FDA lays down the law on gene therapy, offering guidances on both diseases and procedures
As first announced in January, the FDA’s Center for Biologics Evaluation and Research (CBER) on Wednesday offered a swath of new draft guidance documents on human gene therapies — offering a look at what it expects from an industry that’s beginning to take off.
The six guidance documents focus on developing hemophilia, rare disease and retinal disorder gene therapies, and include one on chemistry, manufacturing and control (CMC) information, one on long term follow-up observational studies collecting data on adverse events and one on the testing of retroviral vector-based therapies.
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