‘Pull Every Lever’: FDA’s Marks Worries Gene Therapies Could Falter Without Help To Lower Cost
CBER’s director says manufacturing and regulatory issues need to be resolved to ensure the gene therapy sector thrives.
You may also be interested in...
US FDA draft guidance does not specify the number of donor samples to test in nonclinical studies or the process for detecting genetic variants potentially susceptible to off-target effects. An advisory committee found adequate Vertex's analyses, which the sponsor called ‘the most comprehensive evaluation of off-target potential performed to date.’
Some worries about heritable genetic modifications are subsiding and Center for Biologics Evaluation and Research Director Peter Marks said sponsors should consider the US for regulatory advice and clinical trials in the space.
CBER Director Peter Marks said the US may have to consider whether to maintain its ban on the practice of creating embryos to include heritable genetic modifications if other countries allow the practice.