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Analysis

1.WO/2022/008519THERAPEUTIC RNA FOR HPV-POSITIVE CANCER
WO 13.01.2022
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/EP2021/068680 Applicant BIONTECH SE Inventor SAHIN, Ugur
This disclosure relates to the field of therapeutic RNA to treat HPV-positive cancer, in particular anogenital, cervical and penile cancers and cancer in the head and neck region such as cancer in the genital region and head and neck squamous cell carcinoma (HNSCC). Disclosed herein are compositions, uses, and methods for treatment of HPV-positive cancers. Administration of therapeutic RNAs to a patient having HPV-positive cancer disclosed herein can reduce tumor size, prolong time to progressive disease, and/or protect against metastasis and/or recurrence of the tumor and ultimately extend survival time.
2.WO/2022/002783ADJUVANTS
WO 06.01.2022
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/EP2021/067472 Applicant GLAXOSMITHKLINE BIOLOGICALS SA Inventor HANON, Emmanuel Jules
The present invention relates to immunisation using carrier-formulated mRNA in conjunction with squalene emulsion adjuvants, and to related aspects.
3.WO/2021/262909LNP COMPOSITIONS COMPRISING MRNA THERAPEUTICS WITH EXTENDED HALF-LIFE
WO 30.12.2021
Int.Class C12N 15/67
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
67General methods for enhancing the expression
Appl.No PCT/US2021/038780 Applicant MODERNATX, INC. Inventor REID, David
The disclosure features a polynucleotide encoding a polypeptide, which polynucleotide comprises: a 5'UTR described herein; a coding region comprising a payload and a stop element described herein; and a 3'UTR described herein, and LNP compositions comprising the same. The polynucleotides and/or LNP compositions of the present disclosure can: increase the level and/or activity of the payload by increasing the half-life and/or duration of expression of the polynucleotide encoding the payload or of the payload polypeptide. Also disclosed herein are methods of treating a disease or disorder in a subject using the LNP compositions of the present disclosure.
4.WO/2021/245611MODIFIED BETACORONAVIRUS SPIKE PROTEINS
WO 09.12.2021
Int.Class A61K 39/215
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
215Coronaviridae, e.g. avian infectious bronchitis virus
Appl.No PCT/IB2021/054903 Applicant GLAXOSMITHKLINE BIOLOGICALS SA Inventor BIANCUCCI, Marco
Betacoronavirus Spike proteins, or fragments thereof, including substitution mutations designed to increase stability, decrease the risk of antibody dependent enhancement, or both; and that are useful in, for example, immunogenic compositions.
5.20210369762COMPOSITIONS AND METHODS OF TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US 02.12.2021
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 17200661 Applicant Avidity Biosciences, Inc. Inventor Barbora MALECOVA

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.

6.WO/2021/242870COMPOSITIONS AND METHODS FOR GENOME EDITING
WO 02.12.2021
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/US2021/034272 Applicant SHAPE THERAPEUTICS INC. Inventor BOSE, Debojit
Described herein are compositions for targeting and editing genomes. Also described herein are methods for targeting and editing genomes utilizing the compositions in the instant disclosure.
7.WO/2021/239825NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF CNNM4 IN A CELL
WO 02.12.2021
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/EP2021/064077 Applicant SILENCE THERAPEUTICS GMBH Inventor SCHAEPER, Ute
The invention relates to nucleic acid products that interfere with or inhibit CNNM4 (Cyclin M4) gene expression. It further relates to therapeutic uses of CNNM4 inhibition for the treatment of diseases, such as liver diseases including non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), liver steatosis, liver fibrosis, liver cirrhosis, liver cancer and other diseases associated with magnesium dysregulation.
8.WO/2021/243174DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF SAMD9L
WO 02.12.2021
Int.Class C12Q 1/6881
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
6881for tissue or cell typing, e.g. human leukocyte antigen probes
Appl.No PCT/US2021/034794 Applicant EMENDOBIO INC. Inventor EMMANUEL, Rafi
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: 1-20246 and compositions, methods, and uses thereof.
9.20210371494CIRCULAR RNA COMPOSITIONS AND METHODS
US 02.12.2021
Int.Class C07K 14/725
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
725T-cell receptors
Appl.No 17384460 Applicant Massachusetts Institute of Technology Inventor Alexander Wesselhoeft

Circular RNA and transfer vehicles, along with related compositions and methods are described herein. In some embodiments, the inventive circular RNA comprises group I intron fragments, spacers, an IRES, duplex forming regions, and an expression sequence. In some embodiments, the expression sequence encodes a chimeric antigen receptor (CAR). In some embodiments, circular RNA of the invention has improved expression, functional stability, immunogenicity, ease of manufacturing, and/or half-life when compared to linear RNA. In some embodiments, inventive methods and constructs result in improved circularization efficiency, splicing efficiency, and/or purity when compared to existing RNA circularization approaches.

10.WO/2021/243058BIALLELIC KNOCKOUT OF SARM1
WO 02.12.2021
Int.Class C12N 9/12
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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
10Transferases (2.)
12transferring phosphorus containing groups, e.g. kinases (2.7)
Appl.No PCT/US2021/034583 Applicant EMENDOBIO INC. Inventor EMMANUEL, Rafi
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: 1-12105 and compositions, methods, and uses thereof.