The invention relates to methods of monitoring treatment response, disease resolution, and disease progression in subjects having inflammatory bowel disease (IBD).
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
2.
DIAGNOSIS AND TREATMENT OF DISEASES AND CONDITIONS OF THE INTESTINAL TRACT
The invention relates to methods of predicting a patient's responsiveness to treatment (e.g., vedolizumab treatment) for inflammatory bowel disease (IBD).
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
3.
Database processing system for determining whether an entity affects a transition
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.
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.
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
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.
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.
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
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
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 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
8.
SYSTEMS AND METHODS FOR DESIGNING AND CONDUCTING CLINICAL TRIALS AND BIOMARKER VALIDATION STUDIES
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.
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
9.
COMPOSITIONS AND METHODS FOR TREATING INFLAMMATORY BOWEL DISEASE
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.
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
10.
COMPOSITIONS AND METHODS FOR TREATING COLORECTAL CANCER
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.
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
11.
SYSTEMS AND METHODS FOR DESIGNING AND CONDUCTING CLINICAL TRIALS AND BIOMARKER VALIDATION STUDIES
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.
G16H 10/20 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for electronic clinical trials or questionnaires
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).
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).
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.
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.
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
17.
SYSTEMS AND METHODS FOR ASSOCIATING COMPOUNDS WITH PHYSIOLOGICAL CONDITIONS USING FINGERPRINT ANALYSIS
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.
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.
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.
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
20.
COMPOSITIONS AND METHODS FOR PURIFYING POLYRIBONUCLEOTIDES
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.
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
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.
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.
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.
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
The present disclosure relates, generally, to compositions and methods for producing, purifying, and using circular RNA encoding an antifusogenic polypeptide.
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.
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.
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
31.
METHODS FOR PREDICTING TUMOR CONTENT USING EPIGENETIC SIGNATURES
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.
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
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
32.
COMPOSITIONS AND METHODS FOR PURIFYING POLYRIBONUCLEOTIDES
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.
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
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.
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.
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
37.
CONDENSED AZINES AS INHIBITORS OF CYCLIC ADP RIBOSE HYDROLASE
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).
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
38.
METHODS FOR ENRICHMENT OF CIRCULAR RNA UNDER DENATURING CONDITIONS
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.
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.
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.
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.
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
43.
DIAGNOSIS AND TREATMENT OF DISEASES AND CONDITIONS OF THE INTESTINAL TRACT
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
44.
DIAGNOSIS AND TREATMENT OF DISEASES AND CONDITIONS OF THE INTESTINAL TRACT
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
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.
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 31/341 - 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 31/4184 - 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
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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).
Provided herein are macromolecules that conditionally induce a cellular effector function (e.g., a biological or therapeutic activity) based on the presence of a disease signature ligand, compositions comprising the same, and methods of using the same.
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
C07K 14/71 - ReceptorsCell surface antigensCell surface determinants for growth factorsReceptorsCell surface antigensCell surface determinants for growth regulators
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
The present application is directed to methods for identifying and characterizing molecules that mimic binding sites of specific binders on various binding surfaces.
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
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
The invention relates generally to tRNA-based effector molecules for use in inserting a missense mutation into an open reading frame (ORF) of a gene, e.g., for treatment of a repeat expansion disease.
The invention relates generally to tRNA-based effector molecules comprising a locked nucleotide acid (LNA) moiety, as well as compositions and methods relating thereto.
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
Methods and compositions for modulating a target genome are disclosed. The composition may comprise a first RNA encoding a polypeptide comprising a retrotransposase reverse transcriptase domain and a retrotransposase endonuclease domain. The composition may also comprise a second RNA comprising a sequence that binds the polypeptide and a heterologous object sequence. The composition may insert the sequence of the heterologous object sequence into a target DNA.
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 phenylketonuria (PKU) are described.
The present disclosure features compositions including polyribonucleotides having one or more expression augmenting elements or spacer elements. The polyribonucleotides described herein may increase the stability of the polyribonucleotide and/or may increase the expression of a polynucleotide cargo encoded by the polyribonucleotide.
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. Improved gRNA scaffolds compatible with St1Cas9 are described.
The present disclosure features compositions including polyribonucleotides having one or more expression augmenting elements or spacer elements. The polyribonucleotides described herein may increase the stability of the polyribonucleotide and/or may increase the expression of a polynucleotide cargo encoded by the polyribonucleotide.
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 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
Disclosed herein are compositions including a plurality of plant messenger packs, (e.g., including a plant extracellular vesicle (EV), or segment, portion, or extract thereof), that are modified to have enhanced cell uptake (e.g., animal plant cell uptake, bacterial cell uptake, or fungal cell uptake), e.g., for use in a variety of agricultural or therapeutic methods.
A01N 25/22 - Biocides, pest repellants or attractants, or plant growth regulators, characterised by their forms, or by their non-active ingredients or by their methods of applicationSubstances for reducing the noxious effect of the active ingredients to organisms other than pests containing ingredients stabilising the active ingredients
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
74.
CLEAVABLE LINKER-CONTAINING IONIZABLE LIPIDS AND LIPID CARRIERS FOR THERAPEUTIC COMPOSITIONS
The present disclosure relates to a lipid compound of formula (Ia) or (AL-GI):
The present disclosure relates to a lipid compound of formula (Ia) or (AL-GI):
The present disclosure relates to a lipid compound of formula (Ia) or (AL-GI):
having various cleavable linkers defined by the variables Z1 and Z2 and Z10 and Z20. The present disclosure also relates to a lipid carrier or lipid nanoformulation employing the lipid compound, and the use of the lipid compound in a pharmaceutical composition as well as for a method of delivering an effector, e.g., a therapeutic agent.
C07D 241/08 - Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having one or two double bonds between ring members or between ring members and non-ring members with oxygen atoms directly attached to ring carbon atoms
C07D 251/10 - Heterocyclic compounds containing 1,3,5-triazine rings not condensed with other rings having two double bonds between ring members or between ring members and non-ring members
C07D 265/32 - 1,4-OxazinesHydrogenated 1,4-oxazines not condensed with other rings with oxygen atoms directly attached to ring carbon atoms
This disclosure provides, for example, double stranded DNA (dsDNA) molecules comprising a chemically modified cytosine nucleotide. In some embodiments, the dsDNA molecules comprise a therapeutic payload sequence. In some embodiments, the dsDNA molecules are resistant to endonuclease digestion and/or resistant to immune sensor recognition, and supports expression of a therapeutic payload encoded in the dsDNA molecules. The disclosure also provides, for example, pharmaceutical compositions comprising dsDNA molecules comprising a chemically modified cytosine nucleotide.
This disclosure provides, for example, double stranded DNA (dsDNA) molecules comprising a chemically modified uridine nucleotide. In some embodiments, the dsDNA molecules comprise a therapeutic payload sequence. In some embodiments, the dsDNA molecules are resistant to endonuclease digestion and/or resistant to immune sensor recognition, and supports expression of a therapeutic payload encoded in the dsDNA molecules. The disclosure also provides, for example, pharmaceutical compositions comprising dsDNA molecules comprising a chemically modified uridine nucleotide.
This disclosure provides, for example, double stranded DNA (dsDNA) molecules comprising a chemically modified uridine nucleotide. In some embodiments, the dsDNA molecules comprise a therapeutic payload sequence. In some embodiments, the dsDNA molecules are resistant to endonuclease digestion and/or resistant to immune sensor recognition, and supports expression of a therapeutic payload encoded in the dsDNA molecules. The disclosure also provides, for example, pharmaceutical compositions comprising dsDNA molecules comprising a chemically modified uridine nucleotide.
This disclosure provides, for example, double stranded DNA (dsDNA) molecules comprising a chemically modified cytosine nucleotide. In some embodiments, the dsDNA molecules comprise a therapeutic payload sequence. In some embodiments, the dsDNA molecules are resistant to endonuclease digestion and/or resistant to immune sensor recognition, and supports expression of a therapeutic payload encoded in the dsDNA molecules. The disclosure also provides, for example, pharmaceutical compositions comprising dsDNA molecules comprising a chemically modified cytosine nucleotide.
Provided herein are, inter alia, compositions (e.g., vaccine compositions (e.g., vaccine booster compositions)) comprising a hIL-10R binding agent (e.g., a hIL-10R binding protein (or a nucleic acid molecule comprising the same)) and optionally an immunogen (e.g., an immunogenic protein (or a nucleic acid molecule encoding the same)). Further provided herein are methods of utilizing hIL-10R binding agents (e.g., hIL-10R binding proteins (or nucleic acid molecules comprising the same)), including, e.g., in methods of vaccination, e.g., as vaccine boosters.
C07K 14/44 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from protozoa
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
Methods and compositions for modulating a target genome are disclosed. For instance, the disclosure provides site-specific recombinases (e.g., serine recombinases, e.g., serine integrases) capable of directing insertion of an insert DNA, or portion thereof, into a desired site in a target genome.
The disclosure provides antifungal compositions comprising conidial germination-inhibiting (CGI) factors, CGI factor precursors, CGI factor fragments, and CGI factor motifs and fungicides. In addition, the disclosure provides methods of controlling or inhibiting fungal growth and infection, as well as methods of treating a fungal disease using the compositions comprising CGI factors, CGI factor precursors, CGI factor fragments, and CGI factor motifs and fungicides.
Disclosed herein are viroid-derived polynucleotides for the modification of plants and methods of using such polynucleotides in a variety of agricultural and commercial methods. Specifically, the disclosure provides a method of delivering to a plant a composition comprising a recombinant polynucleotide comprising: (i) a single-stranded RNA (ssRNA) viroid sequence and (ii) a heterologous RNA sequence comprising or encoding an effector, wherein the effector has a biological effect on the plant, and wherein the viroid is potato spindle tuber viroid (PSTVd) or eggplant latent viroid (ELVd).
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 sickle cell disease (SCD) are described.
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.
The methods of the disclosure can be used to enrich a population of circular polyribonucleotides in a mixture of linear polyribonucleotides and circular polyribonucleotides.
The methods of the disclosure can be used to enrich a population of circular polyribonucleotides in a mixture of linear polyribonucleotides, circular polyribonucleotides, and linear polydeoxyribonucleotides.
Methods of associating a test compound with a compound property. One or more datasets including: for each of a plurality of cell lines and each of a plurality of compounds: for each respective exposure condition: a corresponding response signature for the respective compound in the respective cell line under the respective exposure condition is obtained. A correlation is determined for each unique combination of exposure conditions for a respective pair of compounds based on the corresponding response signature. A weight is determined for each respective pair of compounds based on the determined correlations. A plurality of compound clusters is formed, where each cluster represents compounds that satisfy one or more weight criteria with respect to a particular compound. A compound property of the test compound is determined from properties of one or more compounds in a one or more compound clusters that contain the test compound.
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 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
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. Heterologous gene modifying systems for disrupting the TRAC gene and/or the B2M gene are described.
C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
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 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
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.
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 40/00 - ICT specially adapted for biostatisticsICT specially adapted for bioinformatics-related machine learning or data mining, e.g. knowledge discovery or pattern finding
Disclosed herein are methods, non-transitory computer readable media, and systems for determining a signal informative for presence or absence of a cancer in a sample. Generally, the signal includes phased sequencing information of cell-free DNA in which methylation sequence information and/or mutation sequence information can be attributed to various sources (e.g., to a maternal chromosome or to a paternal chromosome). Individual-specific differences between the maternal and paternal chromosomes can be informative markers to create haplotype-specific sequence information (e.g., phase sequencing information) informative for presence or absence of cancer.
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
96.
COMPOSITIONS AND METHODS FOR DELIVERY OF THERAPEUTIC AGENTS TO BONE
Macromolecule compositions and related methods that effect targeted delivery of therapeutic agents to effector targets in bone tissue 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 bone tissue are described.
A61K 47/68 - 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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 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
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
Compositions comprising donor cells, acceptor cells, and methods involving the same are described herein. 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.
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.
Disclosed herein are achromosomal dynamic active system (ADAS) derived from a parent bacterial cell, comprising at least one genetic loss-of-function alteration in a lytic enzyme to increase the stability of ADAS. Also disclosed are methods of interrupting sporulation in parent bacterial cells in combination with a lytic enzyme deletion or other loss-of-function mutation.
patents
