The concept of using pre-existing immunogenicity as an exclusion criteria for gene therapy clinical trials is changing. BioAgilytix’s Chief Scientific Officer, Dr. Jim McNally, explores why.
A new clinical trial by St. Jude Children’s Research Hospital for the treatment of SCID-X1 highlights a major advancement in gene therapy safety over recent years, in this case spurred by scientists who were courageous enough to push forward the development of safer vectors to overcome serious adverse events (SAEs) that were resulting from the previous generation of gene therapies developed for SCID-X1.
The recent approval of Zynteglo further builds credibility to the specific and emerging class of cell therapies engineered via “ex vivo” gene therapy. We explain how these cell-based gene therapies work, and how they lend themselves to a host of translatable techniques for future therapeutic candidates.
By Dr. Arno Kromminga and Dr. Lydia Michaut
Gene therapy has experienced an exciting resurgence in the post-genomic era, but unwanted immunogenicity is still a concern. Our latest blog gives insight into the current concepts for designing an immunogenicity assessment strategy fitting the specific needs of a given gene therapy compound.
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