Resumen de: US20260166134A1
An mRNA, an mRNA vaccine formulation, and a preparation method and use thereof are provided. An amino acid sequence of the mRNA is set forth in SEQ ID NO: 2. Studies have demonstrated that the mRNA vaccine formulation prepared from the mRNA having the amino acid sequence set forth in SEQ ID NO: 2 can effectively stimulate dendritic cell maturation and secretion of pro-inflammatory cytokines, thereby activating effector T cells to kill prostate cancer cells. A preparation method of the mRNA vaccine formulation is simple and suitable for industrial-scale production. By using lipid nanoparticles (LNPs) as a carrier, the mRNA vaccine formulation achieves high encapsulation efficiency, enabling robust induction of immune responses in vivo with high immunogenicity.
Resumen de: US20260166063A1
0000 The present disclosure relates, in general, to compositions comprising phosphatidylcholine and phosphatidylcholine derivatives, e.g., DLPC (1,2-dilauroyl-sn-glycero-3-phosphocholine) or DPPC, for the treatment of fibrosis, including liver fibrosis and associated conditions such as fatty liver disease, non-alcoholic steatohepatitis (NASH) and cirrhosis, or lung fibrosis and conditions associated with lung fibrosis.
Resumen de: CN121532174A
The present disclosure describes a lipid-polymer hybrid nanoparticle and a method of synthesizing such a nanoparticle or a composition containing such a nanoparticle. These nanoparticles are made from a biodegradable polymer-based micellar core surrounded by a lipid-based shell, wherein a majority of the agent is present on the inner periphery of such nanoparticles due to physical adhesion to lipid molecules. Only a small amount of medicament is encapsulated in the micelle core. Thus, the lipid-based shell becomes the primary excipient part of the nanoparticle, and the core containing the biodegradable polymer becomes the secondary excipient part of the nanoparticle.
Resumen de: WO2024254459A2
Disclosed are methods of treating complex regional pain syndrome in a subject by administering a therapeutic MSC secretome product made by a method comprising culturing bone marrow-derived MSCs under conditions that include oxygen tension below 5% and a culture media with a pH below 7.
Resumen de: WO2026127735A1
The present invention relates to a PD-L1-targeting PROTAC peptide, PROTAC nanoparticles comprising same, and a composition for cancer prevention or treatment containing same as an active ingredient, wherein in cancer tissues, the PD-L1 receptor is subjected to a direct lysosomal degradation pathway and a PROTAC-mediated degradation pathway, thereby not only inducing PD-L1 protein degradation but also fundamentally blocking PD-L1 protein regeneration, thus inhibiting the transmission of immunosuppressive signals in the interaction between cancer cells and T cells to activate immune responses, so that the effect of inducing the death of cancer cells can be continuously and effectively achieved.
Resumen de: WO2026127822A1
There is provided compound comprising a structure represented by general formula (1): wherein R1, R3, R4, R5, and R6 are each independently H, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; R7 is optionally substituted alkylene, optionally substituted alkenylene, or optionally substituted alkynylene; R10 is optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; k is 0 or 1; n and l are each independently ≥ 1; A comprises galactose and/or a derivative(s) thereof; and B comprises a lipid and/or a derivative(s) thereof
Resumen de: US20260166075A1
0000 The disclosure relates to pharmaceutical compositions including Ir/Cu nanoenzymes, methods of treatment for neurodegenerative disease or disorder, and kits including the compositions. The Ir/Cu nanoenzymes can suppress oxidative stress in neuronal cells and inhibit aggregation of alpha-synuclein.
Resumen de: US20260167972A1
A nanoparticle complex contains short-interfering RNA (siRNA) and one or more amino acid derivatives. The nanoparticles may be spherical or micelle-like. The complex may have a mixture of spherical/micelle-like and fibril-like nanoparticles, or a mixture of spherical/micelle-like, sheet-like and/or fibril-like nanoparticles. The complexes may be used as part of a method of delivering RNA or DNA into a cell, where the RNA or DNA is mixed with one or more amino acid derivatives, the mixture is diluted into a cell culture medium, and a cell is dosed with the diluted mixture. The RNA may be small interfering RNA (siRNA). The method may reduce messenger RNA (mRNA) levels and/or protein levels related to a specific gene or genes by at least 50% and exhibit cell viabilities equal to or exceeding 40% after 48 hours.
Resumen de: US20260166171A1
The present disclosure features a method of preparing a population of payload-associated cell complexes (PACCs), as well as related methods of use thereof.
Resumen de: WO2026127823A1
There is provided a compound comprising a structure represented by general formula (1) wherein A comprises a sterol and/or a derivative(s) thereof; B comprises: (i) a carbohydrate and/or a derivative(s) thereof; or (ii) an oligopeptide or a polypeptide comprising carbohydrate and/or a derivative(s) thereof; R1 and R3 are each independently H, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; R2 is optionally substituted alkylene, optionally substituted alkenylene, or optionally substituted alkynylene; R4 is –H or –C(=O)R, where R is optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; p = 0 or 1; and q = 0 or 1.
Resumen de: WO2026123105A1
An AI-based automated self-driving laboratory system comprises a machine learning module (MLM) operatively coupled to a laboratory module (LM). Pretrained with a set of molecules to learn their structures and properties, the MLM is configured to produce an initial set of molecules for the LM to synthesize and analyze based on the learned structures and properties, and receive feedback of structures and properties of the initial set for further training and produce a next iteration for the LM to synthesize and analyze. The LM is configured to synthesize and analyze the initial set, relay the initial set's structures and properties for the iterative training, receive from the MLM, and synthesize and analyze the next iteration. The MLM is iteratively retrained to produce the next iteration for iterative synthesis and analysis by the LM until at least one termination criterion is met. An ionizable lipid of Formula I is disclosed.
Resumen de: WO2026125660A1
The present invention refers to Particles comprising circular RNA and compositions thereof as well as methods of their use.
Resumen de: WO2026128814A1
Disclosed are solid compositions, comprising a lipid nanoparticle, a nucleic acid, and an antioxidant; wherein the nucleic acid and the antioxidant are encapsulated by the lipid nanoparticle.
Resumen de: WO2026128792A1
Disclosed is a thermally stable, single-dose mRNA-LNP (lipid nanoparticle) platform.
Resumen de: WO2026127586A1
The present invention relates to a novel lipid and a preparation method therefor and, more particularly, to a lipid and a preparation method therefor, in which the lipid can be ionized to form a complex with an anionic drug, allowing various molecular designs according to a target tissue or organ, and thus is useful for tissue- or organ-specific drug delivery.
Resumen de: WO2026128506A1
The present disclosure provides an ionizable lipid of Formula (I) or Formula (II).
Resumen de: WO2026125793A2
The present invention relates to nanostructured lipid carriers comprising a lipid core of liquid and solid lipids, particularly a triglyceride having a fatty acid chain of 12 to 16 carbon atoms and a mixture of triglycerides of medium-chain saturated fatty acids and covered by a surfactant layer, in which at least one of said surfactants is phosphatidylcholine. Said nanostructured lipid carriers are characterised in that they do not comprise any active ingredient or agent. The invention also relates to the (non-therapeutic) use of the nanostructured lipid carriers for inhibiting the formation of biofilms, as well as their therapeutic use as a medicament for treating or preventing diseases caused by biofilm-forming bacteria.
Resumen de: US20260166175A1
The present disclosure provides for a polymer comprising repeating units of a cyclodextrin comprising α-cyclodextrin (α-CD), γ-cyclodextrin (γ-CD), or any combination thereof, and a crosslinker. Further provided herein are a particle and cryogel comprising the same. Also provided herein are methods of using and making thereof.
Resumen de: US20260166176A1
Alterations of the 5′GT splice motif or 3′AG splice motif result in severe missplicing and are prevalent in many genetic diseases, including cystic fibrosis (CF). While protein-targeted modulator therapies are currently available for treatment of CF, individuals with these canonical splice site variants (CSSVs) are among the ˜10% who remain untreated. The most common CF-causing variant in individuals of African descent is a CSSV, c.2988+1G>A. While >75% of individuals with a CSSV have a modulator eligible in trans allele, only 50% of the 450 individuals bearing c.2988+1G>A are eligible. Thus, there is a particular unmet need for a treatment for these individuals. CRISPR/Cas9-mediated adenine base editing (ABE) is an efficient and targeted genome editing method correct G>A variants. We electroporated NRCH-ABE8e mRNA and a previously optimized sgRNA to non-differentiated human primary nasal (HNE) or bronchial (HBE) epithelial cells from individuals with CF compound heterozygous for c.2988+1G>A. After differentiation of edited cells genomic editing and recovery of CFTR channel function were assessed. In primary HBEs and primary HNEs, we observed an allelic conversion to WT of 74.7% and 81.3%, respectively, at the +1 site. Interestingly, we also observed high levels of editing at adjacent adenines (+3, +7), which would have a modest effect on mRNA splicing (˜20% reduction). However, this did not preclude recovery of CFTR channel function. Compared to WT/WT HBEs and HNEs, un
Resumen de: WO2026127599A1
The present invention relates to a novel lipid and a method for preparing same and, more particularly, to a lipid and a method for preparing same, wherein the lipid is ionizable to form a complex with an anionic drug and allows for various molecular designs depending on a target tissue or organ, thereby being useful for tissue- or organ-specific drug delivery.
Resumen de: AU2025344951A1
The present disclosure relates to ionisable lipids of Formula I and related structures that comprise a headgroup, core, and hydrophobic substituents. The lipids are capable of forming lipid particles, including lipid nanoparticles (LNPs), in combination with additional lipids such as neutral lipids, charged lipids, structural lipids, or PEGylated lipids. The lipid particles are suitable for delivery of therapeutic agents, including polynucleotides, peptides, antibodies, and small molecules. Compositions comprising the ionisable lipids and methods of forming and using the lipid particles are also provided.
Resumen de: AU2025344428A1
The present disclosure relates to ionisable lipids of Formula I and related structures that comprise a headgroup, core, and hydrophobic substituents. The lipids are capable of forming lipid particles, including lipid nanoparticles (LNPs), in combination with additional lipids such as neutral lipids, charged lipids, structural lipids, or PEGylated lipids. The lipid particles are suitable for delivery of therapeutic agents, including polynucleotides, peptides, antibodies, and small molecules. Compositions comprising the ionisable lipids and methods of forming and using the lipid particles are also provided.
Resumen de: AU2024414252A1
The present invention pertains to the technical field of pharmaceutical preparations, and provides a novel solution-type quetiapine composition for transnasal administration. In the provided composition, quetiapine is completely dissolved therein in the form of a solution and administered transnasally. Compared with oral dosage forms on the market, the provided quetiapine solution-type transnasal preparation has particularly useful pharmacokinetic and pharmacodynamic profiles. Compared with other disclosed transnasal nano-emulsion forms, the composition of the present invention still has surprising pharmacokinetic and pharmacodynamic profiles, shows good clinical application prospects, and is expected to meet unmet clinical needs. On this basis, the present invention also provides a further improved high-saturation dissolution concentration technical solution and a further improved good chemical stability technical solution for preparing the quetiapine solution-type transnasal preparation.
Resumen de: AU2024401935A1
Aspects of the disclosure relate to compositions and methods for improving the stability of lipid nanoparticles (LNPs). In some embodiments, the LNPs comprise one or more active pharmaceutical ingredients (API) encapsulated therein. The disclosure is based, in part, on compositions that directly or indirectly reduce the degradation (e.g., oxidation, hydrolysis, etc.) of one or more lipids components of the lipid nanoparticle. In some embodiments, the compositions comprise a citrate drug product matrix, EDTA drug product matrix, methionine drug product matrix, and/or a tryptophan drug product matrix. The disclosure also provides methods for storing compositions contemplated herein as well as methods for improving the stability of the API.
Nº publicación: AU2024389988A1 18/06/2026
Solicitante:
METAGENOMI THERAPEUTICS INC
METAGENOMI THERAPEUTICS, INC.
Resumen de: AU2024389988A1
Described herein are methods, compositions, and systems derived from uncultivated microorganisms useful supplementing liver enzyme deficiencies.