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We are a genome editing company developing genetic medicines for the fight against rare pediatric diseases. GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV based gene therapies With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types.
Prescos is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
At Prevail Therapeutics, we are pioneering innovative therapies that aim to cure neurodegenerative disorders, such as Parkinson`s disease. Our mission is to translate breakthrough advances in human genetics and gene therapy technology into life-changing medicines that slow, stop and reverse the neurodegenerative process. Our novel gene therapies target patients with urgent unmet needs, where there are currently no available therapies to change the progressive course of their diseases. Prevail was formed in 2017, and as an emerging biotechnology company, we are growing rapidly. We are building a team of dedicated, creative and talented individuals who share our passion for serving patients with neurodegenerative diseases. We pride ourselves on following great science, and we value collaboration, integrity and agility. We are based in the Alexandria Center for Life Science in New York City.
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.
Greffex™ is a privately held, multi-national company, headquartered in Aurora, Colorado. Established in 1999, Greffex has developed a novel genetic platform for the development and production of vaccines. Our proprietary GREVAX™ Universal Platform delivers vaccines in a faster, more affordable and more efficient manner. Once a pathogen has been identified, Greffex`s team designs and produces a GREVAX™ vaccine in as little as 4 weeks. The state-of-the-art flexibility of the GREVAX™ Universal Platform makes it the ideal delivery vehicle for vaccines against a wide variety of infectious disease, including but not limited to Pandemic Influenza strains, Anthrax and Dengue Fever, as well as Malaria, Hepatitis C, Respiratory Syncytial Virus (RSV), Ebola, HPV, Plague, Tuberculosis and a host of other vaccine candidates.