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1 / 447
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/2026/082899RNA FOR PREVENTING OR TREATING TUBERCULOSIS
WO - 23.04.2026
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/EP2025/079965 Applicant BIONTECH SE Inventor ATES, Louis Simon
The present disclosure provides agents and methods for preventing or treating tuberculosis using RNA. The RNA encoding variants of antigens of Mycobacterium tuberculosis ox of fragments thereof is formulated and administered in a way that the variants are produced by cells of a subject, in particular after intramuscular or intravenous administration of the RNA.
4.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.
5.WO/2026/085437LYME DISEASE VACCINE AND METHOD OF USE THEREOF
WO - 23.04.2026
Int.Class A61K 39/02
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
02Bacterial antigens
 Appl.No PCT/US2025/051464 Applicant HEALTH RESEARCH, INC. Inventor LIN, Yi-Pin
Provided is an immunogenic composition for use in raising immunity against the agent of Lyme disease. The composition is based on variants of CspZ and comprises a polypeptide of SEQ ID NO: 1, except that (a) the amino acid whose position corresponds to amino acid 188 of SEQ ID NO: 1 is any amino acid other than tyrosine; and the amino acid whose position corresponds to amino acid 192 of SEQ ID NO: 1 is any amino acid other than tyrosine; and (b) one or both of the amino acids acid whose position corresponds to amino acid 164 of SEQ ID NO: 1 may be an amino acid other than inosine and an amino acid whose position corresponds to amino acid 168 of SEQ ID NO: 1 may be an amino acid other than cysteine. Also provided is a polynucleotide encoding any of the foregoing polypeptides and a method of vaccinating against Lyme disease comprising administering the polypeptide or RNA encoding the polypeptide to a subject.
6.WO/2026/078565MESSENGER RNA ENCODING CAS9 FOR USE IN GENOME-EDITING SYSTEMS
WO - 16.04.2026
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 PCT/IB2025/060141 Applicant CRISPR THERAPEUTICS AG Inventor WANG, Albert
The present disclosure provides improved mRNAs encoding a site-directed endonuclease for use in a CRISPR/Cas system. Also provided herein are delivery systems for use of the CRISPR/Cas system in methods of in vivo and ex vivo genome editing.
7.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.
8.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.
9.2026064992β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
10.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.
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