BioCentury | Dec 2, 2020
Distillery Therapeutics

Skeletal muscle-specific derivative of myosin inhibitor tool compound for spasticity and muscle stiffness

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...
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 | Nov 14, 2019
Targets & Mechanisms

New roles for HDACs in the cytoplasm and muscle disorders

While HDACs are known for their epigenetic function, NIBR researchers have discovered cytoplasmic HDAC4 substrates involved in muscle physiology. The findings suggest blocking HDAC4 deacetylation activity could treat muscle atrophy and other musculoskeletal disorders. HDACs...
BioCentury | Nov 28, 2017
Distillery Techniques


TECHNOLOGY: SNPs SNPs on GDF1, MYH6 and FLT4 could help predict the risk of subtypes of congenital heart disease. Whole-exome sequencing of 2,871 congenital heart disease patients identified associations between severe disease in the Ashkenazi...
BioCentury | Oct 13, 2017

Seeking surrogates

Thanks to flaws in PTC Therapeutics Inc. ’s trials, the recent FDA advisory committee meeting to discuss Translarna ataluren did nothing to clarify what level of dystrophin expression would be necessary to support an accelerated...
BioCentury | Sep 15, 2016
Targets & Mechanisms

KOR-recting MS

Although the failed Phase II multiple sclerosis trial of Biogen Inc. 's anti-LINGO mAb in June dealt a blow to myelin repair, a slew of compounds acting by different mechanisms lies behind it in clinical...
BioCentury | Jul 30, 2015
Targets & Mechanisms

Muscling in on atherosclerosis

A finding from the University of Virginia overturning assumptions about the biology of atherosclerotic plaques could prompt companies to rethink their therapeutic strategies for the disease. Using a series of in vitro and in vivo...
BioCentury | Jun 25, 2015
Distillery Therapeutics

Therapeutics: Myosin heavy chain 9 (MYH9); leucine-rich repeat-containing G protein-coupled receptor 5 (LGR5; GPR49)

...inhibiting MYH9 could help treat colitis. In a mouse model of chemical-induced colitis, levels of MYH9...
...MYH9 expression or vehicle. Next steps could include identifying and developing druggable MYH9 inhibitors. TARGET/MARKER/PATHWAY: Myosin heavy chain 9...
...expression or vehicle. Next steps could include identifying and developing druggable MYH9 inhibitors. TARGET/MARKER/PATHWAY: Myosin heavy chain 9 (MYH9...
BioCentury | Mar 12, 2015
Distillery Therapeutics

Therapeutics: Core-binding factor β subunit (CBFB; CBF-β); myosin heavy chain 11 smooth muscle (MYH11; SMMHC); runt-related transcription factor 1 (RUNX1)

Cancer INDICATION: Acute myelogenous leukemia (AML) In vitro, mouse and patient sample studies suggest a small molecule protein-protein inhibitor could help treat the inv(16) subtype of AML. The subtype is driven by an oncogenic CBFB-SMMHC...
BioCentury | Sep 11, 2014
Distillery Therapeutics

Indication: Cardiovascular disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Cardiovascular disease Cardiomyopathy; heart failure Myosin heavy chain 7 cardiac muscle-b (MYH7) Mouse studies suggest a cluster of long noncoding RNAs (lncRNAs) encoded by Myh7 loci...
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