DNA- and RNA-based gene therapies have huge potential in enabling the pursuit of novel therapeutic targets. However, the broad uptake of gene therapies has been limited by challenges with delivery, manufacturing, safety and commercialization. Delivery remains a key hurdle, as therapeutic modulation requires tissue-specific localization of DNA/RNA payloads; however, systemically administered payloads must resist degradation or excretion before reaching their targets, while minimizing immunogenicity.
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Competing Interests
The authors of this article are employees of McKinsey & Company, a management consultancy that works with the world’s leading biopharmaceutical and biotechnology companies. The research for this specific article was funded by McKinsey’s Life Sciences practice.
