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1.WO/2024/197309PEG TARGETING COMPOUNDS FOR DELIVERY OF THERAPEUTICS
WO 26.09.2024
Int.Class A61K 47/54
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
Appl.No PCT/US2024/021351 Applicant MODERNATX, INC. Inventor SEEPERSAUD, Mohindra
Provided herein are targeting compounds (e.g., a compound of Formula I, a stereoisomer thereof, a tautomer thereof, and/or a pharmaceutically acceptable salt thereof), lipid nanoparticle (LNP) compositions comprising such targeting compounds and the use thereof. The LNP compositions described herein may further comprise one or more selected from ionizable lipids, PEG-lipids, phospholipids, and structural lipids.
2.WO/2024/194153RSV-F-ENCODING NUCLEIC ACIDS
WO 26.09.2024
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/EP2024/056893 Applicant GLAXOSMITHKLINE BIOLOGICALS SA Inventor BARROWS, Nicholas John
The present disclosure provides inter alia, a recombinant ribonucleic acid (RNA) encoding a respiratory syncytial virus fusion (RSV-F) protein comprising an F2 and an F1 domain; wherein the RSV-F protein further comprises, relative to a wild-type RSV-F sequence, the substitution of a residue for a C residue in both of the F2 and F1 domains.
3.WO/2024/197307PEG TARGETING COMPOUNDS FOR DELIVERY OF THERAPEUTICS
WO 26.09.2024
Int.Class A61K 47/50
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
Appl.No PCT/US2024/021344 Applicant MODERNATX, INC. Inventor SEEPERSAUD, Mohindra
Provided herein are targeting compounds (e.g., a compound of Formula I, a stereoisomer thereof, a tautomer thereof, and/or a pharmaceutically acceptable salt thereof), lipid nanoparticle (LNP) compositions comprising such targeting compounds and the use thereof. The LNP compositions described herein may further comprise one or more selected from ionizable lipids, PEG-lipids, phospholipids, and structural lipids.
4.WO/2024/193965RSV-F-ENCODING NUCLEIC ACIDS
WO 26.09.2024
Int.Class C12N 15/45
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
45Paramyxoviridae, e.g. measles virus, mumps virus, Newcastle disease virus, canine distemper virus, rinderpest virus, respiratory syncytial viruses
Appl.No PCT/EP2024/055127 Applicant GLAXOSMITHKLINE BIOLOGICALS SA Inventor BIANCUCCI, Marco
The present disclosure provides inter alia, a recombinant nucleic acid encoding a respiratory syncytial virus fusion (RSV-F) protein comprising a cytoplasmic tail; wherein, relative to a cytoplasmic tail according to SEQ ID NO: 3 or 4, 2-20 residues are deleted from the cytoplasmic tail of the RSV-F protein.
5.WO/2024/197310PEG TARGETING COMPOUNDS FOR DELIVERY OF THERAPEUTICS
WO 26.09.2024
Int.Class A61K 47/54
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
Appl.No PCT/US2024/021352 Applicant MODERNATX, INC. Inventor SEEPERSAUD, Mohindra
Provided herein are targeting compounds (e.g., a compound of Formula I, a stereoisomer thereof, a tautomer thereof, and/or a pharmaceutically acceptable salt thereof), lipid nanoparticle (LNP) compositions comprising such targeting compounds and the use thereof. The LNP compositions described herein may further comprise one or more selected from ionizable lipids, PEG-lipids, phospholipids, and structural lipids.
6.WO/2024/192257MRNA THERAPEUTICS FOR DISORDERS OF VASCULAR PERMEABILITY
WO 19.09.2024
Int.Class A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Appl.No PCT/US2024/019965 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor PARHIZ, Hamideh
The present invention relates to compositions and methods for pulmonary targeted delivery of VE-cadherin and methods of use thereof for treating or preventing vascular permeability disorders, such as acute respiratory stress syndrome (ARDS).
7.WO/2024/192351TRANSIENT MODIFIED-RNA BASED ELEVATED EXPRESSION OF FLI-1 TO AUGMENT HEMATOPOIETIC STEM AND PROGENITOR CELL EXPANSION
WO 19.09.2024
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/US2024/020161 Applicant CORNELL UNIVERSITY Inventor ITKIN, Tomer
The present disclosure is directed to a method of activation and expansion of adult non-activated hematopoietic stem and progenitor cells (HSPCs). The present disclosure reveals that Fli-1 activity is essential during regenerative hematopoiesis. Fli-1 directs activation programs while manipulating cellular sensory and output machineries, enabling HSPCs co-adoptability with a stimulated vascular niche. Applying FLI-1 transient modified-mRNA transduction into lethargic adult human mobilized HSPCs, enables their niche-mediated expansion and superior engraftment capacities. The present disclosure provides a population of regenerated and expanded adult hematopoietic stem and progenitor cells (HSPCs). The present disclosure also provides methods of treating a subject in need of activated and expanded adult HSPCs by administering the regenerated and expanded HSPCs to the subject.
8.WO/2024/192233TARGETED DELIVERY OF THERAPEUTIC AGENTS TO THE CENTRAL NERVOUS SYSTEM AND METHODS OF USE FOR TREATMENT OF NEUROLOGICAL CONDITIONS
WO 19.09.2024
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/US2024/019916 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor BRENNER, Jacob, S.
The present invention relates to compositions and methods for targeted delivery of therapeutic anti-inflammatory agents to the blood brain barrier to restore blood brain barrier function. The delivery method can comprise various nanoparticles. The invention also relates to methods of treating or preventing neurological conditions using the described compositions. The compositions can include use of cell editing processes such as CRISPR/Cas systems.
9.WO/2024/184489MANUFACTURE OF MESSENGER RNA WITH KP34 POLYMERASE
WO 12.09.2024
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/EP2024/056094 Applicant SANOFI Inventor CUI, Jianping
The present disclosure provides methods and compositions for improving the in vitro transcription (IVT) of messenger RNA using Klebsiella phage KP34 RNA polymerase. In particular, the present disclosure provides a method for manufacturing messenger RNA comprising: (a) providing a DNA template comprising a nucleic acid sequence encoding an mRNA transcript for expression of a polypeptide or protein; (b) contacting the DNA template with a Klebsiella phage KP34 RNA polymerase under conditions suitable for IVT of the mRNA transcript.
10.20240299579GENE EDITING TO IMPROVE JOINT FUNCTION
US 12.09.2024
Int.Class A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Appl.No 18006455 Applicant Orthobio Therapeutics, Inc. Inventor Peter J. Millett

The present disclosure provides compositions and methods for treating joint disorders that are characterized by an inflammatory component. In some aspects, the compositions and methods are to prevent the progression of osteoarthritis and other arthritides and to treat osteoarthritis and other arthritides in a mammalian joint.