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1.WO/2022/132765BIALLELIC KNOCKOUT OF B2M
WO 23.06.2022
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 PCT/US2021/063302 Applicant EMENDOBIO INC. Inventor IZHAR, Lior
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-1538 and compositions, methods, and uses thereof.
2.20220195535Methods for Detecting and Treating Esophageal Cancer
US 23.06.2022
Int.Class C12Q 1/6886
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
6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
6883for diseases caused by alterations of genetic material
6886for cancer
Appl.No 17604255 Applicant The Johns Hopkins University Inventor Stephen J. Meltzer

The present invention relates to the field of cancer. More specifically, the present invention provides compositions

and methods useful for detecting and treating esophageal cancer. In a specific embodiment, a method for identifying a subject having esophageal squamous cell carcinoma (ESCC) comprises (a) extracting genomic DNA from a sample obtained from the subject; (b)

performing a conversion reaction on the genomic DNA in vitro to convert unmethylated cytosine to uracil by deamination; and (c)

detecting nucleic acid methylation of one or more genes in the converted genomic DNA, wherein detecting nucleic acid methylation e identifies the subject as having ESCC. The one or more genes can comprise ZNF542, ZNF132, cg20655070, TAC1 and SLC35F1. In a

more specific embodiment, the one or more genes comprise ZNF542 and ZNF132 and can further comprise detecting the nucleic acid N methylation of one or more of cg20655070, TAC1 and SLC35F1.

3.WO/2022/132756BIALLELIC KNOCKOUT OF TRAC
WO 23.06.2022
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 PCT/US2021/063290 Applicant EMENDOBIO INC. Inventor IZHAR, Lior
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-1932 and compositions, methods, and uses thereof.
4.2022528696アンチコドンループを延長した合成転移RNA
JP 15.06.2022
Int.Class A61P 1/04
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
1Drugs for disorders of the alimentary tract or the digestive system
04for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
Appl.No 2021559312 Applicant アークタラス テラピュウティクス、インク Inventor イグナトヴァ、ゾヤ
延長したアンチコドンループを有する合成転移RNAに関する。フレームシフト変異に関連する神経線維腫症のような遺伝子疾患の治療に有用な合成抑制因子転移RNAを提供する。前記合成転移RNAは、4ヌクレオチドアンチコドンまたは5ヌクレオチドアンチコドンを有する延長アンチコドンループを含む。
【選択図】図1
5.WO/2022/119881DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF LRRK2
WO 09.06.2022
Int.Class C12N 15/90
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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
Appl.No PCT/US2021/061334 Applicant EMENDOBIO INC. Inventor EMMANUEL, Rafi
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-103,606 and compositions, methods, and uses thereof.
6.20220162612SYNTHETIC TRANSFER RNA WITH EXTENDED ANTICODON LOOP
US 26.05.2022
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 17602251 Applicant Arcturus Therapeutics, Inc. Inventor Zoya IGNATOVA

The invention relates to a synthetic transfer RNA with an extended anticodon loop. The invention provides a synthetic suppressor transfer RNA useful for the treatment of a genetic disease like neurofibromatosis associated with a frameshift mutation. The synthetic transfer RNA comprises an extended anticodon loop having a four-nucleotide anticodon or a five-nucleotide anticodon.

7.20220133775P-ETHOXY NUCLEIC ACIDS FOR IGF-1R INHIBITION
US 05.05.2022
Int.Class A61K 31/7125
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
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
Appl.No 17530051 Applicant Bio-Path Holdings, Inc. Inventor Ana Ashizawa

Provided herein are methods of treating cancer or an autoimmune disease comprising administering a liposome that comprises neutral phospholipids and a P-ethoxy oligonucleotide that targets a IGF-1R-encoding polynucleotide.

8.20220136013DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF STAT1
US 05.05.2022
Int.Class C12N 15/90
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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
Appl.No 17433977 Applicant EMENDOBIO INC. Inventor David Baram

RNA molecules comprising a guide sequence portion having 17-25 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ NOs 1-37365 and compositions, methods, and uses thereof.

9.20220098621CRISPR COMPOSITIONS AND METHODS FOR PROMOTING GENE EDITING OF RIBOSOMAL PROTEIN S19 (RPS19) GENE
US 31.03.2022
Int.Class C12N 15/90
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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
Appl.No 17428880 Applicant EmendoBio Inc. Inventor David Baram

RNA molecules comprising a guide sequence portion having 17-25 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs 1-20465 and compositions, methods, and uses thereof.

10.20220073991METHODS AND COMPOSITIONS FOR PREDICTING AND/OR MONITORING CARDIOVASCULAR DISEASE AND TREATMENTS THEREFOR
US 10.03.2022
Int.Class C12Q 1/6883
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
6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
6883for diseases caused by alterations of genetic material
Appl.No 17466786 Applicant Cardio Diagnostics, Inc. Inventor Meeshanthini V. Dogan

This document describes methods and compositions for predicting cardiovascular disease (CVD). Specifically, this document describes methods and compositions for determining the methylation status of at least one CpG locus and the sequence of at least one single nucleotide polymorphism (SNP) that are predictive for the incidence (e.g., one-year, three-year, five-year incidence) of CVD.