Regulatory requirements for clinical trial and marketing authorisation application for gene therapy medicinal products
Schüle, S.; Renner, M.; Longhurst, S.; Narayanan, G. · Bundesgesundheitsblatt - Gesundheitsforschung - Gesundheitsschutz · 2009 · Heft 12 · S. 30 bis 37
Bibliografische Angaben
Zusammenfassung
Over the last two decades, clinical trials using gene therapy medicinal products (GTMPs) have been carried out for a large number of rare, inherited monogeneic disorders as well as common multigeneic diseases such as cancer, cardiovascular and infectious diseases including AIDS. Despite some early difficulties and setbacks, the gene therapy field has slowly progressed and, nowadays, offers the promise of novel treatments for a growing number of diseases. On the other hand, gene therapy approaches are often associated with additional risks due to limited clinical experience with a given gene transfer system, long…
