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Analysis

1 / 252
1.20250312376COMPOSITIONS AND METHODS FOR MODULATING A GENOME IN T CELLS, INDUCED PLURIPOTENT STEM CELLS, AND RESPIRATORY EPITHELIAL CELLS
US - 09.10.2025
Int.Class A61K 35/17
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
14Blood; Artificial blood
17Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
 Appl.No 18860423 Applicant FLAGSHIP PIONEERING INNOVATIONS VI, LLC Inventor Barrett Ethan Steinberg

Methods and compositions for modulating a target genome are disclosed. For instance, gene modifying systems may be used to insert a heterologous object sequence (e.g., encoding a chimeric antigen receptor) into a target cell. The target cell may be, e.g., a T cell, induced pluripotent stem cell, or respiratory epithelial cell.

2.20250302925CONJUGATED CRISPR-CAS COMPLEXES
US - 02.10.2025
Int.Class A61K 38/46
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
 Appl.No 18833834 Applicant Synthego Corporation Inventor Travis MAURES

Provided herein are polynucleotides and CRISPR effector proteins configured to be covalently bound together in a CRISPR complex. The polynucleotides can be further modified to modulate the activity of the CRISPR complex. Modification of the polynucleotide and CRISPR effector protein can be used to improve the efficacy of target binding and/or cleavage.

3.20250304994Circular RNA Compositions and Methods
US - 02.10.2025
Int.Class C12N 15/85
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
 Appl.No 19234626 Applicant Orna Therapeutics, Inc. Inventor Robert Alexander Wesselhoeft

Circular RNA, along with related compositions and methods are described herein. In some embodiments, the inventive circular RNA comprises intron segments, spacers, an IRES, duplex forming regions, and an expression sequence. In some embodiments, circular RNA of the invention has improved expression, functional stability, immunogenicity, ease of manufacturing, and/or half-life when compared to linear RNA. In some embodiments, the disclosed methods and constructs result in improved translation when compared to existing RNA approaches. In some embodiments, the disclosed methods and constructs result in improved circularization efficiency, splicing efficiency, and/or purity when compared to existing RNA circularization approaches.

4.20250302987Targeted Lipid Nanoparticles
US - 02.10.2025
Int.Class A61K 47/69
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
69the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
 Appl.No 19083329 Applicant Zitra Medicines Pty Ltd Inventor Denzil Furtado

The present invention relates to engineered targeted lipid nanoparticles (LNPs) comprising a nucleic acid, and compositions thereof, wherein the LNPs or compositions are capable of traversing the blood brain barrier (BBB) and delivering nucleic acid cargoes to a target tissue or cell in the central nervous system. In one aspect, the invention relates to the treatment of a neurological disease or disorder with a LNP or composition of the invention.

5.20250305002RECRUITMENT IN TRANS OF GENE EDITING SYSTEM COMPONENTS
US - 02.10.2025
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 18690120 Applicant Flagship Pioneering Innovation VI, LLC Inventor Anne Helen Bothmer

The disclosure provides, e.g., compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell's genome at one or more locations in a DNA sequence in a cell, tissue, or subject.

6.20250302860DELIVERY OF RNA TO TRIGGER MULTIPLE IMMUNE PATHWAYS
US - 02.10.2025
Int.Class A61K 31/7088
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
 Appl.No 18954638 Applicant GLAXOSMITHKLINE BIOLOGICALS SA Inventor Andrew GEALL

RNA encoding an immunogen is co-delivered to non-immune cells as the site of delivery and also to immune cells which infiltrate the site of delivery. The responses of these two cell types to the same delivered RNA lead to two different effects, which interact to produce a strong immune response against the immunogen. The non-immune cells translate the RNA and express the immunogen. Infiltrating immune cells respond to the RNA by expressing type I interferons and pro-inflammatory cytokines which produce a local adjuvant effect which acts on the immunogen-expressing non-immune cells to upregulate major histocompatibility complex expression, thereby increasing presentation of the translated protein to T cells. The effects on the immune and non-immune cells can be achieved by a single delivery of a single RNA e.g., by a single injection.

7.1020250141840혈색소병증의 치료를 위한 조성물 및 방법
KR - 29.09.2025
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 1020257030429 Applicant 노파르티스 아게 Inventor 보이타노, 앤소니 에드워드
본 발명은 혈색소병증(hemoglobinopathies) 치료를 위한 게놈 편집 시스템, 시약 및 방법에 관한 것이다.
8.120712350用于药物用途的RNA制剂
CN - 26.09.2025
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 202480012929.8 Applicant 生物技术公司 Inventor 乌尔·沙欣
本公开内容涉及包含一个或更多个miRNA结合序列的RNA，其中所述一个或更多个miRNA结合序列与存在于其中不期望所述RNA表达的细胞中的miRNA结合。在施用之后，特别是在肌内或静脉内施用之后，所述RNA递送至细胞，使得由所述RNA编码的多肽在某些细胞中表达，同时抑制在其他细胞中的表达。在一些实施方案中，这样的细胞包含内皮细胞。本文中所述的RNA组合物允许通过所述RNA在对象中表达药物活性肽或多肽，同时降低或避免由药物活性肽或多肽在某些细胞或组织中的表达而导致的不期望作用的风险。
9.20250295746THERAPEUTIC RNA FOR LUNG CANCER
US - 25.09.2025
Int.Class A61K 39/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
 Appl.No 18699947 Applicant BIONTECH SE Inventor Ugur SAHIN

This disclosure relates to the field of RNA to treat lung cancer, in particular non-small-cell lung carcinoma (NSCLC). Lung cancer is the third most frequent malignancy in women and the second most frequent malignancy in men. NSCLC accounts for about 85% of all lung cancers. Disclosed herein are compositions, uses, and methods for treatment of lung cancers. Administration of therapeutic RNAs to a patient having lung cancer disclosed herein can reduce tumor size, prolong time to progressive disease, and/or protect against metastasis and/or recurrence of the tumor and ultimately extend survival time.

10.20250295744TREATMENT INVOLVING NON-IMMUNOGENIC RNA FOR ANTIGEN VACCINATION AND PD-1 AXIS BINDING ANTAGONISTS
US - 25.09.2025
Int.Class A61K 39/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
 Appl.No 18696518 Applicant BioNTech SE Inventor Ugur SAHIN

The present disclosure relates to methods and agents for antigen vaccination and inducing effective antigen-specific immune effector cell responses such as T cell responses. These methods and agents are, in particular, useful for the treatment of diseases characterized by diseased cells expressing an antigen the immune effector cells are directed to. In some embodiments, the present disclosure relates to methods comprising administering to a subject (i) non-immunogenic RNA encoding a peptide or polypeptide comprising an epitope for inducing an immune response against an antigen in the subject, i.e., non-immunogenic RNA encoding vaccine antigen; and (ii) a PD-1 axis binding antagonist such as an anti-PD-1 antibody and/or an anti-PD-L1 antibody.

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