Gene Therapy Manufacturing Remains Large Hurdle For Development, US FDA's CBER Director Says
Scale-up in particular is a challenge and FDA wants to help lower the barrier, CBER Director Peter Marks tells rare disease conference.
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Agency's agreement with rare disease advocacy group will facilitate patient meetings with review divisions, as well as enhance communication of safety information.
FDA wants more companies to adopt continuous manufacturing, which could expand the US manufacturing base.
Lengthy safety registries, extensive premarket testing for replication competent retrovirus, and manufacturing submission advice likely; pending CBER documents informed by review and approval of Novartis' Kymriah, Kite's Yescarta, and Spark's Luxturna.