For Immediate Release
Challenges in Bringing Gene Therapy to the Market
Contact: Kathryn Ryan
914-740-2250
kryan@liebertpub.com
New Rochelle, NY, February 22, 2024—In a paradox of the field’s own success, unintended challenges remain in bringing gene therapy from the lab bench to the market. According to a new article in the peer-reviewed journal Human Gene Therapy, the market for gene therapy is on a steady ascent, yet challenges persist, included the cost, with the average cost of a gene therapy being between $1 million and $2 million per dose. Click here to read the article now.
Heather Gray-Edwards, from the University of Massachusetts Chan Medical School, and coauthors, describe the “Great Abandonment,” in which the pharmaceutical industry has shelved, abandoned, or rolled back development in more than 50 rare disease programs since 2021. “While classified as ‘rare,’ there are over 7,000 diseases, each impacting fewer than 200,000 Americans and collectively affecting around 30 million people in the United States,” stated the authors.
New approaches are needed to optimize both the approval and delivery of these lifesaving treatments. The authors point to the U.S. Food and Drug Administrations’ START Pilot Program as an example of a strategy to support novel drugs targeting rare diseases.
“Our position is that the biotechnology industry has benefited greatly from rare disease patients and families volunteering to participate in their trials, at times with significant risk,” says Editor in Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School. “It is only fair that the industry that has gained so much from rare disease patients would continue to provide access for those patients to the life-saving therapies that have come from the work.”
About the Journal
Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, and an esteemed international editorial board. Human Gene Therapy is available in print and online. Complete tables of contents and a sample issue are available on the Human Gene Therapy website.
About the Publisher
Mary Ann Liebert, Inc. is a global media company dedicated to creating, curating, and delivering impactful peer-reviewed research and authoritative content services to advance the fields of biotechnology and the life sciences, specialized clinical medicine, and public health and policy. For complete information, please visit the Mary Ann Liebert, Inc. website.