycloalkyl or heteroaryl is attached to the sulfur via a carbon ring atom, and the cycloalkyl, heterocycloalkyl, or heteroaryl is substituted with 0,1,2, or 3 R2 groups; each R2 is independently halo, CN, N(RN)2 C1-3alkyl, C1-3haloalkyl, C1-3alkoxy, oxo, CO2RN, or C(0)C1-3alkyl; and each RN is independently H or C1-3alkyl. Also provided are formulations comprising the oligonucleotides disclosed herein, and methods of treating diseases and disorders using the oligonucleotides and formulations thereof disclosed herein.
8.WO/2025/096203TARGETING A CHIMERIC RNA FOR TREATING CANCER
WO 08.05.2025
Int.Class C12Q 1/6886
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
6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
6883for diseases caused by alterations of genetic material
6886for cancer
Appl.No PCT/US2024/051600 Applicant UNIVERSITY OF VIRGINIA PATENT FOUNDATION Inventor LI, Hui
Chimeric RNAs and their protein products, including BCR-ABL in chronic myelogenous leukemia and EML4-ALK4 in lung cancer, have been well-established as ideal biomarkers and drug targets for malignant cancers. Using RNA-Sequencing data from The Cancer Genome Atlas (TCGA) database, a chimeric RNA SLC2A11-MIF was identified that is present in nearly 50% of CRC samples but absent in non-cancer colon tissue. Disclosed herein is a method for diagnosing and treating cancers expressing this chimeric RNA and protein.
9.20250134978RNA CANCER VACCINES
US 01.05.2025
Int.Class A61K 39/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
Appl.No 18807315 Applicant ModernaTX, Inc. Inventor Nicholas Valiante

The disclosure relates to cancer ribonucleic acid (RNA) vaccines, as well as methods of using the vaccines and compositions comprising the vaccines. In particular, the disclosure relates to concatemeric mRNA cancer vaccines encoding several cancer epitopes on a single mRNA construct, i.e. poly-epitope mRNA constructs or poly-neo-epitope constructs. The disclosure further relates to p53 and KRAS mutations, as well as incorporation of immune enhancers such as STING, e.g. mRNA constructs further encoding an immune stimulator or adjuvant. The disclosure further relates to inclusion of universal T cell epitopes, such as tetanus or diphtheria toxins to elicit an enhanced immune response.

10.119895037用于增强靶mRNA表达的系统及其用途
CN 25.04.2025
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 202380058918.9 Applicant 约翰霍普金斯大学 Inventor 杰弗里·科勒
本文提供了核酸药剂,其是或包含一种或多种核酸分子,该一种或多种核酸分子一起包含(a)与靶mRNA杂交的互补元件;和(b)多聚腺苷酸元件。在一些实施方式中,靶mRNA是与病症相关的基因的活性等位基因的mRNA,该病症与由mRNA进行的蛋白质表达降低相关,其中该病症是单倍剂量不足病症。