A draft guidance on how sponsors can provide sufficient CMC information for human gene therapy (GT) INDs is among a series of six GT draft guidances released by FDA. Also addressed are gene therapies targeted for the treatment of hemophilia, retinal disorders, and rare diseases. The two additional drafts address testing for replication-competent retrovirus during the manufacture of retroviral vector-based products and follow-up patient monitoring, and long term follow up. In a statement, FDA Commissioner Gottlieb highlighted the agency’s efforts to provide a policy framework for GT development, review and reimbursement that will support their advancement.
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