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Analysis

1 / 5,938
1.WO/2024/153118IMPLEMENTATION OF DIRECTED EVOLUTION AND DARWINIAN ADAPTATION IN MAMMALIAN CELLS BY MEANS OF REPLICABLE RNA
WO - 25.07.2024
Int.Class C12N 15/40
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
31Genes encoding microbial proteins, e.g. enterotoxins
33Genes encoding viral proteins
40Proteins from RNA viruses, e.g. flaviviruses
 Appl.No PCT/CN2024/072737 Applicant PEKING UNIVERSITY Inventor MA, Liang
An orthogonal alphavirus RNA replication system, i.e., an RNA replicase-assisted continuous in vivo evolution (REPLACE) system, and an REPLACE method. An RNA-based evolution element makes REPLACE of mammalian cells possible. According to the method, more than one billion replicable RNA copies can be continuously diversified and selected, and RNA replication and powerful inducible RNA mutation by means of replicase restriction are achieved. According to the method, new functions of fluorescent proteins, transcription factors and miniature Cas proteins (dCasMINI) are evolved or existing functions are improved. Cells equipped with REPLACE can adapt to challenges from the exterior or interior of the cells by continuously evolving the replicable RNA carrying cancer-related key genes (i.e., MEK1 and KRAS). An RNA-based evolution platform will provide a new high-performance tool kit for mammalian synthetic biology, and assist in adaptive engineering modification of mammalian cells and tissues.
2.WO/2024/155938LIPIDOID COMPOUNDS AND RELATED COMPOSITIONS AND USES
WO - 25.07.2024
Int.Class C07C 219/12
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
CACYCLIC OR CARBOCYCLIC COMPOUNDS
219Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
02having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
04the carbon skeleton being acyclic and saturated
12having at least one of the hydroxy groups esterified by a carboxylic acid having the esterifying carboxyl group bound to a carbon atom of a ring other than a six-membered aromatic ring
 Appl.No PCT/US2024/012245 Applicant POSEIDA THERAPEUTICS, INC. Inventor RYCHAK, Joshua
Compositions comprising lipidoid compounds, methods of preparing such compositions, and the use of these compositions in gene delivery applications are disclosed.
3.WO/2024/153247COMPOUND AS CTPS1 INHIBITOR
WO - 25.07.2024
Int.Class C07D 401/14
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
401Heterocyclic 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
14containing three or more hetero rings
 Appl.No PCT/CN2024/073373 Applicant HANGZHOU INNOGATE PHARMA CO., LTD. Inventor ZHANG, Hancheng
Provided in the present invention is a class of compounds. Specifically, provided in the present invention is a compound having a structure as shown in formula (I), or an optical isomer, pharmaceutically acceptable salt, prodrug, deuterated derivative, hydrate and solvate thereof. The compound can effectively inhibit CTPS1, and is used for treating or preventing diseases or disorders associated with the activity or expression quantity of CTPS1.
4.WO/2024/153953mRNA COMPOSITIONS
WO - 25.07.2024
Int.Class A61K 39/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
 Appl.No PCT/GB2024/050155 Applicant SISAF LTD Inventor SAFFIE-SIEBERT, Roghieh Suzanne
Disclosed is an mRNA vector composition, comprising: mRNA; one or more particles comprising hydrolysable silicon; and one or more lipids, characterised in that the mRNA is mRNA which is not chemically modified. Also disclosed are related products, methods and uses.
5.WO/2024/153324RNA FORMULATIONS FOR PHARMACEUTICAL USE
WO - 25.07.2024
Int.Class C12N 15/11
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
 Appl.No PCT/EP2023/051117 Applicant BIONTECH SE Inventor SAHIN, Ugur
The present disclosure relates to RNA comprising one or more miRNA binding sequences, wherein the one or more miRNA binding sequences bind to miRNA that is present in cells in which expression of the RNA is not desired. Delivering the RNA to cells after administration, in particular after intramuscular or intravenous administration, allows expression of a polypeptide encoded by the RNA in certain cells while expression in other cells is repressed. In some embodiments, such cells comprise endothelial cells. RNA compositions described herein allow expression of a pharmaceutically active peptide or polypeptide by the RNA in a subject while reducing or avoiding the risks of undesired effects resulting from expression of the pharmaceutically active peptide or polypeptide in certain cells or tissues.
6.WO/2024/155739POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR TREATMENT OF NEURODEGENERATIVE DISEASES
WO - 25.07.2024
Int.Class C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
 Appl.No PCT/US2024/011890 Applicant APTAH BIO, INC. Inventor BRUNO QUINTA DE SOUZA LEAL, Caio
This disclosure concerns an engineered polynucleotide that interacts with a pre-mRNA and a spliceosome to regulate gene expression. The engineered polynucleotide may have stem- loop structure that recruits the spliceosome and targeting sequences that are complementary to a target sequence at an exon-intron splice junction and may include nucleotides with 2' modifications and phosphorothioate linkages. The engineered polynucleotide can be administered to a subject to treat a neurodegenerative disease.
7.WO/2024/155740POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR TREATMENT OF CANCER
WO - 25.07.2024
Int.Class C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
 Appl.No PCT/US2024/011891 Applicant APTAH BIO, INC. Inventor BRUNO QUINTA DE SOUZA LEAL, Caio
This disclosure provides an engineered polynucleotide that interacts with a pre-mRNA and a spliceosome to regulate gene expression. The engineered polynucleotide may have stem¬ loop structure that recruits the spliceosome and targeting sequences that are complementary to a target sequence at an exon-intron splice junction and may include nucleotides with 2' modifications and phosphorothioate linkages. The engineered polynucleotide can be administered to a subject to treat a cancer.
8.WO/2024/155770COMPOSITIONS FOR MODULATING KRAS EXPRESSION AND USES THEREOF
WO - 25.07.2024
Int.Class C12N 15/85
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
 Appl.No PCT/US2024/011938 Applicant MOLECULAR AXIOM, LLC Inventor CHEN, Lishan
Described herein are compounds, compositions, and methods for modulating KRAS expression, such as mutated KRAS expression, and/or downstream signaling pathways. Also described herein are compounds, compositions, and methods for treating a disease or condition associated with mutated KRAS. In some embodiments, the compound comprises at least one antisense oligonucleotide that, upon being delivered into a cell, hybridizes to an endogenous KRAS mRNA, which leads to the degradation of the KRAS mRNA. In some embodiments, the antisense oligonucleotide hybridizes to an mRNA encoding a mutated KRAS protein, such as a KRAS protein comprising a G12C mutation, a G12V mutation, a G12D mutation, or a G12A mutation.
9.WO/2024/155741PRIME EDITING-MEDIATED READTHROUGH OF PREMATURE TERMINATION CODONS (PERT)
WO - 25.07.2024
Int.Class C12N 15/11
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
 Appl.No PCT/US2024/011892 Applicant THE BROAD INSTITUTE, INC. Inventor LIU, David, R.
Aspects of the disclosure relate to methods, compositions, and systems for editing an endogenous tRNA into a suppressor tRNA, or alternatively, replacing said endogenous tRNA with a suppressor tRNA with using prime editing. Additional aspects relate to compositions comprising the prime editing machinery, pegRNAs, and/or complexes comprising the prime editor and pegRNA that are capable of editing and/or replacing an endogenous tRNA to yield a suppressor tRNA. In some aspects, the disclosure further relates to polynucleotides encoding one or more nucleic acid sequences encoding the prime editor and/or pegRNA, cells comprising the polynucleotides and complexes comprising the prime editor and pegRNA, kits comprising any one of the compositions, complexes, polynucleotides, vectors, and/or cells disclosed herein, and/or delivery systems for administering any one of the compositions, complexes, polynucleotides, vectors to a subject in need thereof. Additional aspects relate to methods for inserting a new suppressor tRNA gene into a target site in a genome (e.g., a safe harbor locus site) using prime editing.
10.WO/2024/155681METHODS AND SYSTEMS FOR DETECTING AND ASSESSING LIVER CONDITIONS
WO - 25.07.2024
Int.Class C12Q 1/6827
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
6813Hybridisation assays
6827for detection of mutation or polymorphism
 Appl.No PCT/US2024/011793 Applicant HEPTA BIO, INC. Inventor AMINI, Hamed
In an aspect, the present disclosure provides a method for identifying whether a subject has or is at an increased risk of developing a liver disease (e.g., non-alcoholic fatty liver disease, non-alcoholic steatohepatitis, hepatitis, cirrhosis, or cancer), comprising providing a cell-free deoxyribonucleic acid (cfDNA) sample derived from the subject, assaying the cfDNA sample or a derivative thereof to determine a methylation pattern or a methylation level of DNA molecules of the cfDNA sample, processing the methylation pattern or the methylation level using a trained machine learning (ML) algorithm to generate an output indicative of whether the cfDNA sample is positive for the liver disease, and based at least in part on the output, generating an electronic report that is indicative of the subject having or being at the increased risk of developing the liver disease.
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