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Analysis

1 / 364
1.WO/2025/227005RNAI AGENTS FOR INHIBITING EXPRESSION OF MYOCILIN (MYOC), COMPOSITIONS THEREOF, AND METHODS OF USE
WO - 30.10.2025
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/US2025/026321 Applicant ARROWHEAD PHARMACEUTICALS, INC. Inventor LI, Xiaokai
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Myocilin (MYOC) gene. The MYOC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an MYOC gene. Pharmaceutical compositions that include one or more MYOC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described MYOC RNAi agents to ocular tissue, in vivo, provides for inhibition of MYOC gene expression and a reduction in MYOC activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including glaucoma, e.g., primary open-angle glaucoma (POAG).
2.WO/2025/226876SEQUENCING LIBRARY NORMALIZATION METHODS
WO - 30.10.2025
Int.Class C12Q 1/6806
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
6806Preparing nucleic acids for analysis, e.g. for polymerase chain reaction assay
 Appl.No PCT/US2025/026068 Applicant WATCHMAKER GENOMICS, INC. Inventor RANIK, Martin
This disclosure provides methods and compositions for normalizing polynucleotide concentration and enriching polynucleotides from a sample.
3.WO/2025/226652NUCLEIC ACID SEQUENCES FOR REDUCING CHEMORESISTANCE AND TREATING DISEASE
WO - 30.10.2025
Int.Class A61K 31/7115
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
 Appl.No PCT/US2025/025725 Applicant THE CLEVELAND CLINIC FOUNDATION Inventor LI, Xiaoxia
Provided herein are compositions, systems, kits, and methods for reducing chemoresistance (e.g., to platinum containing chemotherapeutics) and treating certain diseases, using nucleic acid sequences (e.g., aptamers and antisense sequences) that bind to certain mRNAs (e.g., WTAP), or bind a SEFIR domain of an ACT1 protein, or bind to or inhibit mRNA sequences that bind to Act1 protein.
4.20250327070COMPOSITIONS AND METHODS FOR INHIBITING COMPLEMENT FACTOR B
US - 23.10.2025
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 18292187 Applicant ASTRAZENECA IRELAND LIMITED Inventor Melissa Lasaro

Described herein are oligonucleotides (e.g., RNAi oligonucleotides) containing sense and antisense strands for targeting complement factor B (CFB) mRNA. The RNAi oligonucleotide may be used to inhibit CFB expression, levels, and/or activity in a cell. Also, described herein are methods for using an oligonucleotide (e.g., an RNAi oligonucleotide) for the prophylaxis or treatment of a disease, disorder, or condition mediated by complement pathway activation or dysregulation.

5.WO/2025/221909USES OF TYPE II AND TYPE I INORGANIC PYROPHOSPHATASES
WO - 23.10.2025
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/US2025/024998 Applicant CODEXIS, INC. Inventor FORGET, Stephanie, Marie
The present disclosure provides uses of type II and variant type I and type II inorganic pyrophosphatases under nonnatural conditions present in industrial applications. In another aspect, the present disclosure describes compositions and methods of using inorganic pyrophosphatases with nucleoside triphosphates and in coupled reactions with a second enzyme.
6.WO/2025/221925RNA LIGASE MEDIATED OLIGONUCLEOTIDE SYNTHESIS
WO - 23.10.2025
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 PCT/US2025/025017 Applicant CODEXIS, INC. Inventor FORGET, Stephanie, Marie
The present disclosure relates to methods of using single-stranded RNA ligase for synthesis of oligonucleotides, including oligonucleotides containing a conjugate moiety. In some embodiments, the present disclosure further provides recombinant single-stranded RNA ligases, polynucleotides encoding the recombinant single-stranded RNA ligases, and compositions of the single-stranded RNA ligases.
7.20250325702COMPOSITIONS AND METHODS FOR EDITING A TRANSTHYRETIN GENE
US - 23.10.2025
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 19209589 Applicant Beam Therapeutics Inc. Inventor Genesis Lung

Compositions for gene modification related to base editor systems, and methods of using the same to treat or prevent conditions associated with the extracellular deposition in various tissues of amyloid fibrils formed by the aggregation of misfolded transthyretin (TTR) proteins. Such conditions include, but are not limited to, polyneuropathy due to hereditary transthyretin amyloidosis (hATTR-PN) and hereditary cardiomyopathy due to transthyretin amyloidosis (hATTR-CM), both associated with autosomal dominant mutations of the TTR gene, and an age-related cardiomyopathy associated with wild-type TTR proteins (ATTRwt), also known as senile cardiac amyloidosis.

8.WO/2025/217126POLYNUCLEOTIDES, 3' CHAIN TERMINATING TAILS, LIGATION AND USES THEREOF
WO - 16.10.2025
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 PCT/US2025/023599 Applicant CONVERGENCE BIO, INC. Inventor WANG, Chong
The present disclosure provides constructs and methods for RNA ligation of 3' chain terminating tails and uses for improving nucleic acid stability and protein expression.
9.20250320494INHIBITORS OF EXPRESSION AND/OR FUNCTION
US - 16.10.2025
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 18869034 Applicant E-Therapeutics PLC Inventor Alan Victor WHITMORE

The present invention relates to inhibitors, and compositions containing inhibitors, and uses of the same in the treatment or prevention of a disease related to a disorder of haemostasis, such as haemophilia.

10.20250319036BISPHOSPHONATE LIPIDS, LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME, AND METHODS OF USE THEREOF FOR TARGETED DELIVERY
US - 16.10.2025
Int.Class A61K 9/51
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules
51Nanocapsules
 Appl.No 18865098 Applicant The Trustees of the University of Pennsylvania Inventor Michael J. Mitchell

Described herein, in some aspects, are bisphosphonate lipid compounds, lipid nanoparticles (LNPs) thereof, and methods of use thereof. In various embodiments, the LNP selectively targets a cell of interest (e.g., a bone cell and/or bone marrow cell, such as a stem cell, stroma cell, osteoblast, osteocyte, osteoclast, bone lining cell, local mesenchymal cell, progenitor cell, mononuclear blood-borne precursor cell, B cell, endothelial cell, granulocytes, T cell, monocytic lineage, B cell lineage, monocytes, cancer cell, tumor cell, tumor cell that metastasizes to bone, blood cancer cell, and multiple myeloma cell, inter alia). In other aspects, the present disclosure relates to methods for in vivo delivery of therapeutic agents to prevent or treat diseases, disorders, or conditions using the LNP compositions of the disclosure.

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