Creating A Scalable Cell And Gene Therapy Ecosystem

Optimism abounds about the potential for cell and gene therapies to revolutionize patient care. We’ve seen groundbreaking drugs come to market for treating spinal muscular atrophy, metachromatic leukodystrophy, and retinal disease. Advances in CAR-T cell therapy are giving cancer patients new confidence. Every breakthrough in research holds promise for better patient outcomes, and not just for those with rare diseases; increasingly, there’s hope that discoveries will result in improved care for more common conditions and even lead to one-time cures.

Tempering that optimism is a mix of systemic obstacles that limit the effectiveness and reach of these innovations. Many new drugs come with hefty price tags. Gene therapies for treating hemophilia...