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As Intellia recently unveiled its latest promising data around one in a series of potentially game-changing gene therapies, the FDA on Tuesday sought to further encourage the field with new draft guidance on what should be submitted in a clinical trial application and what potentially concerning safety issues to track for these genome editing products.
The agency is upfront about the risks of genome editing, as Allogene was hit with a clinical hold and preclinical studies suggest potential risks. The draft guidance points to several specific risks associated with genome editing, including off-target editing, unintended consequences of on- and off-target editing, and the unknown long-term effects of on- and off-target editing.
As such concerns linger, sponsors may be on the hook for a long and expensive follow-up to any clinical work. As part of efforts to further ensure the safety of such developing products, the guidance recommends at least 15 years of long-term follow-up after product administration, which is in line with past guidance on gene editing.
The agency also calls for adequate monitoring of any off-target editing and adequate...