The heart of market access: opportunities and challenges for cell and gene therapy development for orphan and prevalent cardiovascular diseases

Heart disease is the leading cause of death globally, and there is a need for better medicines. No cell and gene therapies (CGTs) for heart disease are approved, but a new generation of companies are advancing promising science. The pipeline of CGTs is mostly focused on in vivo AAV-based therapies for prevalent cardiovascular (CVD) conditions, in contrast to broader trends favoring an initial focus on rare diseases seen in other therapeutic areas. CGTs for orphan heart disease indications have relevant benchmarks that could be used to justify the value and price for a one-time, potentially curative therapy. Significant challenges stand in the way of the development, approval, pricing, and adoption of even highly effective CGTs for prevalent CVD indications. Overcoming these will require scientific breakthroughs; heavy investment in CGT manufacturing technology and capacity; commercial and financial sophistication; and a focus on the needs of patients.

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