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MapLight Therapeutics is developing targeted, highly effective therapeutics to improve the lives of those with difficult-to-treat brain disorders. Today, there are few therapeutic options for people living with conditions such as schizophrenia, Alzheimer`s disease, Parkinson`s, and autism spectrum disorder. Available treatments are limited by modest efficacy and significant side effects. MapLight is committed to redefining this standard of care. The company`s unique discovery platform combines novel, proprietary technologies to uncover the individual circuits that misfire in brain disorders and treat those circuits with effective, safe therapeutics. MapLight was founded in 2019 by a team of renowned neuroscientists who led the discovery of the two groundbreaking technologies, optogenetics and STARmap.
We founded Cedilla at a time of fundamental and rapidly growing insight into the mechanisms underlying protein stability. Small molecules that degrade or stablize target proteins are precedented, but have been discovered serendipitously. Cedilla is taking an integrated approach to discover new drugs with this mode of action. Our present focus is to develop novel medicines in oncology, but this approach will also be applicable to other fields, including central nervous system disorders and genetic diseases. Cedilla launched with $56.2 million in Series A funding from Third Rock Ventures.
BioMarin is a global pharmaceutical company focused on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We remain steadfast to our original mission—to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mostly children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options and are usually ignored. Time is critical to patients with rare diseases, and we strive to quickly develop important therapies for them. The efficiency and speed of our research, development, manufacturing, and commercial efforts is at the heart of our ability to urgently deliver therapies. Our track record of developing and commercializing new treatments has been significantly faster than the industry average and is a remarkable accomplishment that is ingrained in our culture. The company`s product portfolio comprises five marketed products and multiple clinical and pre-clinical product candidates.
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.
Vysis, Inc. is a Downers Grove, IL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.