Disclosed are synthetic nanocarrier compositions with coupled adjuvant compositions as well as related methods.

The invention relates to pharmaceutical compositions having casein kinase 1 delta (CK1δ) inhibitors and to the use of the inhibitors in the treatment of neurodegenerative disorders such as Alzheimer's disease.

Compounds of formula (I) can modulate the activity of one or more S 1P receptors. Sphingosine 1-phosphate (S IP) is a lysophospholipid mediator that evokes a variety of cellular responses by stimulation of five members of the endothelial cell differentiation gene (EDG) receptor family, namely S1P1, S1P2, S1P3, S1P4, and S1P5 (formerly EDG1, EDG5, EDG3, EDG6 and EDG8). The EDG receptors are G-protein coupled receptors (GPCRs) and on stimulation propagate second messenger signals via activation of heterotrimeric G-protein alpha (Ga.) subunits and beta-gamma (G( )y) dimers.

The present disclosure relates to dimers of engineered polypeptides having a binding affinity for the neonatal Fc receptor FcRn, and provides an FcRn binding dimer, comprising a first monomer unit, a second monomer unit and an amino acid linker, wherein said first and second monomer unit search comprises an FcRn binding motif. Said FcRn binding dimer binds FcRn with higher capacity compared to said first monomer unit or second monomer unit alone. The present disclosure also relates to the use of said FcRn binding dimer as an agent for modifying pharmacokinetic and pharmacodynamic properties and as a therapeutic agent.

The present disclosure provides compounds useful as inhibitors of SARM1 NADase activity, compositions thereof, and methods of using the same. The present disclosure provides compounds useful for treating a neurodegenerative or neurological disease or disorder, compositions thereof, and methods of using the same.

An object of the present invention is to provide an adsorbent for oral administration capable of adsorbing large quantities of indole in the presence of bile acid.

The above problem can be solved by an adsorbent for oral administration comprising a spherical activated carbon, the activated carbon having a specific surface area determined by the BET method of 800 m.sup.2/g or more, a bulk density of from 0.3 g/mL to 0.8 g/mL, a volume of pores having a diameter less than 3 nm of 0.3 mL/g or more, and a micropore/mesopore ratio (Vm) determined by Formula (1): Vm=Vmic/Vmet (1) wherein Vmic is a volume of pores having a diameter less than 3 nm, and Vmet is a volume of pores having a diameter from 3 nm to 50 nm; of 3.0 or more.

The present invention relates to a cysteine releasing pharmaceutical preparation(s) for novel use in preventing and/or treating alcohol flushing and alcohol induced hypersensitivity reactions. Additionally, the invention relates to a method for eliminating histamine releasing effect of locally formed acetaldehyde in the digestive tract.

Immunoprotective primary mesenchymal stems cells (IP-MSC) which episomally express immunoreactive polypeptides that specifically target a pathogen (e.g., an infectious species of virus, bacterium, or parasite) or toxin are described herein. The immunoreactive polypeptides can be, e.g., full antibodies, single-chain antibodies (ScFV), Fab or F(ab).sub.2 antibody fragments, diabodies, tribodies, and the like). Optionally IP-MSC are trasfected to express one or more other immunomodulating polypeptides, e.g., a cytokine such as an interleukin (e.g., IL-2, IL-4, IL-6, IL-7, IL-9, and IL-12), an interferon (e.g., IFNα, IFβ, or IFNω), and the like, which can enhance the effectiveness of the immunoreactive polypeptides.

Wnt signaling agonist compositions and methods for their use are provided. Wnt signaling agonists of the invention comprise a frizzled binding moiety, which is fused or conjugated to an LRP5 or LRP6 binding moiety.

The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.