Aug 2026· Cell and Gene Therapy Insights· Vol 12, pp. 735–740· 0 citations
TL;DR
The financial and regulatory barriers to patient access, the field’s perceived image problem, and what it would take for policymakers to evaluate gene therapy with the same measured risk–benefit lens applied to other medicines are discussed.
Abstract
“Five years from now, when we have treated and cured many people with sickle cell disease, it will begin to feel mundane.” Abi Pinchbeck, Editor, Cell and Gene Therapy Insights, speaks with Paula Cannon, Distinguished Professor of Molecular Microbiology and Immunology, Keck School of Medicine of USC, about the evolution of gene therapy from a bold engineering challenge to an increasingly routine clinical reality. They discuss the financial and regulatory barriers to patient access, the field’s perceived image problem, and what it would take for policymakers to evaluate gene therapy with the same measured risk–benefit lens applied to other medicines.
The biological and regulatory challenges that need to be overcome are examined to bridge the gap between experimental innovation and clinically accessible therapies for patients with inherited skin diseases.
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This abstract book presents five undergraduate research proposals developed for a genetics research case competition organized by the Global Society for Genetics and Genome Biology Queen’s Chapter at Queen’s University in Kingston, Ontario, Canada. The competition focused on advancing gene therapy by considering scient...
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“As a physician, my goal is to develop a therapy that all intended patients, and healthcare systems, can afford and deliver.”Abi Pinchbeck, Commissioning Editor, Cell & Gene Therapy Insights , speaks with Dr Frank Borriello, CEO, Alloplex Biotherapeutics, about a non-engineered, autologous approach to cancer immunother...
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Although each individual rare genetic disorder is uncommon, collectively they represent a significant global health challenge. The clustered regularly interspaced short palindromic repeats (CRISPR) genome-editing technology has attracted substantial attention in medicine due to its adaptability, simple design, high eff...
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