Scientific Wave Pushes CBER Into Brighter Light

What are US regulators doing to prepare for adequate review of the upcoming wave of gene therapy applications, and what other actions could CBER take to ensure it is able to facilitate the expected onslaught of breakthroughs in this field? Consultant Nancy Bradish Myers weighs in.  

rough colored ocean wave falling down at sunset time
The over 900 active investigational new drug applications in the US are proof the gene therapy wave continues to gather strength and speed. • Source: Shutterstock

Scientific leaps forward have opened the floodgates of interest and investment in gene therapy. With the advent of safe and effective gene therapy vectors, such as Adeno Associated Virus (AAV) vectors, the US Food and Drug Administration has seen a “surge” in gene therapy products in early development, as noted by Center for Biologics Evaluation and Review Director Peter Marks and then-FDA Commissioner Scott Gottlieb last year.

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