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 annually in the US?

• 10

• 40

• 20

• 30

What has been the standard of care for the disease until now?

• Gene therapy

• Stem cell transplant

• Radiation therapy

• Chemotherapy

What is the median follow-up time for the new gene therapy according to the New England Journal of Medicine?

• 10.2 months

• 18.7 months

• 12.5 months

• 16.4 months

What is one proposed method to make the therapy financially accessible to families?

• Single upfront payment

• Government subsidy

• Payment over time based on success

• Crowdfunding

Why did Mustang choose to focus on this gene therapy despite primarily working on cancer therapies?

• It was a more profitable market

• They received a government grant

• They had an experienced team in gene and cell therapy

• They wanted to diversify their portfolio

What other rare disease is mentioned as a potential target for gene therapy?

• Sickle cell anemia

• Wiskott Aldrich syndrome

• Cystic fibrosis

• Huntington's disease

What is Mustang's goal regarding the commercialization of their gene therapy program?

• Abandon the program

• Sell the program to a larger company

• License the technology to other firms

• Bring it to market themselves