Hemophilia, a rare X-linked, inherited bleeding disorder, affects over 1.1 million people worldwide. Approximately 90% of those people are male, and an estimated 33,000 live in the United States. The disease is classified into different subtypes. Hemophilia A and hemophilia B are the two most common. With hemophilia A, there is a missing or defective blood clotting factor VIII; with hemophilia B, it’s factor IX. Of the two, hemophilia A is more prevalent.

Currently approved treatments for hemophilia A include rebalancing agents, factor replacement therapy, a factor VIII mimetic antibody and gene therapy. The sky-high hopes for gene therapy have been brought down to earth by how they have fared in the market, where the combination of multimillion-dollar price tags and lingering uncertainti

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