Access to Gene Therapies for Rare Diseases: Barry Byrne, MD, PhD


The director of the Powell Gene Therapy Center at the University of Florida discussed improving access to gene therapies for patients with rare diseases worldwide.

“Access is a really important issue in the rare disease community, because we're representing patients worldwide... we've spent a considerable effort on understanding manufacturing technology that will lower costs and improve access... That, hopefully, will benefit patients in areas where third party payers don't have the same resources as in the US and Europe. We look forward to making that available to a wider community.”

While gene therapy may be able to treat many previously incurable diseases, being treated with a gene therapy often comes with a hefty price tag. Differences in healthcare systems and third-party payers further complicate gaining access to gene therapies. Shortages in labor and materials due to the COVID-19 pandemic are also currently exacerbating the issue.

GeneTherapyLive spoke with Barry Byrne, MD, PhD, director, Powell Center for Gene Therapy, University of Florida, and advisory committee member, FDA Cellular, Tissue, and Gene Therapies Advisory Committee, to learn more about improving access to gene therapies for patients with rare diseases worldwide. He also discussed recent challenges with manufacturing shortages due to the COVID-19 pandemic.

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