The latest developments on the gene therapy frontier Gene therapy has reached clinical reality with three dozen FDA approvals, but mounting safety concerns have intensified regulatory scrutiny. As developers navigate seven-figure price tags and limited adoption, the field balances breakthrough potential against sobering setbacks. INCLUDED IN THIS TRENDLINE Scribe’s Benjamin Oakes on a rare IPO and bringing CRISPR to the masses UniQure to file gene therapy for approval in reversal reflecting major shifts at FDA Regenxbio to resubmit gene therapy as FDA backtracks on another drug rejection
