DISEASE CATEGORY: Musculoskeletal; ophthalmic disease INDICATION: Myotonic dystrophy type 1 (DM1); ophthalmic A Scripps Research team led by Expansion Therapeutics Inc. co-founder Matthew Disney has designed an RNA-targeting small molecule that could treat DM1 and Fuchs...

DISEASE CATEGORY: Cancer INDICATION: Breast cancer Inhibiting the RNA epigenetic reader protein YTHDF3, which recognizes the m6A methylation mark, could treat brain metastasis in breast cancer. In breast cancer patients, high expression of YTHDF3 mRNA was associated with...

DISEASE CATEGORY: Infectious disease INDICATION: Sepsis Inhibiting the scaffold protein SQSTM1 or its extracellular receptor INSR could treat bacterial sepsis. Among 40 patients with bacterial sepsis, survivors had lower peripheral blood mononuclear cell mRNA levels of SQSTM1...

DISEASE CATEGORY: Cancer INDICATION: Brain cancer Inhibiting epigenetic regulator BMI1 alone or in combination with BCL-2 inhibition could treat diffuse intrinsic pontine glioma (DIPG), an aggressive childhood brain cancer. A screen in two human DIPG...

DISEASE CATEGORY: Autoimmune disease INDICATION: Lupus Promoting the expression of the metalloprotease MYSM1 could treat systemic lupus erythematous (SLE). MYSM1 mRNA and protein levels were lower in peripheral blood mononuclear cells (PBMCs) from 14 SLE...

DISEASE CATEGORY: Ophthalmic disease INDICATION: Ocular hypertension Penn researchers showed that NLY01 from Neuraly Inc., could treat ocular hypertension. In mice modelling ocular hypertension, the pegylated exenatide reduced retinal ganglion cell death and mRNA levels of...

DISEASE CATEGORY: Autoimmune disease INDICATION: Multiple sclerosis (MS) Extracellular vesicles derived from oligodendrocytes could prevent and treat MS. The vesicles were collected from the supernatant of mature oligodendrocytes differentiated from mouse oligodendrocyte progenitor cells. In mouse models...

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Mitochondrial disease A PFKM inhibitor could treat MELAS (mitochondrial encephalopathy, lactic acidosis and stroke-like episodes) syndrome, which is most often caused by mutations in mitochondrially encoded MT-TL1. A cell-based screen of 17,568 small...

DISEASE CATEGORY: Cancer INDICATION: Breast cancer; colorectal cancer; melanoma PCSK9 inhibition could treat melanoma, breast, colorectal and other cancers, and enhance the efficacy of PD-1 inhibitors. In cohorts of melanoma, liver, pancreatic, bladder, lung, kidney and...

DISEASE CATEGORY: Neurology INDICATION: Alzheimer’s disease (AD) Blocking MCL1, which inhibits apoptosis, could treat Alzheimer’s disease. A high-throughput screen in HEK cells of 2,204 drug candidates or FDA-approved drugs yielded 20 agents, including an MCL1...

DISEASE CATEGORY: Cardiovascular INDICATION: Atherosclerosis Targeting the ER cargo receptor SURF4 could treat atherosclerosis and dyslipidemia. In a mouse model of atherosclerosis, liver-specific heterozygous or homozygous SURF4 deletion reduced cholesterol and triglyceride levels in plasma...

DISEASE CATEGORY: Musculoskeletal INDICATION: Spasticity A small molecule inhibitor of fast skeletal muscle myosin isoforms could treat spasticity and muscle stiffness. The compound is a morpholine derivative of blebbistatin, a tool compound inhibitor of multiple...

DISEASE CATEGORY: Musculoskeletal INDICATION: Osteoarthritis Inhibiting the tyrosine protein kinase receptor ROR2 could treat osteoarthritis. Expression levels of the ROR2 ligand WNT5A were higher in human osteoarthritic cartilage than in healthy cartilage. In a mouse fibroblast...

DISEASE CATEGORY: Infectious disease INDICATION: Sepsis Inhibiting the myeloid receptor TREML4 could treat sepsis. In mice with acute polymicrobial sepsis, systemic knockout of TREML4 prevented macroscopic thymic involution and increased proportions of mature and immature...

DISEASE CATEGORY: Cardiovascular INDICATION: Cardiomyopathy Inhibiting AQP1, a water channel expressed in cardiac and vascular tissues, could treat hypertrophic cardiomyopathy. Among 26 patients with aortic stenosis, high AQP1 mRNA expression in cardiac biopsies was associated with...