A61K47/54

Rapadocins, inhibitors of equilibrative nucleoside transporter 1 and uses thereof

A compound of Formula I, ##STR00001##
and its analogs are provided. Compositions that include Formula I can be used to inhibit human equilibrative nucleoside transporter 1, increase adenosine signaling and produce effects that include increasing antiviral activity, increasing antiparasitic activity, increasing alcohol tolerance, decreasing pain protecting from ischemia as well as many other conditions.

Compositions and methods of modulating macrophage activity
11554178 · 2023-01-17 · ·

Disclosed herein, inter alia, are compositions and methods of modulating macrophage activity. Provided is a method of treating a disease (e.g., a macrophage-associated disease, autoimmune disease, inflammatory disease, or a cancer of an organ in the intraperitoneal cavity), the method including intraperitoneally administering to a subject in need thereof a therapeutically effective amount of a nanoparticle composition or pharmaceutical composition. Provided is a silica nanoparticle non-covalently bound to a plurality of nucleic acids, wherein the silica nanoparticle has a net positive charge in the absence of the plurality of nucleic acids. Provided is a pharmaceutical composition including a nanoparticle composition as described herein, and a pharmaceutically acceptable excipient.

SMALL MOLECULE DEGRADERS OF SHP2 PROTEIN
20230218765 · 2023-07-13 ·

The present disclosure provides compounds represented by Formula I: and the salts or solvates thereof, wherein R1, R3, L, Y, and B1 are as defined in the specification. Compounds having Formula I are SHP2 protein degraders useful for the treatment of cancer and other diseases.

TRANSMEMBRANE PROTEASE, SERINE 6 (TMPRSS6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS6 gene and to methods of preventing and treating a TMPRSS6-associated disorder, e.g., a disorder associated with iron overload and/or a disorder of ineffective erythropoiesis, e.g., hereditary hemochromatosis, β-thalassemia (e.g., β-thalassemia major and β-thalassemia intermiedia), polycythemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, Parkinson's Disease, Alzheimer's Disease or Friedreich's Ataxia.

Phosphorylcholine conjugates and uses thereof
11696935 · 2023-07-11 · ·

The present invention provides phosphorylcholine conjugates and pharmaceutical compositions comprising same for the prevention or treatment of autoimmune diseases. In particular, the conjugates of the present invention are effective in treating autoimmune diseases associated with pathological inflammation.

Core-shell microneedle devices and uses thereof

The present disclosure relates to microneedle devices and methods for treating a disease (for example, diabetes) using a degradable cross-linked gel for self-regulated delivery of a therapeutic agent (for example, insulin).

Core-shell microneedle devices and uses thereof

The present disclosure relates to microneedle devices and methods for treating a disease (for example, diabetes) using a degradable cross-linked gel for self-regulated delivery of a therapeutic agent (for example, insulin).

Synthesis of spherical nucleic acids using lipophilic moieties

Spherical nucleic acids (SNA) carrying self-aggregating oligonucleotides are described herein. Compositions of the SNA include discrete nanostructures that are not aggregated. Related methods are also described.

METHODS FOR CONTROLLING EXTRACORPOREAL MEMBRANE OXYGENATION (ECMO) COAGULATION
20230012024 · 2023-01-12 ·

The present application provides for a method for controlling coagulation in a subject in need of extracorporeal membrane oxygenation. The method comprises administering an effective amount of an anticoagulant agent that directly inhibits one or more steps in a coagulation pathway to the subject, wherein the effective amount of the anticoagulant agent is capable of controlling coagulation during extra corporeal membrane oxygenation for a coagulation control time of at least 4 hours.

USE OF FGFR INHIBITORS FOR TREATMENT OF IDIOPATHIC SHORT STATURE

The present invention describes methods for treating idiopathic short stature. Described herein are also methods of increasing a subject’s height. The methods involve administering an effective amount of a pan FGFR inhibitor or a selective FGFR inhibitor to the subject. An example of an FGRF inhibitor used in the methods described herein is erdafitinib.