01:25 , Nov 28, 2017 |  BC Innovations  |  Distillery Techniques

Biomarkers

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...
19:11 , Oct 13, 2017 |  BioCentury  |  Regulation

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...
07:00 , Sep 15, 2016 |  BC Innovations  |  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...
07:00 , Jul 30, 2015 |  BC Innovations  |  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...
07:00 , Jun 25, 2015 |  BC Innovations  |  Distillery Therapeutics

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

Gastrointestinal disease INDICATION: Colitis Mouse studies suggest inhibiting MYH9 could help treat colitis. In a mouse model of chemical-induced colitis, levels of MYH9 were higher at sites of colonic epithelial injury and on LGR5-positive stem...
07:00 , Mar 12, 2015 |  BC Innovations  |  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...
07:00 , Sep 11, 2014 |  BC Innovations  |  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...
08:00 , Nov 7, 2013 |  BC Innovations  |  Distillery Therapeutics

Indication: Cardiovascular disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Cardiovascular disease Cardiomyopathy Myosin heavy chain 7 cardiac muscle-b (MYH7) Mouse studies suggest RNAi that targets a disease-causing mutation in MYH7 could help prevent hypertrophic cardiomyopathy...
08:00 , Jan 31, 2013 |  BC Innovations  |  Distillery Techniques

Technology: Disease models

Approach Summary Licensing status Publication and contact information Disease models Induced pluripotent stem (iPS) cell-derived models of familial hypertrophic cardiomyopathy (HCM) Patient-derived iPS cell models of familial HCM could help identify new treatments for the...
07:00 , Jul 12, 2012 |  BC Innovations  |  Distillery Therapeutics

Indication: Cardiovascular disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Cardiovascular disease Myocardial infarction (MI) Myosin heavy chain 6 cardiac muscle-a (MYH6) Mouse studies suggest blocking autoimmune activity against cardiac myosin could help reduce damage following...