Provided are methods of treating a disease or condition characterized by a deficiency of or a defect in an iron transporter using a small molecule. For example, the method may increase transepithelial iron transport, or it may increase iron release. Additionally, the small molecule may be hinokitiol, or it may be selected from the group consisting of amphotericin B, calcimycin, nonactin, deferiprone, purpurogallin, and maltol. Also provided is a method of identifying a compound capable of treating a disease or condition characterized by a deficiency of or a defect in an iron transporter.

A solid particulate formulation comprising soluble ferric pyrophosphate and a sachet comprising the solid particulate formulation of soluble ferric pyrophosphate for adding to a dialysis solution are provided. Improved methods of administering soluble ferric pyrophosphate comprising the solid particulate formulations and kits comprising the solid particulate formulation and a dialysis concentrate formulation are also disclosed.

The invention concerns a trans-splicing RNA (tsRNA) molecule comprising one or multiple unstructured binding domains; a cell or vector comprising said tsRNA; and a method for killing cells or treating a disease using said tsRNA.

This invention relates to JAK1 selective inhibitors, particularly pyrrolo[2,3-d]pyrimidine and pyrrolo[2,3-b]pyridine derivatives, and their use in treating myelodysplastic syndromes (MDS).

The present invention relates to novel pharmaceutical formulations of an antibody against human P-selectin, especially SEG101, or an antibody having at most 3 amino acid difference from crizanlizumab, and processes for the preparation thereof and uses of the formulations.

The compound (S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrroll-2(1H)-yl)-3-hydroxy-2-phenylpropan-1-one, or a pharmaceutically acceptable salt thereof, is useful to increase the affinity of hemoglobin for oxygen. Methods and compositions for the treatment of a hemoglobinopathies are provided herein, including certain pharmaceutical compositions for activating PKR.

The compound (S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrroll-2(1H)-yl)-3-hydroxy-2-phenylpropan-1-one, or a pharmaceutically acceptable salt thereof, is useful to increase the affinity of hemoglobin for oxygen. Methods and compositions for the treatment of a hemoglobinopathies are provided herein, including certain pharmaceutical compositions for activating PKR.

Compositions, containers and kits for low temperature storage of a specified quantity of packed red blood cells (RBCs) include L-camitine, HES and blood plasma proteins. Methods of storing a packed RBC blood product, transfusing a packed RBC blood product into a subject, and treating anemia, ischemia, hypoxia, a hemoglobin disorder, or a hematopoietic disorder in a subject (e.g., a human) include the compositions, containers, kits and blood products.

The present invention relates to bifunctional compounds, which find utility as modulators of targeted ubiquitination, especially inhibitors of a variety of polypeptides and other proteins which are degraded and/or otherwise inhibited by bifunctional compounds according to the present invention. In particular, the present invention is directed to compounds, which contain on one end a VHL ligand which binds to the ubiquitin ligase and on the other end a moiety which binds a target protein such that the target protein is placed in proximity to the ubiquitin ligase to effect degradation (and inhibition) of that protein. The present invention exhibits a broad range of pharmacological activities associated with compounds according to the present invention, consistent with the degradation/inhibition of targeted polypeptides.

The present invention provides compositions comprising tolerizing immune modified particles (TIMPs) and methods for using and making said TIMPs. In particular, carrier polymer is covalently conjugated with antigenic peptide before particle formation, which allows for exquisite control of particle size and antigen encapsulation (e.g., for use in eliciting induction of immunological tolerance).