BioCentury | Oct 31, 2020
Distillery Therapeutics

Blocking ROR2 for osteoarthritis

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...
BioCentury | Oct 31, 2020
Distillery Therapeutics

TREML4 as a sepsis target

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...
BioCentury | Oct 30, 2020
Distillery Therapeutics

Targeting water channel AQP1 for hypertrophic cardiomyopathy

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...
BioCentury | Oct 30, 2020
Distillery Therapeutics

KSP as a neuroblastoma target

DISEASE CATEGORY: Cancer INDICATION: Neuroendocrine tumors Inhibiting the mitotic kinesin KSP could treat neuroblastoma, a pediatric sympathetic nervous system malignancy. In neuroblastoma patients, high KSP expression in tumors was associated with high-risk neuroblastoma, advanced tumor...
BioCentury | Oct 28, 2020
Distillery Therapeutics

Small molecule ERG, FLI1 inhibitor identified for retinopathy of prematurity

DISEASE CATEGORY: Ophthalmic INDICATION: Retinopathy of prematurity A tool compound that inhibits multiple E26 transformation-specific transcription factors, including ERG and FLI1, could treat retinopathy associated with premature birth. In the oxygen-induced mouse model of retinopathy...
BioCentury | Oct 28, 2020
Distillery Therapeutics

Inhibiting IL-33-driven inflammation to treat brain cancer

DISEASE CATEGORY: Cancer INDICATION: Brain cancer Targeting nuclear accumulation of IL-33 could treat glioblastoma multiforme by reducing recruitment of pro-tumorigenic innate immune cells to the tumor microenvironment. In patients with glioblastoma multiforme, high expression of...
BioCentury | Oct 28, 2020
Distillery Therapeutics

STK25 and TAO1 identified as schistosomiasis targets

DISEASE CATEGORY: Infectious disease INDICATION: Infectious Inhibiting the serine/threonine kinases STK25 and TAO1 in parasitic flatworms could treat schistosomiasis. An RNAi screen of 2,320 protein-encoding genes in adult Schistosoma mansoni identified 19 genes encoding protein kinases involved in...
BioCentury | Oct 28, 2020
Distillery Therapeutics

LIMS1 identified as NSCLC target

DISEASE CATEGORY: Cancer INDICATION: Non-small cell lung cancer (NSCLC) Targeting the focal adhesion protein LIMS1 could treat NSCLC. In patients with lung adenocarcinoma, LIMS1 expression was higher in tumor tissue compared with normal lung tissue,...
BioCentury | Oct 28, 2020
Distillery Therapeutics

Targeting the NMDA receptor-TRPM4 complex for neurodegeneration

DISEASE CATEGORY: Neurology INDICATION: Stroke; neurology Disruption of the NMDA receptor-TRPM4 complex could treat ischemic stroke and other neurodegenerative diseases with NMDA receptor involvement. In mouse hippocampal neurons deprived of oxygen and glucose or treated...
BioCentury | Oct 27, 2020
Distillery Therapeutics

PCF11 binding site on E3 ubiquitin ligase MAGEA11 identified as neuroblastoma target

DISEASE CATEGORY: Cancer INDICATION: Neuroendocrine tumors Interrupting the interaction between the oncogenic E3 ubiquitin ligase MAGEA11 and its substrate PCF11 could treat neuroendocrine tumors. Crystallographic analysis of the MAGEA11-PCF11 complex identified a shallow cleft on...
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