Business-in-Brief

Advanced medicine

• The FDA’s Center for Biologics Evaluation and Research (CBER) unveiled six new draft guidance documents covering gene therapy. The guidance documents focus on i) rare disease therapies, ii) retinal disorder therapies, iii) haemophilia therapies, iv) chemistry, manufacturing and control (CMC) information, v) long-term follow-up observational studies that collect data on adverse events, and vi) retroviral vector-based therapies testing.
• Japanese scientists will use induced pluripotent stem (iPS) cells in a Parkinson’s trial for the first time, following successful animal studies. In an attempt to replace the lost dopaminergic cells responsible for Parkinson’s, the researchers, led by Jun Takahashi at Kyoto University’s Center for iPS Cell Research and Application (CiRA), will derive dopaminergic progenitors from iPS cells and inject roughly five million of them into the forebrain. Sumitomo Dainippon Pharma hopes to manufacture and start selling cellular medicine based on the data from the clinical trials by March 2023.