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1.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.
2.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.
3.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.
4.WO/2026/080612COMPOSITIONS AND METHODS FOR SMALL CIRCULAR RNA-BASED IMMUNOTHERAPIES
WO 16.04.2026
Int.Class A61K 39/215
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
215Coronaviridae, e.g. avian infectious bronchitis virus
Appl.No PCT/US2025/050083 Applicant VIRGINIA COMMONWEALTH UNIVERSITY Inventor ZHU, Guizhi
Provided herein are compositions and methods for inducing an immune response using small circular ribonucleic acid (circRNA) molecules. In particular, the compositions comprise a small circRNA encoding at least one antigen or immunogenic peptide fragment, wherein the composition is self-adjuvanted and configured to elicit a T-cell–mediated immune response.
5.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.
6.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.
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
8.2026511794免疫細胞阻害分子の発現のための活性化免疫細胞に標的化された脂質ベースのナノ粒子及びその使用
JP 14.04.2026
Int.Class A61P 37/06
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
37Drugs for immunological or allergic disorders
02Immunomodulators
06Immunosuppressants, e.g. drugs for graft rejection
Appl.No 2025557021 Applicant オーエスイー・イミュノセラピューティクス Inventor ニコラ・ポワリエ
本発明は、活性化免疫細胞表面に発現される標的に特異的に結合することができる抗原結合ドメインと、前記活性化免疫細胞の活性阻害タンパク質をコードする1つ又は幾つかのmRNA分子とを含む、脂質ベースのナノ粒子、及びその使用に関する。
9.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.
10.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.