process validation for expedited approval drugs

White Paper:

The High Bar for Gene Therapy CMC

How drug developers can step up to meet regulatory expectations for gene therapy chemistry manufacturing and controls

After well publicized stumbling blocks in the late-90s, vigor was restored to the gene therapy field in 2017 when the U.S. FDA greenlighted two chimeric antigen receptor (CAR) T-cell treatments for acute lymphoblastic leukemia, followed by the first directly administered gene therapy, developed to treat a hereditary form of vision loss.

Years later, there are still only a handful of approvals in the space (two gene therapies and five CAR-T cell therapies),
but pipelines are brimming with potential.

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