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Janpix is discovering and developing monovalent small molecule degraders of Signal Transducer and Activator of Transcription (“STAT”) proteins. Most members of this family of transcription factors have been known for some time and the relevance of STAT3 & STAT5, in particular, in certain cancers is well validated. Although disrupting the function of STAT proteins is very challenging, Janpix has developed new chemistry to address this complex problem.
Amisure International is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California.
Organovo (NYSE MKT: ONVO) designs and creates functional, three-dimensional human tissues for medical research and therapeutic applications. The Company is collaborating with pharmaceutical and academic partners to develop human biological disease models in three dimensions. These 3D human tissues have the potential to accelerate the drug discovery process, enabling treatments to be developed faster and at lower cost. In addition to numerous scientific publications, their technology has been featured in The Wall Street Journal, Time Magazine, The Economist, and other outlets. Organovo is changing the shape of medical research and practice.
Exonics Therapeutics was launched in February 2017 to advance the research of our scientific founder, Dr. Eric Olson and his laboratory at the University of Texas Southwestern Medical Center (UTSW) to develop treatments for patients with neuromuscular diseases. Dr. Olson is one of the world’s leading experts in the study of muscle cells and the application of gene editing to treat these types of diseases. In particular, Dr. Olson’s laboratory has used adeno-associated virus (AAV) to deliver a CRISPR/Cas9 technology that can identify and repair exon mutations to restore the production of dystrophin, a protein that helps stabilize and protect muscle fibers. Dystrophin is the protein missing in boys with Duchenne. The loss of dystrophin causes Duchenne.