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1.WO/2024/092086COMPOSITIONS AND METHODS FOR REGENERATING CORNEAL ENDOTHELIUM
WO 02.05.2024
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 PCT/US2023/077845 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor ROMPOLAS, Panteleimon
The invention relates to regenerating corneal endothelium to treat vision loss in a subject in need thereof. The invention includes a lipid nanoparticle (LNP) capable of regenerating corneal endothelium in a subject in need thereof. The LNP comprises: (a) at least one nucleoside-modified ribonucleic acid (RNA) encoding at least one pro-growth factor, wherein each pro-growth factor is encoded by a distinct nucleoside-modified RNA; and (b) at least one ionizable lipid. The invention also includes pharmaceutical compositions comprising the LNP of the invention, a kit comprising the pharmaceutical composition, and a method of regenerating corneal endothelium in a subject in need thereof.
2.WO/2024/091918METHODS OF LIPID NANOPARTICLE PRODUCTION IN CROSS-MIXERS
WO 02.05.2024
Int.Class B01F 23/41
Appl.No PCT/US2023/077606 Applicant MODERNATX, INC. Inventor SMITH, Michael H.
In some aspects, a method can include feeding an eLNP solution to a first inlet, the first inlet splitting into a first sub-inlet and a second sub-inlet, the first sub-inlet connected to a first side of a central region of a mixer, the second sub-inlet connected to a second side of the central region of the mixer, feeding an mRNA solution to a second inlet, the second inlet connected to a third side of the central region of the mixer, the third side orthogonal to the first side and the second side, mixing the eLNP and the mRNA in the central region to form encapsulated eLNP, and transferring the encapsulated eLNP out of the central region via an outlet, the outlet coupled to a fourth side of the central region, the fourth side opposite the third side.
3.WO/2024/089638NUCLEIC ACID BASED VACCINE
WO 02.05.2024
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/IB2023/060807 Applicant GLAXOSMITHKLINE BIOLOGICALS SA Inventor PETSCH, Benjamin
The present invention is directed to nucleic acids suitable for use in treatment or prophylaxis of an infection with a coronavirus, such as a Coronavirus SARS-CoV-2 variant, or a disorder related to such an infection, such as COVID-19. The present invention is also directed to compositions, and vaccines. The compositions and vaccines comprise at least one of said nucleic acid sequences, and nucleic acid sequences in association with a lipid nanoparticle (LNP). The invention is also directed to first and second medical uses of the nucleic acids, the composition, the vaccine, and the kit, and to methods of treating or preventing a coronavirus infection, such as a Coronavirus infection from a SARS-CoV-2 variant.
4.WO/2024/092209VITAMIN-BASED LIPIDS AND LIPID NANOPARTICLES COMPRISING THE SAME
WO 02.05.2024
Int.Class C07D 311/04
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
311Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings
02ortho- or peri-condensed with carbocyclic rings or ring systems
04Benzo pyrans, not hydrogenated in the carbocyclic ring
Appl.No PCT/US2023/078044 Applicant ADVANCED RNA VACCINE (ARV) TECHNOLOGIES, INC. Inventor XU, Jiangsheng
Described are compounds, compositions, and methods for delivery of therapeutic, diagnostic, or prophylactic agents (for example, a nucleic acid).
5.WO/2024/089633RNA MOLECULES ENCODING RSV-F AND VACCINES CONTAINING THEM
WO 02.05.2024
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/IB2023/060798 Applicant PFIZER INC. Inventor CHEN, Wei
The present disclosure relates to RNA molecules encoding a respiratory syncytial virus (RSV). The present disclosure further relates to compositions comprising the RNA molecules formulated in a lipid nanoparticle (RNA-LNP). The present disclosure further relates to the use of the RNA molecules, RNA-LNPs and compositions for the treatment and/or prevention of RSV infection-induced acute respiratory tract illness, including pneumonia and bronchitis.
6.20240132877GENOME EDITING SYSTEMS COMPRISING REPAIR-MODULATING ENZYME MOLECULES AND METHODS OF THEIR USE
US 25.04.2024
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 18104461 Applicant Editas Medicine, Inc. Inventor Cecilia Cotta-Ramusino

This application provides improved methods of genome editing. The genome editing systems described herein comprise a RNA-guided nuclease molecule and a Repair-Modulating Enzyme Molecule (RMEM).

7.20240131181Therapeutic Targeting of Lipid Nanoparticles
US 25.04.2024
Int.Class A61K 47/69
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
69the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
Appl.No 18526587 Applicant The Trustees of the University of Pennsylvania Inventor Vladimir Muzykantov

The present invention relates to compositions comprising a delivery vehicle conjugated to a targeting domain, wherein the delivery vehicle comprises at least one agent, and wherein the targeting domain specifically binds to an endothelial marker. The invention also relates to methods of treating or preventing neurological or pulmonary conditions using the described compositions.

8.20240131049CIRCULAR RNA COMPOSITIONS AND METHODS
US 25.04.2024
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 18320126 Applicant Orna Therapeutics, Inc. Inventor Brian Goodman

Disclosed herein are circular RNAs and transfer vehicles, along with related compositions and methods of treatment. The circular RNAs can comprise group I intron fragments, spacers, an IRES, duplex forming regions, and/or an expression sequence, thereby having the features of improved expression, functional stability, low immunogenicity, ease of manufacturing, and/or extended half-life compared to linear RNA. Pharmaceutical compositions comprising such circular RNAs and transfer vehicles are particularly suitable for efficient protein expression in immune cells in vivo. Also disclosed are precursor RNAs and materials useful in producing the precursor or circular RNAs, which have improved circularization efficiency and/or are compatible with effective circular RNA purification methods.

9.WO/2024/086614TARGETED LNP-MRNA WITH MINIMAL OFF-TARGET EXPRESSION AND METHODS OF USE THEREOF
WO 25.04.2024
Int.Class C12N 15/09
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
Appl.No PCT/US2023/077142 Applicant THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA Inventor PARHIZ, Hamideh
The present invention relates to compositions for effective delivery of an agent to a target cell while minimizing off-target expression of the therapeutic agent by administering therapeutic agents that have been modified with an inhibitory domain which inhibits expression of the agent in non-target cells, as well as methods of use thereof for delivery of therapeutic agents.
10.20240131193MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF CYTOPLASMIC AND CYTOSKELETAL PROTEINS
US 25.04.2024
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 18161598 Applicant Modema TX, Inc. Inventor Antonin DE FOUGEROLLES

The invention relates to compositions including polynucleotides encoding polypeptides which have been chemically modified by replacing the uridines with 1-methyl-pseudouridine to improve one or more of the stability and/or clearance in tissues, receptor uptake and/or kinetics, cellular access by the compositions, engagement with translational machinery, mRNA half-life, translation efficiency, immune evasion, protein production capacity, secretion efficiency, accessibility to circulation, protein half-life and/or modulation of a cell's status, function, and/or activity.