Sanford Burnham Prebys Medical Discovery Institute

Provided herein are small molecule modulators of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1), compositions comprising the compounds of Formula (XI), and methods of using the compounds and compositions comprising the compounds

Provided herein are small molecule active metabotropic glutamate subtype-2 and -3 receptor negative allosteric modulators (NAMs), compositions comprising the compounds, and methods of using the compounds and compositions.

Described herein are EphA4 receptor antagonists, pharmaceutical compositions containing EphA4 antagonists, and methods and uses of treating an EphA4-based disease, disorder or pathology in an individual using EphA4 receptor antagonists.

Disclosed herein are compounds that abrogate the activity of HIPK4 by inhibiting the enzyme, inducing its degradation, or both. Also disclosed are methods for using the disclosed compounds as male contraceptives.

Provided herein are compounds, compositions, and methods for lowering expression levels of runt-related transcription factor 1 (RUNX1) in a cell, tissue or animal. Further provided are methods of improving memory and cognitive functioning in individuals with Down syndrome (DS) using an antisense compound targeted to a RUNX1 nucleic acid. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of decreasing inflammation and slowing cognitive decline in individuals with dementia.

The present disclosure is directed to compounds, compositions, formulations and methods of use thereof in the treatment and prevention of ULK mediated diseases, including cancer.

Disclosed herein are methods of diagnosing and treating MYC-driven cancers by detecting a PVT1 splice variant in a biological sample from a subject.

Methods and compositions related to treating pancreatic cancer are described.

Disclosed are compositions and methods useful for targeting and internalizing molecules into cells of interest and for penetration by molecules of tissues of interest. The compositions and methods are based on peptide sequences that are selectively internalized by a cell, penetrate tissue, or both. The disclosed internalization and tissue penetration is useful for delivering therapeutic and detectable agents to cells and tissues of interest.

Provided herein, inter alia, are compositions and kits comprising epicardial-derived paracrine factors (such as, hypoglycosylated follistatin-like 1 (FSTL1)) for treating and repairing damage to cardiac tissue caused by cardiovascular disease, myocardial infarction (MI), other ischemic events, or cardiac-growth deficiency, as well as methods for using the same.