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1.WO/2025/224230ANTISENSE OLIGONUCLEOTIDES FOR THE TREATMENT OF FATTY LIVER DISEASE
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/EP2025/061190 Applicant PROQR THERAPEUTICS II B.V. Inventor POTMAN, Marko
The present invention relates to antisense oligonucleotides (AONs) that can mediate RNA editing by binding to a target RNA nucleic acid molecule, preferably an RNA transcript molecule, in a cell and recruiting an endogenous deaminating enzyme in the cell to deaminate a target adenosine in the target RNA molecule to an inosine. The target RNA molecule is a transcript molecule form the human PNPLA3 gene that encodes the I148M variant of the Patatin-Like Phospholipase domain-containing protein 3 (PNPLA3), and the target adenosine is the adenosine in the AUG codon coding for methionine (M) at position 148 of the PNPLA3 I148M variant protein. The deamination of the adenosine changes the amino acid to a valine (V). The RNA editing of the adenosine will result in a reversion of the functionality of the PNPLA3 protein that is similar or equal to the functionality of the wildtype PNPLA3 protein, which will result in lowered fattening of the liver, thereby lowering the risk of suffering from disorders related to non-alcoholic fatty liver disease (NAFLD) caused by the I148M variant.
2.WO/2025/224704INJECTION ELUTION METHODS FOR AFFINITY CHROMATOGRAPHY
WO 30.10.2025
Int.Class B01D 15/42
BPERFORMING OPERATIONS; TRANSPORTING
01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
DSEPARATION
15Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
08Selective adsorption, e.g. chromatography
42characterised by the development mode, e.g. by displacement or by elution
Appl.No PCT/IB2025/054347 Applicant WATERS TECHNOLOGIES CORPORATION Inventor MURIITHI, Beatrice
The present disclosure is directed to methods of performing affinity chromatography. The injection elution methods disclosed herein afford rapid and robust elution of a target analyte from an affinity chromatography column, therefore improving workflow efficiency. Further, the single injection elution methods result in highly concentrated samples, mitigating the need for sample concentration or manipulation.
3.WO/2025/222304HCV GENOTYPE3 E2 ENVELOPE PROTEIN FOR NEUTRALIZING INFECTIVITY OF HCV STRAINS
WO 30.10.2025
Int.Class A61K 39/29
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
29Hepatitis virus
Appl.No PCT/CA2025/050602 Applicant THE GOVERNORS OF THE UNIVERSITY OF ALBERTA Inventor HOUGHTON, Michael
The present disclosure provides compositions comprising E2 envelope glycoprotein from HCV Gt3 strain S52. Surprisingly, this composition provides an immune response effective against heterologous HCV strains, e.g., Gt1 strain H77 and Gt2 strain J6. These compositions may be used for inducing an immune response to HCV Gt1-Gt3, e.g., Gt1 strain H77, Gt2 strain J6, and Gt3 strain S52 in an individual.
4.WO/2025/226189PIPERIDINE COMPOUNDS AS PDE5 INHIBITORS
WO 30.10.2025
Int.Class A61K 31/451
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non-condensed pyridines; Hydrogenated derivatives thereof
445Non-condensed piperidines, e.g. piperocaine
451having a carbocyclic ring directly attached to the heterocyclic ring, e.g. glutethimide, meperidine, loperamide, phencyclidine, piminodine
Appl.No PCT/SA2025/050008 Applicant KING SAUD UNIVERSITY Inventor ABDEL-AZIZ, Alaa Abdel-moenes
Novel 1,4-disubstituted piperidine derivatives, a method of synthesizing said compounds, a pharmaceutical composition comprising said compounds and a suitable carrier, and a method of using the compounds. The 1,4-disubstituted piperidine derivatives compounds, identified as PDE5 inhibitors, are useful for the treatment of erectile dysfunction.
5.WO/2025/223684METHODS AND COMPOSITIONS FOR STIMULATING IMMUNE RESPONSE
WO 30.10.2025
Int.Class A61K 39/39
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
39characterised by the immunostimulating additives, e.g. chemical adjuvants
Appl.No PCT/EP2024/071234 Applicant BIONTECH SE Inventor SAHIN, Ugur
The present invention relates to methods and compositions for stimulating an immune response. In particular, the present invention relates to compositions comprising an immunostimulatory RNA comprising sequences derived from an Influenza A virus nudeoprotein-encoding RNA molecule that act as adjuvants and/or immunostimulatory agents to enhance host immune responses, in particular as adjuvants for cancer vaccines.
6.WO/2025/226835GENE THERAPY TREATMENT OF MTRES1 RELATED DISEASES AND DISORDERS
WO 30.10.2025
Int.Class A61P 25/28
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
28for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
Appl.No PCT/US2025/026002 Applicant EMPIRICO INC. Inventor GOTTESMAN, Omri
Disclosed herein are compositions comprising a MTRES1 gene therapy. Also provided herein are methods of treating conditions associated with MTRES1 gene mutations that include providing a MTRES1 gene therapy.
7.WO/2025/224705DIRECT AFFINITY TO SIZE-EXCLUSION CHROMATOGRAPHY METHODS AND SYSTEMS THEREOF
WO 30.10.2025
Int.Class B01D 15/34
BPERFORMING OPERATIONS; TRANSPORTING
01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
DSEPARATION
15Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
08Selective adsorption, e.g. chromatography
26characterised by the separation mechanism
34Size-selective separation, e.g. size-exclusion chromatography; Gel filtration; Permeation
Appl.No PCT/IB2025/054349 Applicant WATERS TECHNOLOGIES CORPORATION Inventor MURIITHI, Beatrice
The present disclosure is directed to methods of performing direct affinity-size exclusion chromatography, wherein there is a direct elution of the sample from the affinity chromatography column to the SEC column. The methods described herein allow for rapid and robust purification of target analytes from heterogeneous samples, and mitigate the need for complicated valve switching, buffer exchange, or other sample manipulation.
8.WO/2025/226701PROTEOLYTICALLY STABLE U1-AGATOXIN-TA1B VARIANT POLYPEPTIDES FOR PEST CONTROL
WO 30.10.2025
Int.Class C07K 14/435
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
Appl.No PCT/US2025/025796 Applicant VESTARON CORPORATION Inventor SCHNEIDER, Kyle Douglas
The application concerns insecticidal proteins and peptides called TVP, which are non-natural peptide variations based upon the U1-agatoxin-Talb toxin derived from the Hobo spider, leading to proteolytically stable variants of the U1-agatoxin-Ta1b. The application also provides polynucleotides and genes encoding these TVPs and their expression in plants; also methods of producing these nucleotides and peptides, processes, production techniques, compositions and formulations of these TVPs, combinations thereof, combinations comprising TVP genes and/or peptides and, organisms expressing these TVPs. These products are useful for the control of pests.
9.WO/2025/226656HEPATITIS B VIRUS MRNA VACCINES
WO 30.10.2025
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/US2025/025733 Applicant MODERNATX, INC. Inventor DIPIAZZA, Anthony T.
Provided herein are messenger ribonucleic acid vaccines for Hepatitis B virus infection and related methods of treatment.
10.WO/2025/226912CELL-TYPE SPECIFIC DELIVERY OF GENE EDITORS AND METHODS OF USE THEREOF
WO 30.10.2025
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/US2025/026135 Applicant SRI INTERNATIONAL Inventor LUEBKE, Kevin, James
The present invention relates to compositions for effective targeted delivery of a gene editing agent or transcriptional modulator to a target cell, as well as methods of use thereof for the treatment of diseases including cancer and genetic diseases. In some embodiments, the invention relates to a composition for targeted delivery of a genome editing agent or transcriptional modulator, wherein the composition comprises a delivery vehicle comprising a targeting moiety specific for binding to a cell or tissue of interest. In some embodiments, the genome editing agent comprises a ribonucleoprotein (RNP) complex comprising a CRISPR Cas protein and a guide RNA.