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CHEM:(HPHXOIULGYVAKW-IOSLPCCCSA-N)

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Analysis

1 / 217
1.WO/2026/090085TREATMENT OF CD33 RELATED DISEASES AND DISORDERS
WO - 30.04.2026
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/US2025/051727 Applicant EMPIRICO INC. Inventor GOTTESMAN, Omri
Disclosed herein are compositions comprising an oligonucleotide that targets CD33. The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating conditions associated with CD33 gene mutations that include providing an oligonucleotide that targets CD33 in a subject.
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/2026/085437LYME 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.
5.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.
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.
8.2026064992β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
9.2026511794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。
10.2026510866ミトコンドリアアミドキシム還元成分１（ＭＡＲＣ１）の発現を阻害するためのＲＮＡｉ剤、その医薬組成物、及び使用方法
JP - 10.04.2026
Int.Class A61K 31/713
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
713Double-stranded nucleic acids or oligonucleotides
 Appl.No 2025553762 Applicant アローヘッド　ファーマシューティカルズ　インコーポレイテッド Inventor ジョナサン　バン　ダイク
本開示は、ミトコンドリアアミドキシム還元成分1(MARC1)遺伝子発現を阻害することができるRNAi剤、例えば、二本鎖RNAi剤、に関する。MARC1 RNAi剤を包含する医薬組成物及びその使用方法もまた開示される。本明細書に開示されるMARC1 RNAi剤は、肝細胞への送達を容易にするために、N-アセチル-ガラクトサミンを含んでいるリガンドを含めた標的化リガンドにコンジュゲートされてもよい。インビボにおけるMARC1 RNAi剤の送達は、MARC1遺伝子発現の阻害をもたらす。RNAi剤は、非アルコール性脂肪性肝炎(NASH)、非アルコール性脂肪性肝疾患(NAFLD)、アルコール性脂肪性肝疾患、自己免疫性肝炎、肝線維症、肝硬変、高い血中コレステロール値、高トリグリセリド血症、肝臓疾患、及び/又は他のMARC1関連疾患を含めた、MARC1遺伝子発現によって部分的に媒介される疾患、障害、又は症状の治療方法に使用できる。
【選択図】なし
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