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1.WO/2023/004150COMPOSITIONS AND METHODS FOR TREATMENT OF NEURODEGENERATIVE DISEASES
WO 26.01.2023
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/US2022/038070 Applicant CONFLUENCE THERAPEUTICS, INC. Inventor MARCUSSON, Eric G.
The disclosure relates to nucleic acid-based compositions for inhibiting pathological polynucleotide binding protein aggregates and methods of using these nucleic acid-based compositions in treating neurodegenerative diseases.
2.WO/2023/285560AGENTS ENCODING CLDN6 AND CD3 BINDING ELEMENTS FOR TREATING CLDN6-POSITIVE CANCERS
WO 19.01.2023
Int.Class C07K 16/28
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18against material from animals or humans
28against receptors, cell surface antigens or cell surface determinants
Appl.No PCT/EP2022/069659 Applicant BIONTECH SE Inventor SAHIN, Ugur
The present invention generally relates to binding agents that are at least bispecific for the binding to CD3 and CLDN6, i.e., they are capable of binding to at least CD3 and CLDN6. Specifically, the present invention relates to RNA encoding these binding agents which may be used in the treatment or prevention of cancer in a subject.
3.WO/2023/288111POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR GENE EXPRESSION REGULATION
WO 19.01.2023
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/US2022/037391 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 phorphorothioate linkages.
4.WO/2023/283342OLIGONUCLEOTIDES AND VIRAL UNTRANSLATED REGION (UTR) FOR INCREASING EXPRESSION OF TARGET GENES AND PROTEINS
WO 12.01.2023
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/US2022/036367 Applicant TEMPLE UNIVERSITY - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION Inventor HU, Wenhui
A novel, small (21-mer oligonucleotide) and unique cz's-regulatory coding motif can greatly enhance the production of a variety of different types of proteins ranging from viral transcripts/proteins, endogenous gene products, vaccines, antibodies to engineered recombinant proteins in mammalian cells. The combination of novel peptide tag(s) having specified short amino acid sequences or derivatives thereof and the untranslated region (UTR) of viruses (snUTR) enhanced production of tagged proteins, including viral transcripts/proteins, endogenous gene products, vaccine, antibody, engineered recombinant proteins in a cell both in vitro, ex vivo and in vivo.
5.WO/2023/283134UTILIZATION OF ANTIBODIES TO SHAPE ANTIBODY RESPONSES TO AN ANTIGEN
WO 12.01.2023
Int.Class C07K 16/10
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
16Immunoglobulins, e.g. monoclonal or polyclonal antibodies
08against material from viruses
10from RNA viruses
Appl.No PCT/US2022/035968 Applicant REGENERON PHARMACEUTICALS, INC. Inventor MURPHY, Andrew
Described herein are methods and compositions for directing an antibody response in a subject away from one or more first epitopes of an antigen (e.g., immunodominant epitopes of a vaccine antigen) and towards one or more second epitopes of the antigen by administering one or more antibodies targeting the one or more first epitopes of the antigen.
6.WO/2023/283576P7 CONTAINING NUCLEOSIDE-MODIFIED MRNA-LIPID NANOPARTICLE LINEAGE VACCINE FOR HEPATITIS C VIRUS
WO 12.01.2023
Int.Class A61K 39/29
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
39Medicinal preparations containing antigens or antibodies
12Viral antigens
29Hepatitis virus
Appl.No PCT/US2022/073463 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor REAGAN, Erin, Kathleen
There is an urgent need to develop a prophylactic HCV vaccine, and to determine if therapeutic vaccines can aid in the treatment of chronically infected patients. Described are compositions comprising a nucleoside-modified RNA molecules encoding a HCV p7 protein in combination with at least one additional HCV antigen, adjuvant, or a combination thereof, and their use for inducing an immune response against HCV.
7.11547673Coronavirus vaccine
US 10.01.2023
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 17233396 Applicant BioNTech SE Inventor Ugur Sahin

This disclosure relates to the field of RNA to prevent or treat coronavirus infection. In particular, the present disclosure relates to methods and agents for vaccination against coronavirus infection and inducing effective coronavirus antigen-specific immune responses such as antibody and/or T cell responses. Specifically, in one embodiment, the present disclosure relates to methods comprising administering to a subject RNA encoding a peptide or protein comprising an epitope of SARS-CoV-2 spike protein (S protein) for inducing an immune response against coronavirus S protein, in particular S protein of SARS-CoV-2, in the subject, i.e., vaccine RNA encoding vaccine antigen.

8.20230002760CRISPR/CAS-RELATED METHODS, COMPOSITIONS AND COMPONENTS
US 05.01.2023
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 17572403 Applicant Editas Medicine, Inc. Inventor Gordon Grant Welstead

CRISPR/Cas-related compositions and methods which provide for efficient gene editing of eukaryotic cells using modified gRNAs.

9.20230000773NOVEL HEADGROUP LIPID COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF THERAPEUTIC AGENTS
US 05.01.2023
Int.Class A61K 9/127
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Liposomes
Appl.No 17761863 Applicant ModernaTX, Inc. Inventor Kerry E. BENENATO

The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.

10.WO/2022/269518COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
WO 29.12.2022
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 PCT/IB2022/055799 Applicant NOVARTIS AG Inventor BELEW, Muluken Shambel
The present invention is directed to compositions and methods for the treatment of hemoglobinopathies.