Enzyme Replacement – PharmaLive

Sangamo Scores Bragging Rights for First-in-Human Gene Editing Trial

Blood, Clinical Trials, DNA, Enzyme Replacement, Enzyme Replacement Therapy (ERT), Fast Track Designation, FDA, Gene Editing, Gene Therapy, Genomics, Headquarters, Hemophilia B, Hunter Syndrome, Hurler-Scheie Syndrome, Liver Cells

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.

November 15, 2017/by BioSpace

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 Diseases

Concerted effort by governments, regulators, and industry is rapidly expanding development of new therapies for the more than 7,000 known rare diseases.

June 8, 2016/by Administrator