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At Spark Therapeutics, we don`t follow footsteps. We create the path. We were born of innovation, springing from the curiosity, imagination and dedication of remarkable scientists and healthcare visionaries. Our mission is seemingly impossible to others, but not to us: Challenge the inevitability of genetic disease by discovering, developing and delivering treatments in ways unimaginable – until now. Since our founding, we have been committed to bringing a wide range of expertise to build a fully integrated gene therapy company focused on inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver, such as hemophilia. We are seeking talented individuals with diverse experiences, abilities and interests who have the curiosity, courage and drive to reimagine a new health care paradigm. Join us on a journey through uncharted territory – seeking to bring gene therapy for genetic diseases to people worldwide. The resilience of the people we serve is our inspiration to break barriers, as we strive to turn genes into medicine for those with inherited diseases. Spark is a member of the Roche Group.
Aileron Therapeutics, based in Cambridge, Massachusetts, is a biopharmaceutical company developing and advancing a revolutionary class of drugs called Stapled Peptides. The company`s proprietary Stapled Peptides platform is a breakthrough approach to creating drugs for highly sought after biological targets using its novel peptide stabilizing technologies.
REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO`s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO`s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO`s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.
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.
Just Evotec Biologics is an integrated design company that delivers advanced biotherapeutics discovery, CDMO capabilities and FIH to commercial manufacturing. They focus on accelerating and expanding access to biotherapeutics through scientific and tec...