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Stimulating mucosal-associated invariant T cells for melanoma, breast cancer

DISEASE CATEGORY: Cancer INDICATION: Melanoma; breast cancer A previously reported ribitylaminouracil-based synthetic antigen designed to stimulate mucosal-associated invariant T cells (MAITs) could treat melanoma and breast cancer. In a mouse model of melanoma, ex vivo pulsing of tumor...

CaMKII gene therapy to preserve retinal ganglion cells

DISEASE CATEGORY: Ophthalmic; neurology INDICATION: Glaucoma CaMK2 gene therapy could treat eye conditions involving damage to retinal ganglion cells, including glaucoma. In four mouse models of retinal...

Dual targeting of CTLA-4 and CD47 for colon cancer

DISEASE CATEGORY: Cancer INDICATION: Colon cancer A bispecific fusion protein targeting CTLA-4 and CD47 could promote immunity against solid tumors by depleting immunosuppresive tumor Tregs. The heterodimeric fusion protein combined a mAb targeting the checkpoint...

Blocking unfolded protein response, integrated stress response for necrotizing fasciitis, lung cancer

DISEASE CATEGORY: Infectious disease; cancer INDICATION: Streptococcus; lung cancer Targeting the unfolded protein response and integrated stress response mediators PERK and eIF-2α could treat group...

Brain metabolite analog to treat ischemic stroke

DISEASE CATEGORY: Neurology INDICATION: Stroke An analog of γ-hydroxybutyrate (GHB), an endogenous brain metabolite of  the neurotransmitter GABA, could treat ischemic stroke. In vitro and cell-based studies revealed the kinase CAMK2A as the most likely target...

Enhancing NK cell cancer therapies with bispecific antibodies

DISEASE CATEGORY: Cancer INDICATION: B cell lymphoma; breast cancer Bispecific antibodies targeting both an NK cell activating receptor and a tumor antigen could enhance the efficacy of NK cell-based cancer...

ESK981 identified as PIKFYVE inhibitor and prostate cancer candidate

DISEASE CATEGORY: Cancer INDICATION: Prostate cancer A University of Michigan-led team showed that ESK981, a multi-tyrosine kinase inhibitor that also inhibits the autophagy regulator PIKFYVE, could treat...

VEGF to prevent aging-related diseases

DISEASE CATEGORY: Musculoskeletal; hepatic; cancer; cardiovascular INDICATION: Sarcopenia; osteoporosis; non-alcoholic steatohepatitis (NASH); cancer (unspecified) Increasing VEGF expression could help prevent aging-related diseases including sarcopenia, osteoporosis, NASH and cancer. In a transgenic mouse model that expresses circulating...

Targeting CHMP7 for sporadic and familial ALS

DISEASE CATEGORY: Neurology INDICATION: Amyotrophic lateral sclerosis (ALS) Inhibition of CHMP7, a mediator of nuclear pore complex quality control, could treat sporadic and familial forms of ALS, where the most common genetic cause of disease is...

Targeting CD93 to treat pancreatic cancer, melanoma

DISEASE CATEGORY: Cancer INDICATION: Pancreatic cancer; melanoma Inhibiting the adhesion protein CD93 or its interaction with IGFBP7 could treat pancreatic cancer, melanoma and other cancers by normalizing tumor vasculature and improving immune cell infiltration and...

Inhibiting multiple cell death pathways to extend neutrophil shelf life for transfusion

DISEASE CATEGORY: Hematology INDICATION: Transfusion Simultaneously targeting multiple cell death pathways could extend the shelf life of granulocyte transplants. In cultures of primary human neutorphils, a cocktail of the antioxidant N-acetyl cysteine, the lysosomal membrane permeabilization...

PARIS farnesylation for Parkinson’s disease

DISEASE CATEGORY: Neurology INDICATION: Parkinson’s disease (PD) Farnesylation of the transcriptional repressor PARIS could help treat PD by alleviating disease-associated inhibition of the metabolic regulator...

STING agonism as treatment for pain from bone metastases

DISEASE CATEGORY: Cancer; neurology INDICATION: Breast cancer; lung cancer; pain STING agonists could treat pain resulting from bone metastases and bone destruction. In a mouse model of lung cancer with bone metastasis, a tool compound...

Targeting SMPD3 to treat polycystic kidney disease

DISEASE CATEGORY: Renal INDICATION: Polycystic kidney disease (PKD) Inhibiting exosome generation by targeting the enzyme SMPD3 could treat autosomal dominant PKD. In a mouse proximal tubule cell line, a tool compound inhibitor of SMPD3 reduced the...

ISM1 as a therapy for diabetes, fatty liver disease

DISEASE CATEGORY: Endocrine/metabolic; hepatic INDICATION: Diabetes; liver disease The adipokine ISM1 could treat diabetes and non-alcoholic fatty liver disease (NAFLD). High levels of ISM1 RNA in subcutaneous mature adipocytes from obese patients were associated with...