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Analysis

1.WO/2026/006213METHODS OF MAKING MODIFIED BRANCHED RNAS
WO 02.01.2026
Int.Class C07H 1/00
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
1Processes for the preparation of sugar derivatives
Appl.No PCT/US2025/034884 Applicant THE BROAD INSTITUTE, INC. Inventor WANG, Xiao
Disclosed herein are branched RNA oligonucleotides and methods of making the same. The branched RNA oligonucleotides may be made by using a brancher phosphoramidite. Also provided are compositions comprising one or more of the branched RNA oligonucleotides provided herein, and methods of using said compositions for therapeutic applications.
2.WO/2026/000033DOUBLE STRANDED RNA MOLECULES WITH A TO G SUBSTITUTIONS
WO 02.01.2026
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/AU2025/050684 Applicant COMMONWEALTH SCIENTIFIC AND INDUSTRIAL RESEARCH ORGANISATION Inventor ZHONG, Chengcheng
The present invention relates to double-stranded RNA molecules having A to G substitutions, precursor RNA molecules thereof, and their use in modifying cells such as for gene silencing.
3.WO/2026/006483SNCA VECTORS AND ENGINEERED GUIDE RNAS
WO 02.01.2026
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/US2025/035296 Applicant SHAPE THERAPEUTICS INC. Inventor BURLEIGH, Stephen
Disclosed herein are plasmids encoding engineered guide RNAs and compositions comprising the same. Also disclosed herein are AAVs containing engineered guide RNAs. Also disclosed herein are methods of treating diseases or conditions in a subject by administering engineered guide RNAs or pharmaceutical compositions described herein.
4.WO/2026/006558COMPOSITIONS AND METHODS FOR INCREASING CXCL9 AND CXCL10 GENE EXPRESSION
WO 02.01.2026
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/US2025/035432 Applicant FLAGSHIP LABS 114, INC. Inventor O'DONNELL, Charles, W.
The present invention provides agents and compositions for increasing expression of a CXCL9 and/or CXCL10 gene by targeting expression control region associated with CXCL9 and/or CXCL10 gene and methods of use thereof for treating a disease or disorder associated with CXCL9 and/or CXCL10, e.g., cancer.
5.WO/2026/006528ENGINEERING SELECTIVE RESISTANCE TO TARGETED THERAPIES
WO 02.01.2026
Int.Class A61K 35/545
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
48Reproductive organs
54Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
545Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
Appl.No PCT/US2025/035376 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor GILL, Saar
The present disclosure provides modified stem cells, in particular hematopoietic stem cells engineered to express a variant of a B-cell lymphoma 2 (Bcl-2) family protein, wherein the variant confers resistance to a cytotoxic inhibitor of the Bcl-2 family protein. Also provided are methods and uses of the modified cells for therapeutic applications.
6.WO/2026/003754NOVEL REVERSE TRANSCRIPTASES AND USES THEREOF
WO 02.01.2026
Int.Class C12N 15/10
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
10Processes for the isolation, preparation or purification of DNA or RNA
Appl.No PCT/IB2025/056459 Applicant LIFE EDIT THERAPEUTICS, INC. Inventor KELSO, Drew
Compositions and methods for binding to a target sequence of interest are provided. The compositions find use in editing a target sequence of interest via polymerase editing. Compositions comprise: reverse transcriptases (RTs) (and polynucleotides encoding the same); DNA-binding polypeptides (and polynucleotides encoding the same); polymerase editor (PE)-enhancing polypeptides (and polynucleotides encoding the same); fusion proteins comprising an RT and a heterologous polypeptide, where the heterologous polypeptide can include a DNA-binding polypeptide and a PE-enhancing polypeptide; polymerase editors (PEs) comprising an RT and a DNA-binding polypeptide, optionally further including a PE-enhancing polypeptide (or one or more polynucleotides encoding any or all components of the PE); and PE systems comprising the PEs and one or more polymerase editing guide RNAs (PEgRNAs) (or polynucleotide(s) encoding the same). Vectors and host cells comprising the polynucleotides are also provided.
7.WO/2026/006334DUX4 VECTORS AND ENGINEERED GUIDE RNAS
WO 02.01.2026
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/US2025/035079 Applicant SHAPE THERAPEUTICS INC. Inventor BURLEIGH, Stephen
Disclosed herein are plasmids encoding engineered guide RNAs and compositions comprising the same. Also disclosed herein are AAVs containing engineered guide RNAs. Also disclosed herein are methods of treating diseases or conditions in a subject by administering engineered guide RNAs or pharmaceutical compositions described herein.
8.WO/2026/006604FEEDER CELL REPLACEMENT
WO 02.01.2026
Int.Class C12N 5/0783
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
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07Animal cells or tissues
071Vertebrate cells or tissues, e.g. human cells or tissues
078Cells from blood or from the immune system
0783T cells; NK cells; Progenitors of T or NK cells
Appl.No PCT/US2025/035496 Applicant LYELL IMMUNOPHARMA, INC. Inventor CHEUNG, Alexander
The present disclosure provides methods of expanding immune cells ex vivo or in vitro comprising culturing the immune cells in a medium comprising a feeder cell replacement, e.g., a PCS, comprising a surface-exposed CD3 agonist. In some aspects, the immune cells comprise T cells, TILs, NK cells, Tregs, or any combination thereof. In some aspects, a population of expanded TILs are administered to a subject in need thereof.
9.WO/2026/006493REAGENTS AND METHODS FOR AMPLIFICATION REACTIONS
WO 02.01.2026
Int.Class C12Q 1/6848
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
6844Nucleic acid amplification reactions
6848characterised by the means for preventing contamination or increasing the specificity or sensitivity of an amplification reaction
Appl.No PCT/US2025/035307 Applicant ECOLOGENIX, LLC. Inventor WANGH, Lawrence Joel
This disclosure relates to method for preparing a mixture of at least one Hot-Start-Like Reagent and a DNA polymerase prior to assembly of a complete master-mix for amplification of double-stranded amplicons in a closed-reaction vessel, the method comprising combining at least one Hot-Start-Like (HSL) Reagent and at least one DNA polymerase at an operative ratio of units of said reagent to units of said DNA polymerase. The HSL Reagent being comprised of one or two non-amplifiable single-stranded oligonucleotides and said operative ratio being determined by quantitative end-point melt-curve analysis of a fluorescent dye that binds to double-stranded DNA molecules in said closed-reaction vessel.
10.WO/2026/0017082,5-DIKETOPIPERAZINE COMPOUND HAVING ANTI-TUMOR EFFECT AND USE THEREOF IN PREPARATION OF DRUG FOR TREATING AND/OR PREVENTING NEUTROPENIA
WO 02.01.2026
Int.Class C07D 403/06
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
403Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/167
02containing two hetero rings
06linked by a carbon chain containing only aliphatic carbon atoms
Appl.No PCT/CN2025/100804 Applicant QINGDAO HAIHE BIOTECHNOLOGY CO., LTD Inventor LI, Wenbao
Disclosed in the present invention are a 2,5-diketopiperazine compound having an anti-tumor effect and use thereof in the preparation of a drug for treating and/or preventing neutropenia. The 2,5-diketopiperazine compound has a novel structure, has good activity and water solubility, and can overcome the defects that the poor water solubility of plinabulin is not conducive to drug preparation and leads to difficulty in clinical administration. The 2,5-diketopiperazine compound represented by formula (I) of the present invention or a pharmaceutically acceptable salt thereof can be used for treating and/or preventing neutropenia and improving toxic and side effects in clinical tumor treatment, and meanwhile, has an anti-tumor effect. Also disclosed in the present invention is use of such compounds in combination with a granulocyte colony-stimulating factor (G-CSF) biological formulation in a drug for treating and/or preventing neutropenia.