What Are The Key Ethical And Regulatory Considerations As Gene Editing Technologies Advance Toward Widespread Clinical Use?

As gene editing therapies continue to evolve, their promise to treat previously incurable diseases grows—but so too does the urgency to ensure their safety and reliability. Experts across the field are rising to the challenge, each contributing innovative solutions to manage the risks associated with these powerful technologies.

Christopher Bohl, Ph.D., highlights how traditional ADME in vitro systems are being repurposed to detect off-target hepatotoxicity —a vital step, even for therapies not aimed at the liver. Meanwhile, Mission Bio’s Vanee Pho, Ph.D., discusses the value of single-cell, multi-omic platforms to characterize allelic variants and editing outcomes with exceptional accuracy, helping regulators and developers track both intended and unintended genomic changes. Steve Becker of Broken String Biosciences emphasizes the high financial stakes associated with poor off-target characterization and presents emerging tools that quickly and thoroughly assess genome editing-induced DNA breaks. Lastly, Jakob Reiser, Ph.D., of Vector BioMed outlines how improvements in lentiviral vector manufacturing are reducing costs and turnaround time.

Together, these insights reflect the biotech community's collective commitment to balancing innovation with safety. Explore how these critical advancements in gene therapy safety testing are evolving the way to treat previously incurable diseases.