Mustang Bio CEO on Next Steps for 'Bubble Boy' Cure

The transcript discusses the potential of gene therapy to treat rare diseases, focusing on patient statistics, treatment viability, manufacturing capacity, and FDA approval. It addresses cost and reimbursement strategies for families and highlights Mustang's focus on gene therapy, product development, and market strategy. The potential for gene therapy to address other rare diseases is also explored.

How many new patients are diagnosed with the disease in the US each year?

What is the standard of care for patients until gene therapy is approved?

What is the expected lifespan of patients treated with the therapy?

What challenges are associated with the manufacturing process for gene therapy?

How does the reimbursement model for the therapy differ from traditional payment methods?

What are some other rare diseases that could potentially benefit from gene therapy?

What is Mustang's goal regarding the development and marketing of their gene therapy?