Absstract of: WO2026142373A1
The present invention relates to a novel compound having a targeting site introduced through a biodegradable linker and a method for preparing same, and more specifically, to a compound useful for tissue- or organ-specific drug delivery by enabling various molecular designs according to target tissues or organs, and a method for preparing same.
Absstract of: WO2026141452A1
The present disclosure provides an ionizable lipid that can be used for nucleic acid delivery to the cytoplasm. An ionizable lipid according to the present disclosure is, for example, a compound represented by formula (I) or a pharmaceutically acceptable salt thereof. In the formula, L1 represents –(CH2)n-, L2 represents –(CH2)m-, n represents an integer of 1-5, m represents an integer of 1-5, X1 and X2 each independently represent –OC(O)- or –OC(O)O-, Y represents -OC(O)- or –OC(O)O-, R1 and R2 each independently represent a C2-C25 alkyl group or a C2-C25 alkenyl group which may be substituted with at least one C4-C12 alkoxy group, the total number of carbon atoms of either R1 or R2 is 4-30, P is represented by formula P-1 or formula P-2, R3 represents a C1-C5 alkyl group which may be substituted with a hydroxy group, R4 and R5 each independently represent a hydrogen atom or a C1-C5 alkyl group, or R4 and R5 are bonded to each other to form a C2-C5 alkylene group, p represents 0 or 1, q represents an integer of 0-2, R6 and R7 each independently represent a C1-C5 alkyl group, r represents an integer of 1-5, and * represents a linking site.
Absstract of: WO2026143047A1
Disclosed herein are a messenger RNA (mRNA) comprising an open reading frame (ORF) encoding a protein of interest, a heterologous 5' untranslated region (UTR) and/or a heterologous 3' UTR for highly expressing the protein of interest. A method for synthesizing the mRNA and uses of the mRNA are also provided.
Absstract of: US20260183418A1
0000 The invention provides ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to target cells, such as immune cells and hematopoietic stem cells, and methods of making and using, such lipids and targeted lipid nanoparticles.
Absstract of: WO2026141632A1
The present invention addresses the problem of providing: lipid nanoparticles that enable selective delivery of a nucleic acid to a target cell; or a method for measuring the optimal density for selective delivery of a nucleic acid to a target cell. Provided are lipid nanoparticles that include: a nucleic acid encapsulated in the lipid nanoparticles; a cationic lipid; a sterol or a sterol derivative; an antibody selected from a VHH antibody, an Fab antibody, and an scFv antibody; a targeting polyalkylene glycol-modified lipid; and a non-targeting polyalkylene glycol-modified lipid. The antibody targets an antigen on a T-cell. The targeting polyalkylene glycol-modified lipid is a lipid in which the antibody is linked to polyalkylene glycol. The non-targeting polyalkylene glycol-modified lipid is a lipid not linked to the antibody. The density of the VHH antibody existing on the surfaces of the lipid nanoparticles in a state capable of binding to the antigen is 0.004-2.300 per 100 nm2 of the nanoparticle surfaces. The density of the Fab antibody existing on the surfaces of the lipid nanoparticles in a state capable of binding to the antigen is 0.003-1.113 per 100 nm2 of the nanoparticle surfaces. The density of the scFv antibody existing on the surfaces of the lipid nanoparticles in a state capable of binding to the antigen is 0.003-1.113 per 100 nm2 of the nanoparticle surfaces.
Absstract of: WO2026141628A1
The purpose of the present invention is to provide a lipid nanoparticle that makes it possible to efficiently introduce a drug into a cell, and a constituent lipid of this lipid nanoparticle. Provided is a lipid nanoparticle comprising a nucleic acid encapsulated in the lipid nanoparticle, a cationic lipid, and a targeted polyalkylene glycol-modified lipid linked to a target binding site, wherein the target binding site targets a target site on a target cell.
Absstract of: WO2026141629A1
The purpose of the present invention is to provide lipid nanoparticles that make it possible to efficiently introduce a drug into a cell, and a constituent lipid constituting the aforementioned lipid nanoparticles. Provided are lipid nanoparticles comprising a nucleic acid encapsulated in the lipid nanoparticles, a cationic lipid, and a targeting polyalkylene-glycol-modified lipid linked to a target binding site, the target binding site targeting a target site on a target cell.
Absstract of: WO2026141630A1
The purpose of the present invention is to provide: a lipid nanoparticle that enables efficient introduction of a drug into a cell; and constituent lipids of the lipid nanoparticle. Provided is a lipid nanoparticle comprising a nucleic acid encapsulated in the lipid nanoparticle, a cationic lipid, and a targeted polyalkylene glycol-modified lipid linked to a target-binding site, wherein the target-binding site targets a target site on a target cell.
Absstract of: WO2026141627A1
The purpose of the present invention is to provide lipid nanoparticles capable of efficiently introducing drugs into cells, and constituent lipids of the lipid nanoparticles. Provided are lipid nanoparticles including nucleic acids encapsulated within the lipid nanoparticles, cationic lipids, and targeted polyalkylene glycol-modified lipids linked to a target binding site, wherein the target binding site targets a target site on a target cell.
Absstract of: WO2026143008A1
A lipid nanoparticle and a method of delivering a nucleic acid encapsulated in the lipid nanoparticle to a cell or a subject. The lipid nanoparticle may contain 55-65 mol% of a cationic lipid to the total lipid amount of the lipid nanoparticle; 30-40 mol% of a sterol to the total lipid amount of the lipid nanoparticle; 1-3 mol% of 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE) to the total lipid amount of the lipid nanoparticle; and 1-5 mol% of 1,2-dimyristoyl-rac-glycero-3-methoxypolyethylene glycol (DMG-PEG) to the total lipid amount of the lipid nanoparticle. The lipid nanoparticle may also contain 55-65 mol% of a cationic lipid to a total lipid amount of the lipid nanoparticle; 30-40 mol% of a sterol to the total lipid amount of the lipid nanoparticle; 4-6 mol% of 1,2-dioleoyl-sn-glycero-3-phosphocholine (DOPC) to the total lipid amount of the lipid nanoparticle; and 1-5 mol% of 1,2-dimyristoyl-rac-glycero-3-methoxypolyethylene glycol (DMG-PEG) to the total lipid amount of the lipid nanoparticle.
Absstract of: WO2026142012A1
The present invention relates to a lipid, a preparation method therefor, and a composition of nanoparticles for drug delivery comprising same and, more specifically, to: a lipid and a preparation method therefor, wherein the lipid is ionizable and capable of forming a complex with anionic drugs and allows various molecular designs depending on a target tissue or organ, thereby being useful for tissue- or organ-specific drug delivery; and a drug delivery composition in which a drug is encapsulated within a nanoparticle structure formed by the lipid and a polymer and a preparation method therefor.
Absstract of: WO2026142343A1
The present invention relates to an antibody-drug conjugate (ADC) targeting a CT83 (KK-LC-1) antigen. The ADC can induce concentration-dependent apoptosis in CT83-expressing cancer cells and exhibits nanomolar (nM)-level binding affinity, and thus can exhibit excellent target selectivity and anti-tumor efficacy. Therefore, the ADC according to the present invention may be applied as an effective target-based anticancer therapeutic platform that can be utilized in the treatment of various CT83-expressing cancer cells.
Absstract of: WO2024254709A1
The present disclosure provides a therapeutic nanodisk, the therapeutic nanodisk comprising: a lipid-binding polypeptide; a lipid bilayer and a therapeutic agent, wherein the therapeutic agent may be of use for treating, preventing a central nervous system disease, disorder, trauma or injury; or as a diagnostic agent for diagnosing a central nervous system disease, disorder, trauma or injury. The lipid bilayer may be 1,2-Dimyristoyl-sn-glycero-3-phosphocholine (DMPC) and the therapeutic agent may be a nucleic acid polymer. Further provided are methods for administration of the therapeutic nanodisk to treat, prevent or diagnose the central nervous system disease, disorder, trauma or injury and uses of such therapeutic nanodisks.
Absstract of: MX2025014916A
Compounds are provided having the following Formula (I) or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R1, R2, R3, G1, G2, L1, and L2 are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.
Absstract of: WO2025040724A1
A method for preparing nucleic acid-containing lipid nanoparticles (NALNP) from a nucleic acid-containing neutral pH ionic salt solution and a lipid solution is provided. The method includes combining the nucleic acid-containing neutral pH ionic salt solution and the lipid solution and forming a combined mixture. The method further includes introducing an aqueous solution of lower ionic concentration than that of the neutral pH ionic salt solution into the combined mixture, thereby creating an ionic flux and forming the nucleic acid-containing lipid nanoparticles (NALNP).
Absstract of: MX2025014915A
Compounds are provided having the following Formula (I): (I) or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein G1, R1, R2, R3, L1, and L2 are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.
Absstract of: WO2025045767A1
This invention presents novel lipid and lipidoid nanoparticle (LNP and LiNP) formulations enhanced with specifically characterized surfactants. These surfactants exhibit unique Langmuir isotherm properties, ensuring optimal stabilization of the nanoparticles. The described formulations improve the stability, reduce aggregation, and mitigate challenges faced during purification, such as filtration clogging or fouling. Integral to the innovation is a method for determining the suitability of surfactants as stabilizers based on predetermined Langmuir isotherm values and filtration speeds. The stabilized nanoparticles, when formulated with therapeutic agents, have demonstrated potential in medical applications, particularly in the realm of mRNA delivery, vaccination and immunization.
Absstract of: WO2024259356A1
Compounds are provided having the following Formula (I): or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein G1, G2, R1, R2, R3, L1a, L1b, and L2 are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.
Absstract of: WO2025040904A1
The present invention relates to a pharmaceutical composition comprising nanoparticles of a cyanine dye and a therapeutic agent. The present invention also relates to the use of said pharmaceutical composition in therapy.
Absstract of: EP4768468A2
0001 The invention relates to mRNA comprising lipid nanoparticles and their medical uses. The lipid nanoparticles of the present invention comprise a cationic lipid according to formula (I), (II) or (III) and/or a PEG lipid according to formula (IV), as well as an mRNA compound comprising an mRNA sequence encoding an antigenic peptide or protein. The invention further relates to the use of said lipid nanoparticles as vaccines or medicaments, in particular with respect to influenza or rabies vaccination.
Absstract of: EP4768472A1
The present invention relates to a drug delivery material and an application thereof. Specifically provided is a nanoparticle composition comprising a lipid component, the lipid component comprising a compound represented by formula (I). The nanoparticle composition of the present invention is delivered only at the site of administration.
Absstract of: WO2025046399A1
The present invention relates to nanocomplexes comprising functionalized gold nanoparticles for the delivery of genome editing systems, genome editing systems, compositions comprising them and their uses. Furthermore, the present invention relates to the process for the functionalization of said nanoparticles and the preparation of said nanocomplexes.
Absstract of: WO2025043212A2
Disclosed herein are functionalized branched poly-lysine compounds and uses thereof, as well as delivery systems in which polymeric nanocarriers are functionalized with branched poly-lysine compounds. Also provided herein are uses of the functionalized branched poly-lysine compounds and the delivery systems for treating joint diseases such as osteoarthritis, and in delivering pharmaceutically active compounds to cartilage and subchondral bone.
Absstract of: AU2024328597A1
In some aspects, the present disclosure provides methods for generating CAR T cells in situ. The present disclosure provides lipid nanoparticles that selectively target a spleen cell, in particular, a lymphocyte such as a T cell. The lipid nanoparticle provided herein contain a five component composition that includes a permanently anionic lipid giving the lipid nanoparticle an apparent pKa of less than 6.
Nº publicación: EP4766763A1 01/07/2026
Applicant:
PARK JONG GU [KR]
JIE OOI YING [SG]
CLEARLAB SG PTE LTD [SG]
CHAN PARK MARY B [SG]
Clearlab SG Pte Ltd
Jie, Ooi, Ying
Chan-Park, Mary, B.
Park, Jong, Gu
Absstract of: WO2025041111A1
Provided herein are hyperbranched poly(β-amino esters) polymers that serve as effective transfection carriers of small interfering RNA (siRNA) for RNA interference (RNAi) mediated gene silencing therapy. These disclosed polymers exhibit outstanding gene silencing efficiency in both easy-to-transfect and hard-to-transfect cells. Furthermore, they are biodegradable and non-toxic.