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

Video

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.

Related Videos
Xandra Breakefield, PhD, on Trying New Approaches to AAV Therapy for Glioblastoma
Nathan Yozwiak, PhD, on Researching AAV Gene Therapy Delivery to the Brain
Salvador Rico, MD, PhD, on Informing Clinical Trials With Natural History Studies in Dravet
Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials
Janice Chen, PhD, the cofounder and chief technology officer of Mammoth Biosciences
Sekar Kethiresan, MD, on Following up VERVE-101 With Next-Generation Editing Therapies
Maria Escolar, MD, the chief medical officer of Forge Biologics
Leigh Ramos-Platt, MD, on Allowing Access and Ensuring Preparation for Gene Therapies
John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies
Erika Fullwood Augustine, MD, MS, the associate chief science officer of the Kennedy Krieger Institute
© 2024 MJH Life Sciences

All rights reserved.