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IC:C12N15/00

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1 / 97,607
1.825771RNAI AGENTS FOR INHIBITING EXPRESSION OF ATAXIN-2 (ATXN2), COMPOSITIONS THEREOF, AND METHODS OF USE
NZ - 31.10.2025
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 825771 Applicant ARROWHEAD PHARMACEUTICALS, INC. Inventor PEI, Tao
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Ataxin-2 (ATXN2) gene. The ATXN2 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an ATXN2 gene. Pharmaceutical compositions that include one or more ATXN2 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described ATXN2 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of ATXN2 gene expression and a reduction in ATXN2 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including spinocerebellar ataxia type 2 (SCA2) or amyotrophic lateral sclerosis (ALS.)
2.826078ANTIBODY SPECIFICALLY BINDING TO CLAUDIN18.2 AND PREPARATION METHOD THEREFOR AND USE THEREOF
NZ - 31.10.2025
Int.Class C07K 16/28
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
28against receptors, cell surface antigens or cell surface determinants
 Appl.No 826078 Applicant GRACELL BIOTECHNOLOGIES (SHANGHAI) CO., LTD. Inventor DONG, Qi
Provided is an anti-Claudin18.2 nanobody or a binding fragment thereof. The antibody has good specificity, and immune effector cells targeting Claudin18.2 which are prepared from the antibody show a good therapeutic effect in treating or ameliorating diseases having positive expression of Claudin18.2.
3.825945FORMULATIONS FOR OLIGONUCLEOTIDE DELIVERY
NZ - 31.10.2025
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 825945 Applicant ALNYLAM PHARMACEUTICALS, INC. Inventor PHUNG, Andy
The disclosure relates to compositions and methods for delivery of nucleic acid therapeutics, particularly iRNA agents to target locations and genes residing in the central nervous system (CNS) of a subject.
4.826100ADENO-ASSOCIATED VIRUS COMPLEX WITH IMPROVED EXPRESSION OF RUNX3 GENE AND USES FOR PREVENTING OR TREATING KRAS MUTATED LUNG CANCER
NZ - 31.10.2025
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 826100 Applicant GENECRAFT INC. Inventor BAE, Suk Chul
Provided is an adeno-associated virus (AAV) complex for expression of an RUNX3 gene including an asymmetrically modified inverted terminal repeat (ITR). The AAV complex has asymmetric ITRs in which one of the two ITRs is modified, thereby increasing self-replication efficiency in host cells and increasing expression efficiency of a delivered gene, and therefore, compared to existing AAV complexes, the AAV complex has an advantage of improved productivity and gene expression efficiency.
5.826170ZINC FINGER PROTEIN TRANSCRIPTION FACTORS FOR REPRESSING NAV1.7 EXPRESSION
NZ - 31.10.2025
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 826170 Applicant SANGAMO THERAPEUTICS, INC. Inventor SAMIE, Mohammad
The present disclosure provides zinc finger fusion proteins that inhibit expression of Nav1.7 in the nervous system, and methods of using the proteins to treat pain disorders.
6.826372COMPOSITIONS FOR AND METHODS OF ENGINEERING THE TRANSCRIPTOME
NZ - 31.10.2025
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 826372 Applicant Duke University Inventor ASOKAN, Aravind
Disclosed herein are CRISPR-free compositions for and methods of generating chimeric RNA molecules via trans-splicing and methods of treating and/or preventing a genetic disease or disorder using chimeric RNA molecules generated via trans-splicing.
7.826371COMPOSITIONS COMPRISING BRAIN-TROPIC AAVS AND METHODS OF USE THEREOF
NZ - 31.10.2025
Int.Class C12N 15/861
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
861Adenoviral vectors
 Appl.No 826371 Applicant Duke University Inventor ASOKAN, Aravind
Recombinant adeno-associated virus (rAAV) vectors are leading gene delivery platforms, and several rAAV-mediated therapies have recently been approved for clinical use. Disclosed herein are compositions comprising AAV capsid proteins demonstrating improved tropism and improved transduction efficiency for one or more cells in the human CNS, and methods of using AAV particles and AAV vectors comprising these AAV capsid proteins to efficiently deliver a gene of interest or a transgene to target cells or tissues and to treat a subject in need thereof.
8.826351MIVELSIRAN COMPOSITIONS AND METHODS OF USE THEREOF
NZ - 31.10.2025
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 826351 Applicant ALNYLAM PHARMACEUTICALS, INC. Inventor BOSTWICK, Bret Lee
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as Alzheimer's disease (e.g., early onset Alzheimer's disease), using such dsRNAi agents and compositions.
9.825755ANTIBODY, NUCLEIC ACID, CELL AND DRUG
NZ - 31.10.2025
Int.Class C07K 16/28
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
28against receptors, cell surface antigens or cell surface determinants
 Appl.No 825755 Applicant NB HEALTH LABORATORY CO., LTD. Inventor TAKASAKI Jun
The present invention provides an antibody that specifically binds to an extracellular domain of human CCR7 and has a heavy chain CDR1 including an amino acid sequence represented by SEQ ID NO: 25, a heavy chain CDR2 including an amino acid sequence represented by SEQ ID NO: 27, a heavy chain CDR3 including an amino acid sequence represented by SEQ ID NO: 29, a light chain CDR1 including an amino acid sequence represented by SEQ ID NO: 35, a light chain CDR2 including an amino acid sequence represented by SEQ ID NO: 37, and a light chain CDR3 including an amino acid sequence represented by SEQ ID NO: 39.
10.825693METHOD FOR TRANSFORMING BACTERIUM BELONGING TO GENUS BIFIDOBACTERIUM
NZ - 31.10.2025
Int.Class C12N 15/63
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
 Appl.No 825693 Applicant KABUSHIKI KAISHA YAKULT HONSHA Inventor WATANABE, Yohei
Provided is a method for efficiently transforming a bacterium belonging to the genus Bifidobacterium. The method for transforming a bacterium belonging to the genus Bifidobacterium comprises bringing the bacterium belonging to the genus Bifidobacterium as a recipient into contact with a bacterium or linear DNA as a donor containing a DNA to be introduced into the recipient, and performing stationary culture, wherein the culture medium used in the stationary culture has a pH of 6.0 or more and less than 10.0 and satisfies the following requirement: when the pH is 6.0 or more and less than 8.5, the content x (mass%) of sugars that can be assimilated by the bacterium belonging to the genus Bifidobacterium as the recipient is in the range of 0 < x ≤ 0.2×(pH)-1.2; and when the pH is 8.5 or more and less than 10.0, x is in the range of 0.2×(pH)-1.7 < x ≤ 0.2×(pH)-1.2.
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