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1.WO/2024/216289SYSTEMS AND METHODS FOR EARLY-STAGE CANCER DETECTION AND SUBTYPING
WO 17.10.2024
Int.Class G16B 40/30
GPHYSICS
16INFORMATION AND COMMUNICATION TECHNOLOGY SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
40ICT specially adapted for biostatistics; ICT specially adapted for bioinformatics-related machine learning or data mining, e.g. knowledge discovery or pattern finding
30Unsupervised data analysis
Appl.No PCT/US2024/024682 Applicant EXAI BIO, INC. Inventor ALIPANAHI, Babak
Embodiments described herein provide a neural network based cancer detection and subtyping tool for predicting the presence of a tumor, its tissue of origin, and its subtype using small RNA sequencing (smRNA-seq) data, for example, the oncRNA count data. Specifically, the AI-based cancer detection and subtyping tool uses variational Bayes inference and semi-supervised training to adjust for batch effects and learn a low dimensional distribution explaining biological variability of the data. A method is also provided for determining the likely subtype(s) in a cancer sample.
2.WO/2024/216029COMPOSITIONS AND METHODS FOR MOLECULAR MIMICRY
WO 17.10.2024
Int.Class C40B 30/06
CCHEMISTRY; METALLURGY
40COMBINATORIAL TECHNOLOGY
BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES, IN SILICO LIBRARIES
30Methods of screening libraries
06by measuring effects on living organisms, tissues or cells
Appl.No PCT/US2024/024265 Applicant FLAGSHIP PIONEERING INNOVATIONS VII, LLC Inventor ACKER, Daniel, William Menon
The present application is directed to methods for identifying and characterizing molecules that mimic binding sites of specific binders on various binding surfaces.
3.20240344098METHODS AND SYSTEMS FOR CAPPING NUCLEIC ACID MOLECULES
US 17.10.2024
Int.Class C12P 19/34
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
19Preparation of compounds containing saccharide radicals
26Preparation of nitrogen-containing carbohydrates
28N-glycosides
30Nucleotides
34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
Appl.No 18750619 Applicant Quantoom Biosciences S.A. Inventor Kristof VANDEKERCKHOVE

Described herein are systems for capping nucleic acid. Also described herein are methods for capping nucleic acid with the systems described herein.

4.WO/2024/214071COMPOSITIONS AND METHODS FOR THE TREATMENT OF HUNTINGTON'S DISEASE BY EDITING THE MUTANT HUNTINGTIN GENE
WO 17.10.2024
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/IB2024/053622 Applicant LIFEEDIT THERAPEUTICS, INC. Inventor BORDEN, Philip
Compositions and methods for cleaving a mutant huntingtin (mutHTT) allele are provided. Compositions include CRISPR RNAs, guide RNAs, and nucleic acid molecules encoding the same. Vectors and host cells comprising the nucleic acid molecules are also provided. Further provided are RNA-guided nuclease (RGN) systems for cleaving a mutHTT allele, wherein the RGN system comprises an RNA-guided nuclease and a guide RNA. The compositions find use in cleaving or modifying a mutHTT allele, and/or modifying the expression of a mutHTT allele. The compositions are additionally useful for treating Huntington's disease (HD), particularly in an allele-specific manner.
5.20240344060COMPOSITIONS AND METHODS FOR THE MODULATION OF ADAPTIVE IMMUNITY
US 17.10.2024
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 18505709 Applicant Locanabio, Inc. Inventor David A. NELLES

Disclosed are compositions and methods for simultaneously providing a gene therapy and preventing an adaptive immune response to a cell modified by the gene therapy by the immune system of a subject. In some embodiments, compositions of the disclosure modify a level of expression of an RNA molecule associated with a disease or disorder as well as inhibit expression or activity of a component of an adaptive immune response to mask the modified cell from a subject's immune system.

6.WO/2024/216217HIV VACCINE
WO 17.10.2024
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/US2024/024503 Applicant BIONTECH SE Inventor LE DOUCE, Valentin
The present invention concerns compositions comprising an RNA molecule, wherein the RNA molecule comprises an expression cassette encoding an immunogenic peptide, which peptide comprises at least two fragments, wherein each fragment comprises at least one epitope, wherein the epitope is derived from an amino acid sequence encoded by the human immunodeficiency virus (HIV) and medical preparations comprising such compositions. Furthermore, the invention concerns methods for preventing or treating HIV.
7.20240342206mRNAS FOR TREATMENT OR PROPHYLAXIS OF LIVER DISEASES
US 17.10.2024
Int.Class A61K 31/7105
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
Appl.No 18293542 Applicant CureVac SE Inventor Tim SONNTAG

The present invention relates to mRNA medicines for use in the therapy and prevention of liver diseases like liver fibrosis, liver cirrhosis, hepatocellular carcinoma (HCC), non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH) or liver cancer, and more particularly to mRNA medicines of this kind which can exhibit excellent therapeutic and preventive effects with respect to liver diseases individually developed or to complications resulting from diseases of these organs. In detail, the present invention relates to an mRNA suitable for treatment or prophylaxis of liver diseases. In particular, the present invention provides mRNAs encoding hepatocyte nuclear factor 4 alpha (HNF4A), human wild type and engineered variants thereof), or a fragment or a variant of any of these peptides or proteins. The present invention concerns said mRNA as well as compositions and kits comprising the mRNA. Furthermore, the present invention relates to the mRNA, compositions or kits as disclosed, preferably LNP formulations or compositions, herein for use as a medicament, in particular for treatment or prophylaxis of a liver disease. The present invention also provides the use of the RNA, compositions or kits as disclosed herein for increasing the expression of said encoded protein, in particular in gene therapy.

8.WO/2024/213788METHOD OF DNA SEQUENCING
WO 17.10.2024
Int.Class C12Q 1/6806
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
6806Preparing nucleic acids for analysis, e.g. for polymerase chain reaction assay
Appl.No PCT/EP2024/060167 Applicant ANILING SL. Inventor CUENCA LEON, Ester
The present invention relates to methods for determining the sequence of nucleic acid molecules. In particular, the present invention provides a method comprising: i. Providing a nucleic acid molecule which comprises a 5' region and a 3' region, wherein the 5' region and the 3' region are covalently linked by a nucleotide sequence to which primers can bind, wherein the base identities in one of the 5' or 3' regions and the base identities in the other region both provide, independently, information on the base identities in the corresponding loci in an original nucleic acid molecule, wherein the molecule further comprises: - One adapter at the 5' end of the molecule; - One adapter at the 3' end of the molecule; ii. Using at least two different primers to sequence the molecule provided in step (i), wherein the at least two different primers bind to at least three, preferably to at least four different regions in the nucleic acid molecule provided in (i), wherein: 1. At least one of the primers is capable of binding at least partially to at least a portion of the adapter at the 5' end of the molecule, to sequence at least part of the 5' region of the nucleic acid molecule provided in (i); 2. At least one of the primers is capable of binding at least partially to the region of the nucleotide sequence which covalently links the 5' region and the 3' region of the nucleic acid molecule provided in (i), to sequence the 3' region of the nucleic acid molecule provided in (i).; 3. At least one of the primers is capable of binding at least partially to at least a portion of the adapter at the 3' end of the molecule, to sequence at least part of the 3' region of the nucleic acid molecule provided in (i); and/or 4. At least one of the primers is capable of binding at least partially the region of the nucleotide sequence which covalently links the 5' region and the 3' region of the nucleic acid molecule provided in a., to sequence the 5' region of the nucleic acid molecule provided in (i).
9.20240342246INTERLEUKIN-12 VARIANTS AND METHODS OF USE
US 17.10.2024
Int.Class A61K 38/20
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
19Cytokines; Lymphokines; Interferons
20Interleukins
Appl.No 18683398 Applicant Yale University Inventor Aaron Ring

The present disclosure provides compositions and methods comprising variant polypeptides of IL-12 with partial agonism relative to wild-type IL-12 for use in therapeutic and non-therapeutic applications.

10.WO/2024/216162SYNTHETIC CIRCUITS AND USES THEREOF
WO 17.10.2024
Int.Class C12N 15/63
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
Appl.No PCT/US2024/024440 Applicant STRAND THERAPEUTICS INC. Inventor KITADA, Tasuku
The present disclosure relates to a synthetic circuit that can be used to selectively express a payload in a target cell. In some aspects, a synthetic circuit comprises a payload sequence comprising a sensor and a regulator sequence comprising a sensor. Also provided herein are methods of using such synthetic circuits to treat a wide range of disease or disorders.