Category Archives: sickle

Gene Therapy and regenerative medicine companies.



Tim Miller, Ph.D., President & CEO
Dallas, TX
PlasmaTech Biopharmaceuticals/Abeona Therapeutics is focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. PlasmaTech’s lead program is a gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB) in collaboration with patient advocate groups, researchers and clinicians. In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDF™) Process and Polymer Hydrogel Technology (PHT™), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary

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Global Blood Therapeutics – respiratory distress syndrome

Global Blood Therapeutics initially focusing on indications with significant unmet need, including idiopathic pulmonary fibrosis (IPF) and acute respiratory distress syndrome (ARDS), in which hypoxia is believed to play a key role in disease pathogenesis and adverse patient outcomes. These are conditions in which the lungs cannot supply adequate oxygen to the blood. While supplemental oxygen therapy is a well-established lifesaving treatment in acute and chronic hypoxemic conditions, it is associated with a number of risks, including injury or infection as a result of intubation. This highlights a significant unmet … Read more