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Passage Bio was launched with the vision to be a first in class fully-integrated biotech company developing life transforming AAV-delivered in vivo therapeutics for the treatment of rare CNS diseases. In collaboration with the University of Pennsylvania Gene Therapy Program, Philadelphia-based Passage Bio plans to advance a portfolio of 5 rare disease indications in the neuro/CNS genetic space through IND-enabling studies using best-in-class AAV technology and know-how. Subsequently, Passage Bio will be responsible for all clinical development and will collaborate with Penn`s Orphan Disease Center to support these efforts.
At Travere Therapeutics, our mission is to identify, develop and deliver life-changing therapies to people living with rare disease. #InRareForLife
Organovo (NYSE MKT: ONVO) designs and creates functional, three-dimensional human tissues for medical research and therapeutic applications. The Company is collaborating with pharmaceutical and academic partners to develop human biological disease models in three dimensions. These 3D human tissues have the potential to accelerate the drug discovery process, enabling treatments to be developed faster and at lower cost. In addition to numerous scientific publications, their technology has been featured in The Wall Street Journal, Time Magazine, The Economist, and other outlets. Organovo is changing the shape of medical research and practice.
Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.
DiaCarta is a translational genomics and personalized diagnostics company that provides ultra-sensitive detection tools for cancer diagnosis, therapy identification and prognosis monitoring. Our mission is to provide ultra-sensitive and advanced technologies that will redefine the way molecular diagnostics and translational genomics impact healthcare treatment plans and the well-being of individuals around the world.