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Analysis

1 / 188
1.WO/2025/250534T CELL TARGETS FOR TUBERCULOSIS VACCINES
WO - 04.12.2025
Int.Class A61K 39/04
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
02Bacterial antigens
04Mycobacterium, e.g. Mycobacterium tuberculosis
 Appl.No PCT/US2025/031027 Applicant THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY Inventor DAVIS, Mark M.
The Mtb protein Rv2140c, specific peptide epitopes derived, and polynucleotide sequences encoding such polypeptides are disclosed herein as a useful TB antigen for generating a protective immune response to development of tuberculosis. In some embodiments the Rv2140c polypeptide or polynucleotide is formulated as a vaccine. In some embodiments the Rv2140c vaccine is administered to a naïve recipient. In some embodiments the Rv2140c vaccine is administered as a booster to a previously vaccinated to exposed individual. In some embodiments the previous vaccination is a BCG vaccination.
2.WO/2025/250751CIRCULAR RNA COMPOSITIONS AND METHODS
WO - 04.12.2025
Int.Class A61P 35/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
 Appl.No PCT/US2025/031358 Applicant ORNA THERAPEUTICS, INC. Inventor EMMANUEL, Akinola
Circular RNA, along with related compositions, methods, and precursor RNA, are described herein. In some embodiments, the composition comprises a transfer vehicle and a circular RNA comprising a 3' self-spliced exon segment, a translation initiation element (TIE), an expression sequence encoding a chimeric antigen receptor (CAR), and a 5' self-spliced exon segment. In some embodiments, the transfer vehicle comprises an ionizable lipid disclosed herein. In some embodiments, the circular RNA and/or composition thereof has improved expression, functional stability, immunogenicity, ease of manufacturing, and/or half-life as compared to linear RNA. Also presented herein are methods and compositions for the manufacture and preparation of circularized RNAs, along with methods of administering said circular RNAs and related compositions to a subject in need thereof for treatment or prevention purposes.
3.WO/2025/250677COMPOSITIONS AND METHODS FOR CORRECTION OF TREX1 MUTATIONS
WO - 04.12.2025
Int.Class C12N 15/52
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
52Genes encoding for enzymes or proenzymes
 Appl.No PCT/US2025/031247 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor MINER, Jonathan
Compositions and methods for correction of RVCL-causing mutations in a TREX1 gene are provided.
4.WO/2025/240659ACTIVATABLE CONSTRUCTS, COMPOSITIONS AND METHODS
WO - 20.11.2025
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 PCT/US2025/029426 Applicant CYTOMX THERAPEUTICS, INC. Inventor FOX, Ellaine Anne Mariano
Activatable constructs and methods of making and treating using the same, comprising a first antigen binding domain (AB1) having specific binding affinity for a first antigen, wherein the AB1 comprises a heavy chain variable domain 1 (HVD1) and a light chain variable domain 1 (LVD1), wherein the HVD1 is covalently coupled directly or indirectly to a first masking moiety (MM1) via a first cleavable moiety (CM1), wherein the LVD1 is covalently coupled directly or indirectly to a second masking moiety (MM2) via a second cleavable moiety (CM2), a second antigen binding domain (AB2) having specific binding affinity for a second antigen, wherein the AB2 comprises a heavy chain variable domain 2 (HVD2) and a light chain variable domain 2 (LVD2), a first dimerization domain (DD1) and a second dimerization domain (DD2).
5.20250352583Treatment of osteoarthritis and/or rheumatoid arthritis with pro-chondrogenic and/or chondrocyte protective factors
US - 20.11.2025
Int.Class A61K 35/28
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
28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
 Appl.No 19272688 Applicant SmartCella Solutions AB Inventor Ran YANG

The present disclosure relates to mesenchymal stem cells (MSCs) for use in a method of treatment of arthritis. The MSCs are transfected with an mRNA construct encoding a pro-chondrogenic and/or chondrocyte protective factor. The pro-chondrogenic and/or chondrocyte protective factor can be WNT3a.

6.WO/2025/237620A COMPUTATIONAL METHOD FOR SMALL RNA BIOMARKER MODIFICATION CLASSIFICATION
WO - 20.11.2025
Int.Class G16B 20/20
GPHYSICS
16INFORMATION AND COMMUNICATION TECHNOLOGY SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
20ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
20Allele or variant detection, e.g. single nucleotide polymorphism detection
 Appl.No PCT/EP2025/060457 Applicant HUMMINGBIRD DIAGNOSTICS GMBH Inventor RAJAKUMAR, Timothy
The present invention relates to a method classifying target RNA modifications in a neural network system, to a method for training a neural network system that is configured to classify target RNA modifications, to a neural network system, to a computer system adapted to classify target RNA modifications, to a computer program and to a non-transitory readable medium.
7.2025170187Ｃ型肝炎ウイルスに対するヌクレオシド修飾ｍＲＮＡ－脂質ナノ粒子系統ワクチン
JP - 17.11.2025
Int.Class C07K 14/08
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005from viruses
08RNA viruses
 Appl.No 2025097034 Applicant ザ　トラスティーズ　オブ　ザ　ユニバーシティ　オブ　ペンシルバニア Inventor ドリュー　ワイスマン
【課題】本発明は、対象におけるC型肝炎ウイルス（HCV）に対する適応免疫応答を誘導するための組成物及び方法に関する。
【解決手段】いくつかの実施形態では、本発明は、HCV抗原、アジュバント、又はそれらの組み合わせをコードするヌクレオシド修飾核酸分子を含む組成物を提供する。例えば、幾つかの実施形態では、本組成物は、HCV抗原、アジュバント、又はそれらの組み合わせをコードするヌクレオシド修飾核酸分子を含むワクチンを含む。
【選択図】なし
8.2025169942ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP - 14.11.2025
Int.Class C12P 21/02
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
21Preparation of peptides or proteins
02having a known sequence of two or more amino acids, e.g. glutathione
 Appl.No 2025097708 Applicant モデルナティエックス　インコーポレイテッド Inventor バンセル、ステファヌ
【課題】細胞内翻訳の効果的な調節およびポリペプチドまたはその断片をコードする核酸の処理を取り巻く無数の障壁に対処する治療法を提供する。
【解決手段】ポリヌクレオチドは、（ａ）目的とするポリペプチドをコードするオープンリーディングフレームであって、修飾されたウリジン、シチジン、アデノシン、およびグアノシンのヌクレオチドからなり、前記修飾ウリジンが、Ｎ１－メチル－シュードウリジンである、オープンリーディングフレームと、（ｂ）５’－ＵＴＲと、（ｃ）少なくとも１つの５’キャップ構造と、（ｄ）３’－ＵＴＲと、（ｅ）結合ヌクレオシドの３’尾部配列とを含む。
【選択図】なし
9.2025537178環状ＲＮＡ組成物
JP - 14.11.2025
Int.Class C07K 19/00
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
19Hybrid peptides
 Appl.No 2025525776 Applicant オーナ　セラピューティクス，　インコーポレイテッド Inventor ベッカー，　エイミー　エム．
本明細書では、ＩＲＥＳと、結合分子をコードする少なくとも１つの発現配列とを含む環状ＲＮＡコンストラクト、その組成物、ならびにがん及び自己免疫疾患に対する処置を含む処置方法が提供される。特に、ＩＲＥＳと、ＣＤ１９結合体、ＨＥＲ２結合体、またはＢＣＭＡ結合体とを含む環状ＲＮＡが、任意選択で、送達媒体と配合されて提供される。また、ＩＲＥＳと、ＣＡＲコンストラクトをコードする少なくとも１つの発現配列とを含む前駆体ポリヌクレオチドも本明細書に記載される。
【選択図】なし
10.20250346927METHODS AND COMPOSITIONS FOR MODULATING A GENOME
US - 13.11.2025
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 19272775 Applicant Flagship Pioneering Innovations VI, LLC Inventor Anne Helen Bothmer

Methods and compositions for modulating a target genome are disclosed. This disclosure relates to novel compositions, systems and methods for altering a genome at one or more locations in a host cell, tissue or subject, in vivo or in vitro. In particular, the invention features compositions, systems and methods for inserting, altering, or deleting sequences of interest in a host genome.

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