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WO/2024/100247A1 |
The present invention relates to artificial nucleic acids for site-directed editing of a target RNA. In particular, the present invention relates to artificial nucleic acids which provide for enhanced editing specificity and avoid undesi...
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WO/2024/099391A1 |
Disclosed herein are a lipid-charged molecule conjugate, an inhalable lipid nanoparticle, a preparation method therefor, and use thereof. The lipid-charged molecule conjugate of the present invention comprises a lipid unit and a charged ...
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WO/2024/098940A1 |
Provided is a bispecific antibody, comprising a first domain specifically binding to an interleukin-6 receptor (IL-6R), and a second domain specifically binding to a programmed cell death receptor-ligand 1 (PD-L1). Further provided is a ...
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WO/2024/099103A1 |
A polypeptide having an analgesic effect and a use thereof. The polypeptide or a construct thereof can be used as a positive allosteric modulator and used in combination with a TRPV1 receptor agonist to enhance an agonistic effect on TRP...
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WO/2024/100549A1 |
The present invention relates to compounds for use for treating and/or preventing tumors by virtue of the ability to reprogram tumor-associated macrophages M2 (TAM-M2) to tumor-associated macrophages M1 (TAM-M1).
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WO/2024/102860A1 |
Strategies, systems, compositions, and methods for genetically modifying cells to include one or more loss-of-function modifications and/or to include one or more gain-of-function modifications, as well as modified cells (and composition...
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WO/2024/100063A1 |
The invention relates to genetically modified hosts cell comprising a recombinant pathway having enhanced production of one or more benzylisoquinoline alkaloids or glycosylated benzylisoquinoline alkaloid, wherein the host cell has been ...
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WO/2024/076688A3 |
Certain embodiments of the invention provide a synthetic genomic safe harbor in the genome of a cell. Certain embodiments provide a method of creating a synthetic genomic safe harbor in a genome. Certain embodiments of the invention prov...
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WO/2024/102977A1 |
Disclosed herein are genetically engineered cells for AAV production. The genetically engineered cell comprises molecular systems for temporal control of expression of genes required for AAV production. Also disclosed herein are methods ...
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WO/2024/100235A1 |
The invention provides immunogenic peptides, polypeptides, and compositions based on group A Streptococcus (GAS) Enn protein, and their use in the therapy, particularly prophylaxis, of GAS infections.
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WO/2024/099310A1 |
An anti-IL-13 long-acting nanobody, a coding sequence encoding the anti-IL-13 nano antibody, a corresponding expression vector, a host cell capable of expressing the nanobody, and a method for producing the nanobody.
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WO/2024/099323A1 |
Provided are a Zn-nicotinamide transport protein and a use thereof. The protein can positively regulate the content of Zn or Fe in Gramineae plant seeds (including embryo and endosperm), and yield and quality are reduced from loss-of-fun...
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WO/2024/102659A1 |
The disclosure provides engineered nucleotide sequences, vectors, and pharmaceutical compositions that can be used in methods for treating a disease or disorder with an associated haploinsufficiency and/or cancer.
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WO/2024/099975A1 |
The invention relates to nanoparticles for Cas protein/gRNA ribonucleoprotein (RNP) complex, siRNA or PMO delivery comprising one or more Cas protein/gRNA RNP complex(es), siRNA(s) or PMO(s) as cargo; and a carrier comprising a sequence-...
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WO/2024/102797A1 |
Provided are polymeric compounds of Formula I which include cationic poly(alpha-amino ester) monomers and lipid functionalized beta-amido carbonate (bAC) monomers, and related compositions and methods for transport and delivery of polyan...
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WO/2024/102773A1 |
The present invention provides computer-implemented methods for predicting and/or screening for a candidate riboswitch as well as related methods for validating said candidate riboswitch.
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WO/2024/102972A1 |
Provided herein are compositions for gene modification related to base editor systems, and methods of using the same to treat or prevent conditions associated with the extracellular deposition in various tissues of amyloid fibrils formed...
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WO/2024/076750A3 |
The present disclosure relates to pharmaceutical compositions comprising engineered macrophages and methods of use thereof. In some aspects, the present disclosure relates to engineered macrophages derived from pluripotent stem cells. In...
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WO/2024/102672A2 |
The present disclosure provides methods for ameliorating or treating a neurological condition or disease in a subject in need of treatment thereof by administering to the subject a therapeutically effective amount of a composition compri...
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WO/2024/100656A1 |
The present invention provides delivery system compositions comprising self- assembling lipid nanoparticles for targeted delivery of therapeutic or diagnostic agents to target cells. The particles are non-covalently attached to a lipidat...
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WO/2024/102985A1 |
The present disclosure is related to spliceosome perturbations. Provided herein are methods of inducing an immune response to a cancer cell. Also provided are methods to treat a subject in need thereof e.g., a subject suffering from cancer.
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WO/2023/250492A3 |
The disclosure provides, e.g., compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell's genome at one or more locations in a DNA sequence in a cell, tissue, or subject. Gene modifying systems fo...
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WO/2024/102833A2 |
A composition that includes a micro-RNA mimic containing one or more adenine bases in which at least one adenine base is replaced by 6-mercaptopurine. Also provided is a method for killing a cancer cell by contacting it with a compositio...
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WO/2024/100196A1 |
The present disclosure relates to vectors, such as DNA plasmids, comprising multiple nucleic acid sequences of interest engineered to be co-expressed as separate molecules, pharmaceutical compositions comprising such vectors and the use ...
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WO/2024/102961A1 |
Synthetic nucleic acids are described that can be used for astrocyte-directed expression of heterologous nucleotide sequences. Also described are methods of using the same for astrocyte-directed expression of such nucleotide sequences fo...
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WO/2024/077256A9 |
The present invention discloses methods and platforms for generating protein binding proteins with specificity for native peptide-MHC (pMHC) complexes. The pMHC binding proteins can be used in bi-specific antibodies or for generating CAR...
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WO/2024/100776A1 |
This transformant obtained by introducing a chorismate-pyruvate lyase gene of the following (a), (b), (c), (d), or (e) to a bacterium belonging to the genus Hydrogenophilus can efficiently produce 4-hydroxybenzoic acid by using carbon di...
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WO/2024/102881A1 |
A transgenic Camelina plant genetically modified to be tolerant to glufosinate without exhibiting a decrease in seed yield is disclosed. The transgenic Camelina plant comprises an insertion of a heterologous gene encoding phosphinothrici...
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WO/2023/172916A3 |
Engineered T cells are provided that express (a) a chimeric antigen receptor wherein the antigen binding domain of the CAR binds to human GPC3 ("a GPC-CAR"); and (b) an orthogonal receptor. Also provided are methods of making and using s...
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WO/2024/101936A1 |
One aspect of the present invention relates to a cucumber mosaic virus-based improved recombinant vector. A recombinant vector, according to one embodiment, can significantly improve the efficiency of foreign protein expression in plants...
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WO/2024/054768A3 |
This disclosure concerns methods and compositions for identifying plants that have an Ogura CMS restorer of fertility phenotype. Some aspects concern a zygosity assay to identify, select, and/or construct Ogura CMS restorer of fertility ...
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WO/2023/220599A3 |
Provided are gain-of-activity mutations in components of a type I-F3 Tn7-CRISPR-Cas system. The mutations allow use of systems that contain proteins with the mutations to function with both typical and atypical guide RNAs, and permit alt...
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WO/2024/098269A1 |
The present invention provides a transaminase, the amino acid sequence thereof being set forth in SEQ ID NO. 3. The transaminase can catalyze the conversion of (2Z)-4-oxo-4-[3-(trifluoromethyl)-5,6-dihydro[1,2,4]triazolo
[4,3-a]pyrazin-7...
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WO/2024/103050A1 |
Disclosed are exosomes comprising a Na+ taurocholate co-transporting polypeptide (NTCP) binding motif, such as HBV preSl peptide (PS IP). Disclosed are methods of making exosomes that have been engineered to express a heterologous bindin...
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WO/2024/098360A1 |
Provided is an artificial nucleic acid molecule, which comprises at least one open reading frame and at least one 5-untranslated region element, and has high translation efficiency.
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WO/2024/102850A2 |
The present inventions relate to a breeding system for the production of polyploid seeds, plants, or plant parts where cycles of meiosis, syngamy, and selection are used for interpopulation improvement of progenitor lines, and sexual pol...
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WO/2024/064606A3 |
Compositions comprising an RNA molecule comprising a guide sequence portion having 17- 50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-4886 and methods and uses thereof.
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WO/2024/098142A1 |
Monovalent antibodies such as single-domain antibodies (sdAbs) that are specific for galectin- 13/16 or galectin-9 are described. These monovalent antibodies are able to interfere with the activity of galectin-13/16 or galectin-9, and th...
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WO/2024/100038A1 |
The invention relates to newly identified nucleic acids, ribonucleic acids (RNAs) and deoxyribonucleic acids (DNAs), specifically esiRNAs/ERNAs (effective small interfering RNAs) and RNAs derived therefrom, as well as eASO (effective ant...
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WO/2024/101345A1 |
Provided is an agent containing an RGMa inhibition substance, the agent being for suppressing accumulation of abnormal protein aggregates or for inhibiting uptake of abnormal proteins by nerve cells, particularly for preventing or treati...
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WO/2024/059881A3 |
Nucleic acid products are provided that modulate, in particular interfere with or inhibit AGT gene expression. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first n...
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WO/2024/100176A1 |
The present invention concerns a controlled gene therapy of ocular diseases. This technology can be used in gene therapy protocols and avoids the side effects resulting from a continuous and uncontrolled expression of a transgene.
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WO/2024/102830A2 |
A method for covalently joining linear RNA molecules end-to-end to extend their functionality. A cis-acting ligase ribozyme is used to generate circular RNA in vitro for ectopic protein expression. RNA circularization was confirmed by ma...
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WO/2024/098980A1 |
Provided are CD112R-binding proteins, particularly anti-CD112R antibodies and antigen-binding fragments thereof. Further provided are compositions comprising the CD112R-binding proteins, nucleic acid molecule encoding the antigen-binding...
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WO/2024/064694A3 |
The present disclosure provides compositions and methods for producing transgenic banana plants that exhibit increased resistance to Fusarium oxysporumf.sp. cubense Tropical Race 4 (TR4), and the banana plants and bananas so produced.
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WO/2024/098178A1 |
Provided is a reaction system for preparing a DNA nanoball (DNB), comprising: adapters; an enzyme, wherein the enzyme comprises a DNA ligase; and a buffer solution. The adapters comprise an adapter A and an adapter B. The adapter A compr...
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WO/2024/102434A1 |
Disclosed herein are RNA compositions including one or more polynucleotides encoding one or more gene editing systems, formulated within a lipid reconstructed natural messenger pack (LNMP) comprising natural lipids and an ionizable lipid...
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WO/2024/103063A1 |
Methods, devices, and systems for isolating nucleic acid e.g., in a point of need setting, featuring a housing with a binding chamber therein, the binding chamber comprising a binding component capable of binding nucleic acid or other mo...
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WO/2024/101961A1 |
The present invention relates to cells genetically modified to express a peptide capable of inhibiting the transforming growth factor beta (TGF-β) signaling pathway or a fragment of the peptide.
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WO/2024/103025A1 |
The disclosure provides methods and tools allowing production of large quantities of double stranded RNA (dsRNA) in bacterial cells, including plasmid vectors useful for transforming gram-positive or gram-negative bacterial cells such th...
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