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1 / 476
1.WO/2026/085157ARRHYTHMOGENIC CARDIOMYOPATHY LIPID PATHOGENESIS
WO - 23.04.2026
Int.Class A61K 31/164
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
31Medicinal preparations containing organic active ingredients
16Amides, e.g. hydroxamic acids
164of a carboxylic acid with an aminoalcohol, e.g. ceramides
 Appl.No PCT/US2025/050974 Applicant THE JOHNS HOPKINS UNIVERSITY Inventor CHATTERJEE, Subroto
Arrhythmogenic cardiomyopathy (ACM) is a familial heart disease characterized by progressive myocardial fibrofatty infiltration. Compositions in the treatment of fibrofatty accumulation and its pathogenesis in ACM include activators of glycosphingolipid (GSL) glycosyltransferase, β-1,4-galactosyltransferase- V (β-1,4-GalT-V).
2.WO/2026/085428IONIZABLE AMPHIPHILIC JANUS DENDRIMERS, DELIVERY SYSTEMS COMPRISING THE SAME, AND METHODS OF USE THEREOF
WO - 23.04.2026
Int.Class A61K 39/385
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
385Haptens or antigens, bound to carriers
 Appl.No PCT/US2025/051447 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor PERCEC, Virgil
The invention relates to ionizable amphiphilic Janus dendrimers with different linker lengths. In some embodiments, the different linkers enable delivery of the agent to a specific target of interest (e.g., kidney, heart, lung, liver, lymph nodes, spleen, or any combination thereof). In some embodiments, the invention relates to nanoparticles comprising said ionizable amphiphilic Janus dendrimers. In some embodiments, the invention relates to compositions comprising said ionizable amphiphilic Janus dendrimers. In some aspects, the present invention also relates to methods of inducing an adaptive immune response in a subject comprising administering to the subject an effective amount of said nanoparticle or said composition.
3.WO/2026/085452OPTIMIZED FORMULATION OF IONIZABLE AMPHIPHILIC JANUS DENDRIMER DELIVERY SYSTEMS, AND METHODS OF USE THEREOF
WO - 23.04.2026
Int.Class C07D 295/15
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
295Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
04with substituted hydrocarbon radicals attached to ring nitrogen atoms
14substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
145with the ring nitrogen atoms and the carbon atoms with three bonds to hetero atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
15to an acyclic saturated chain
 Appl.No PCT/US2025/051492 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor ATOCHINA-VASSERMAN, Elena
Nanoparticles comprising at least one amphiphilic Janus dendrimer are described as well as methods of delivering an agent to a target of interest (e.g., lung, liver, lymph nodes, and spleen) using said nanoparticle and methods of inducing an adaptive immune response in a subject comprising administering to the subject an effective amount of a composition comprising at least one nucleoside-modified RNA encoding at least one antigen and at least one amphiphilic Janus dendrimer.
4.WO/2026/080920MRNA VACCINE FOR PEANUT ALLERGY TREATMENT
WO - 16.04.2026
Int.Class A61K 39/35
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
35Allergens
 Appl.No PCT/US2025/050635 Applicant UKKO INC. Inventor OFRAN, Yanay
Disclosed herein are lipid nanoparticles comprising messenger RNA (mRNA) molecules encoding deepitoped Ara h 1 and/or de-epitoped Ara h 2, vaccines comprising the nanoparticles and methods for inducing desensitization to peanuts and/or immunomodulation of a response to peanuts in a subject allergic to peanuts.
5.WO/2026/080865ENGINEERED ADENYLYL-SULFATE KINASE VARIANTS
WO - 16.04.2026
Int.Class C12N 9/12
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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
10Transferases (2.)
12transferring phosphorus containing groups, e.g. kinases (2.7)
 Appl.No PCT/US2025/050530 Applicant CODEXIS, INC. Inventor BERMUDEZ, Ericka
The present invention provides engineered adenylyl-sulfate kinase polypeptides capable of adding a phosphate group to the 3' position of the sugar of a natural or modified nucleoside diphosphate, as well as compositions and methods of utilizing these engineered polypeptides.
6.WO/2026/080581ANTITUMOR REAGENTS FOR INCREASING SENSITIVITY OF MALIGNANT TUMOR CELLS TO CHEMOTHERAPY AND IMMUNOTHERAPY
WO - 16.04.2026
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/US2025/050022 Applicant TAIPEI MEDICAL UNIVERSITY Inventor TSAI, Kun-Chih, Kelvin
The present disclosure relates to anti-tumor reagents for increasing the sensitivities of solid tumor cells to death stimuli induced by immunotherapy reagents, chemotherapy reagents, immune cell therapy, or radiation therapy. Said anti-tumor reagents suitable for use in the invention are selected from the group comprising a) a polynucleotide complementary to the transcript of the nuclear corepressor 2 gene with a nucleotide sequence shown in SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, or SEQ ID NO: 5 that can induce the sufficient and the specific degradation and/or reduce the amount of the transcript of the nuclear corepressor 2 gene, or a derivative thereof, or b) a polynucleotide with a nucleotide sequence which comprises a contiguous segment having at least 70%, at least 80%, or at least 90% sequence identity to the polynucleotide set forth in a) when optimally aligned, or a derivative thereof. A preferred embodiment includes the delivery of said polynucleotide to said solid tumor cells with a nanoparticle, such as lipid nanoparticle, liposome, micelle, virosome, nucleic acid complex, extracellular vesicle, and exosome.
7.2026064992β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物
JP - 14.04.2026
Int.Class C12N 15/09
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
 Appl.No 2025244943 Applicant エディタス・メディシン、インコーポレイテッド Inventor ジェニファー・リア・ゴリ
【課題】β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連組成物および方法を提供する。
【解決手段】ＨＢＧ１、ＨＢＧ２、並びにＨＢＧ１及びＨＢＧ２調節領域からなる群から選択される領域内に位置する標的ドメインに相補的な標的化ドメインを含むガイドＲＮＡ（ｇＲＮＡ）分子であって、前記標的化ドメインが特定のヌクレオチド配列を含む、ｇＲＮＡ分子が提供される。
【選択図】なし
8.WO/2026/0758204-1BB BINDING MOLECULES, NUCLEIC ACIDS ENCODING SAME, AND METHODS OF USE
WO - 09.04.2026
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/US2025/046895 Applicant MODERNATX, INC. Inventor FARRINGTON, Graham
Described herein are specific single domain antibodies and binding molecules comprising such single domain antibodies that specifically bind to 4-1BB (human, or human and cynomolgus 4-1BB), as well as nucleic acids encoding the same. Also provided are nucleic acids (e.g., mRNA) encoding such single domain antibodies and binding molecules and delivery vehicles (e.g., lipid nanoparticles) encapsulating said nucleic acids. The disclosure also features methods of using such antibodies, binding molecules, nucleic acids, and delivery vehicles encapsulating said nucleic acids to treat various diseases such as cancer.
9.WO/2026/075844METHODS AND COMPOSITIONS FOR SINGLE-STRANDED RNA PURIFICATION
WO - 09.04.2026
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 PCT/US2025/047503 Applicant LIFE TECHNOLOGIES CORPORATION Inventor POTTER, Robert
Methods and compositions for purifying a nucleic acid preparation. The methods include contacting a nucleic acid preparation including single-stranded ribonucleic acid (ssRNA) with a double-stranded ribonucleic acid binding protein (dsRBP) that specifically binds to double-stranded ribonucleic acid (dsRNA) and is optionally immobilized on a solid support before or after the dsRBP-dsRNA complex is formed to remove dsRNA from a mixed nucleic acid preparation.
10.2026510601ＡＡＶＳ１セーフハーバー部位でのノックイン戦略
JP - 08.04.2026
Int.Class C12N 15/12
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
12Genes encoding animal proteins
 Appl.No 2025555272 Applicant エメンドバイオ・インコーポレイテッド Inventor エマニュエル、ラフィ
配列番号１～２４１９５のいずれかに示す配列内の連続する１７～５０ヌクレオチドを有するガイド配列部分を含むＲＮＡ分子、並びにその組成物、方法及び用途。
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