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Analysis

1.WO/2026/087678TREATMENT FOR RENAL DISORDERS
WO 30.04.2026
Int.Class A61K 31/7125
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
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
Appl.No PCT/EP2025/080654 Applicant SAPREME TECHNOLOGIES B.V. Inventor BUJNY, Miriam Verena
The invention relates to the fields of therapy and drug delivery. It is based on an unexpected finding that a combination of an oligonucleotide-based medicament with a saponin component comprising a penta-cyclic triterpene saponin of a 12,13-dehydrooleanane aglycone core, not only does not appear to be associated with oligonucleotide medicament-induced nephrotoxic effects after systemic administration in vivo, but also that it allows the oligonucleotide-based medicament to enter and act on its nucleic acid target inside of the kidney cells instead of remaining unproductively trapped in renal subcellular compartments or being eliminated into the urine via the renal glomerular filtration system. In line with this finding, herein disclosed are therapeutic combinations, compositions, and formulations, as well as methods of treatment involving their administration, which comprise an oligonucleotide-based medicament that acts on an intracellular nucleic acid target in the kidney cells, and a saponin component that enables the effective release of said medicament inside of the kidney cells, thus allowing its use at a much lower and safer dose. The provision of the presented herein therapeutic combinations enables effective modulation of gene expression in the kidney cells and, therefore, it opens not only new treatment options for kidney diseases, but also for diseases of other organs which affect the kidneys and/or which are affected by inefficient or impaired kidney function.
2.WO/2026/088212RECOMBINANT DNA MOLECULES AND CONSTRUCTS ENCODING Β- GLUCOCEREBROSIDASE (GCASE) ENZYME, AND METHODS THEREOF
WO 30.04.2026
Int.Class C12N 9/24
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.)
24acting on glycosyl compounds (3.2)
Appl.No PCT/IN2025/051693 Applicant MICRO CRISPR PVT. LTD. Inventor CHOKSI, Arpankumar Ajaybhai
The present invention relates to recombinant DNA molecule encoding β-glucocerebrosidase (GCase) enzyme. The present invention also relates to recombinant DNA construct, recombinant vector, recombinant host cell, mRNA molecule, delivery system, pharmaceutical compositions and method of treating or managing symptoms of Gaucher disease in a subject.
3.WO/2026/085245METHOD FOR SEQUENTIAL PREPARATION OF NUCLEIC ACID LIBRARIES
WO 23.04.2026
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/US2025/051110 Applicant NATERA, INC. Inventor CONSTANTIN, Tudor Pompiliu
The present disclosure provides methods, systems and compositions for amplifying and sequencing nucleic acids, including sequential preparation of a plurality of DNA libraries from the same original nucleic acids. Such methods may comprise extracting nucleic acids from a sample; ligating an adaptor having a capture moiety to the extracted nucleic acids; preparing a first library of amplified DNA by performing a first amplification on the adapted nucleic acids and separating the adapted nucleic acids from the first amplified DNA; sequencing at least a portion of the first library of amplified DNA and generating a first set of sequence reads; preparing a second library of amplified DNA by performing a second amplification on the adapted nucleic acids and separating the adapted nucleic acids from the second amplified DNA; and sequencing at least a portion of the second library of amplified DNA and generating a second set of sequence reads.
4.WO/2026/085427METHODS OF SELECTIVELY EXPRESSING PRODUCTS IN EBV-INFECTED CELLS, AND COMPOSITIONS FOR PRACTICING THE SAME
WO 23.04.2026
Int.Class C12N 15/90
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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
Appl.No PCT/US2025/051445 Applicant THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY Inventor LANZ, Tobias V.
The present disclosure provides a method for selectively expressing a product in an EBV-infected cell, the method comprising: contacting an EBV-infected cell with a sensor RNA comprising: (i) a first nucleotide sequence comprising a sensor nucleotide sequence that hybridizes to a target RNA present in the EBV-infected cell, wherein the sensor nucleotide sequence comprises one or more editable codons, (ii) a second nucleotide sequence encoding a first cleavage domain, and (iii) a third nucleotide sequence encoding a product.
5.WO/2026/085157ARRHYTHMOGENIC CARDIOMYOPATHY LIPID PATHOGENESIS
WO 23.04.2026
Int.Class A61K 31/164
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
31Medicinal preparations containing organic active ingredients
16Amides, e.g. hydroxamic acids
164of a carboxylic acid with an aminoalcohol, e.g. ceramides
Appl.No PCT/US2025/050974 Applicant THE JOHNS HOPKINS UNIVERSITY Inventor CHATTERJEE, Subroto
Arrhythmogenic cardiomyopathy (ACM) is a familial heart disease characterized by progressive myocardial fibrofatty infiltration. Compositions in the treatment of fibrofatty accumulation and its pathogenesis in ACM include activators of glycosphingolipid (GSL) glycosyltransferase, β-1,4-galactosyltransferase- V (β-1,4-GalT-V).
6.WO/2026/085428IONIZABLE AMPHIPHILIC JANUS DENDRIMERS, DELIVERY SYSTEMS COMPRISING THE SAME, AND METHODS OF USE THEREOF
WO 23.04.2026
Int.Class A61K 39/385
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
385Haptens or antigens, bound to carriers
Appl.No PCT/US2025/051447 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor PERCEC, Virgil
The invention relates to ionizable amphiphilic Janus dendrimers with different linker lengths. In some embodiments, the different linkers enable delivery of the agent to a specific target of interest (e.g., kidney, heart, lung, liver, lymph nodes, spleen, or any combination thereof). In some embodiments, the invention relates to nanoparticles comprising said ionizable amphiphilic Janus dendrimers. In some embodiments, the invention relates to compositions comprising said ionizable amphiphilic Janus dendrimers. In some aspects, the present invention also relates to methods of inducing an adaptive immune response in a subject comprising administering to the subject an effective amount of said nanoparticle or said composition.
7.WO/2026/082268METHODS FOR PROCESSING NUCLEIC ACID-CONTAINING SAMPLES
WO 23.04.2026
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/EP2024/078924 Applicant NOSCENDO GMBH Inventor STEVENS, Philip
The present disclosure relates to methods for analyzing nucleic acid molecules present in a sample, wherein the DNA molecules present in the sample or derived from nucleic acids in the sample are contacted with a terminal deoxynucleotidyl transferase to add nucleotides to the 3' end of the DNA molecules in a step-wise fashion and then identifying the contacted DNA molecules by their sequence. The method can be advantageously used in methods of diagnosing diseases caused by pathogenic organisms, as well as detecting the presence of pathogenic organisms in the environment.
8.WO/2026/082899RNA FOR PREVENTING OR TREATING TUBERCULOSIS
WO 23.04.2026
Int.Class A61K 39/04
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
02Bacterial antigens
04Mycobacterium, e.g. Mycobacterium tuberculosis
Appl.No PCT/EP2025/079965 Applicant BIONTECH SE Inventor ATES, Louis Simon
The present disclosure provides agents and methods for preventing or treating tuberculosis using RNA. The RNA encoding variants of antigens of Mycobacterium tuberculosis ox of fragments thereof is formulated and administered in a way that the variants are produced by cells of a subject, in particular after intramuscular or intravenous administration of the RNA.
9.WO/2026/082659METHODS FOR PROCESSING NUCLEIC ACID-CONTAINING SAMPLES
WO 23.04.2026
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/EP2025/079481 Applicant NOSCENDO GMBH Inventor STEVENS, Philip
The present disclosure relates to methods for analyzing nucleic acid molecules present in an obtained sample, such as cell-free DNA molecules, wherein the nucleic acid molecules present in the sample are contacted in the sample with a terminal deoxynucleotidyl transferase to add nucleotides to the 3 ' end of the DNA molecules in a step-wise fashion and then identifying the contacted DNA molecules comprising the added nucleotides by their sequence. The method can be advantageously used in methods of diagnosing diseases caused by pathogenic organisms, as well as detecting the presence of pathogenic organisms in the environment.
10.WO/2026/084761CONFORMATIONALLY TUNABLE LIPID COMPOSITIONS AND THERAPEUTIC USES THEREOF
WO 23.04.2026
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 PCT/US2025/030596 Applicant FLAGSHIP PIONEERING INNOVATIONS VII, LLC Inventor BLAKE, Timothy, Ray
Disclosed are lipid nanoparticle formulations comprising a lipidoid as defined in the application. Also disclosed are nanoparticle compositions comprising a lipidoid of the invention that are capable of delivering a therapeutic agent. The application also discloses a pharmaceutical composition comprising a lipidoid composition of the invention.