Barely a week after announcing it was moving into a new corporate headquarters, Sangamo Therapeutics announced that the company has dosed the first patient in its Phase I/II gene therapy trial.
Incentives, Opportunities Abound as Orphan Drug Demand Grows
Biopharma, Bones, Breakthrough Therapy Designation, CDC, Congress, Department of Health and Human Services (HHS), EMA, Enzyme Replacement, EU, FDA, FDA/Regulatory, glioblastoma Multiforme (GBM) Brain Cancer, Hypophosphatasia (HPP), June 2017, Metabolic Disorders, Orphan Drug Act, Rare DiseasesConcerted effort by governments, regulators, and industry is rapidly expanding development of new therapies for the more than 7,000 known rare diseases.