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Renovacor is a preclinical gene therapy company developing a pipeline of innovative and proprietary AAV based gene therapies for BAG3 gene mutation-associated diseases in areas of high unmet medical need. Renovacor`s therapeutic focus is initially on cardiovascular disease, with a lead program in BAG3 mutation-associated dilated cardiomyopathy.
Loxo Oncology is committed to the discovery, development, and commercialization of targeted cancer therapies with best-in-class potential. Our diverse pipeline reflects the convergence of proven therapeutic technologies with emerging insights into the underlying susceptibilities of cancer and drug resistance. It is an exciting time to develop cancer drugs. Diagnostic methods are yielding strong clues about which cancer features are actionable, “driver alterations,” in contrast to those merely along for the ride as “passenger alterations.” In the last five years, these insights have transformed the standard of care in melanoma, lung cancer and other cancers. Picking a target is just the first step in building a cancer drug. Good chemists build good drugs. The physical properties of a compound have much to do with its ultimate success or failure. Is it stable? Is it soluble? Is it absorbed? Does it reach its target? Does it engage the target? Does it cause toxicity elsewhere in the body before it achieves maximum efficacy in the cancer? In our opinion, drugs that achieve excellent exposure in the body and are highly specific for their intended targets have the highest chances of success. Once an attractive target is selected and a drug with best-in-class properties is constructed, setting up the right clinical development plan is of utmost importance. Our goal is to generate a clinical path that reflects the underlying scientific hypothesis that made the drug interesting in the first place. This often means developing the drug in a population of patients whose tumors possess a specific vulnerability targeted by the drug. Loxo Oncology was built around a team of full-time professionals and engaged scientific advisors who are aligned in choosing the most actionable targets, and pursuing them through disciplined clinical trial approaches. Our license and collaboration agreement with Array Biopharma and our experienced team allow us to construct drugs with best-in-class properties from the ground up.
BioWa is a Princeton, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Clinical Glycoproteomics + Artificial Intelligence
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.