US FDA's Authority Over CRISPR Is Adequate, NASEM Finds, But Off-Label Challenge May Emerge
National Academies’ report on the fast-advancing genome-editing technologies points out that ways to improve musculature in dystrophy patients will almost certainly raise issues about treatment use in narrow population versus enhancement use in broader population. In most cases, however, specificity of genome-edited products will help limit use of the new therapies to targeted populations.
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Newly formed biotech company hopes to avoid patent melee and use gene editing technology to develop therapy for up to 80% of boys with DMD; trial outcomes to drive terms of future licensing deals.
Regenerative Medicine In Cures: Conditional Approval Dropped In Lieu Of ‘Breakthrough’-Style Approach
Push to open up new US pathway for regenerative medicine helped the 21st Century Cures bill make it to the finish line in the lame duck Congress – but only after the proposal has been dramatically revised to fit into the established BLA pathway.
Multiple simultaneous sponsors, foreshortened regulatory reviews, and a packet of unanswered post-approval efficacy issues make for what should be very competitive – and inherently comparative – post-market assessment period for the upcoming SARS-CoV-2 vaccines.