Potential Adverse Outcomes of Gene Therapy
Gene therapy may warrant long-term follow-up of research subjects due to the risk of delayed adverse events. The FDA cites potential risks of adverse outcomes following exposure to human gene therapy products that are summarized as follows1:
“Integration activity” of the gene therapy product: Raises the potential for disruption of critical host genes that could result in malignancies.
Genome editing activity: Genome editing-based products impart their biological activity through site-specific changes in the human genome, but may also have off-target affects that raise the risk of malignancies and impaired gene function.
Prolonged expression: A gene therapy product where the therapeutic gene encodes growth factors, raising the potential for unregulated cell growth and malignancies.
Latency: A gene therapy product using, for example, a herpesvirus, has the potential for reactivation from latency, raising the risk of delayed adverse events related to a symptomatic infection.
Establishment of persistent infections: Gene therapy products that are replication-competent viruses and bacteria, such as listeria-based bacterial vectors, have the potential to cause persistent infections in immunocompromised patients.
REFERENCE
1. FDA. Long-Term Follow-Up After Administration of Human Gene Therapy Products. Draft Guidance for Industry. July 2018. Available at: https://bit.ly/2O4C3VE
Gene therapy may warrant long-term follow-up of research subjects due to the risk of delayed adverse events.
Subscribe Now for Access
You have reached your article limit for the month. We hope you found our articles both enjoyable and insightful. For information on new subscriptions, product trials, alternative billing arrangements or group and site discounts please call 800-688-2421. We look forward to having you as a long-term member of the Relias Media community.