Abstract

In some embodiments, the subject matter of this disclosure relates to systems and methods for assessing patient health and disease states. An example method includes: obtaining a set of patient data for a patient; supplementing the set of patient data with a plurality of possible values for a parameter missing from the set of patient data; using a foundation model to generate a plurality of bioprofiles based on the set of patient data and the plurality of possible values for the parameter; using the plurality of bioprofiles and a fine-tuned model to make a plurality of disease state predictions for the patient; and facilitating performance of a test to determine a value for the parameter.

IPC Classes  ?

• G16H 10/20 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for electronic clinical trials or questionnaires
• G16H 50/50 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for simulation or modelling of medical disorders

Abstract

The present disclosure relates, generally, to compositions and methods for producing, purifying, and using circular RNA.

IPC Classes  ?

• C12N 15/67 - General methods for enhancing the expression
• C12N 15/74 - Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora

Abstract

Disclosed are lipidoid compounds having the structure of formula (X) or formula Disclosed are lipidoid compounds having the structure of formula (X) or formula Disclosed are lipidoid compounds having the structure of formula (X) or formula wherein the groups are as defined in the application. Also disclosed are nanoparticle compositions comprising a lipidoid of the invention that are capable of delivering a therapeutic agent. The application also discloses pharmaceutical compositions comprising a lipidoid composition of the invention.

IPC Classes  ?

• A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
• A61K 9/51 - Nanocapsules
• A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
• A61K 39/00 - Medicinal preparations containing antigens or antibodies
• A61K 39/215 - Coronaviridae, e.g. avian infectious bronchitis virus
• C07C 237/08 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
• C07C 237/20 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton containing six-membered aromatic rings
• C07D 207/06 - Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with radicals, containing only hydrogen and carbon atoms, attached to ring carbon atoms
• C07D 211/12 - Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with radicals containing only carbon and hydrogen atoms attached to ring carbon atoms with only hydrogen atoms attached to the ring nitrogen atom
• C07D 233/64 - Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with substituted hydrocarbon radicals attached to ring carbon atoms, e.g. histidine

Abstract

Low background regions (LBRs) refer to genomic regions comprising one or more CpG sites that are differentially methylated in cancer and non-cancer samples. Disclose methods involve identifying LBRs comprising one or more CpG sites whose methylation statuses sufficiently distinguish between samples that contain a presence of a cancer signature and samples that do not contain a presence of a cancer signature. LBRs enable improved detection of presence or absence of cancer signatures while using smaller samples and fewer consumable reagents.

IPC Classes  ?

• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer

Abstract

inter aliae.g.,e.g.,e.g.,e.g.,e.g.,e.g.,e.g., MASH).

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides

Abstract

The disclosure provides, in various embodiments, antibodies, antigen-binding fragments thereof, and compositions (e.g., pharmaceutical composition) comprising said antibodies, or antigen-binding fragments thereof, that bind to thymic stromal lymphopoietin (TSLP) and methods for using the same in the treatment of a subject having a TSLP-associated disease or condition.

IPC Classes  ?

• A61P 11/00 - Drugs for disorders of the respiratory system
• A61P 11/06 - Antiasthmatics
• C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons

Abstract

Synthetic endornaviral satellite RNA molecules and satellite particles containing the same are disclosed. The synthetic endornaviral satellite RNA molecules can include coding and/or non-coding cargo sequences, and are heritable through generations of plants. Also disclosed are methods of using the endornaviral satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

Provided herein are, inter alia, agents (e.g., RNAi agents, dsRNA agents) comprising a sense strand and an antisense strand targeting CIDEB (e.g., hCIDEB); and methods of manufacturing and pharmaceutical compositions comprising the same. Further provided herein are methods of utilizing the agents (e.g., RNAi agents, dsRNA agents) including, e.g., methods of inhibiting or decreasing CIDEB expression (e.g., mRNA expression), methods of treating CIDEB associated diseases, and methods of treating liver diseases (e.g., MASH).

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

e.g. e.g., a therapeutic agent, such as a nucleic acid, to a target tissue or organ. More particularly, the disclosure relates to the use of plant viral capsid proteins, for example, protein complexes comprising plant viral capsid proteins, for delivering therapeutic agents, such as nucleic acids, to target tissues and organs.

IPC Classes  ?

• A61K 35/76 - VirusesSubviral particlesBacteriophages
• A61K 9/51 - Nanocapsules
• A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
• A61K 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

inter aliae.g.,e.g.,e.g.,e.g.,e.g., autoimmune) diseases.

IPC Classes  ?

• C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
• C07K 14/705 - ReceptorsCell surface antigensCell surface determinants

Abstract

The present disclosure relates to therapeutic agents and combinations thereof (e.g., pharmaceutical compositions) for the treatment of a viral infection in a subject, tissue or cell.

IPC Classes  ?

• A61K 31/352 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. cannabinols, methantheline
• A61K 31/675 - Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
• A61K 31/7072 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
• A61K 36/16 - Ginkgophyta, e.g. Ginkgoaceae (Ginkgo family)
• A61P 31/14 - Antivirals for RNA viruses

Abstract

Synthetic partitiviral satellite RNA molecules and satellite particles containing the same are disclosed. Also disclosed are methods of using the partitiviral satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Disclosed is a tiered, multipart method for tracking tumor heterogeneity across samples obtained from a subject at different timepoints. Each sample undergoes at least an intra-individual analysis to generate background-corrected methylation information. The change in the background-corrected methylation information across the different samples is informative for tracking a change in the tumor heterogeneity. The change in tumor heterogeneity is useful e.g., for providing a guided therapy.

IPC Classes  ?

• G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment
• C12Q 1/6806 - Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
• C12Q 1/6809 - Methods for determination or identification of nucleic acids involving differential detection
• C12Q 1/686 - Polymerase chain reaction [PCR]
• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
• G16B 20/20 - Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
• G16B 30/10 - Sequence alignmentHomology search
• G16B 40/10 - Signal processing, e.g. from mass spectrometry [MS] or from PCR
• G16B 40/20 - Supervised data analysis
• G16H 20/10 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients

Abstract

Disclosed is a tiered, multipart method for tracking tumor heterogeneity across samples obtained from a subject at different timepoints. Each sample undergoes at least an intra-individual analysis to generate background-corrected methylation information. The change in the background-corrected methylation information across the different samples is informative for tracking a change in the tumor heterogeneity. The change in tumor heterogeneity is useful e.g., for providing a guided therapy.

IPC Classes  ?

• C12Q 1/6869 - Methods for sequencing
• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
• G16B 20/20 - Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
• G16H 10/40 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for data related to laboratory analysis, e.g. patient specimen analysis
• G16H 15/00 - ICT specially adapted for medical reports, e.g. generation or transmission thereof
• G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment

Abstract

The disclosure provides, inter alia, dual-layer, semi-permeable, fibrosis-resistant, hydrogel polymer enclosures, optionally containing cells, such as mammalian cells, where the enclosures are suitable for administration to a subject in need thereof and further wherein the enclosure are scalable for large volumes of delivered cells. The disclosure further provides methods of making and using the enclosures, for example, in therapeutic methods, such as treating disorders of the liver or other cells and organs treatable with, for example secretory and/or catalytic cells.

IPC Classes  ?

• A61K 9/06 - OintmentsBases therefor
• A61K 9/50 - Microcapsules
• A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
• A61K 35/545 - Embryonic stem cellsPluripotent stem cellsInduced pluripotent stem cellsUncharacterised stem cells
• A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
• A61K 38/36 - Blood coagulation or fibrinolysis factors
• A61K 38/37 - Factors VIII
• A61K 38/39 - Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
• C12N 5/00 - Undifferentiated human, animal or plant cells, e.g. cell linesTissuesCultivation or maintenance thereofCulture media therefor
• C12N 5/0775 - Mesenchymal stem cellsAdipose-tissue derived stem cells

Abstract

The disclosure provides pharmaceutical compositions comprising a therapeutically effective amount of compound (A), compound (B), compound (C), compound (D), compound (E), compound (F), compound (G), compound (H), compound (J), compound (K), compound (L), compound (M), compound (N), compound (O), compound (P), or compound (Q) or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable excipient: Also provided are dosage units comprising one or more of compound (A), compound (B), compound (C), compound (D), compound (E), compound (F), compound (G), compound (H), compound (J), compound (K), compound (L), compound (M), compound (N), compound (O), compound (P), or compound (Q) or the pharmaceutical compositions described herein, methods of treating an inflammatory bowel disease in a subject in need thereof, or methods of modulating an inflammatory bowel disease marker in a subject in need thereof. The disclosure provides pharmaceutical compositions comprising a therapeutically effective amount of compound (A), compound (B), compound (C), compound (D), compound (E), compound (F), compound (G), compound (H), compound (J), compound (K), compound (L), compound (M), compound (N), compound (O), compound (P), or compound (Q) or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable excipient: Also provided are dosage units comprising one or more of compound (A), compound (B), compound (C), compound (D), compound (E), compound (F), compound (G), compound (H), compound (J), compound (K), compound (L), compound (M), compound (N), compound (O), compound (P), or compound (Q) or the pharmaceutical compositions described herein, methods of treating an inflammatory bowel disease in a subject in need thereof, or methods of modulating an inflammatory bowel disease marker in a subject in need thereof.

IPC Classes  ?

• C07D 417/14 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
• A61K 31/427 - Thiazoles not condensed and containing further heterocyclic rings
• A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
• C07D 277/10 - Heterocyclic compounds containing 1,3-thiazole or hydrogenated 1,3-thiazole rings not condensed with other rings having one double bond between ring members or between a ring member and a non-ring member with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached to ring carbon atoms

Abstract

e.g.,e.g.,e.g.,e.g.,e.g., plastic products).

IPC Classes  ?

• C12N 9/18 - Carboxylic ester hydrolases
• C08J 11/10 - Recovery or working-up of waste materials of polymers by chemically breaking down the molecular chains of polymers or breaking of crosslinks, e.g. devulcanisation
• C12N 15/52 - Genes encoding for enzymes or proenzymes

Abstract

e.g.,e.g.,e.g.,e.g.,e.g., plastic products).

IPC Classes  ?

• C12N 9/18 - Carboxylic ester hydrolases
• C08J 11/10 - Recovery or working-up of waste materials of polymers by chemically breaking down the molecular chains of polymers or breaking of crosslinks, e.g. devulcanisation

Abstract

Provided herein are esterases (e.g., PETases) (and functional fragments, functional variants, and domains thereof), nucleic acid molecules encoding the same, and compositions comprising the same. The disclosure further relates to methods of utilizing the esterases (e.g., PETases) (or nucleic acid molecules encoding the same), including, e.g., in methods of degrading polyester containing products (e.g., plastic products).

IPC Classes  ?

• C12N 9/18 - Carboxylic ester hydrolases
• C12N 15/52 - Genes encoding for enzymes or proenzymes
• C08J 11/10 - Recovery or working-up of waste materials of polymers by chemically breaking down the molecular chains of polymers or breaking of crosslinks, e.g. devulcanisation

Abstract

e.g.,e.g.,e.g.,e.g.,e.g., plastic products).

IPC Classes  ?

• C12N 9/18 - Carboxylic ester hydrolases
• C08J 11/10 - Recovery or working-up of waste materials of polymers by chemically breaking down the molecular chains of polymers or breaking of crosslinks, e.g. devulcanisation
• C08J 11/00 - Recovery or working-up of waste materials

Abstract

e.g.,e.g.,e.g.,e.g.,e.g., plastic products).

IPC Classes  ?

• C12N 9/18 - Carboxylic ester hydrolases
• C08J 11/10 - Recovery or working-up of waste materials of polymers by chemically breaking down the molecular chains of polymers or breaking of crosslinks, e.g. devulcanisation
• C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression

Abstract

A feature computational module encodes a signal generated by processing a biological sample, given one or more health-condition-informative regions related to an analyte, by using metrics based on marker information occurring within specified windows within a sequence of sites of interest within the health-condition informative regions related to the analyte. Each window has a specified position within a sequence of sites of interest in the health-condition informative region, and a specified size. The size is specified in terms of a number of consecutive sites of interest within the analyte. A metric is thus computed for a plurality of positions within the health-condition informative region. In each metric, a first function of respective marker information for an instance of an analyte for a window is used to compute a respective value for each instance of the analyte in the window. A second function of these respective values is computed to provide one or more values for the one or more metrics for the window.

IPC Classes  ?

• G16B 40/10 - Signal processing, e.g. from mass spectrometry [MS] or from PCR

Abstract

Low background regions (LBRs) refer to genomic regions comprising one or more CpG sites that are differentially methylated in cancer and non-cancer samples. Disclose methods involve identifying LBRs comprising one or more CpG sites whose methylation statuses sufficiently distinguish between samples that contain a presence of a cancer signature and samples that do not contain a presence of a cancer signature. LBRs enable improved detection of presence or absence of cancer signatures while using smaller samples and fewer consumable reagents.

IPC Classes  ?

• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof
• G01N 33/574 - ImmunoassayBiospecific binding assayMaterials therefor for cancer
• G16B 40/00 - ICT specially adapted for biostatisticsICT specially adapted for bioinformatics-related machine learning or data mining, e.g. knowledge discovery or pattern finding
• G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems

Abstract

e.g.,e.g.,e.g.,e.g.,e.g., plastic products).

IPC Classes  ?

• C12N 9/18 - Carboxylic ester hydrolases
• C08J 11/10 - Recovery or working-up of waste materials of polymers by chemically breaking down the molecular chains of polymers or breaking of crosslinks, e.g. devulcanisation
• C12N 15/52 - Genes encoding for enzymes or proenzymes

Abstract

e.g.,e.g.,e.g.,e.g.,e.g., plastic products).

IPC Classes  ?

• C12N 9/18 - Carboxylic ester hydrolases
• C08J 11/10 - Recovery or working-up of waste materials of polymers by chemically breaking down the molecular chains of polymers or breaking of crosslinks, e.g. devulcanisation
• C12N 15/52 - Genes encoding for enzymes or proenzymes

Abstract

The present disclosure relates, generally, to methods for producing, purifying, and using circular RNA from a prokaryotic system.

IPC Classes  ?

• C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
• C12N 1/20 - BacteriaCulture media therefor
• C12N 9/00 - Enzymes, e.g. ligases (6.)ProenzymesCompositions thereofProcesses for preparing, activating, inhibiting, separating, or purifying enzymes
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

The invention relates generally to tRNA-based effector molecules (TREMs) comprising an asialoglycoprotein receptor (ASGPR) binding moiety, as well as compositions and methods relating thereto.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Analyzing whether an individual has a neoplasm, such as an early-stage cancer, is based on detection of novel cancer-associated biomarkers in a biological sample. In one aspect, a process begins with a liquid biopsy from the individual, e.g., a blood sample, which is subjected to several sample processing steps resulting in a processed sample, such as a next generation sequencing (NGS) sample library. The processed sample contains cancer-informative cell-free DNA (“cfDNA”) from the liquid biopsy. The processed sample is subjected to DNA sequencing, to detect certain low-abundance cfDNA, for example by using a next generation DNA sequencer. Sequencer data output from the DNA sequencer is processed by a data processing system to provide a classification for the individual. For example, the data processing system can determine a likelihood of the individual having a neoplasm such as an early-stage cancerous tumor based on that individual's liquid biopsy.

IPC Classes  ?

• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
• G16B 20/20 - Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection

Abstract

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 for treating cystic fibrosis, e.g., in subjects having a mutation resulting in F508del, are described.

IPC Classes  ?

• C12N 15/11 - DNA or RNA fragmentsModified forms thereof
• C12N 9/12 - Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
• C12N 9/22 - Ribonucleases
• C12N 15/90 - Stable introduction of foreign DNA into chromosome

Abstract

e.g.,e.g.,e.g.,e.g.,e.g.,e.g., genetic diseases).

IPC Classes  ?

• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
• C12N 9/22 - Ribonucleases
• C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression

Abstract

e.g.e.g.e.g., to treat diseases, such as cancer, or conditions involving dysregulated immune response or other disorders associated with aberrant AhR signaling.

IPC Classes  ?

• C07D 471/04 - Ortho-condensed systems
• A61P 35/00 - Antineoplastic agents
• A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings

Abstract

Disclosed herein are compositions (e.g., inhibitory agents, such as inhibitory nucleic acid molecules) and methods for treating subjects having or at risk of developing non-alcoholic fatty liver disease (NAFLD), such as non-alcoholic fatty liver (NAFL) or non-alcoholic steatohepatitis (NASH). Such methods can slow, inhibit, or even reverse further progression of NAFLD.

IPC Classes  ?

• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics

Abstract

Disclosed herein are methods, non-transitory computer readable media, systems, and kits for performing a multiple tiered analysis for identifying individuals with a health condition for monitoring, treating, and/or enrolling the individuals in a clinical trial. Specifically, the multiple tiered analysis involves a first screen, which eliminates a large proportion of individuals who are identified as not at risk for a health condition, and a subsequent second analysis which detects presence of a health condition in the remaining individuals. The second analysis includes an intra-individual analysis, which involves combining sequence information from target nucleic acids and reference nucleic acids obtained from the individual. The target nucleic acids include signatures that may be informative for determining presence or absence of the health condition and the reference nucleic acids include baseline biological signatures of the individual. Altogether, the multiple tiered analysis achieves improved performance and accurate identification of individuals with the health condition.

IPC Classes  ?

• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
• C12Q 1/6869 - Methods for sequencing
• G16B 5/20 - Probabilistic models
• G16B 20/20 - Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
• G16B 40/00 - ICT specially adapted for biostatisticsICT specially adapted for bioinformatics-related machine learning or data mining, e.g. knowledge discovery or pattern finding

Abstract

Macromolecule compositions and related methods that effect targeted delivery of therapeutic agents to effector targets in a desired cell, tissue and/or organ of interest while minimizing or avoiding undesirable delivery to other cells, tissues or organs are provided. Compositions and methods related to macromolecules, such as an ANDbody™, that include an effector target binding domain specific for an effector target, and an address binding domain specific for an address target are described.

IPC Classes  ?

• C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
• A61K 39/00 - Medicinal preparations containing antigens or antibodies
• C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
• C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
• C07K 16/40 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against enzymes

Abstract

Disclosed are lipidoid compounds having the structure of formula (I), or a pharmaceutically acceptable salt thereof: Disclosed are lipidoid compounds having the structure of formula (I), or a pharmaceutically acceptable salt thereof: Disclosed are lipidoid compounds having the structure of formula (I), or a pharmaceutically acceptable salt thereof: wherein the groups are as defined in the application. Also disclosed are nanoparticle compositions comprising a lipidoid of the invention that are capable of delivering a therapeutic agent. The application also discloses pharmaceutical compositions comprising a lipidoid composition of the invention.

IPC Classes  ?

• C07C 219/06 - Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
• A61K 9/51 - Nanocapsules
• A61K 39/00 - Medicinal preparations containing antigens or antibodies
• A61K 39/215 - Coronaviridae, e.g. avian infectious bronchitis virus
• C07C 229/16 - Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
• C07C 271/12 - Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals
• C07C 271/20 - Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to carbon atoms of hydrocarbon radicals substituted by nitrogen atoms not being part of nitro or nitroso groups
• C07C 323/52 - Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
• C07C 327/34 - Esters of monothiocarboxylic acids having sulfur atoms of esterified thiocarboxyl groups bound to carbon atoms of hydrocarbon radicals substituted by carboxyl groups with amino groups bound to the same hydrocarbon radicals
• C07C 329/06 - Esters of monothiocarbonic acids having sulfur atoms of thiocarbonic groups bound to acyclic carbon atoms

Abstract

Disclosed herein are compositions (e.g., inhibitory agents, such as inhibitory nucleic acid molecules) and methods for treating subjects having or at risk of developing non-alcoholic fatty liver disease (NAFLD), such as non-alcoholic fatty liver (NAFL) or non-alcoholic steatohepatitis (NASH). Such methods can slow, inhibit, or even reverse further progression of NAFLD.

IPC Classes  ?

• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
• A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics

Abstract

Disclosed are lipidoid compounds having the structure of formula (I), or a pharmaceutically acceptable salt thereof: formula (I) wherein the groups are as defined in the application. Also disclosed are nanoparticle compositions comprising a lipidoid of the invention that are capable of delivering a therapeutic agent. The application also discloses pharmaceutical compositions comprising a lipidoid composition of the invention.

IPC Classes  ?

• C07C 219/16 - Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having at least one of the hydroxy groups esterified by an inorganic acid or a derivative thereof
• C07C 271/12 - Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals
• C07C 271/20 - Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to carbon atoms of hydrocarbon radicals substituted by nitrogen atoms not being part of nitro or nitroso groups
• C07C 327/30 - Esters of monothiocarboxylic acids having sulfur atoms of esterified thiocarboxyl groups bound to carbon atoms of hydrocarbon radicals substituted by nitrogen atoms, not being part of nitro or nitroso groups
• C07C 329/06 - Esters of monothiocarbonic acids having sulfur atoms of thiocarbonic groups bound to acyclic carbon atoms

Abstract

Macromolecule compositions and related methods that effect targeted delivery of IL-22 polypeptides or fragments or variants thereof to effector targets in a desired cell, tissue and/or organ of interest while minimizing or avoiding undesirable delivery to other cells, tissues, or organs are provided. Compositions and methods related to macromolecules, such as an ANDbody™, that include an IL-22 polypeptide or fragment or variant thereof and an address binding domain specific for an IL-10 family address target are described. Also provided are IL-22 mutein polypeptides that have altered (e.g., decreased) affinity for the IL-22 receptor.

IPC Classes  ?

• C07K 14/54 - Interleukins [IL]
• A61K 38/20 - Interleukins
• A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
• C12N 15/62 - DNA sequences coding for fusion proteins
• C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells

Abstract

The disclosure provides, for example, a double stranded DNA (dsDNA) molecule comprising chemically modified nucleotides. In some embodiments, the dsDNA molecule comprises an upstream DNA end form which is a closed end, a downstream DNA end form which is a closed end, and a double stranded region comprising a sense strand and an antisense strand, in which the sense strand comprises one or more chemically modified nucleobases, and the antisense strand is free of chemically modified nucleobases.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

Abstract

Described herein are platforms for use in targeted therapy development for an individual or group of individuals. Targeted therapy development as described herein comprises developing a therapeutic or therapeutic modality that is specific to or customized to an individual or group of individuals.

IPC Classes  ?

• G16H 20/40 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to mechanical, radiation or invasive therapies, e.g. surgery, laser therapy, dialysis or acupuncture
• G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment

Abstract

The invention relates to methods of monitoring treatment response, disease resolution, and disease progression in subjects having inflammatory bowel disease (IBD).

IPC Classes  ?

• C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

Abstract

A database processing system performs a first database access call accessing a first construct representing a differential component amount between a normal and different state. This identifies a plurality of components and, for each, a corresponding first association between (a) a change in amount of the respective component in a first plurality of first component datasets and a second plurality of second component datasets and (b) a change in state between the normal and different state. A second database access call accesses a second construct representing a measure of differential component amount between a native and exposed sample. The second construct identifies all or a portion of the plurality of components and, for each, a corresponding second association between a change of amount of the respective component between a third and fourth plurality of component datasets. The first associations and corresponding second associations determine whether the entity affects the transition.

IPC Classes  ?

• G06F 16/28 - Databases characterised by their database models, e.g. relational or object models
• G16B 50/00 - ICT programming tools or database systems specially adapted for bioinformatics

Abstract

The invention relates to methods of predicting a patient's responsiveness to treatment (e.g., vedolizumab treatment) for inflammatory bowel disease (IBD).

IPC Classes  ?

• C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

Abstract

Provided herein are SARS-CoV-2 spike proteins and polypeptides (e.g., SARS-CoV-2 spike proteins and polypeptide immunogens (and immunogenic fragments and immunogenic variants thereof)) comprising at least one set of amino acid substitutions, and nucleic acid molecules encoding the same. Further provided herein are compositions (e.g., pharmaceutical compositions) and vaccines comprising the same for use in e.g., the prevention, treatment, and/or amelioration of a SARS-CoV-2 infection.

IPC Classes  ?

• A61K 39/215 - Coronaviridae, e.g. avian infectious bronchitis virus
• A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
• A61K 9/51 - Nanocapsules
• A61K 39/00 - Medicinal preparations containing antigens or antibodies
• B82Y 5/00 - Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
• C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
• C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof

Abstract

The disclosure provides, in various embodiments, polypeptides (e.g., antibodies and antigen binding fragments thereof) that specifically bind to a thymic stromal lymphopoietin (TSLP) (e.g., a full-length human TSLP). The disclosure also provides, in various embodiments, fusion proteins comprising one or more of the polypeptides, polynucleotides encoding the polypeptides, vectors and host cells suitable for expressing the polypeptides, and methods for treating a TSLP-associated disease or condition.

IPC Classes  ?

• C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
• A61P 11/06 - Antiasthmatics

Abstract

e.g.,e.g.,e.g.,e.g.,e.g.,e.g., genetic diseases).

IPC Classes  ?

• C12N 9/22 - Ribonucleases
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof
• C12N 15/52 - Genes encoding for enzymes or proenzymes
• C12N 15/62 - DNA sequences coding for fusion proteins

Abstract

Disclosed are lipidoid compounds having the structure of formula (X) or formula (I): wherein the groups are as defined in the application. Also disclosed are nanoparticle compositions comprising a lipidoid of the invention that are capable of delivering a therapeutic agent. The application also discloses pharmaceutical compositions comprising a lipidoid composition of the invention.

IPC Classes  ?

• C07C 237/20 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton containing six-membered aromatic rings
• C07C 237/08 - Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
• C07D 233/64 - Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with substituted hydrocarbon radicals attached to ring carbon atoms, e.g. histidine
• A61K 9/1272 - Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers

Abstract

In some embodiments, the subject matter of this disclosure relates to systems and methods for designing and conducting clinical trials and biomarker validation studies. An example method includes: obtaining access to a trained computer-implemented model; providing, to the trained computer-implemented model, a plurality of sets of values for a plurality of features, each set of values corresponding to a candidate individual from a set of candidate individuals; receiving, from the trained computer-implemented model, a prediction of a disease state for each set of values; identifying, from the predictions of the disease state, a group of participants from the set of candidate individuals; and facilitating at least one of a clinical trial or a biomarker validation study involving the group of participants.

IPC Classes  ?

• G16H 10/20 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for electronic clinical trials or questionnaires
• G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
• G16H 50/70 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for mining of medical data, e.g. analysing previous cases of other patients

Abstract

The invention relates to diagnostic and therapeutic methods for inflammatory bowel disease (IBD) and to physiologically acceptable compositions of isolated bacterial strains, lysates thereof, and supernatants therefrom for use in treating IBD.

IPC Classes  ?

• C12Q 1/689 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for detection or identification of organisms for bacteria

Abstract

The invention relates to diagnostic and therapeutic methods for colorectal cancer (CRC) and to physiologically acceptable compositions of isolated bacterial strains, lysates thereof, and supernatants therefrom for use in treating CRC.

IPC Classes  ?

• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
• C12Q 1/689 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for detection or identification of organisms for bacteria

Abstract

In some embodiments, the subject matter of this disclosure relates to systems and methods for designing and conducting clinical trials and biomarker validation studies. An example method includes: obtaining access to a trained computer-implemented model; providing, to the trained computer-implemented model, a plurality of sets of values for a plurality of features, each set of values corresponding to a candidate individual from a set of candidate individuals; receiving, from the trained computer-implemented model, a prediction of a disease state for each set of values; identifying, from the predictions of the disease state, a group of participants from the set of candidate individuals; and facilitating at least one of a clinical trial or a biomarker validation study involving the group of participants.

IPC Classes  ?

• G16H 10/20 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for electronic clinical trials or questionnaires

Abstract

Provided herein are Cas endonucleases (and functional fragments, functional variants, and domains thereof), nucleic acid molecules encoding the same, and systems comprising the same. The disclosure further relates to methods of utilizing the Cas endonucleases (or nucleic acid molecules encoding the same), including, e.g., in methods of editing a nucleic acid molecule (e.g., a gene) and methods of treating diseases (e.g., genetic diseases).

IPC Classes  ?

• C12N 15/90 - Stable introduction of foreign DNA into chromosome
• C12N 9/22 - Ribonucleases
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof

Abstract

Provided herein are Cas endonucleases (and functional fragments, functional variants, and domains thereof), nucleic acid molecules encoding the same, and systems comprising the same. The disclosure further relates to methods of utilizing the Cas endonucleases (or nucleic acid molecules encoding the same), including, e.g., in methods of editing a nucleic acid molecule (e.g., a gene) and methods of treating diseases (e.g., genetic diseases).

IPC Classes  ?

• C12N 9/22 - Ribonucleases
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof
• C12N 15/90 - Stable introduction of foreign DNA into chromosome

Abstract

e.g.,e.g.,e.g.,e.g., the prevention, treatment, and/or amelioration of a SARS-CoV-2 infection.

IPC Classes  ?

• A61K 39/12 - Viral antigens
• A61P 31/14 - Antivirals for RNA viruses

Abstract

Synthetic endornaviral satellite RNA molecules and satellite particles containing the same are disclosed. The synthetic endornaviral satellite RNA molecules can include coding and/or non-coding cargo sequences, and are heritable through generations of plants. Also disclosed are methods of using the endornaviral satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

The disclosure provides compositions, pharmaceutical preparations, and uses of circular polyribonucleotides complexed with one or more targeting moieties. The disclosure further discloses methods of using targeting moieties complexed with a circular polyribonucleotide to achieve functional delivery of a polypeptide to a cell.

IPC Classes  ?

• A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound

Abstract

Systems and methods for associating a compound with physiological conditions are provided. A fingerprint of a compound chemical structure is obtained and inputted to a model that outputs one or more calculated activation scores. Each activation score represents a cellular constituent module in a set of modules, where each module includes a subset of cellular constituents and a first module in the set of modules is associated with the physiological condition. When the activation score for the first module satisfies a threshold criterion, the compound is identified as associated with the physiological condition. In some aspects, each activation score represents a perturbation signature associated with the physiological condition and the compound is identified when the activation score for a first perturbation signature satisfies a threshold criterion. Systems and methods for training a model that associates compounds with physiological conditions are also provided.

IPC Classes  ?

• C12N 5/0789 - Stem cellsMultipotent progenitor cells
• A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
• A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
• G16C 20/30 - Prediction of properties of chemical compounds, compositions or mixtures
• G16C 20/70 - Machine learning, data mining or chemometrics

Abstract

Compositions comprising donor cells, acceptor cells, and methods involving the same are described herein. In some embodiments, the donor cell is deficient in at least one endogenous function, for instance cytotoxic activity. In some embodiments, the donor cell comprises a T cell receptor (TCR) and a cargo, and the acceptor cell comprises an MHC. In some embodiments, the TCR facilitates transfer of the cargo to the acceptor cell. In some embodiments, the donor cell comprises a chimeric antigen receptor (CAR) and a cargo, and the acceptor cell comprises an antigen bound by the CAR. In some embodiments, the CAR facilitates transfer of the cargo to the acceptor cell.

IPC Classes  ?

• C12N 15/90 - Stable introduction of foreign DNA into chromosome
• A61K 39/00 - Medicinal preparations containing antigens or antibodies
• C07K 14/725 - T-cell receptors
• C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
• C12N 9/22 - Ribonucleases
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof

Abstract

Disclosed herein are methods and compositions for predicting tumor content e.g., tumor content in a sample and tumor burden of an individual. Generally, methods and compositions involve determining methylation statuses of two or more sequential CpG sites in one or more genomic locations using nucleic acids of the obtained sample. The methylation statuses of the two or more sequential CpG in genomic locations are used to distinguish between reads of nucleic acids that are likely derived from cancer and reads of other nucleic acids that are unlikely to be derived from cancer. Distinguishing reads that are likely derived from cancer enables the prediction of tumor content in a sample.

IPC Classes  ?

• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
• G16B 20/20 - Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection

Abstract

The present disclosure relates to compositions and methods for separating and/or purifying polyribonucleotides. The polyribonucleotide may be separated from a mixture of polyribonucleotides with an oligonucleotide that hybridizes to a target region of the polyribonucleotide.

IPC Classes  ?

• C07H 21/02 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
• A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
• C07H 1/08 - SeparationPurification from natural products
• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA

Abstract

This disclosure provides compositions, pharmaceutical preparations, and methods relating to circular polyribonucleotides encoding an immunogen and a multimerization domain useful in the development and production of vaccines.

IPC Classes  ?

• A61K 39/215 - Coronaviridae, e.g. avian infectious bronchitis virus
• A61K 39/00 - Medicinal preparations containing antigens or antibodies
• A61K 39/145 - Orthomyxoviridae, e.g. influenza virus
• C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
• C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof

Abstract

e.g.,e.g.,e.g.,e.g., genetic diseases).

IPC Classes  ?

• C12N 9/22 - Ribonucleases
• C12N 15/52 - Genes encoding for enzymes or proenzymes
• C12N 15/62 - DNA sequences coding for fusion proteins
• A61K 38/45 - Transferases (2)
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
• C12N 9/12 - Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
• C12N 9/78 - Hydrolases (3.) acting on carbon to nitrogen bonds other than peptide bonds (3.5)

Abstract

e.g.,e.g.,e.g.,e.g., genetic diseases).

IPC Classes  ?

• C12N 9/22 - Ribonucleases

Abstract

The disclosure provides, for example, single stranded, covalently closed DNA that does not form a double stranded structure longer than 100 base pairs. The ssDNA may encode an effector sequence, for instance a therapeutic protein. The ssDNA may comprise a nuclear targeting sequence (NTS). In some embodiments, the ssDNA shows decreased activation of the innate immune system compared to an otherwise similar dsDNA.

IPC Classes  ?

• C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
• A61K 9/51 - Nanocapsules
• A61K 38/21 - Interferons
• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides

Abstract

Described herein are platforms for use in targeted therapy development for an individual or group of individuals. Targeted therapy development as described herein comprises developing a therapeutic or therapeutic modality that is specific to or customized to an individual or group of individuals.

IPC Classes  ?

• G16H 20/40 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to mechanical, radiation or invasive therapies, e.g. surgery, laser therapy, dialysis or acupuncture
• G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment

Abstract

Plasmodiae.g.,Plasmodiae.g.,Plasmodia e.g.,e.g., in pharmaceutical compositions and methods of treating diseases.

IPC Classes  ?

• A61P 33/06 - Antimalarials
• C12N 1/10 - ProtozoaCulture media therefor
• C12R 1/90 - Protozoa

Abstract

The invention relates generally to tRNA-based effector molecules having a non-naturally occurring modification and methods relating thereto.

IPC Classes  ?

• A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
• A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
• A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof
• C12N 15/67 - General methods for enhancing the expression

Abstract

The present disclosure relates, generally, to compositions and methods for producing, purifying, and using circular RNA encoding an antifusogenic polypeptide.

IPC Classes  ?

• C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses
• A61K 35/00 - Medicinal preparations containing materials or reaction products thereof with undetermined constitution
• C07K 14/16 - HIV-1

Abstract

The present disclosure relates, inter alia, to perturbagens and methods, including ex vivo methods, for directing a change in the cell state of T cells, e.g., naïve T cells, effector T cells and exhausted T cells. The present disclosure also relates to methods for mitigating or preventing T cell exhaustion, including contacting cells with a perturbagen ex vivo. Further, the present disclosure relates to methods for treating diseases or disorders characterized by, e.g., production of exhausted T cells, production of abnormal number of exhausted T cells, or production of high number of T cells.

IPC Classes  ?

• A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
• A61P 35/00 - Antineoplastic agents
• G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing

Abstract

Provided herein are engineered Plasmodia comprising a heterologous nucleic acid molecule encoding a therapeutic protein and compositions (e.g., pharmaceutical compositions) comprising the same; as well as methods of making engineered Plasmodia comprising a heterologous nucleic acid molecule encoding a therapeutic protein and compositions (e.g., pharmaceutical compositions) comprising the same. The engineered Plasmodia provided herein are useful e.g., in pharmaceutical compositions and methods of treating diseases.

IPC Classes  ?

• C12N 15/79 - Vectors or expression systems specially adapted for eukaryotic hosts
• C07K 14/705 - ReceptorsCell surface antigensCell surface determinants
• C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons

Abstract

e.g.,e.g., tumor content in a sample and tumor burden of an individual. Generally, methods and compositions involve determining methylation statuses of two or more sequential CpG sites in one or more genomic locations using nucleic acids of the obtained sample. The methylation statuses of the two or more sequential CpG in genomic locations are used to distinguish between reads of nucleic acids that are likely derived from cancer and reads of other nucleic acids that are unlikely to be derived from cancer. Distinguishing reads that are likely derived from cancer enables the prediction of tumor content in a sample.

IPC Classes  ?

• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
• C12Q 1/6869 - Methods for sequencing
• G16B 20/20 - Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
• G16B 30/00 - ICT specially adapted for sequence analysis involving nucleotides or amino acids
• G16B 30/10 - Sequence alignmentHomology search
• G16B 40/20 - Supervised data analysis
• G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems

Abstract

The present disclosure relates to compositions and methods for separating and/or purifying polyribonucleotides. The polyribonucleotide may be separated from a mixture of polyribonucleotides with an oligonucleotide that hybridizes to a target region of the polyribonucleotide and be available for use as a therapeutic agent.

IPC Classes  ?

• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA

Abstract

Methods and compositions for modulating a target genome are disclosed.

IPC Classes  ?

• C12N 15/90 - Stable introduction of foreign DNA into chromosome
• C12N 9/22 - Ribonucleases
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof
• C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle

Abstract

Synthetic endornaviral satellite RNA molecules and satellite particles containing the same are disclosed. The synthetic endornaviral satellite RNA molecules can include coding and/or non-coding cargo sequences, and are heritable through generations of plants. Also disclosed are methods of using the endornaviral satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

e.g.,e.g.,e.g.,e.g.,e.g., the prevention, treatment, and/or amelioration of a SARS-CoV-2 infection.

IPC Classes  ?

• A61K 39/00 - Medicinal preparations containing antigens or antibodies

Abstract

In some embodiments, methods and corresponding systems are disclosed for providing associated biopolymer sequence(s) to conform to a reference structure. The reference structure includes a target complex and the one or more associated biopolymer sequences. The biopolymer sequences are obtainable by the method, including embedding a graph representation using a neural network. The graph representation is featurized from the reference structure and includes a topology of the biopolymer with monomers as nodes and interactions between monomers as edges. The methods, in certain embodiments, further include processing the graph representation with a graph neural network or equivariant neural network that iteratively updates node and edge embeddings with a learned parametric function. The methods may further include converting the embedded graph representation to an energy landscape using a decoder. The methods can further include obtaining one or more biopolymer sequences from the energy landscape.

IPC Classes  ?

• G16B 15/20 - Protein or domain folding
• G16B 15/30 - Drug targeting using structural dataDocking or binding prediction
• G16B 40/00 - ICT specially adapted for biostatisticsICT specially adapted for bioinformatics-related machine learning or data mining, e.g. knowledge discovery or pattern finding

Abstract

The disclosure provides compounds, in part, compounds of Formula (I) or Formula (II) and their use in treating medical diseases or disorders, such as neurodegenerative diseases, e.g., Parkinson's disease. Pharmaceutical compositions and methods of making compounds of the disclosure are provided. The compounds are contemplated to be modulators, e.g., inhibitors, of cyclic ADP ribose hydrolase (CD38).

IPC Classes  ?

• C07D 277/30 - Radicals substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
• C07D 401/04 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings directly linked by a ring-member-to-ring- member bond
• C07D 401/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
• C07D 403/04 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
• C07D 403/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
• C07D 405/14 - Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
• C07D 417/04 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
• C07D 491/048 - Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
• A61K 31/4353 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
• A61P 25/00 - Drugs for disorders of the nervous system

Abstract

The present disclosure is directed to methods for the enrichment of circular polyribonucleotides (circRNA), e.g., from population of polyribonucleotides containing circRNA and linear polyribonucleotides (linRNA), where the enrichment is performed under denaturing conditions. Also disclosed are compositions including a population of polyribonucleotides containing circRNA and linRNA in a solution under denaturing conditions. Further within the scope of the present disclosure are compositions containing an enriched population of circRNA, such as a composition that was produced by exposing the composition to one or more denaturing conditions.

IPC Classes  ?

• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof

Abstract

This disclosure relates generally to membrane-bound compositions, in particular exophers having a diameter between 1 and 20 microns induced from human cells, and uses thereof.

IPC Classes  ?

• C12N 5/0793 - Neurons
• C12N 5/077 - Mesenchymal cells, e.g. bone cells, cartilage cells, marrow stromal cells, fat cells or muscle cells

Abstract

The invention relates generally to tRNA-based effector molecules having a non-naturally occurring modification and methods relating thereto.

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 9/51 - Nanocapsules

Abstract

Disclosed herein are plant messenger packs (PMPs) encapsulating one or more exogenous polypeptides. Also disclosed are methods of producing a PMP comprising an exogenous polypeptide.

IPC Classes  ?

• A61K 36/28 - Asteraceae or Compositae (Aster or Sunflower family), e.g. chamomile, feverfew, yarrow or echinacea
• A61K 9/00 - Medicinal preparations characterised by special physical form
• A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
• A61K 9/19 - Particulate form, e.g. powders lyophilised
• A61K 31/164 - Amides, e.g. hydroxamic acids of a carboxylic acid with an aminoalcohol, e.g. ceramides
• A61K 36/31 - Brassicaceae or Cruciferae (Mustard family), e.g. broccoli, cabbage or kohlrabi
• A61K 36/752 - Citrus, e.g. lime, orange or lemon
• A61K 36/754 - Evodia
• A61K 36/87 - Vitaceae or Ampelidaceae (Vine or Grape family), e.g. wine grapes, muscadine or peppervine
• A61K 38/28 - Insulins
• C07K 14/475 - Growth factorsGrowth regulators
• C07K 14/52 - CytokinesLymphokinesInterferons
• C07K 14/575 - Hormones
• C07K 14/62 - Insulins
• C07K 14/705 - ReceptorsCell surface antigensCell surface determinants
• C07K 14/81 - Protease inhibitors

Abstract

The invention relates to diagnostic and therapeutic methods for inflammatory bowel disease. Disclosed is a method of differentiating between ulcerative colitis (UC) and Crohn's disease (CD) in a subject having an inflammatory bowel disease (IBD) comprising determining a level of one or more of SEQ ID NOs: 1-590 in a sample from the subject.

IPC Classes  ?

• C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

Abstract

The invention relates to diagnostic and therapeutic methods for colorectal cancer.

IPC Classes  ?

• C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
• C12Q 1/689 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for detection or identification of organisms for bacteria

Abstract

The invention relates to diagnostic and therapeutic methods for inflammatory bowel disease (IBD).

IPC Classes  ?

• C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

Abstract

Methods and compositions for modulating a target genome are disclosed.

IPC Classes  ?

• A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
• C12N 9/22 - Ribonucleases
• C12N 15/11 - DNA or RNA fragmentsModified forms thereof

Abstract

The present disclosure relates, inter alia, to perturbagens and methods for directing a change or inhibiting a change in the cell state of a hematopoietic progenitor cell. It also relates to methods for increasing or decreasing a quantity of neutrophils, monocytes or immediate progenitors thereof and/or the ratios thereof. Further, the present disclosure relates to methods for treating diseases or disorders characterized by, at least, abnormal ratios of neutrophils to monocytes and/or abnormal numbers thereof.

IPC Classes  ?

• A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
• A61K 31/166 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the carbon atom of a carboxamide group directly attached to the aromatic ring, e.g. procainamide, procarbazine, metoclopramide, labetalol
• A61K 31/167 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the nitrogen atom of a carboxamide group directly attached to the aromatic ring, e.g. lidocaine, paracetamol
• A61K 31/18 - Sulfonamides
• A61K 31/341 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide not condensed with another ring, e.g. ranitidine, furosemide, bufetolol, muscarine
• A61K 31/35 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom
• A61K 31/365 - Lactones
• A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
• A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
• A61K 31/513 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
• A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
• A61K 31/535 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and at least one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
• G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing

Abstract

Synthetic Martellivirales satellite RNA molecules and satellite particles containing the same are disclosed. Synthetic Martellivirales satellite RNA molecules which contain internal heterologous RNA virus (HRV) amplicons are also disclosed. Also disclosed are methods of using the Martellivirales satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

Synthetic Ghabrivirales satellite RNA molecules and satellite particles containing the same are disclosed. Synthetic Ghabrivirales satellite RNA molecules which contain internal heterologous RNA virus (HRV) amplicons are also disclosed. Also disclosed are methods of using the Ghabrivirales satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

Synthetic endornavirus satellite RNA molecules and satellite particles containing the same are disclosed. Synthetic endornavirus satellite RNA molecules which contain internal heterologous TNA virus (HRV) amplicons are also disclosed. Also disclosed are methods of using the endornavirus satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

Synthetic Tombusviridae satellite RNA molecules and satellite particles containing the same are disclosed. Synthetic Tombusviridae satellite RNA molecules which contain internal heterologous RNA virus (HRV) amplicons are also disclosed. Also disclosed are methods of using the Tombusviridae satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

Synthetic Secoviridae satellite RNA molecules and satellite particles containing the same are disclosed. Synthetic Secoviridae satellite RNA molecules which contain internal heterologous RNA virus (HRV) amplicons are also disclosed. Also disclosed are methods of using the Secoviridae satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

Synthetic Solemoviridae satellite RNA molecules and satellite particles containing the same are disclosed. Synthetic Solemoviridae satellite RNA molecules which contain internal heterologous RNA virus (HRV) amplicons are also disclosed. Also disclosed are methods of using the Solemoviridae satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

Synthetic Tymovirales satellite RNA molecules and satellite particles containing the same are disclosed. Synthetic Tymovirales satellite RNA molecules which contain internal heterologous RNA virus (HRV) amplicons are also disclosed. Also disclosed are methods of using the Tymovirales satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

Synthetic partitivirus satellite RNA molecules and satellite particles containing the same are disclosed. Synthetic partitivirus satellite RNA molecules which contain internal heterologous TNA virus (HRV) amplicons arc also disclosed. Also disclosed arc methods of using the partitivirus satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

Synthetic amalgavirus satellite RNA molecules and satellite particles containing the same are disclosed. Synthetic amalgavirus satellite RNA molecules which contain internal heterologous RNA virus (HRV) amplicons are also disclosed. Also disclosed are methods of using the amalgavirus satellite RNA molecules and satellite particles containing the same to change plant phenotypes, improve plant stress resistance, and improve plant pest and pathogen resistance.

IPC Classes  ?

• C12N 15/82 - Vectors or expression systems specially adapted for eukaryotic hosts for plant cells

Abstract

The present disclosure provides methods for increasing the quantity and/or the ratios of erythroblasts, reticulocytes, and/or erythrocytes, or progenitors thereof, in which any of these cells express HbF (e.g. HbF+ and/or HbFhigh cells); increasing the quantity of erythrocytes and/or the ratios of erythrocytes to other related cells. The present disclosure relates, inter alia, to perturbagens and methods for directing a change in the cell state of a progenitor cell. The present disclosure further provides methods for treating diseases or disorders characterized by, for example, oxygen delivery deficiencies and/or reduced expression and/or activity of hemoglobin; abnormal erythron distribution and/or physiology or an erythrocyte deficiency; and diseases or disorders characterized by, at least, abnormal ratios and/or abnormal numbers of megakaryocytes, proplatelets, and/or platelets or immediate progenitors thereof.

IPC Classes  ?

• C12N 5/078 - Cells from blood or from the immune system
• A61K 35/18 - Erythrocytes
• A61K 35/19 - PlateletsMegacaryocytes
• A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid

Abstract

The present disclosure relates, inter alia, to perturbagens and methods for directing a change in the cell state of an intestinal stem cell and/or a basal cell. It also relates to methods for increasing a quantity of goblet progenitors, goblet cells, Paneth cells, and/or enteroendocrine cells or immediate progenitors thereof and/or the ratios thereof, and methods for decreasing the function and quantity of goblet cells or immediate progenitors thereof. Further, the present disclosure relates to methods for treating diseases or disorders characterized by, at least, abnormal function, abnormal ratios and/or abnormal numbers of goblet progenitors, goblet cells, Paneth cells, and/or enteroendocrine cells, or immediate progenitors thereof.

IPC Classes  ?

• G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing
• A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
• C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

Abstract

Provided herein are polynucleotides (e.g., plasmids), including transfer polynucleotides and landing pad polynucleotides, which are useful, e.g., in the generation of cell and virion libraries each expressing and encoding a protein of interest (e.g., viral entry proteins). The libraries described herein are further useful, e.g., in methods of assessing functional characteristics of the proteins of interest (e.g., neutralization of viral entry proteins by one or more antibodies).

IPC Classes  ?

• C12N 15/867 - Retroviral vectors
• C07K 14/015 - Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
• C07K 14/155 - Lentiviridae, e.g. human immunodeficiency virus [HIV], visna-maedi virus or equine infectious anaemia virus
• C12N 15/864 - Parvoviral vectors

Abstract

e.g.,e.g.,e.g.,e.g.,e.g.,e.g., neutralization of viral entry proteins by one or more antibodies).

IPC Classes  ?

• C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
• C12N 15/64 - General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
• C12N 15/86 - Viral vectors

Abstract

inter aliae.g.,e.g.,e.g.,e.g.,e.g.,e.g.,e.g., nonalcoholic steatohepatitis (NASH) and nonalcoholic fatty liver disease (NAFLD)).

IPC Classes  ?

• C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
• A61K 31/713 - Double-stranded nucleic acids or oligonucleotides