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Enzyvant is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 in the first half of 2018. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.
At Karuna, our purpose is to create and deliver transformative medicines for people living with psychiatric and neurological conditions. Through the cultivation of our deep expertise in neuroscience, and inventive thinking around drug discovery and development, we are advancing a pipeline of novel medicines with the goal of providing a meaningful difference in the lives of those affected by these conditions. Our culture is defined by our passion to serve those affected by mental illness. We are purpose-driven and intentional. We know impactful innovations don`t come easy, but are borne from passion and hard work. Together, we are working toward a goal bigger than ourselves.
Avectas is a cell engineering technology business developing a unique delivery platform to enable the ex vivo manufacture of gene-modified cell therapy products, which will retain high in vivo functionality. Our vision is to position the non-viral SOLUPORE® cell engineering technology to be integrated into manufacturing processes for multiple autologous and allogeneic therapies and commercialised through development and license agreements.
Omeros Corporation is one of the leading companies in the Healthcare, Pharmaceuticals, and Biotech sector.
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.