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1.WO/2026/090278SYSTEMS, COMPOSITIONS, AND METHODS FOR CONVERTING A CELL TO A RETINAL PHOTORECEPTOR INDUCED NEURON (PR-IN)
WO 30.04.2026
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/US2025/052052 Applicant THE REGENTS OF THE UNIVERSITY OF CALIFORNIA Inventor WAHLIN, Karl
Provided herein are recombinant expression systems for converting a cell to a retinal photoreceptor induced neuron (PR-iN) that include (a) a nucleic acid sequence encoding a gene cassette comprising one or more of ASCL1, NEUROG2, OTX2, CRX, LHX4, NRL, NR2E3, NEUROD1, IKZF1, IKZF4, MEF2C, MEF2D, MEIS2, ONECUT1, KLF16, SALL3, ZIC3, POU2F1, POU2F2, RORA, RORB, RXRG, or THRB, and (b) a nucleic acid sequence encoding a porcine teschovirus 2A (p2A) peptide sequence. Additionally, the recombinant expression systems disclosed herein can be used to treat retinal degenerative disorders.
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/2026/085335BIDIRECTIONAL REP-CAP PLASMID FOR PRODUCTION OF AAV
WO 23.04.2026
Int.Class C12N 15/864
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
864Parvoviral vectors
Appl.No PCT/US2025/051266 Applicant FORGE BIOLOGICS, INC. Inventor PADEGIMAS, Linas
The present disclosure provides improved adenoviral plasmids for the production of recombinant adeno-associated viruses.
4.WO/2026/085440SERIAL VACCINATIONS USING MANNAN ADJUVANTS
WO 23.04.2026
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/US2025/051470 Applicant THE CHILDREN'S MEDICAL CENTER CORPORATION Inventor KARP, Jeffrey M.
Provided herein are serial vaccination methods to induce an immune response against a pathogen (e.g., Beta coronavirus) comprising administering a first composition comprising a nucleic acid encoding a first antigen (e.g., a first Beta coronavirus antigen) and administering a second composition comprising a nucleic acid encoding an second antigen (e.g., a second Beta coronavirus antigen), and wherein the first composition and/or the second composition comprises an adjuvantation system comprising a mannan (e.g., a plant mannan or a fungal mannan).
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/2026/085427METHODS 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.
7.WO/2026/084768HELPER PROTEINS FOR INCREASED SECRETION
WO 23.04.2026
Int.Class A61K 38/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
38Medicinal preparations containing peptides
Appl.No PCT/US2025/037270 Applicant HELIX NANOTECHNOLOGIES INC Inventor GILL, Taylor
Disclosed herein is a nucleic acid expression system comprising (i) a polynucleotide comprising a payload sequence, and (ii) at least one polynucleotide that encodes one or more helper polypeptides. Methods of making and using the disclosed nucleotide expression system, polypeptides, and compositions are also provided.
8.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.
9.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.
10.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.