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1.20250327070COMPOSITIONS AND METHODS FOR INHIBITING COMPLEMENT FACTOR B
US 23.10.2025
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 18292187 Applicant ASTRAZENECA IRELAND LIMITED Inventor Melissa Lasaro

Described herein are oligonucleotides (e.g., RNAi oligonucleotides) containing sense and antisense strands for targeting complement factor B (CFB) mRNA. The RNAi oligonucleotide may be used to inhibit CFB expression, levels, and/or activity in a cell. Also, described herein are methods for using an oligonucleotide (e.g., an RNAi oligonucleotide) for the prophylaxis or treatment of a disease, disorder, or condition mediated by complement pathway activation or dysregulation.

2.20250320519SYNTHETIC CIRCULAR RNA COMPOSITIONS AND METHODS OF USE THEREOF
US 16.10.2025
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 18868935 Applicant SHANGHAI CIRCODE BIOMED CO., LTD. Inventor Yun YANG

The present disclosure relates to compositions, methods, processes, kits and devices for the selection, design, preparation, manufacture, formulation, and/or use of a polynucleotide having an Internal Ribosome Entry Site (IRES) sequence, an IRES-like sequence or a combination thereof. In particular, the present disclosure relates to compositions, methods, processes, kits and devices for the selection, design, preparation, manufacture, formulation, and/or use of a circular polynucleotide (e.g., a circular RNA). The present disclosure also relates to a method of improving expression, functional stability, immunogenicity, ease of manufacturing and/or half-life of a therapeutic product encoded by the circular RNA.

3.20250319036BISPHOSPHONATE LIPIDS, LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME, AND METHODS OF USE THEREOF FOR TARGETED DELIVERY
US 16.10.2025
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 18865098 Applicant The Trustees of the University of Pennsylvania Inventor Michael J. Mitchell

Described herein, in some aspects, are bisphosphonate lipid compounds, lipid nanoparticles (LNPs) thereof, and methods of use thereof. In various embodiments, the LNP selectively targets a cell of interest (e.g., a bone cell and/or bone marrow cell, such as a stem cell, stroma cell, osteoblast, osteocyte, osteoclast, bone lining cell, local mesenchymal cell, progenitor cell, mononuclear blood-borne precursor cell, B cell, endothelial cell, granulocytes, T cell, monocytic lineage, B cell lineage, monocytes, cancer cell, tumor cell, tumor cell that metastasizes to bone, blood cancer cell, and multiple myeloma cell, inter alia). In other aspects, the present disclosure relates to methods for in vivo delivery of therapeutic agents to prevent or treat diseases, disorders, or conditions using the LNP compositions of the disclosure.

4.20250320491CRISPR-CPF1-RELATED METHODS, COMPOSITIONS AND COMPONENTS FOR CANCER IMMUNOTHERAPY
US 16.10.2025
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 19200416 Applicant EDITAS MEDICINE, INC. Inventor Gordon Grant Welstead

CRISPR/Cpf1-related compositions and methods for treatment of cancer.

5.20250319160USE OF IFN-LAMBDA MRNA FOR TREATING VIRAL INFECTIONS
US 16.10.2025
Int.Class A61K 38/21
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
17from animals; from humans
19Cytokines; Lymphokines; Interferons
21Interferons
Appl.No 19248327 Applicant ethris GmbH Inventor Carsten Rudolph

The present invention relates to pharmaceutical compositions comprising an mRNA encoding an IFN-λ polypeptide for use in treating a viral-induced disorder, preferably a viral-induced respiratory disorder, such as COVID-19.

6.WO/2025/212831METHODS OF INDUCING AN ANTI-HSV B-CELL IMMUNE RESPONSE
WO 09.10.2025
Int.Class A61K 39/245
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
245Herpetoviridae, e.g. herpes simplex virus
Appl.No PCT/US2025/022857 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor FRIEDMAN, Harvey
The present disclosure provides a combination comprising ribonucleotide sequences that encode herpes simplex virus (HSV) glycoproteins, including those involved in virus entry and immune evasion and related methods of treating, suppressing, inhibiting, or preventing an HSV infection. The present disclosure further provides a related formulation comprising ribonucleotide sequences that encode HSV glycoproteins, wherein one or more of the ribonucleotides are fully or partially encapsulated within a lipid nanoparticle, wherein the lipid nanoparticle comprises (a) a polymer-conjugated lipid; (b) a cationic lipid; and (c) one or more neutral lipids. Finally, the present disclosure provides methods of inducing an HSV glycoprotein-specific B-cell response in a subject comprising administering to the subject a composition comprising the HSV glycoprotein.
7.WO/2025/212851mRNA COMPOSITIONS AND USES THEREOF IN VARICELLA ZOSTER VIRUS VACCINES
WO 09.10.2025
Int.Class A61K 39/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
Appl.No PCT/US2025/022899 Applicant ORBITAL THERAPEUTICS, INC. Inventor BESIN, Gilles
The present disclosure relates to mRNA sequences encoding an antigen from varicella zoster virus. The mRNA sequences described herein are used as vaccines and compositions for preventing or treating diseases resulting from varicella zoster virus, including shingles. Methods for formulating and preparing the vaccines are also described.
8.20250312376COMPOSITIONS 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.

9.2025148442RNAによる単純ヘルペス ウイルスI型およびその他の関連するヒトヘルペス ウイルスの根絶
JP 07.10.2025
Int.Class A61P 31/22
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
12Antivirals
20for DNA viruses
22for herpes viruses
Appl.No 2025116242 Applicant テンプル ユニヴァーシティ オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション Inventor ハリリ、カメル
【課題】ヘルペスウイルスの感染力を阻害するための組成物および方法を提供する。
【解決手段】組成物は、a)CRISPR関連(Cas)ペプチドまたはCasペプチドをコードする単離された核酸を含む。b)単離されたガイド核酸、またはガイド核酸をコードする単離された核酸であって、ガイド核酸は、ヘルペスウイルスゲノム中の標的配列に実質的に相補的なヌクレオチド配列を含む。
【選択図】図1A-1
10.20250304994Circular 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.