Hemophilia Seen As Good Testing Ground For Commercializing Gene Therapy

With a well characterized genetic driver, a clear marker to show whether it's working, and available treatments that carry high annual costs, hemophilia may be a good testing ground for commercializing gene therapy, but payers still have to work out how to reimburse the one-time medicines.

3-D Rendering Red Blood Cells

Hemophilia may be one of the clearest examples of a win-win situation for the gene therapy field, because it's a disease with a well-characterized genetic cause, a clear marker of whether treatment is working, regulatory support for novel agents and the currently available therapies are incredibly expensive.

The Alliance for Regenerative Medicine (ARM) hosted a panel on Oct

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