Gene Editing: Off-Target Effects Should Be A Key Safety Focus During Development, US FDA Says
Clinical development programs for human gene therapy products that incorporate genome editing should address the potential risks of off-target editing as well as unintended consequences of on-target effects, agency says in new draft guidance.
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In final guidance documents on development of CAR-T cell products and human genome therapies incorporating genome editing, US FDA says that sponsors should plan for continued follow-up, with funding, even if they cease operations or withdraw the IND.
Gene Editing: US Panel Review Of Vertex’s Exa-Cel To Focus On Nonclinical Assessment Of Off-Target Effects
Advisory committee discussion on the adequacy of Vertex’s analyses, using in silico and cellular methods, of off-target alterations with the gene therapy for sickle cell disease could help guide nonclinical assessment of future products made with CRISPR/Cas9-mediated gene editing.
Regulators want to help smooth the process of bringing a gene therapy to market, hoping to iron out ‘surprises’ and even address reimbursement issues for the sake of patients.