A61K31/711

Functionally-interdependent shape switching nucleic acid nanoparticles

The description provides a molecular switch comprising at least two nanoparticles, wherein a first nanoparticle comprises DNA and/or RNA oligonucleotides, and a second nanoparticle which is complementary to the first nanoparticle comprises reverse complementary DNA and/or RNA oligonucleotides of the first nanoparticle; wherein the complementary nanoparticles interact under physiological conditions leading to thermodynamically driven conformational changes in the first and second nanoparticles leading to their re-association to release one or more duplexes comprising said DNA and/or RNA oligonucleotides and the reverse complementary DNA and/or RNA oligonucleotides, and wherein the nanoparticles are not rings and have no single stranded toeholds.

Enhanced organogenesis through manipulation of LIN28/LET-7/DIS3L2

Described herein are methods of prolonging or reactivating organogenesis in a subject in need thereof (e.g., a subject that has impaired organ function such as a prematurely born infant). The methods comprise increasing the expression or activity of Lin28A or Lin28B proteins, inhibiting the expression or activity of let-7 family microRNAs, and/or inhibiting the expression or activity of Dis3L2 exonuclease.

CHITOSAN POLYPLEX-BASED LOCALIZED EXPRESSION OF IL-12 ALONE OR IN COMBINATION WITH TYPE-I IFN INDUCERS FOR TREATMENT OF MUCOSAL CANCERS

The present disclosure relates to methods and compositions comprising derivatized-chitosan polyplexes reversibly coated with a polyanion-containing block co-polymer for the localized expression of IL-12 in mucosal tissues, preferably in combination with an IFN-1 activator/inducer, for use in cancer immunotherapy.

METHOD OF TREATING HBV INFECTION USING A CORE PROTEIN ALLOSTERIC MODULATOR

The present invention relates to method of treating HBV infection in a human patient, wherein the method comprises administration of a therapeutically effective amount of Compound (I), or a pharmaceutically acceptable salt thereof.

METHOD OF TREATING HBV INFECTION USING A CORE PROTEIN ALLOSTERIC MODULATOR

The present invention relates to method of treating HBV infection in a human patient, wherein the method comprises administration of a therapeutically effective amount of Compound (I), or a pharmaceutically acceptable salt thereof.

MUCUS PENETRATING PARTICLE COMPOSITIONS AND METHODS OF USE THEREOF ENHANCING IMMUNE RESPONSE
20220370488 · 2022-11-24 ·

Mucus penetrating nanoparticles for inducing, increasing, or enhancing an immune response typically include core of a blend of a biodegradable hydrophobic polymer and a hydrophilic polymer, wherein ≥50% of the biodegradable polymer is conjugated to the hydrophilic polymer, and the hydrophilic polymers forms a coating on the particle. The particles encapsulate a cargo, typically an antigen, adjuvant or other immunomodulator, or a nucleic acid encoding the antigen, or combination thereof. Pharmaceutical compositions including an effective amount of particles to induce an immune response in a subject in need thereof are also provided. Methods of inducing an immune response are also provided, and typically include administering to a subject, preferably via the respiratory tract, the pharmaceutical composition. In some embodiments, the subject has cancer or an infection of the lung.

MUCUS PENETRATING PARTICLE COMPOSITIONS AND METHODS OF USE THEREOF ENHANCING IMMUNE RESPONSE
20220370488 · 2022-11-24 ·

Mucus penetrating nanoparticles for inducing, increasing, or enhancing an immune response typically include core of a blend of a biodegradable hydrophobic polymer and a hydrophilic polymer, wherein ≥50% of the biodegradable polymer is conjugated to the hydrophilic polymer, and the hydrophilic polymers forms a coating on the particle. The particles encapsulate a cargo, typically an antigen, adjuvant or other immunomodulator, or a nucleic acid encoding the antigen, or combination thereof. Pharmaceutical compositions including an effective amount of particles to induce an immune response in a subject in need thereof are also provided. Methods of inducing an immune response are also provided, and typically include administering to a subject, preferably via the respiratory tract, the pharmaceutical composition. In some embodiments, the subject has cancer or an infection of the lung.

MUCUS PENETRATING PARTICLE COMPOSITIONS AND METHODS OF USE THEREOF ENHANCING IMMUNE RESPONSE
20220370488 · 2022-11-24 ·

Mucus penetrating nanoparticles for inducing, increasing, or enhancing an immune response typically include core of a blend of a biodegradable hydrophobic polymer and a hydrophilic polymer, wherein ≥50% of the biodegradable polymer is conjugated to the hydrophilic polymer, and the hydrophilic polymers forms a coating on the particle. The particles encapsulate a cargo, typically an antigen, adjuvant or other immunomodulator, or a nucleic acid encoding the antigen, or combination thereof. Pharmaceutical compositions including an effective amount of particles to induce an immune response in a subject in need thereof are also provided. Methods of inducing an immune response are also provided, and typically include administering to a subject, preferably via the respiratory tract, the pharmaceutical composition. In some embodiments, the subject has cancer or an infection of the lung.

NUCLEIC ACID COMPLEX

The object of the present invention is to provide a nucleic acid agent that is efficiently delivered to the nervous system, for example, the central nervous system to which drug delivery can be prevented by BBB, and produces an antisense effect on the target transcriptional product at the delivery site, and a composition comprising the same. In an embodiment, the present invention provides a double-stranded nucleic acid complex formed by annealing a first nucleic acid strand that hybridizes to a part of a target transcriptional product and has an antisense effect on the target transcriptional product, and a second nucleic acid strand that comprises a base sequence complementary to the first nucleic acid strand and is bound to a C.sub.22-35 alkyl group optionally substituted with a hydroxy group or an analog thereof.

NUCLEIC ACID COMPLEX

The object of the present invention is to provide a nucleic acid agent that is efficiently delivered to the nervous system, for example, the central nervous system to which drug delivery can be prevented by BBB, and produces an antisense effect on the target transcriptional product at the delivery site, and a composition comprising the same. In an embodiment, the present invention provides a double-stranded nucleic acid complex formed by annealing a first nucleic acid strand that hybridizes to a part of a target transcriptional product and has an antisense effect on the target transcriptional product, and a second nucleic acid strand that comprises a base sequence complementary to the first nucleic acid strand and is bound to a C.sub.22-35 alkyl group optionally substituted with a hydroxy group or an analog thereof.