Gene Editing: Exa-Cel Panel Review Raises Practical Concerns About Theoretical Risks
US FDA advisory committee members questioned how potential off-target effects with Vertex’s gene therapy for sickle cell disease should be conveyed to patients and families, and whether labeling should say anything about these theoretical risks.
You may also be interested in...
Vertex’s Casgevy has made history as the world’s first approved CRISPR gene editing therapy after the UK’s MHRA authorized the medicine ahead of its US and European regulatory counterparts.
Great Britain is first to approve the groundbreaking CRISPR-based therapy for sickle cell and beta thalassemia. Vertex is yet to reveal its price, however, and wants to play down early expectations of its commercial potential.
US FDA draft guidance does not specify the number of donor samples to test in nonclinical studies or the process for detecting genetic variants potentially susceptible to off-target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.’